Pharmacists: Unlocking Market Domination Through Strategic Drug Repurposing

The Dawn of Drug Repurposing: A Paradigm Shift in Pharmaceutical Innovation

The pharmaceutical industry constantly seeks groundbreaking innovations to address pressing medical needs and drive market growth. For decades, the traditional drug discovery pipeline has been the cornerstone of this endeavor, a laborious and often perilous journey from concept to market. This conventional path is characterized by immense time commitments, astronomical costs, and a dishearteningly high rate of failure. Imagine embarking on a quest where only one in ten ventures succeeds, and each attempt drains billions of dollars and spans over a decade. This is the reality of traditional drug development.

However, a revolutionary alternative has emerged, redefining how the industry approaches therapeutic innovation: drug repurposing. Also known as drug repositioning or reprofiling, this strategic approach involves identifying novel therapeutic applications for existing drugs that extend beyond their original indications.¹ It is a pivotal strategy in pharmaceutical development, encompassing not only entirely new uses for approved drugs but also exploring different formulations or even novel combinations of existing medicines.³ This innovative pathway promises a more efficient and economical route to bringing life-saving treatments to patients.

The growing acceptance and formalization of drug repurposing signify a fundamental shift in pharmaceutical research and development strategy. What once might have been considered an opportunistic side-project has evolved into a recognized, strategic pillar of innovation. Historically, many successful instances of drug repurposing occurred serendipitously, through accidental observations in clinical settings, as seen with drugs like sildenafil or aspirin.³ Today, however, the landscape has transformed. The consistent reference to drug repurposing as a “notable strategy” ¹, a “core approach” ⁹, and a “pivotal strategy” ⁴ underscores its maturation from chance discovery to systematic methodology.⁸ This evolution is largely driven by mounting economic pressures within the pharmaceutical sector and rapid advancements in technology. Companies are no longer merely waiting for fortunate accidents; they are actively building dedicated capabilities and processes around this approach.

This paradigm shift carries a profound implication: it places a greater emphasis on leveraging existing knowledge and vast datasets, moving away from an exclusive obsession with discovering novel molecules. This reorientation opens doors for diverse expertise, particularly for professionals like pharmacists, who possess a deep, intimate understanding of existing drugs and their real-world applications. The pharmacological knowledge and extensive clinical data that pharmacists routinely handle ¹ are precisely what drug repurposing endeavors seek to leverage. This inherent alignment positions pharmacists to become increasingly central to the innovation process, transforming their traditional roles into vital strategic contributions.

What Exactly is Drug Repurposing? Redefining Drug Development

To truly appreciate the transformative power of drug repurposing, it is essential to grasp its fundamental definition and how it diverges from the conventional drug development paradigm. Drug repurposing is fundamentally about identifying new therapeutic uses for drugs that are already approved or are still under investigation.² It is not about inventing new chemical entities from scratch, but rather about unlocking the latent potential within compounds whose basic properties are already known. This can involve finding entirely new therapeutic applications, developing different formulations for existing drugs, or even creating novel combinations of medicines.³ The overarching goal is to swiftly identify compounds with established safety profiles and known therapeutic advantages that may prove efficacious for other indications.²

This approach offers a profound advantage: it allows researchers to bypass many of the arduous and resource-intensive early stages of traditional drug development. The established safety profile of existing drugs is the primary enabler for this acceleration. Since these drugs have already undergone extensive preclinical testing and Phase I safety studies in humans, much of the foundational work establishing their safety can be omitted.³ This direct skipping of early development stages translates directly into reduced timelines, lower costs, and significantly mitigated risks for the repurposing project. This foundational advantage is what makes the entire strategy not just appealing, but increasingly viable and attractive for pharmaceutical companies.

Furthermore, the focus on “new therapeutic uses” for “existing or investigational drugs” ² significantly expands the pool of potential candidates. This means the opportunity landscape for repurposing extends far beyond currently marketed drugs. It includes compounds that may have failed in late-stage clinical trials due to inadequate efficacy for their original indication, or even those that were shelved for strategic business reasons, such as poor market prospects or incompatibility with a company’s evolving disease priorities.⁹ This inclusion of previously abandoned or unapproved drugs is crucial because it transforms what might have been considered costly failures into valuable assets. This implies a strategic shift within the industry, moving from a mindset of “what new molecules can we invent?” to a more expansive view of “what existing assets can we unlock value from?” This broader perspective is a key driver of the growing interest in drug repurposing.

Beyond the Original: A Historical Perspective on Repurposed Successes

The concept of drug repurposing, while now a formalized strategy, has a rich history punctuated by remarkable successes, often born from unexpected observations. These historical examples serve as compelling proof-of-concept, illustrating the profound capacity of existing compounds to influence human biological pathways in unforeseen ways.

Perhaps one of the most well-known examples is sildenafil citrate, famously marketed as Viagra®. Initially developed by Pfizer for the treatment of hypertension and angina, its unexpected efficacy in treating erectile dysfunction was observed during clinical trials.¹ This serendipitous discovery led to its repurposing and subsequent blockbuster success. Later, sildenafil found yet another life, repurposed for the treatment of pulmonary arterial hypertension.⁴

Another poignant example is thalidomide. This drug gained notoriety in the 1960s for causing severe congenital disorders when prescribed as a sedative to pregnant women, leading to its withdrawal from the market.¹ However, decades later, its immunomodulatory properties were recognized, and it was successfully repurposed for the treatment of leprosy (specifically, erythema nodosum leprosum) and multiple myeloma.¹ This journey from a troubled past to a life-saving therapy underscores the dynamic nature of drug evaluation.

Aspirin, one of the oldest and most widely used drugs, also boasts a significant repurposing story. Initially marketed in 1899 as an analgesic and anti-inflammatory agent, it was later repurposed in the 1980s, at low doses, as an antiplatelet aggregation drug to prevent cardiovascular events.⁷ Its protective effect against cancer is now being explored, particularly for colorectal cancer, based on its inhibition of COX-2.⁷

More recent examples highlight repurposing’s agility in addressing urgent needs. Dexamethasone, a widely used corticosteroid, was found to be effective in treating hospitalized COVID-19 patients, particularly those requiring ventilation.² Similarly, azidothymidine (AZT), originally developed in the 1960s as a cancer therapeutic, was rapidly repurposed for HIV/AIDS during that crisis, advancing from

in vitro testing to clinical use in less than three years.¹¹ Even propranolol, a beta-blocker traditionally used for cardiovascular conditions, was repurposed for infantile hemangiomas after a clinical observation noted its effect on a baby with both a heart condition and a hemangioma.¹³

The recurring theme across these historical successes is “serendipitous observation”.³ These “aha!” moments, born from unexpected clinical findings, underscore the critical importance of vigilance and real-world data in drug discovery. This directly relates to the role of pharmacists, who are often on the front lines of patient care, uniquely positioned to observe patient responses and potential off-label effects. Their daily interactions and deep understanding of medication use make them invaluable contributors to this observational discovery process.

The successful repurposing of drugs like thalidomide, despite their checkered pasts, carries a profound implication: it signifies that the benefit-to-risk ratio of a drug can be dynamically re-evaluated for new indications.¹⁰ A drug’s “failure” in one context does not necessarily mean it is inherently “bad.” Instead, it suggests that its effects need to be understood in relation to specific disease mechanisms and patient populations. This perspective opens up a vast, untapped pool of “failed” or abandoned drugs for potential repurposing, emphasizing the continuous need for careful re-evaluation and a nuanced understanding of pharmacology.

Why Repurpose? The Irresistible Advantages Over Traditional Drug Discovery

The pharmaceutical industry operates under immense pressure to deliver innovative therapies while managing escalating research and development (R&D) costs and high rates of clinical failure. In this challenging environment, drug repurposing emerges as an almost irresistible strategy, offering compelling business and scientific advantages over traditional de novo drug discovery.

Drug repurposing promises significantly faster timelines, substantially lower costs, and considerably less risk compared to developing entirely new drugs.³ This efficiency stems from a fundamental advantage: repurposed drugs bypass many early-stage development hurdles, including extensive preclinical studies and Phase I safety trials, because their safety profiles are already well-established in humans.³ This streamlined process leads to a remarkably higher approval rate. While only about 10% of new drug applications gain market approval, approximately 30% of repurposed drugs successfully navigate the regulatory pathway.⁹

The established safety profile of repurposed drugs directly translates into this higher success rate and significantly lower R&D costs, making it an exceptionally attractive strategy for pharmaceutical companies. Consider the staggering cost of traditional R&D: an average of US$2.23 billion per asset in 2024, with overall declining returns.¹⁷ In stark contrast, repurposing an existing drug costs approximately $300 million.⁸ This dramatic difference in investment, coupled with a higher probability of success, fundamentally alters the risk-reward equation. It directly impacts a company’s return on investment (ROI) and enhances the sustainability of its pipeline, offering a financially prudent path forward.

The higher approval rate for repurposed drugs, approximately three times that of novel compounds, suggests a more predictable and de-risked investment. This predictability can attract more capital and accelerate market entry for critical therapies. Reduced uncertainty ¹⁹ and a lower “risk of failure” ¹⁰ inherent in repurposing mean that companies can pursue projects with greater confidence. This often translates to faster regulatory reviews, such as those facilitated by the 505(b)(2) pathway ³, ultimately leading to quicker patient access to much-needed treatments.

Speed, Cost, and Risk: The Triple Crown of Repositioning

The quantitative advantages of drug repurposing are truly compelling, forming what can be described as the “triple crown” of repositioning: speed, cost-efficiency, and reduced risk.

Repurposed drugs generally achieve regulatory approval much sooner than novel compounds, typically within 3 to 12 years, and often at about half the cost of traditional development.⁹ To put this into perspective, the traditional drug discovery process can take a daunting 10 to 15 years and incur costs ranging from $2 billion to $3 billion to bring a new drug to market.¹⁶ In contrast, repurposing an existing drug costs approximately $300 million and can take around 6 years on average.⁸

This significant reduction in time and cost directly translates into increased accessibility for patients, particularly for conditions where traditional development might be financially unviable. If a treatment can reach patients in 3-12 years instead of 10-15, and for hundreds of millions instead of billions, it means therapies can become available much faster and potentially at a more affordable price point. This is especially critical for rare diseases or during public health crises where the urgency for rapid solutions is paramount. The “urgent need for effective treatments” demonstrated during the COVID-19 pandemic, which saw over 100 repurposed drugs moved into clinical trials and several receiving FDA approval or Emergency Use Authorization ³, serves as a powerful illustration of this causal link.

The inherent speed, cost-effectiveness, and reduced risk associated with drug repurposing position it as a key strategic tool for pharmaceutical companies. Faced with the “looming patent cliff threatening revenue streams” ¹⁷ and intense “patent expiry pressures” ²¹ on their existing portfolios, repurposing offers a vital lifeline. It functions as a powerful “life-cycle management” strategy ²¹, enabling companies to “extend the value of off-patent or low-performing assets”.²¹ This approach is not merely about discovering new drugs; it is fundamentally about maximizing the return on past investments, making it an indispensable component of long-term business strategy and pipeline sustainability.

