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Whether the "blockbuster drug" era is over, as some claim, or not, coming up with brand new blockbuster drugs is difficult. Bringing one of these high-earning drugs to market can take well over a decade and billions of dollars.
It is faster and less costly to find new uses for existing drugs, and this type of repurposing is gaining traction. After all, existing drugs have already been shown to be safe, so if they happen to work for other diseases, drug development costs and risks both decrease. Furthermore, new technologies are empowering researchers to systematically evaluate existing drugs against other diseases.
Between the years 2014 and 2020, more than one-quarter trillion dollars of pharmaceutical sales worldwide are at risk due to patent expiration. And in many cases, loss of patent exclusivity signals the end of a product's revenue stream. For small molecule drugs, brand unit share falls by an average of 16 percent within the first year of a generic being on the market.
Therefore, it behooves pharma companies to plan and manage loss of exclusivity, because there are ways of preserving meaningful value for the brand name product. One way of doing this has been drug repurposing - developing new, protectable uses for brand name drugs.
In the repurposing process, medicines that have already been through clinical trials get to skip early stages of development and go straight to Phase II trials. This can easily save three to five years. Not only can pharma giants test failed drugs for possible new applications, they can also offer compounds to other researchers. Some of these may be academic researchers, while others may be researchers accessed through partnerships with rival drug companies. Even when partnering with rivals, however, the benefits can be substantial due to the far lower costs of drug repurposing when compared with bringing a new drug to market.
New therapies are protected from generic competition to help underwrite the huge investment required for new drug development. Until 2003, it was easier for "pioneer" drug manufacturers to get injunctive relief from new generic entrants by filing multiple, follow-on patent applications for minor improvements to existing drugs. But this type of "evergreening" became more difficult after the FDA overhauled its Orange Book patent listing procedures to discourage the practice.
Drug patent "evergreening" is not as easy as it used to be
This prompted many pioneer drug manufacturers to develop new protectable uses for their drugs, as well as adding patent-protected language to drug labels to make it harder for generic manufacturers to avoid infringing the patents, helping limit generics to non-protected conditions.
When pharmaceutical companies submit patent applications for a new medical use for an existing drug, they must support the new use with evidence the drug is effective for the specified use. Additionally, the pharma company must establish novelty and inventiveness by providing substantial literature or history of testing in the new therapeutic context. One potentially sticky situation is that experimental data should provide support for the new claimed use, but that same data should not anticipate that use. The legal choreography can be tricky, but still well worthwhile compared to the costs of gaining approval for a brand new molecule.
Enforcing patents for new drug uses can be a challenge, however, if only because it can be difficult to identify when infringement occurs. For example, suppose a drug is prescribed for cross-purpose use, when a medication has regulatory approval for both patented and non-patented indications. While a generic may only be marketed for the non-patented indication, it can still be prescribed off-label for the patented indication. The FDA does not prevent physicians from prescribing drugs off-label for patented uses, and many insurers insist on generic substitution wherever possible. Identifying and challenging infringement can be difficult in these situations.
Repurposing existing drugs for new indications looks to be one of the most promising options for getting valuable new medical treatments to patients. The existing body of FDA-approved drugs may well offer effective treatment for many medical conditions, without the time and expense involved in bringing a brand new molecule onto the market. Pharmaceutical investors can benefit from using the DrugPatentWatch patent application database, which brings together the latest data on tentative approvals, international patent applications, upcoming patent expirations, trade names and generics.