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In November 2018, the Government Accountability Office (GAO) released a report titled “FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue.” It follows up on the FDA’s Orphan Drug Modernization Plan of June 2017, a plan designed to help eliminate a mounting backlog of applications for orphan drug designation.
Rare diseases affect 200,000 or fewer Americans, yet altogether, 30 million Americans are affected by them.
The FDA plan introduced actions like collaboration among different review divisions and reduction in discretionary burdens on the Office of Orphan Products Development (OOPD). Additionally, it developed a standard designation template to promote consistent and efficient review of applications. The GAO report focuses considerable attention on those templates, having found inconsistencies in the completeness of data entered into them.
Addressing Growing Demand for Orphan Drug Designation
Increased interest in drug patents for rare disease medications, as well as incentives offered under the Orphan Drug Act (ODA), have caused a backlog of applications at the FDA.The 2017 Orphan Drug Modernization Plan was designed to address this increase, but implementing the recommendations has not been without problems. Recommendations made in the GAO report are intended to address those problems, put the Modernization Plan back on track, and ultimately result in more orphan drugs being approved by clearing the application backlog.
Office of Orphan Products Development Challenges
The GAO report has focused on the standard designation templates introduced by the Modernization Plan. On these templates, OOPD reviewers enter information from the orphan drug status requests, which is then used in assessment of the request. However, the GAO noticed missing information in the majority of these templates.
Furthermore, reviewers receive no instruction for using the information in the evaluation process. Finally, the OOPD didn’t always verify the data to make sure it was accurate and complete. The GAO recommended that the FDA improve the consistency of information entered into the review templates.
Incomplete information on standard templates has caused problems with the review of orphan drug designation requests.
Barriers to Orphan Drug Development
Additionally, the GAO report cited the most common reason for denial of orphan drug designation: inadequacy of the manufacturer’s scientific rationale in its application. The manufacturer, in other words, may not have provided adequate data to support their scientific rationale, or did not use the strongest available model for drug testing.
The report cited an increase in orphan drug approvals from 2008 through 2017, with the FDA approving 77 orphan drugs for market in 2017 alone. According to the GAO, the two biggest barriers to orphan drug development currently are a need for more basic scientific research and difficulty in recruiting populations for clinical trials.
Drug patents for rare disease treatments have increased substantially during the 35 years the Orphan Drug Act has been in effect, and that’s good news. But the approval process for these important drugs will need to be fine-tuned as interest in developing them grows, to ensure the approval process is thorough, timely, and fair.
Rare diseases affect an estimated 30 million Americans, and only about 5% of those diseases have FDA-approved treatments. The Orphan Drug Act offers incentives like tax credits and exclusive marketing rights for developers of drug patents for rare diseases.
The growth of orphan drug designations is expected to continue due to medical advances, particularly in genetically-targeted care. But with technological and medical advances, the Orphan Drug program will be forced to revisit its approval process periodically so that it remains efficient and thorough, bringing innovative drugs for rare diseases to market in a timely manner.