Addressing Unmet Needs: From Rare Diseases to Global Crises

One of the most profound impacts of drug repurposing lies in its ability to provide solutions for conditions often overlooked or neglected by traditional R&D due to market size constraints or inherent complexities. The conventional drug discovery model, driven by commercial viability, often prioritizes large market opportunities, leaving millions of patients with rare diseases or those affected by sudden, emerging health crises without adequate treatment options. Drug repurposing fills this critical gap, offering a pathway to hope where none traditionally existed.

Drug repurposing is exceptionally relevant for addressing rare and neglected diseases, conditions for which conventional drug development is frequently financially unviable.⁴ Developing novel drugs for these conditions can be financially challenging due to small patient populations and uncertain market returns.⁴ However, the inherently lower cost and reduced risk of repurposing make it “more economically viable”.⁴ This directly leads to the development of treatments for conditions that would otherwise be ignored by traditional pharmaceutical investment. This causal chain—from financial viability to R&D investment and ultimately to patient access—is a powerful testament to repurposing’s impact on public health. It facilitates earlier access to much-needed medicines for serious or life-threatening diseases, including pediatric conditions.³

Beyond chronic and rare conditions, the strategic value of drug repurposing became acutely evident during public health crises. The COVID-19 pandemic served as a real-world stress test, showcasing the unparalleled agility of repurposing. During this global emergency, repurposed drugs were rapidly investigated and deployed, with over 100 unique compounds moved into clinical trials, and several ultimately receiving FDA approval or Emergency Use Authorization.⁴ This rapid deployment capability demonstrates repurposing’s strategic importance for global health preparedness and resilience. It positions repurposing not merely as a commercial strategy but as a public health imperative, providing “timely therapeutic options” ²³ when novel treatments are years away. This highlights the critical need for continued investment and policy support to bolster this vital innovation pathway.

The Pharmacist’s Foundation: A Deep Well of Expertise

To truly grasp the indispensable role pharmacists can play in drug repurposing, it is crucial to understand the comprehensive training and unique daily interactions that form their foundational expertise. Pharmacists are not merely dispensers of medication; they are highly trained healthcare professionals and medicines experts possessing a unique and complex body of knowledge and skills.²⁴ Their education equips them to prepare, dispense, and store prescription drugs, but critically, it also trains them to educate patients on safe use, advise on potential side effects, and manage intricate medication regimens.²⁵ As medication therapy has grown in complexity, the pharmacist’s role has expanded significantly beyond traditional dispensing ²⁶, creating a natural alignment with the evolving needs of drug repurposing.

The evolution of the pharmacist’s role from a purely transactional dispenser to a clinical expert deeply involved in patient care and medication management creates a natural and powerful alignment with the demands of drug repurposing. Snippets reveal that their role has “expanded as medication therapy has increased in complexity” ²⁶, now encompassing “comprehensive medication management” ²⁷, extensive “patient education” ²⁷, and crucial “coordinating care transitions”.²⁶ This ongoing trend of increasing clinical involvement means pharmacists are already performing many functions that generate valuable insights relevant to repurposing, even if these contributions are not explicitly labeled as such within a drug discovery context.

Furthermore, the “unique and complex body of knowledge and skills” ²⁴ that pharmacists possess positions them as significantly underutilized assets in the broader drug discovery pipeline, particularly in the realm of repurposing where real-world insights are paramount. If, as historical examples demonstrate, drug repurposing often stems from astute clinical observations ³, who better to make those observations than the healthcare professional most intimately involved with medication use and patient outcomes? Their expertise in “medication action, dosing, adverse effects, and drug interactions” ²⁸ directly corresponds to the type of data needed to identify and evaluate repurposing candidates. This suggests a significant strategic opportunity for the pharmaceutical industry to better integrate pharmacists into their R&D processes, leveraging their frontline perspective.

Masters of Medication: Pharmacotherapy Knowledge at the Core

At the very core of a pharmacist’s value proposition in drug repurposing lies their unparalleled pharmacotherapy knowledge. Pharmacists possess an in-depth understanding of medications and disease states, applying specialized knowledge of the scientific and clinical use of drugs, including their mechanisms of action, optimal dosing, potential adverse effects, and complex drug interactions.²⁸ They are, quite literally, “medicines experts” ²⁴, rigorously trained to assess medication appropriateness, effectiveness, and safety for each individual patient.²⁶ This comprehensive understanding allows them to identify untreated health problems that could potentially be improved or resolved with appropriate medication therapy.²⁸

A pharmacist’s deep understanding of pharmacokinetics (how drugs move through the body), pharmacodynamics (how drugs affect the body), and the intricate web of drug-disease interactions enables them to hypothesize new therapeutic uses based on a drug’s known mechanisms, even if those uses have not been explicitly studied for a new indication. When pharmacists “understand how the medications work” ²⁷ and can “assess medication appropriateness, effectiveness, and safety for each individual patient” ²⁶, they are, in essence, performing a micro-repurposing evaluation with every patient interaction. Their granular knowledge of “drug action, dosing, adverse effects, and drug interactions” ²⁸ constitutes the raw material necessary for target-based or knowledge-based repurposing methods.¹⁰ This establishes a direct and powerful link between their core competency and the generation of new repurposing opportunities.

This expertise extends beyond the realm of single drugs to encompass complex polypharmacy scenarios, where patients often take multiple medications simultaneously. In such situations, pharmacists are uniquely equipped to spot subtle effects or interactions that might suggest a drug’s broader therapeutic potential or previously unrecognized off-target effects. They “comprehensively review a patient’s full medication regimen to ensure medications work well together and avoid problems (e.g., interaction)”.²⁶ This holistic view means they are perfectly positioned to observe “off-target effects” ³⁰ or instances of “polypharmacology” ¹¹ – where a drug acts on multiple biological targets – which are key drivers for drug repurposing. Their daily practice, therefore, functions as a living laboratory for drug repurposing discovery, generating invaluable, nuanced data that computational methods alone might miss.

Comprehensive Medication Management: Beyond Dispensing

The pharmacist’s role in comprehensive medication management extends far beyond the transaction of dispensing pills; it is a dynamic, patient-centric practice that generates invaluable real-world data for drug repurposing. Pharmacists actively provide comprehensive medication management, which includes resolving medication-related problems through various channels such as telephone calls, home visits, and direct communication with other healthcare clinicians.²⁷ They are adept at identifying medication discrepancies, particularly during critical care transitions, such as a patient moving from hospital to home. This meticulous attention helps prevent errors like unintentional nonadherence or duplication of medication therapy.²⁷ Furthermore, pharmacists regularly collaborate with prescribers to adjust medications, initiate new therapies, modify dosages, or even discontinue unnecessary medications, a process known as “deprescribing”.²⁷ Their adherence to the “five rights” of medication use—right dose, right medication, right patient, right time, right route—underpins every aspect of their practice, ensuring patient safety and optimal outcomes.²⁶

The pharmacist’s active involvement in medication management, including deprescribing and the identification of medication discrepancies, can reveal situations where a drug’s effect (or lack thereof) in a specific clinical context might signal a new therapeutic opportunity or an unexpected interaction. When pharmacists “suggest that medications are stopped or reduced, especially when the risk outweighs the benefit and side effects are present” ²⁷, they are essentially observing drug performance in a nuanced, real-world setting. This iterative process of optimizing existing therapies can inadvertently highlight a drug’s unexpected benefits or reveal an underlying disease mechanism that an existing drug could effectively address. This establishes a direct, practical link between their daily clinical activities and the generation of novel repurposing hypotheses.

Moreover, by diligently ensuring medication adherence and providing thorough patient education ²⁶, pharmacists inherently improve the quality of real-world data. This enhanced data quality makes it significantly more reliable for subsequent computational and observational repurposing efforts. “Real-world data (RWD) are direct observations from clinical patients that may be seen as valuable readouts of drug effects directly on human bodies”.⁵ If pharmacists are actively improving patient adherence and their understanding of their medications, they are implicitly improving the

quality and accuracy of the RWD generated from patient outcomes. Cleaner, more accurate RWD is absolutely crucial for the effective functioning of artificial intelligence (AI) and machine learning algorithms in identifying repurposing signals.⁵ This highlights an indirect, yet profoundly vital, contribution of pharmacists to the broader drug repurposing ecosystem.

Competency Area	Core Pharmacist Skills	Direct Alignment with Drug Repurposing	Relevant Snippet IDs
Pharmacotherapy Knowledge	In-depth understanding of drug mechanisms, pharmacokinetics, pharmacodynamics, adverse effects, and drug interactions across various disease states.²⁴	Identifying new indications based on known molecular targets or unexpected effects; hypothesizing new therapeutic uses from a drug’s established profile; evaluating target specificity for new indications.¹⁰	¹⁰
Medication Management	Providing comprehensive medication management; identifying discrepancies during care transitions; collaborating with prescribers for dosage adjustments, initiation, or deprescribing; ensuring “five rights” of medication use.²⁶	Observing drug performance in real-world settings; identifying unexpected benefits or interactions that signal repurposing opportunities; improving RWD quality through adherence and accurate record-keeping.⁵	⁵
Patient Interaction & Education	Educating patients on safe and effective medication use, potential side effects, and adherence strategies; addressing patient concerns; building trust.²⁶	Fostering patient understanding and engagement, which can facilitate data collection for repurposing studies; improving accuracy of patient-reported outcomes; enhancing patient safety in off-label use scenarios.¹	¹
Pharmacovigilance	Detecting, assessing, understanding, and preventing adverse drug reactions (ADRs); monitoring drug performance post-market; utilizing pharmacovigilance systems.³⁴	Identifying beneficial off-target effects or novel therapeutic mechanisms from unexpected reactions; transforming safety monitoring into a proactive discovery engine for repurposing candidates.³⁰	³⁰
Data Interpretation & Digital Literacy	Accessing and interpreting patient data from EHRs; leveraging digital health tools for remote monitoring and workflow optimization; understanding data-driven clinical decisions.²⁷	Contributing to the richness and accuracy of RWD; validating and refining AI-generated repurposing hypotheses; advocating for and utilizing integrated digital health platforms for research.⁵	⁵

Pharmacists in Action: Driving Drug Repurposing Across the Lifecycle

Having established the profound foundational expertise of pharmacists, the focus now shifts to their active participation in driving drug repurposing across its entire lifecycle. Pharmacists are not merely passive observers in this innovative approach; they are integral players whose unique contributions can accelerate discovery, ensure safety, and optimize patient outcomes. Their crucial role stems from the vast opportunities inherent in drug repurposing.³⁰ Pharmacists are involved from the initial identification of compounds with repurposing potential, through supporting rigorous development processes, and continuing with vital post-market safety monitoring.¹⁹

The increasing complexity of modern drug development, coupled with the rise of data-driven methodologies, necessitates a highly multidisciplinary team approach. Within this collaborative ecosystem, the unique, patient-centric insights offered by pharmacists become absolutely critical. Evidence indicates that pharmacists are consistently “part of multidisciplinary teams” ³⁹ and regularly collaborate with physicians and other health professionals.²⁸ This highlights a broader trend towards integrated healthcare and drug development. Drug repurposing, with its reliance on diverse data sources—from clinical observations and electronic health records (EHRs) to advanced artificial intelligence (AI) analyses—naturally benefits immensely from such a collaborative model. In this environment, pharmacists bring the crucial “real-world” perspective, grounding theoretical possibilities in practical patient experience.

Furthermore, the pharmacist’s involvement throughout the entire drug lifecycle, from initial observation of a potential signal to rigorous clinical trials and ongoing post-market surveillance, creates a continuous feedback loop. This holistic engagement can significantly accelerate and de-risk repurposing efforts. By identifying signals, contributing to validation studies, and then meticulously monitoring outcomes, pharmacists establish a virtuous cycle for drug repurposing. This comprehensive, continuous involvement represents a significant competitive advantage for any organization committed to maximizing the potential of existing therapies.

Identifying the Hidden Gems: Pharmacists as Repurposing Detectives

Pharmacists possess a unique vantage point that positions them as frontline “detectives” in the search for hidden gems within the pharmaceutical landscape. Their daily interactions with patients and deep understanding of medication effects make them invaluable in spotting early signals for drug repurposing.

Serendipitous Discoveries and Clinical Observations: The “Aha!” Moments

Many of the most celebrated drug repurposing success stories, such as sildenafil and aspirin, originated from serendipitous observations of their efficacy for conditions entirely different from their original indications.⁴ Clinical observation plays an undeniably vital role, where unexpected benefits are noticed either during formal clinical trials or through the informal practice of off-label use.⁴ Pharmacists are uniquely positioned to identify these “aha!” moments through their extensive daily patient interactions and their comprehensive medication reviews.¹⁹

The direct and continuous patient interaction that pharmacists engage in, coupled with their profound pharmacotherapy knowledge, creates a fertile ground for these “serendipitous observations” that can spark groundbreaking repurposing hypotheses. Consider how sildenafil’s effect on erectile dysfunction was “unravelled during the clinical trials” ¹⁰, or how propranolol’s impact on hemangiomas was “discovered when treating a heart condition in a baby”.¹³ These instances highlight the immense power of astute clinical observation. Pharmacists, who routinely “consult with the patient’s physicians and other health care providers” ²⁸ and “educate patients about their medications” ²⁷, are constantly observing the nuanced effects of drugs in real-world scenarios. This direct, frontline patient exposure makes them indispensable “detectives” in the repurposing process.

Furthermore, by actively documenting and systematically sharing these clinical observations, even seemingly informal ones, pharmacists can contribute to a richer and more robust real-world data pool. This data, when combined with sophisticated “AI and machine learning” algorithms ⁴, can transform anecdotal evidence into testable hypotheses for systematic repurposing. This bridges the critical gap between individual clinical practice and large-scale drug discovery, creating a pathway for insights from the bedside to inform the laboratory. This underscores the need for improved mechanisms and platforms that enable pharmacists to efficiently report and share such valuable clinical observations.

Proactive Off-Label Use Identification: A Pharmacist’s Vigilance

Off-label drug use, defined as prescribing a medication for indications other than those officially approved by the FDA ³³, is a common and often medically accepted practice. It frequently serves as a precursor to formal drug repurposing. Pharmacists play an integral role in the effective management and evaluation of off-label medication use, critically assessing medications for appropriate use on a case-by-case basis.⁴² They meticulously review indications during the order verification process and proactively communicate with prescribers when off-label use is identified.⁴² Clinical practice areas with a high frequency of off-label drug usage include oncology, rare conditions, psychiatric care, and pediatric practices.⁴² Notable examples of drugs frequently used off-label include gabapentin for anxiety and sleep disorders, and metformin for polycystic ovary syndrome (PCOS) and prediabetes.³³ Pharmacists also bear the responsibility of informing patients when a prescribed medication is being used off-label.³³

The direct involvement of pharmacists in evaluating and managing off-label drug use provides critical real-world efficacy and safety data. This practical experience serves as a direct pipeline for formal drug repurposing initiatives. Off-label use is, in essence, informal drug repurposing already in action.⁴³ When pharmacists “critically evaluate medications for appropriate use on a case-by-case basis” ⁴² and are actively involved in “monitoring for off-label medication use” ⁴², they are generating invaluable, granular data on how a drug performs in a new context. This includes observing “potential safety and toxicity risks, and side effects and adverse events”.² This real-world data is a direct input for formal repurposing efforts, significantly reducing the need for extensive early-stage trials that would otherwise be required for a novel indication.

Furthermore, the pharmacist’s crucial role in disclosing off-label use to patients ³³ highlights their ethical responsibility and positions them as trusted intermediaries within the healthcare system. This transparency with patients is vital for ensuring informed consent.¹ When pharmacists educate patients about their medications, including off-label uses, they build a foundation of trust. This trust can, in turn, facilitate the collection of more accurate patient-reported outcomes and encourage greater patient participation in future research studies. These patient contributions are vital for generating the robust real-world evidence necessary to support formal repurposing applications. This emphasizes the ethical dimension of the pharmacist’s role in driving innovation, where patient trust directly contributes to scientific advancement.

The Power of Pharmacovigilance: Unearthing New Indications from Adverse Events

Sometimes, a drug’s “failure” in one indication due to an adverse effect can paradoxically become its “success” in another, if that “adverse” effect proves therapeutically beneficial elsewhere. Pharmacists are at the forefront of this fascinating intersection, leveraging pharmacovigilance to unearth new repurposing opportunities. Pharmacovigilance (PV) is the comprehensive science and activity dedicated to the collection, detection, assessment, monitoring, and prevention of adverse effects associated with pharmaceutical products.³⁴ Pharmacists play a crucial role within PV systems, actively monitoring drug performance and identifying adverse drug reactions (ADRs) earlier in the post-market phase.³⁴ Intriguingly, PV is increasingly recognized for its contribution to drug repurposing by helping to identify new uses for old drugs.³⁵ Even unexpected adverse reactions (SUSARs) reported during clinical trials can serve as key signals for new indications.³⁵ The journey of thalidomide, repurposed for leprosy and multiple myeloma after its notorious withdrawal due to congenital disorders, powerfully illustrates how even a troubled history can lead to new, life-changing uses.⁴

The active participation of pharmacists in pharmacovigilance, particularly in identifying and reporting adverse drug reactions or unexpected effects, can directly lead to the identification of novel therapeutic mechanisms or off-target effects suitable for repurposing. If a drug exhibits an “undesired effect in one disease state [that] may be beneficial in treating another” ³⁰, pharmacists are uniquely positioned to observe and document this phenomenon. Their “advanced training” enables them to “utilize pharmacovigilance systems interfaced with electronic health records to monitor the performance of the drugs they fill and also identify adverse drug reactions earlier”.³⁴ This direct observation and diligent reporting can serve as the critical initial spark for a repurposing hypothesis, transforming a potential safety concern into a therapeutic opportunity.

Furthermore, integrating pharmacists more deeply into pharmacovigilance systems, especially those augmented with AI-driven tools, has the potential to transform PV from a purely safety-focused activity into a proactive drug discovery engine for repurposing. “AI and machine learning are revolutionizing pharmacovigilance, automating adverse event detection”.³⁵ If pharmacists are empowered with these advanced tools and encouraged to actively look for

beneficial off-target effects, PV evolves into a dual-purpose system: simultaneously ensuring patient safety and identifying new innovation opportunities. This represents a strategic shift from reactive safety monitoring to proactive, data-driven drug discovery.

Drug Name	Original Indication	Repurposed Indication	Key Insight/Mechanism	Relevant Snippet IDs
Sildenafil (Viagra®)	Hypertension, Angina ¹	Erectile Dysfunction, Pulmonary Arterial Hypertension ¹	Unexpected side effect observed during clinical trials ⁴	¹
Thalidomide	Sedative (for morning sickness) ¹	Leprosy (Erythema Nodosum Leprosum), Multiple Myeloma ¹	Re-evaluation of benefit-risk ratio for new indications after withdrawal due to severe adverse effects; immunomodulatory properties ¹	¹
Aspirin	Analgesic, Anti-inflammatory ⁷	Antiplatelet Aggregation (Cardiovascular events), Oncology (Colorectal Cancer prevention) ⁷	Dose-dependent effects; inhibition of COX-1/COX-2; long-term clinical observation ⁷	⁷
Dexamethasone	Corticosteroid (allergies, asthma, arthritis) ¹²	COVID-19 (hospitalized patients requiring ventilation) ²	Efficacy in reducing mortality in a public health crisis; rapid clinical investigation ²	²
AZT (Azidothymidine)	Cancer (environmental retroviruses) ¹¹	HIV/AIDS ¹¹	Emergency drug screening effort during a crisis; potent anti-HIV compound identified ¹¹	¹¹
Propranolol	Cardiovascular conditions (e.g., hypertension) ¹³	Infantile Hemangiomas ¹³	Clinical observation of unexpected effect in a pediatric patient; understanding of mechanism of action (vasoconstriction, angiogenesis inhibition) ¹³	¹³

Navigating the Clinical Labyrinth: Pharmacists in Repurposing Trials

Once a promising drug repurposing candidate has been identified, the journey through clinical trials begins. This phase, while streamlined compared to novel drug development, remains rigorous, demanding meticulous attention to safety, efficacy, and data integrity. Pharmacists are not merely peripheral figures in this labyrinth; they are central navigators, ensuring the scientific and ethical soundness of these crucial studies.

Protocol Design and Medication Management: Ensuring Safety and Efficacy

Pharmacists contribute significantly to the design of clinical trials, ensuring that study protocols are both safe and effective. Their expertise is invaluable in selecting appropriate dosages, routes of administration, and duration of therapy for the new indication.⁴⁴ They meticulously identify potential drug interactions and adverse effects, proactively working to minimize risks to trial participants.⁴⁴ Research pharmacists, in particular, play a pivotal role in facilitating clinical trials through comprehensive protocol review and robust medication management support.³⁹ They bear the critical responsibility for the precise preparation, secure storage, and accurate dispensing of investigational drugs, adhering to stringent guidelines to maintain drug stability and efficacy. This includes ensuring accurate dosing and administration, and maintaining detailed records for regulatory compliance.²⁵

The expertise of pharmacists in medication management and patient safety directly enhances the reliability and ethical conduct of repurposing clinical trials, ultimately reducing the risk of adverse events and improving the quality of trial data. Even with an established safety profile for its original indication, the “safety profile in the new indication needs to be thoroughly evaluated, especially when drugs are combined”.⁴⁵ Pharmacists actively “ensure patient safety first and the quality of the study”.³⁹ By “conducting thorough medication reviews to identify potential interactions or contraindications” and diligently “monitoring adverse drug reactions” ⁴⁶, they directly mitigate risks that could jeopardize patient well-being or compromise trial integrity. This leads to cleaner, more trustworthy trial data, which is absolutely essential for achieving regulatory approval and subsequent market acceptance.

Furthermore, the involvement of pharmacists in critical aspects like “dose adjustments” and understanding “pharmacokinetics and pharmacodynamics” ⁴⁵ for new indications means they are far more than just logistical support. They are active scientific contributors to optimizing repurposed therapies. Repurposing a drug might necessitate significant changes in dosage or formulation to achieve efficacy in a new patient population or for a different disease.² Pharmacists’ deep knowledge of drug properties allows them to provide “expertise and invaluable insight from development and implementation of research protocols”.³⁹ This extends beyond the act of dispensing; it encompasses providing crucial scientific input into how the drug will behave in an entirely new therapeutic context, directly impacting the trial’s success and the ultimate patient benefit.

Patient Monitoring and Data Collection: The Eyes and Ears of the Trial

The success of any clinical trial, particularly for repurposed drugs, hinges on meticulous patient monitoring and the accurate capture of data. Pharmacists, with their inherent patient-facing roles and attention to detail, are uniquely positioned to serve as the “eyes and ears” of the trial, ensuring both patient safety and data integrity. Pharmacists actively monitor participants for adverse drug reactions and interactions, provide comprehensive counseling on proper drug use, and diligently work to ensure patient adherence to the study protocol.⁴⁴ They meticulously track adverse events, monitor medication adherence rates, and collect other vital pharmacological data, all of which contribute significantly to the subsequent analysis and publication of trial findings.⁴⁴ Their ability to develop personalized medication schedules and reminders is also a key strategy to improve patient adherence, a critical factor for trial success.⁴⁶

The direct patient engagement and unwavering focus on adherence and side effect monitoring by pharmacists directly improve the quality and completeness of clinical trial data. This, in turn, leads to more robust evidence for the efficacy and safety of repurposed drugs. If patients fail to adhere to study protocols or neglect to report side effects, the reliability of trial data is severely compromised. Pharmacists “help patients understand the purpose of their medications, how to take them safely and effectively, and potential side effects”.²⁷ This crucial educational role, combined with their proactive “regular check-ins with participants” ⁴⁶, directly enhances patient adherence and the accuracy of reported data. Better, more reliable data subsequently leads to stronger evidence, which is indispensable for gaining regulatory approval—a critical step for market entry.

Furthermore, by serving as a vital bridge between patients and the research team, pharmacists significantly enhance both patient safety and trust in clinical trials. This can have a tangible impact on participation and retention rates, which are fundamental to the success of repurposing studies. Patients often harbor apprehension or skepticism about participating in clinical trials. Pharmacists, as highly accessible and trusted healthcare professionals, are uniquely positioned to “alleviate anxiety and build trust” ⁴⁶ among potential participants. This patient advocacy role is not merely an ethical consideration; it carries a direct practical business implication: higher patient retention in trials translates into less time and financial resources expended on recruitment, thereby accelerating the overall drug development timeline and improving the efficiency of the repurposing pipeline.

Post-Market Optimization: Sustaining Value and Patient Outcomes

The journey of a repurposed drug does not conclude with its initial regulatory approval. In fact, the post-market phase presents a continuous opportunity for optimization, value sustenance, and the identification of further repurposing potential. Pharmacists remain indispensable contributors during this ongoing phase, leveraging their unique position to gather real-world insights and ensure patient well-being.

Real-World Evidence Generation: Informing Future Repurposing

Real-world data (RWD) has emerged as a cornerstone of modern drug development, offering insights that complement and expand upon traditional clinical trial findings. RWD encompasses data collected from diverse sources beyond the controlled environment of traditional clinical trials, including electronic health records (EHRs), insurance claims data, and patient registries.³¹ Real-world evidence (RWE), derived from the analysis of RWD, is increasingly vital for informing patient care, enhancing safety surveillance, guiding therapeutic development, and supporting outcomes research.³¹ RWD studies are notably cost-effective, capable of scaling to encompass vast patient populations, and offer a more accurate representation of real-life drug effects, including interactions with comorbidities and co-medications.⁵ Pharmacists are uniquely positioned to leverage advanced methods, including AI, to optimize the measurement of variables within large healthcare databases and to develop predictive models for adverse drug effects.⁴⁷ The growing importance of RWD is further underscored by legislative frameworks like the 21st Century Cures Act, which actively encourages its use in drug development and repurposing.³¹

Pharmacists’ daily interaction with EHRs and their active role in medication management directly contribute to the richness and accuracy of real-world data. This, in turn, is crucial for generating real-world evidence that can identify new repurposing signals and optimize existing ones. Pharmacists routinely “check for consistency and correctness of a patient’s medication history” within EHRs ³⁶ and perform essential “medication reconciliation” processes.²⁶ This direct, hands-on engagement with patient data means they are not merely consumers of RWD but active contributors to its quality and integrity. High-quality RWD is the lifeblood of advanced computational repurposing methods.⁵ This creates a powerful positive feedback loop: enhanced pharmacist activity leads to superior RWD, which in turn facilitates more effective and efficient drug repurposing.

The increasing reliance on RWE for regulatory approvals, exemplified by initiatives stemming from the 21st Century Cures Act, elevates the pharmacist’s role from primarily a clinical practitioner to a vital data steward and analyst. This shift directly influences the future trajectory of drug development. The 21st Century Cures Act, for instance, aims to “accelerate the FDA drug and medical device approval processes by replacing some of the data requirements from clinical trials with observational data or RWD settings”.³¹ This significant policy shift means that RWE is gaining considerable regulatory weight. Pharmacists, with their unique access to and profound understanding of patient data, become critical in generating this evidence. This underscores a growing need for pharmacists to cultivate stronger data analytics skills and for healthcare systems to provide them with better access to integrated digital tools that facilitate seamless data collection and analysis.

Continuous Pharmacovigilance and Patient Education

The journey of a drug does not end with its market approval; rather, it enters a phase of continuous monitoring and adaptation. Ongoing pharmacovigilance remains paramount post-market to ensure the long-term safety of drugs and to identify any new or rare adverse drug reactions (ADRs) that may emerge in broader patient populations.³⁴ Pharmacists are crucial in this continuous monitoring process, actively detecting ADRs and ensuring the rational use of medications.³⁴ Simultaneously, their role in patient education continues to be vital. They educate patients on safe and effective medication use, potential side effects, and adherence strategies, empowering individuals to manage their health effectively.²⁶

Continuous pharmacovigilance conducted by pharmacists, extending beyond initial approval, can identify long-term or rare beneficial off-target effects. This ongoing vigilance serves as a perpetual source of new repurposing hypotheses. While initial clinical trials establish a drug’s safety and efficacy in controlled settings, long-term use across diverse real-world populations can reveal subtle, unexpected effects—both positive and negative. Pharmacists, through their “continuous monitoring” ³⁴ and their practice of “reviewing proper dosing with patients or providers” ²⁶, are perfectly positioned to detect these nuances. This persistent observation can lead to the “serendipitous discovery” ⁴ of

another repurposing opportunity, potentially extending a drug’s lifecycle and value even further.

Empowering patients through comprehensive education and ongoing adherence support not only improves individual health outcomes but also cultivates a more informed and engaged patient population. This engagement can lead to better self-reporting of drug effects and greater willingness to participate in future research initiatives. “Patients need to understand the importance of taking their medications as prescribed”.²⁷ When pharmacists meticulously educate patients, they foster a more knowledgeable and proactive patient base. This can result in more accurate patient-reported outcomes, which are invaluable sources of RWD, and a greater inclination among patients to participate in follow-up studies or clinical trials for new indications. This concerted effort builds a robust foundation for future patient-centric drug development, where patient insights directly contribute to innovation.

The Strategic Imperative: Business Transformation Through Repurposing

Drug repurposing is not merely a scientific curiosity or a clinical convenience; it is a powerful business strategy driving transformative change across the pharmaceutical industry. This approach offers faster, more cost-effective solutions for addressing unmet medical needs ²³, a critical advantage in today’s competitive landscape. Its growing prominence is rooted in its ability to significantly reduce the time, cost, and risk associated with drug development.¹⁶ The market projections underscore this strategic shift: the global drug repurposing market is forecasted to grow substantially, from an estimated US

29.4billionin2024toUS37.3 billion by 2030, representing a Compound Annual Growth Rate (CAGR) of 4.1%.²¹ Other projections are even more optimistic, estimating a market value of USD 56.25 billion by 2033, growing at a CAGR of 5.42%.⁴⁸ This expansion frames drug repurposing as a key driver for market growth and a potent source of competitive advantage within the pharmaceutical sector.

The rapid growth projections for the drug repurposing market indicate a clear and undeniable industry trend towards valuing efficiency and speed in drug development. This trend is driven by persistent economic pressures and an increasing global demand for affordable therapeutics. The consistent high CAGR projections across multiple sources (ranging from 4.1% to 5.42%) ²¹ signal a robust market shift. This growth is not accidental; it is explicitly “driven by the need for swift and cost-effective drug development”.⁴⁸ This means pharmaceutical companies are actively re-evaluating and re-prioritizing their R&D portfolios to integrate repurposing, recognizing it as a strategic imperative for maintaining financial health and pipeline sustainability.

This market expansion also creates unprecedented opportunities for collaboration, fostering a more dynamic and interconnected innovation ecosystem. The research highlights “partnerships between biotech startups and academic institutions” ²¹ and “collaboration between academia, pharmaceutical companies, and patient advocacy groups”.⁶ The burgeoning growth of the repurposing market incentivizes these diverse collaborations, as no single entity typically possesses all the necessary resources, data, or expertise. This implies a significant shift from traditional siloed R&D models to a more open, collaborative approach, where professionals like pharmacists, serving as crucial connectors between clinical practice and research, can play an increasingly central role.

Market Dynamics and Growth: A Lucrative Frontier

The financial incentives driving the drug repurposing market are compelling, positioning it as a lucrative frontier for pharmaceutical investment.

Investment and ROI: Why Pharma is Leaning In

The economic argument for drug repurposing is undeniably powerful, especially in an era marked by escalating R&D costs and diminishing returns from traditional pipelines. Repurposing offers a significantly lower investment compared to traditional drug discovery.¹⁰ While developing a novel drug can cost an estimated $2 billion to $3 billion, a repurposed drug averages around $300 million.⁸ This dramatic cost differential, coupled with a higher success rate, translates into a compelling return on investment (ROI).⁵⁰ Repurposed drugs have the potential to generate a substantial portion of annual pharmaceutical revenues, with estimates ranging from 25% to 40%.¹⁸ Furthermore, the average forecast internal rate of return (IRR) for top biopharma companies is showing improvement (reaching 5.9% in 2024), a positive trend driven by high-value products in late-stage pipelines, including those addressing critical unmet needs.¹⁷

The stark contrast in development costs ($300 million versus $2-3 billion) and the significantly higher approval rates (30% versus 10%) fundamentally alter the risk-reward profile of drug development. This directly translates into a more favorable ROI, making repurposing a de-risked and highly attractive investment for pharmaceutical companies. These compelling financial figures directly drive “investment in drug repositioning portfolios” ²¹ and enable companies to “exploit return on investment (ROI)”.⁵⁰ This clear causal link explains why the pharmaceutical industry is increasingly “leaning in” to drug repurposing.

Moreover, the ability of repurposed drugs to generate a substantial portion of annual revenue (25-40%) ¹⁸ positions them not merely as stop-gap solutions but as substantial, ongoing revenue streams. This has a significant influence on long-term portfolio management and mergers & acquisitions (M&A) strategies. If repurposed drugs can contribute a quarter to almost half of annual pharmaceutical revenue, they are far from marginal. This sustained revenue potential makes them valuable assets for “life-cycle management” ²¹ and “pipeline replenishment”.¹⁷ This implies that pharmaceutical companies will increasingly integrate repurposing into their core business models, potentially shifting their R&D focus from solely novel entities to a more balanced, hybrid approach that maximizes value from both new and existing assets.

Competitive Advantage: Speed, Cost, and Innovation

In the intensely competitive pharmaceutical market, speed to market and cost efficiency are paramount for securing a competitive edge. Drug repurposing delivers on both fronts, offering a distinct advantage. It significantly reduces development timelines and costs, thereby enabling faster market entry for new therapies.²¹ This efficiency allows companies to address unmet medical needs more quickly ²¹ and can provide crucial “first-mover or fast-follower” advantages in therapeutic areas that are currently underserved.¹⁷ Fundamentally, repurposing serves as a strategic mechanism to extend the value and lifespan of off-patent or low-performing assets within a company’s portfolio.²¹

The inherent efficiency of drug repurposing directly enables companies to gain a “first-mover or fast-follower” advantage, particularly in therapeutic areas with high unmet needs. This leads to increased market share and a clear competitive differentiation. If repurposing allows for “faster market entry” ⁴⁹ and “accelerated approval” ²², companies can seize market opportunities before their competitors. This is particularly true for “rare diseases and unmet medical needs” ²², where the competitive landscape might be less crowded due to the historical financial disincentives of traditional drug development. This speed-to-market is a direct competitive advantage, allowing for quicker revenue generation and stronger market positioning.

By extending the value of existing assets, repurposing transforms a potential liability—such as expiring patents—into a significant opportunity for innovation and sustained profitability. This fundamentally alters competitive dynamics within the industry. “Patent expiry pressures” ²¹ represent a major threat to pharmaceutical companies’ revenue streams. Repurposing allows them to “extend the value of existing assets via new indications” ²¹, effectively turning a challenge into a strategic opportunity. This means that companies are no longer solely competing on who can discover the

newest drug, but increasingly on who can innovate most effectively with their existing portfolio. This fosters a different kind of competitive landscape, one that rewards ingenuity in re-imagining and re-applying known therapies.

Navigating the Regulatory and Intellectual Property Landscape

While the scientific and economic advantages of drug repurposing are clear, successfully bringing a repurposed drug to market requires careful navigation of a complex regulatory and intellectual property (IP) landscape. These frameworks, designed primarily for novel drug development, present unique challenges and opportunities for repurposed therapies.

Streamlined Pathways: The 505(b)(2) and Orphan Drug Designations

Recognizing the unique value proposition of drug repurposing, regulatory agencies worldwide are increasingly introducing streamlined pathways to facilitate their approval. In the U.S., a prime example is the 505(b)(2) route, which allows developers to leverage existing safety and efficacy data from previously approved drugs. This pathway only requires supplemental clinical evidence for the new indication, significantly reducing the scope and cost of new trials.¹² Furthermore, repurposed drugs targeting rare diseases may qualify for Orphan Drug Designation. This designation offers substantial benefits, including extended market exclusivity, valuable tax incentives for clinical research expenses, and direct regulatory assistance from agencies.⁵²

Regulatory support through pathways like 505(b)(2) and Orphan Drug Designation directly reduces the regulatory burden and actively incentivizes investment in repurposing, leading to faster market access for patients. The 505(b)(2) pathway’s allowance to rely on existing data means “fewer supporting studies are required, resulting in shorter timelines and reduced costs”.¹⁸ Orphan Drug Designation, on the other hand, provides crucial “market exclusivity” and “tax incentives”.⁵² These are direct regulatory facilitators that

cause increased commercial interest and accelerate the path to market, benefiting both the pharmaceutical industry through reduced risk and patients through quicker access to needed therapies.

The very existence of these specialized pathways indicates a growing recognition by regulatory bodies of the unique value proposition of drug repurposing. This signals a more adaptable and pragmatic approach to drug approval. If regulators are actively creating specific “streamlined pathways” ²¹, it means they acknowledge that repurposing is fundamentally different from

de novo discovery and therefore requires a tailored regulatory approach. This suggests a maturing regulatory environment that is responsive to innovative drug development strategies, fostering a more favorable and efficient ecosystem for repurposing initiatives.

Intellectual Property Challenges and Strategic Solutions

Despite the clear advantages, intellectual property (IP) barriers represent a common and significant challenge in drug repurposing.⁹ It can be particularly difficult to obtain strong “composition of matter (COM)” claims for repurposed compounds, as the core molecule is already known. However, innovators can still seek IP protection through several strategic avenues, including method-of-use patents (protecting the new therapeutic indication), formulation patents (for novel delivery methods), and combination patents (for new drug combinations).⁹ A critical concern is the limited patent time remaining for compounds that may have failed in later stages of their original development, which can significantly limit the potential return on investment for repurposing efforts.⁹ The practice of “evergreening,” where minor changes are made to an existing drug to extend its patent life, is also a debated practice in this context.⁵³ To overcome the financial disincentives often associated with repurposing generic drugs, public funding and public-private partnerships are increasingly recognized as vital strategic solutions.⁵⁴

The inherent difficulty in securing strong IP for repurposed drugs, especially those that are off-patent or generic, directly disincentivizes traditional pharmaceutical companies from investing heavily. This often leads to a gap in development that academic institutions and non-profit research organizations frequently fill, or it necessitates innovative partnership models. “Poor protection of intellectual property” ⁵⁵ and the challenge of gaining “Composition of matter (COM) claims” ⁹ mean that the “chances of recovering the Research and Development (R&D) costs by the marketing of the drug” are significantly reduced.⁵⁵ This directly

causes commercial hesitation, leading to “nontraditional developers” ¹² stepping in, or requiring “revenue sharing” models ⁵⁶ to make projects viable. This highlights a market inefficiency within the IP framework that demands creative solutions and collaborative approaches.

The strategic focus on “method-of-use patents” ⁵² for repurposed drugs fundamentally shifts the IP strategy. Instead of protecting the molecule itself, the innovation lies in its novel application. This means IP strategy for repurposing moves from the realm of chemistry to the domain of clinical application and therapeutic strategy. This shift elevates the importance of clinical observation, real-world data, and the specialized expertise of professionals like pharmacists who possess an intimate understanding of

how drugs are used in patients and what their effects are beyond the original label.

Leveraging DrugPatentWatch for Patent Landscape Analysis

Navigating the intricate IP landscape is paramount for strategic decision-making in drug repurposing, allowing companies to identify lucrative opportunities and mitigate potential risks. This is where specialized tools become indispensable. DrugPatentWatch, for instance, provides deep knowledge on pharmaceutical drugs, encompassing patents, suppliers, generics, and formulations.⁵⁷ It offers a fully integrated database of drug patents and other critical information, enabling freeform searching and dynamic browsing of both U.S. and international patent data.⁵⁷ This powerful platform assists companies in identifying market entry opportunities, accurately predicting patent expiration dates, and thoroughly assessing competitor research paths.⁵⁷ Crucially, it can also be leveraged to discover future therapeutic indications through sophisticated biopharmaceutical forecasting.⁵⁷

Tools like DrugPatentWatch directly enable pharmaceutical companies to make informed strategic decisions by providing comprehensive, real-time patent intelligence. This directly mitigates IP risks and facilitates the identification of lucrative repurposing opportunities. “Navigating the complex web of intellectual property rights and regulatory considerations… can be challenging”.⁵² DrugPatentWatch “allows pharmaceutical companies and organizations to make better business decisions by having access to a database that covers business intelligence on biologic and small molecule drugs, prices, patents, litigation, and drugs currently in development”.⁵⁷ This direct access to granular patent data

reduces uncertainty and enables proactive strategic planning, which is absolutely crucial for successful market entry and competitive advantage in the repurposing space.

The utility of such platforms underscores the increasing reliance on data analytics and competitive intelligence within the pharmaceutical industry. This transforms IP management from a purely legal function into a strategic business driver for repurposing initiatives. DrugPatentWatch is not merely a tool for legal teams; it is designed for “global business intelligence and forecasting”.⁵⁷ This indicates that IP is no longer a reactive legal hurdle but a proactive strategic asset that can be actively managed for competitive gain. For repurposing, understanding “patent expiry pressures” ²¹ and identifying “future therapeutic indications” ⁵⁷ through rigorous data analysis becomes a core competitive strategy. This highlights the growing need for data-savvy professionals within pharmaceutical companies who can effectively leverage such intelligence.

Feature	Traditional Drug Discovery	Drug Repurposing	Relevant Snippet IDs
Stages	5 stages: Discovery & preclinical, Safety review, Clinical research, FDA review, FDA post-market safety monitoring ¹⁰	4 stages: Compound identification, Compound acquisition, Development, FDA post-market safety monitoring ¹⁰	¹⁰
Timeframe	Generally more time-consuming, 10-15 years on average ¹⁰	Less time-consuming, 3-12 years on average, often 6 years ⁸	⁸
Cost	High investment or cost, $2-3 billion to bring a novel drug to market ¹⁰	Lesser investment, $300 million on average ⁸	⁸
Risk of Failure	More risk of failure, ~90% failure rate (only ~10% gain market approval) ⁹	Less risk of failure, ~30% approval rate ⁹	⁹
Safety Profile	Clinical efficacy and safety profile should be evaluated ¹⁰	Clinical efficacy and safety profiles already exist ¹⁰	¹⁰
Clinical Efficacy	Must be established from scratch	Built upon existing data, focus on efficacy for new indication ¹⁴	¹⁴

The Digital Revolution: AI, Big Data, and the Future Pharmacist

The digital revolution is profoundly reshaping the landscape of drug discovery and development, particularly in the realm of repurposing. Advanced technologies such as data science, computational research, artificial intelligence (AI), machine learning (ML), and sophisticated data mining techniques have significantly expanded the scope and efficiency of drug repurposing efforts.² AI, in particular, possesses the remarkable ability to accelerate drug discovery by rapidly analyzing vast datasets, identifying optimal compounds, and accurately predicting their likely outcomes.⁶² This transformative impact of technology is not merely providing new tools; it is fundamentally altering how drug repurposing is approached and executed.

The widespread adoption of AI and big data in drug repurposing signifies a fundamental shift from purely experimental or serendipitous discovery to a more systematic, predictive, and data-driven approach. The consistent mention of “complex computational algorithms” ³, “AI-driven real-time monitoring” ³⁵, and various “machine learning algorithms” ⁴ across the research indicates that this is more than just about using computers; it is about fundamentally changing the

methodology of drug discovery. This transition moves the industry from a laborious needle-in-a-haystack approach to a more targeted, intelligent search, which directly increases efficiency and success rates in identifying viable repurposing candidates.

This profound technological shift carries a significant implication for the pharmacy profession: it necessitates that pharmacists, traditionally focused on direct clinical practice, also cultivate proficiency in data literacy and digital tools. This evolution is crucial for them to remain central and impactful in the future of drug development and repurposing. If AI “analyzes vast datasets from biological, chemical, and clinical sources” ⁶⁴ and “streamlines processes for clinical pharmacists” ⁶², then pharmacists must understand how to effectively interact with and leverage these sophisticated systems. Their role will increasingly involve critically interpreting AI-generated insights and contributing high-quality, clinically relevant data back into these systems. This highlights a clear need for curriculum adjustments in pharmacy education and ongoing professional development initiatives focused on digital health competencies.

Leveraging Big Data: Unlocking Insights from Electronic Health Records

Vast datasets represent a veritable goldmine of real-world patient information, offering unparalleled insights into drug effects that traditional, controlled clinical trials often cannot capture. These “big health datasets,” including electronic health records (EHRs), insurance claims data, and patient registries, are increasingly recognized as vital sources for drug repurposing.³ Real-world data (RWD) derived from EHRs can guide clinical research at a significantly lower cost, enabling the inclusion of broader patient populations, including those with complex comorbidities and co-medications, thereby providing a more accurate representation of real-life drug effects.³¹ Pharmacists are uniquely positioned to leverage these EHRs to identify medication discrepancies, meticulously manage patient data, and contribute directly to the generation of robust real-world evidence.²⁷

Pharmacists’ direct access to and daily interaction with EHRs position them as critical “human-in-the-loop” components for validating and refining AI-generated repurposing hypotheses derived from big data. While AI systems can efficiently “sift through millions of records” ¹⁶, the raw data in EHRs can often contain “many types of bias” ⁵ or suffer from “poor data quality”.³² Pharmacists, by actively “identifying medication discrepancies” ²⁷ and possessing “access to the patient’s electronic health record” ⁶⁵, can provide crucial clinical context, identify potential data anomalies, and offer invaluable validation for computational findings. This human oversight is absolutely essential to prevent false positives that might arise from data mining and to ensure the clinical relevance and applicability of AI predictions.

The immense potential of RWD for drug repurposing underscores a pressing need for enhanced interoperability and standardized data collection across diverse healthcare systems. This, in turn, makes pharmacists’ advocacy for better digital health tools even more critical. “Data are often siloed and stored in multiple formats” ², and critically, “most clinical management systems don’t integrate well with EHRs”.³⁶ If RWD is to truly “accelerate drug development process” ³¹, these significant barriers must be systematically addressed and overcome. Pharmacists, who experience these data silos and integration challenges on a daily basis, are uniquely positioned to advocate for and help design integrated digital health platforms that facilitate seamless data flow, thereby maximizing the utility of RWD for repurposing research.

Artificial Intelligence and Machine Learning: The Pharmacist’s New Toolkit

Artificial intelligence (AI) and machine learning (ML) are not just buzzwords in drug discovery; they are fundamentally transforming the field, and pharmacists are gaining a powerful new toolkit to harness these technologies.

AI-Driven Candidate Identification and Predictive Analytics

AI and ML are rapidly advancing drug discovery by extracting hidden patterns and previously unidentifiable evidence from vast biomedical datasets.⁴ These sophisticated technologies can analyze complex biological datasets to uncover potential drug-disease interactions that would be impossible for human analysis alone.³ AI models are now capable of identifying optimal compounds, accurately predicting their efficacy and safety profiles, and even repurposing drugs that may have failed in their original clinical trials due to reasons other than safety.⁶² Deep learning (DL), a subset of AI, is particularly adept at identifying intricate patterns among chemical structures, thereby predicting the potential success of a new compound.⁶² Beyond molecular interactions, AI can also predict complex biological phenomena like protein folding and optimize drug candidates for specific targets.⁶⁶

AI’s remarkable ability to process “vast datasets” ⁶⁴ and identify “hidden patterns” ¹⁶ directly accelerates the drug repurposing process by rapidly generating high-confidence candidate hypotheses. This significantly reduces the laborious “trial-and-error” ⁶⁶ phase that has historically plagued drug discovery. Traditional methods are notoriously slow, expensive, and often yield low success rates. AI, by “analyzing data sets, identifying optimal compounds, and predicting their likely” success ⁶², drastically shortens the “compound identification” stage.¹⁰ This speed is a direct result of AI’s computational power, allowing researchers to focus their resources on the most promising candidates, thereby accelerating the entire repurposing pipeline and bringing therapies to patients faster.

The integration of AI in drug repurposing also fundamentally shifts the pharmacist’s role from reactive problem-solving to proactive, predictive intervention. This evolution requires pharmacists to develop new competencies in understanding and interpreting AI-generated insights for direct patient care. If AI can “predict potential patterns among chemical structures” ⁶² or “predict how proteins will fold and optimize drug candidates” ⁶⁶, pharmacists will increasingly receive AI-driven recommendations and alerts. Their new challenge will be to critically evaluate these predictions, integrate them seamlessly with patient-specific clinical data, and translate them into actionable, personalized treatment plans. This elevates their cognitive role within the healthcare team and demands a new, specialized skill set in what is increasingly termed “pharmacointelligence”.⁶²

Digital Health Tools: Enhancing Pharmacist Capabilities

Beyond the complex algorithms of AI, everyday digital health tools and platforms are profoundly empowering pharmacists in their daily practice, indirectly but significantly supporting drug repurposing efforts. These tools, which include wearables like continuous glucose monitors (CGMs) and smart inhalers, as well as digital therapeutics such as therapy apps and AI-powered chatbots, are transforming healthcare delivery.⁶⁷ Pharmacists can effectively leverage these tools for remote medication evaluations, virtual patient consultations, and meticulous tracking of medication adherence.⁶⁷ Digital platforms are increasingly sophisticated, capable of tracking patient adherence, monitoring side effects in real-time, and providing patient-specific data on potential drug interactions.⁶⁷ Furthermore, AI-powered writing assistants are emerging to reduce administrative burdens, thereby freeing up valuable pharmacist time for more complex clinical management and direct patient care.⁶⁷

Digital health tools, by streamlining routine pharmacy tasks and enhancing data collection capabilities, directly free up pharmacists’ time and provide richer, more real-time patient data. This enables pharmacists to focus more intently on complex clinical reasoning, which is highly relevant to drug repurposing. “Pharmacy automation technology, when used to streamline pharmacy operations, such as medication dispensing, inventory management, and data analysis, can create an opportunity for pharmacists to provide this critical guidance”.⁶⁶ If administrative burdens are significantly reduced by tools like AI writing assistants ⁶⁷, pharmacists gain more capacity for “clinical management and direct patient care”.⁶⁷ This directly

causes an increase in their ability to observe, analyze, and contribute nuanced clinical insights that could lead to novel repurposing opportunities.

The integration of digital health tools positions pharmacists as key facilitators in the broader shift towards personalized medicine and data-driven healthcare. This makes their insights not only more granular but also more valuable for precision drug repurposing initiatives. “Pharmacogenomic tools, which use genetic data to personalize medication therapy, are also gaining traction”.⁶⁷ Digital platforms that provide “patient-specific data on potential drug interactions” ⁶⁷ enable more tailored and precise interventions. This move towards highly personalized care, significantly enabled by digital tools, means pharmacists are increasingly working with granular patient data. This detailed data is ideal for identifying specific patient subgroups that might benefit most from repurposed drugs, driving the field towards “precision drug repurposing”.⁵

Conclusion: The Indispensable Role of Pharmacists in a Repurposed Future

Drug repurposing has unequivocally emerged as a vital, efficient, and cost-effective strategy for pharmaceutical innovation. It offers a powerful pathway to address critical unmet medical needs, from rare diseases to global health crises, while simultaneously presenting significant market advantages for the pharmaceutical industry. This transformative approach promises not just new therapies, but a more sustainable and agile model for drug development.

Pharmacists, with their deep pharmacotherapy knowledge, patient-centric approach, and increasing engagement with data and digital tools, are uniquely positioned to contribute across the entire repurposing lifecycle. Their expertise extends from the foundational understanding of drug mechanisms to the nuanced complexities of real-world patient care.

The confluence of the growing strategic importance of drug repurposing and the evolving, data-empowered role of pharmacists creates a synergistic relationship that is critical for future pharmaceutical innovation and sustained market success. The report has established that repurposing is financially attractive and addresses critical needs. It has also demonstrated how pharmacists’ core competencies and their adoption of new technologies align perfectly with the demands of repurposing. This relationship is not merely parallel; it is profoundly interdependent. Optimal repurposing requires the unique contributions of pharmacists, and conversely, pharmacists’ future relevance and impact in drug development are significantly enhanced by their integration into repurposing initiatives. This powerful synergy represents a compelling strategic advantage for business professionals to recognize and act upon.

For pharmaceutical companies and healthcare systems to truly capitalize on this potential, active recognition and integration of pharmacists into drug repurposing strategies are essential. This means moving beyond traditional perceptions of the pharmacist’s role. Proactively investing in and integrating pharmacists into repurposing initiatives is not merely a “nice-to-have”; it is a strategic investment that can yield tangible returns through accelerated development, reduced costs, and enhanced market penetration. If pharmacists can indeed “accelerate results, and improve patient outcomes” ¹⁶, then their strategic integration offers a direct pathway to business success. This implies that companies should consider creating new roles or departments specifically focused on leveraging pharmacists’ unique insights for repurposing.

However, the full potential of pharmacists in drug repurposing can only be realized through systemic changes. This includes robust policy support, improved data infrastructure, and ongoing professional development. While individual pharmacists possess immense capabilities, their collective impact is amplified by supportive systems. For instance, “regulatory bodies should make legislations to encourage pharmacists to be actively involved” in pharmacovigilance systems.³⁴ Furthermore, the current reality where “most pharmacists want to have access to this type of data, but current pharmacy management systems don’t support it” ³⁶ highlights a critical barrier. Addressing these systemic challenges—by building the necessary infrastructure and enacting empowering policies—is paramount. Only then can the individual expertise of pharmacists be fully harnessed and transformed into collective market domination and, most importantly, enhanced patient benefit.

Key Takeaways

Drug repurposing offers a compelling alternative to traditional drug discovery, being significantly faster, cheaper, and less risky, with a success rate of approximately 30% compared to about 10% for novel drugs.
Pharmacists, with their deep pharmacotherapy knowledge, extensive patient interaction skills, and active involvement in medication management and pharmacovigilance, are uniquely positioned to identify and contribute valuable insights to drug repurposing opportunities.
Their critical role spans the entire repurposing lifecycle, from spotting early signals through serendipitous observations and proactive identification of off-label uses, to contributing to rigorous clinical trial design and patient monitoring, and finally, generating crucial real-world evidence post-market.
Advanced technologies, including Artificial Intelligence (AI), machine learning, and big data analytics, are revolutionizing drug repurposing by enabling systematic candidate identification and predictive analytics. Pharmacists’ growing proficiency in leveraging these digital tools is becoming indispensable for optimizing outcomes.
Navigating the complex intellectual property and regulatory landscape for repurposed drugs requires strategic planning. Specialized pathways like the 505(b)(2) route and Orphan Drug Designations streamline approvals, while tools such as DrugPatentWatch are invaluable for comprehensive patent landscape analysis and competitive intelligence.
For pharmaceutical businesses, strategically integrating pharmacists into drug repurposing initiatives provides a clear competitive advantage. This integration enables faster market entry, substantial reductions in R&D costs, and a more efficient approach to addressing critical unmet medical needs.
The future of pharmaceutical innovation will increasingly rely on collaborative, data-driven approaches where pharmacists play an indispensable, proactive role, transforming clinical data and insights into both market domination and profound patient benefit.

Frequently Asked Questions (FAQs)

Q1: How does drug repurposing significantly reduce development costs and timelines?

Drug repurposing drastically cuts down on costs and timelines because it leverages existing drugs that already possess established safety profiles. This crucial advantage means developers can often bypass the lengthy and resource-intensive preclinical testing and Phase I safety trials, moving directly to Phase II efficacy studies for the new indication. For example, a new drug might cost billions of dollars and take 10-15 years to bring to market, whereas a repurposed drug typically averages around $300 million in cost and can reach the market in approximately 6 years. This inherent efficiency fundamentally transforms the economic landscape of pharmaceutical development.

Q2: What specific insights can pharmacists offer in identifying drug repurposing candidates?

Pharmacists operate on the front lines of patient care, providing them with a unique and invaluable vantage point. Their deep knowledge of drug mechanisms, potential adverse effects, and observed patient responses allows them to spot “off-label” uses or unexpected beneficial effects of medications in real-world clinical settings. They are adept at identifying subtle signals from routine patient observations, comprehensive medication reviews, and pharmacovigilance data that might indicate a drug’s potential for an entirely new indication. This makes them crucial “detectives” in the drug repurposing process, capable of identifying opportunities that might otherwise go unnoticed.

Q3: How do AI and big data empower pharmacists in drug repurposing efforts?

Artificial Intelligence (AI) and big data tools are revolutionizing how pharmacists contribute to drug repurposing by enabling them to move beyond manual observation to systematic, data-driven analysis. AI algorithms can efficiently sift through and analyze vast datasets, such as electronic health records (EHRs), to identify hidden patterns and predict potential drug-disease interactions that would be impossible for human analysis alone. For pharmacists, this translates into more accurate candidate identification, optimized dosage predictions, and streamlined workflows. This empowers them to make more precise, data-driven clinical decisions and significantly contribute to the generation of robust real-world evidence for repurposed therapies.

Q4: What are the main intellectual property challenges in drug repurposing, and how can they be addressed?

A primary intellectual property (IP) challenge in drug repurposing is securing robust patent protection, as the original chemical compound may no longer be eligible for patenting. To address this, companies often pursue “method-of-use” patents, which protect the new therapeutic indication, or explore formulation patents for novel delivery methods, or combination patents for new drug combinations. The limited remaining patent life for older drugs can also impact the potential return on investment. Strategic solutions include leveraging specialized regulatory pathways like the 505(b)(2) route and Orphan Drug Designation, forming collaborative public-private partnerships, and utilizing advanced tools such as DrugPatentWatch to navigate the complex patent landscape and identify strategic opportunities.

Q5: Why is integrating pharmacists into a pharmaceutical company’s drug repurposing strategy a “strategic imperative” for market domination?

Integrating pharmacists into a pharmaceutical company’s drug repurposing strategy is a strategic imperative because they serve as a vital bridge between scientific discovery and real-world patient outcomes. Their unique clinical insights, their ability to generate valuable real-world evidence, and their growing proficiency with digital health tools directly contribute to faster, more cost-effective drug development. By leveraging pharmacists’ comprehensive expertise, companies can effectively de-risk investments, significantly accelerate market entry for repurposed therapies, address critical unmet medical needs more efficiently, and ultimately gain a substantial competitive advantage in the rapidly evolving pharmaceutical landscape.

References

What is drug repurposing? (n.d.). Elsevier. Retrieved from
https://www.elsevier.com/industry/drug-repurposing
Introduction. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9336118/
Abstract. (n.d.). DrugRepoCentral. Retrieved from
https://drugrepocentral.scienceopen.com/hosted-document?doi=10.58647/DRUGREPO.24.1.0013
Drug Repurposing: An Effective Tool in Modern Drug Discovery. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9945820/
Drug Repurposing Basics. (n.d.). IQVIA. Retrieved from
https://www.iqvia.com/blogs/2022/05/drug-repurposing-basics
TRADITIONAL DRUG DISCOVERY DRUG REPURPOSING. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9945820/
Table 1. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9945820/
This Core Competency Framework reflects the practice of pharmacists in the early stages of their career, i.e., 0-3 years registered. (n.d.). PSI. Retrieved from
https://www.psi.ie/sites/default/files/2024-06/PSI_Core_Competency_Framework.pdf
Here are some of the areas where pharmacists such as Gates and Clark assist older people with medication management. (n.d.). WSU. Retrieved from
https://pharmacy.wsu.edu/2024/11/25/how-do-pharmacists-help-people-manage-medications/
²⁸ Clinical pharmacists work directly with physicians, other health professionals, and patients to ensure that the medications prescribed for patients contribute to the best possible health outcomes. (n.d.). ACCP. Retrieved from
https://www.accp.com/about/clinicalpharmacists.aspx
³³ When the Food and Drug Administration (FDA) approves a drug for sale in the United States, the approval includes a section entitled “Indications for Use.” (n.d.). Congress.gov. Retrieved from
https://www.congress.gov/crs-product/R45792
⁴ Drug repurposing, a pivotal strategy in pharmaceutical development, involves the identification of new therapeutic applications for existing drugs or the advancement of previously studied but unapproved drugs for indications beyond their original scope. (n.d.). DrugPatentWatch. Retrieved from
https://www.drugpatentwatch.com/blog/drug-repurposing-an-overview/
⁵ As discussed in an editorial by Talevi and Bellera,1 many of the most well-known drugs that have been repurposed over the years, such as sildenafil, aspirin, and valproic acid, were identified through serendipitous observation of their efficacy for conditions other than what they were approved for. (n.d.). HMP Global Learning Network. Retrieved from
https://www.hmpgloballearningnetwork.com/site/frmc/articles/drug-repurposing-expediting-discovery-through-novel-algorithmic-framework
³⁸ Pharmacists Can Play Key Role in Drug Repurposing – YouTube. (n.d.). YouTube. Retrieved from
https://m.youtube.com/watch?v=fvCTVc9srj4
³⁹ Behind The Scenes, Pharmacists Play Key Role In Clinical Research. (n.d.). Mayo Clinic College of Medicine and Science. Retrieved from
https://college.mayo.edu/about/news/news-archive/behind-the-scenes-pharmacists-play-key-role-in-clinical-research/
³⁴ Abstract. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC5065524/
³⁵ What is Pharmacovigilance? (n.d.). CCRPS. Retrieved from
https://ccrps.org/clinical-research-blog/what-is-the-role-of-pharmacovigilance-in-clinical-research
²² Drug repurposing, also known as drug repositioning or reprofiling, refers to the process of identifying new therapeutic uses for existing, ideally registered, drugs that were initially developed for a different indication and even a different target protein. (n.d.). EAHP. Retrieved from
https://eahp.eu/congress_goal/er2-the-second-life-of-drugs-opportunities-and-challenges-of-drug-repurposing/
¹¹ Drug repurposing, also referred to as drug repositioning or reprofiling, is an approach that identifies new clinical applications for approved or investigational drugs outside of the compound’s originally intended uses. (n.d.). Technology Networks. Retrieved from
https://www.technologynetworks.com/drug-discovery/articles/drug-repurposing-strategies-challenges-and-successes-384263
⁶⁵ Impact of Pharmacist Interventions on Patient Outcomes. (n.d.). CPS. Retrieved from
https://perspectives.cps.com/cps-clinical-outcomes-program-impact-of-pharmacist-interventions-on-patient-outcomes
¹⁴ Drug repurposing (or repositioning) offers an alternative path by which drugs can be brought to market — in significantly less time and at significantly lower cost and risk. (n.d.). Xtalks. Retrieved from
https://clinicaledge.xtalks.com/magazine/issue1/drug-repurposing
⁶ The drug repurposing also known as drug reprofiling, involves identifying new therapeutic uses for existing or investigational drug beyond their original therapeutic use. (n.d.). PharmaBiz. Retrieved from
https://pharmabiz.com/ArticleDetails.aspx?aid=174110&sid=21
³¹ Abstract. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC8492393/
³² Real-world evidence (RWE) is clinical evidence on a medical product’s safety and efficacy that is generated using real-world data (RWD) resulting from routine healthcare delivery. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9815890/
⁶² AI technology for the pharmacy field, otherwise known as pharmacointelligence, can help streamline processes for clinical pharmacists, including making more accurate and evidence-based clinical decisions through analyzing a large amount of patient data, medical records, laboratories, and medication profiles. (n.d.). US Pharmacist. Retrieved from
https://www.uspharmacist.com/article/artificial-intelligence-and-the-future-of-specialty-pharmacy
⁷ Examples of repositioned drugs. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC7262062/
⁴ Introduction. (n.d.). DrugPatentWatch. Retrieved from
https://www.drugpatentwatch.com/blog/drug-repurposing-an-overview/
⁸ Computational Drug Repurposing: Approaches and Case Studies. (n.d.). DrugPatentWatch. Retrieved from
https://www.drugpatentwatch.com/blog/computational-drug-repurposing-approaches-and-case-studies/
⁴⁸ Drug Repurposing Market Revenue To Attain USD 56.25 Bn By 2033. (n.d.). Precedence Research. Retrieved from
https://www.precedenceresearch.com/press-release/drug-repurposing-market
⁴⁹ Drug Repurposing Market Size. (n.d.). Straits Research. Retrieved from
https://straitsresearch.com/report/drug-repurposing-market
¹² Regulatory Actions to Advance Generic Drug Repurposing. (n.d.). Duke Health Policy. Retrieved from
https://healthpolicy.duke.edu/sites/default/files/2025-05/Working%20Paper-Regulatory%20Actions%20to%20Advance%20Generic%20Drug%20Repurposing.pdf
⁹ Introduction. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9336118/
⁵² Intellectual Property Rights and Regulatory Considerations for Drug Repurposing. (n.d.). DrugPatentWatch. Retrieved from
https://www.drugpatentwatch.com/blog/intellectual-property-rights-and-regulatory-considerations-for-drug-repurposing/
⁵⁷ DrugPatentWatch provides deep knowledge on pharmaceutical drugs – patents, suppliers, generics, formulation and more. (n.d.). Crozdesk. Retrieved from
https://crozdesk.com/software/drugpatentwatch
³⁰ Pharmacists play a crucial role in drug repurposing due to the vast amount of opportunities that exist in this innovative approach. (n.d.). DrugPatentWatch. Retrieved from
https://www.drugpatentwatch.com/blog/pharmacists-can-play-key-role-in-drug-repurposing/
⁵⁵ Abstract. (n.d.). Der Pharma Chemica. Retrieved from
https://www.derpharmachemica.com/pharma-chemica/drug-repurposing-an-opportunity-in-drug-research-84511.html
⁴¹ The Academy of Managed Care Pharmacy (AMCP) supports off‐label use of U.S Food and Drug Administration (FDA)‐approved pharmaceuticals and related products when medically appropriate, safe, and necessary. (n.d.). AMCP. Retrieved from
https://www.amcp.org/legislative-regulatory-position/label-use-pharmaceuticals-and-related-products
⁴³ In the intricate world of pharmaceuticals, medications often reveal a fascinating duality. (n.d.). DrugPatentWatch. Retrieved from
https://www.drugpatentwatch.com/blog/common-drugs-with-effective-off-label-uses/
⁴⁷ In addition, she leverages causal and counterfactual AI predictions to identify heterogeneity in treatment effects and signals of drug repurposing. (n.d.). University of Florida. Retrieved from
https://pharmacy.ufl.edu/research/artificial-intelligence-empowering-the-future-of-pharmacy/
⁴⁵ Drug repurposing is the process of identifying a new use for an existing medicine or active substance outside the scope of the original indication(s). (n.d.). DrugRepoCentral. Retrieved from
https://drugrepocentral.scienceopen.com/hosted-document?doi=10.58647/DRUGREPO.24.2.0016
²⁵ The Role of Pharmacists in Clinical Research. (n.d.). Headlands Research. Retrieved from
https://headlandsresearch.com/blogs/the-role-of-pharmacists-in-clinical-research/
¹¹ For example, thalidomide, which was notoriously removed from the market in 1961 after it was found to induce severe congenital disorders, was later repurposed as a treatment for leprosy after clinical case studies suggested potential effectiveness. (n.d.). Technology Networks. Retrieved from
https://www.technologynetworks.com/drug-discovery/articles/drug-repurposing-strategies-challenges-and-successes-384263
² Examples of drug repurposing. (n.d.). Elsevier. Retrieved from
https://www.elsevier.com/industry/drug-repurposing
⁶³ Abstract. (n.d.). MDPI. Retrieved from
https://www.mdpi.com/2078-2489/16/2/131
⁶⁴ Recent advancements in artificial intelligence (AI) have significantly enhanced the efficiency and accuracy of drug repurposing efforts. (n.d.). ResearchGate. Retrieved from
https://www.researchgate.net/publication/382878316_Artificial_Intelligence_in_Drug_Repurposing
⁶⁹ Repositioning refers to finding a different application for a drug that was originally developed and approved for a different indication. (n.d.). MDPI. Retrieved from
https://www.mdpi.com/1422-0067/24/13/11000
⁴⁸ Drug Repurposing Market Revenue and Trends 2025 to 2033. (n.d.). Precedence Research. Retrieved from
https://www.precedenceresearch.com/press-release/drug-repurposing-market
⁵⁰ The Drug Repurposing Market was valued at USD 35.33 Bn in 2024 is expected to grow at a CAGR of 4.9% from 2025 to 2032 and reach USD 51.80 Bn by the end of 2032. (n.d.). Maximize Market Research. Retrieved from
https://www.maximizemarketresearch.com/market-report/drug-repurposing-market/273130/
⁵¹ Drug Repurposing Market Size to Hit USD 59.30 Billion by 2034. (n.d.). Precedence Research. Retrieved from
https://www.precedenceresearch.com/drug-repurposing-market
¹⁷ After more than a decade of declining returns on pharmaceutical research and development (R&D), the tide is turning. (n.d.). Deloitte. Retrieved from
https://www.deloitte.com/us/en/Industries/life-sciences-health-care/articles/measuring-return-from-pharmaceutical-innovation.html
²³ The global drug repurposing market is emerging as a transformative approach in the pharmaceutical industry, offering faster, cost-effective solutions for unmet medical needs. (n.d.). EIN Presswire. Retrieved from
https://www.einpresswire.com/article/780644699/drug-repurposing-market-forecasted-to-hit-us-51-8-billion-by-2033-biovista-excelra-fios-genomics-novartis-ag
¹⁸ Additionally, the market generates around 25% of the annual revenue for the pharmaceutical industry. (n.d.). Pharma Int. Retrieved from
https://www.pharm-int.com/resources/advantages-of-drug-repurposing/
⁵⁸ DrugPatentWatch provides a fully integrated database of drug patents and other critical information. (n.d.). Crozdesk. Retrieved from
https://crozdesk.com/software/drugpatentwatch/review
¹⁹ Drug repurposing is an innovate and creative method to find new indications for medications that allows one to circumvent this productivity gap. (n.d.). Pharmacy Times. Retrieved from
https://www.pharmacytimes.com/view/pharmacists-can-play-key-role-in-drug-repurposing
²⁹ These specialized pharmacists are focused on achieving optimal use of medications, emphasizing dosing, monitoring, identification of adverse effects, and economic efficiency to achieve optimal patient outcomes. (n.d.). Elsevier. Retrieved from
https://www.elsevier.es/es-revista-revista-medica-clinica-las-condes-202-articulo-clinical-pharmacists-practitioners-who-are-S0716864016300827
⁹ Introduction. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9336118/
³³ Appendixes. (n.d.). Congress.gov. Retrieved from
https://www.congress.gov/crs-product/R45792
³¹ Abstract. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC8492393/
⁴⁴ What is the Role of Pharmacists in Clinical Trials? (n.d.). Rx Relief. Retrieved from
https://www.rxrelief.com/blog/what-is-the-role-of-pharmacists-in-clinical-trials/
³⁵ Introduction: The Wild World of Drug Safety. (n.d.). CCRPS. Retrieved from
https://ccrps.org/clinical-research-blog/what-is-the-role-of-pharmacovigilance-in-clinical-research
⁶⁶ This advancement is exciting for patients and providers alike, but it will have significant implications for pharmacists, who will need to adapt to new, more complex treatments and stay current with evolving therapies. (n.d.). Swisslog Healthcare. Retrieved from
https://www.swisslog-healthcare.com/en-us/company/new-blog-overview-page/ai-drug-discovery-and-what-it-means-for-pharmacists
⁶⁷ By Jessica Steinhoff. (n.d.). University of Wisconsin-Madison. Retrieved from
https://pharmacy.wisc.edu/2025/03/25/pharmacy-in-the-digital-age/
¹⁰ Table 4. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9945820/
¹¹ Decades later, it was predicted to have anti-angiogenic and tumor-suppressing properties, which led to the success of thalidomide to treat multiple myeloma patients. (n.d.). Technology Networks. Retrieved from
https://www.technologynetworks.com/drug-discovery/articles/drug-repurposing-strategies-challenges-and-successes-384263
¹³ Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, reaching out to regulatory scientific advice at an early stage, and public-private collaboration. (n.d.). Frontiers in Medicine. Retrieved from
https://www.frontiersin.org/journals/medicine/articles/10.3389/fmed.2024.1352803/full
⁴⁸ Drug Repurposing Market Revenue to Attain USD 56.25 Bn by 2033. (n.d.). Precedence Research. Retrieved from
https://www.precedenceresearch.com/press-release/drug-repurposing-market
¹⁷ After more than a decade of declining returns on pharmaceutical research and development (R&D), the tide is turning. (n.d.). Deloitte. Retrieved from
https://www.deloitte.com/us/en/Industries/life-sciences-health-care/articles/measuring-return-from-pharmaceutical-innovation.html
²⁰ Abstract. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC11920972/
⁵⁹ A recent guide from DrugPatentWatch analyzes strategies for cost optimization in generic drug production. (n.d.). GeneOnline. Retrieved from
https://www.geneonline.com/drugpatentwatch-guide-patent-landscape-analysis-key-to-generic-drug-cost-optimization/
⁵³ Let’s dive into the world of drug repurposing and unravel the patent implications that come with it. (n.d.). PatentPC. Retrieved from
https://patentpc.com/blog/patent-implications-of-drug-repurposing
⁵⁶ Executive Summary. (n.d.). Rising Tide Foundation. Retrieved from
https://www.risingtide-foundation.org/wp-content/uploads/2024/02/On-patent-Drug-Repurposing-White-Paper-02-2024.pdf
⁵⁴ Furthermore, regardless of the success of a repurposing trial, commercial parties often lack interest in pursuing marketing authorisation for financial reasons, and academic researchers lack the knowledge, time and funding. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC10627937/
⁴⁰ “Understanding the unique pathogenesis and clinical characteristics of HS as well as the pathogenic overlap between HS and other inflammatory skin diseases may reduce delays in HS diagnosis, improve patient outcomes, and create opportunities to repurpose disease management strategies.” (n.d.). Drug Topics. Retrieved from
https://www.drugtopics.com/view/improved-understanding-of-hidradenitis-suppurativa-can-enhance-patient-outcomes
⁹ Introduction. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC9336118/
⁴⁶ 5 Ways Pharmacists Can Improve Clinical Trials. (n.d.). Rx Relief. Retrieved from
https://www.drugpatentwatch.com/blog/5-ways-pharmacists-can-improve-clinical-trials/
⁶² AI technology for the pharmacy field, otherwise known as pharmacointelligence, can help streamline processes for clinical pharmacists, including making more accurate and evidence-based clinical decisions through analyzing a large amount of patient data, medical records, laboratories, and medication profiles. (n.d.). US Pharmacist. Retrieved from
https://www.uspharmacist.com/article/artificial-intelligence-and-the-future-of-specialty-pharmacy
³⁶ Most have witnessed what digital health does for emergency rooms, doctors’ offices, and even home care patients. (n.d.). My Veta Health. Retrieved from
https://www.myvetahealth.com/veta-health-blog/the-importance-of-engaging-pharmacists-in-the-digital-health-space-1
³⁷ In recent decades, the development of a computerized workflow has allowed pharmacists to automate the process of identifying drug interactions, readily access online therapeutic resources and digitize the storage of patient information. (n.d.). PMC. Retrieved from
https://pmc.ncbi.nlm.nih.gov/articles/PMC8756370/
²¹ Drug repurposing – also known as drug repositioning – involves identifying new therapeutic uses for existing or previously shelved drugs. (n.d.). Business Wire. Retrieved from
https://www.businesswire.com/news/home/20250528921205/en/Drug-Repurposing-Strategic-Research-Business-Report-2024-2025-2030—Patent-Expiry-Pressures-Encourage-Pharma-Companies-to-Extend-Value-of-Existing-Assets-via-New-Indications—ResearchAndMarkets.com
⁵⁸ DrugPatentWatch provides a fully integrated database of drug patents and other critical information. (n.n.). Crozdesk. Retrieved from
https://crozdesk.com/software/drugpatentwatch/review