Landmark NORD Report on Orphan Drugs Authored By HP&M Director Shows FDA Flexibility in Approval

By Kurt R. Karst –      

The first U.S. Conference on Rare Diseases and Orphan Products started with a bang today with the release of a landmark report announcing its findings of flexibility in FDA’s review of potential treatments for patients with rare (“orphan”) diseases.  The conference, which is co-sponsored by the National Organization for Rare Disorders (“NORD”), under the auspices of the Drug Information Association, is taking place in Washington, D.C. from October 11th-13th.  (Collaborating organizations include EURORDIS (the European Rare Disease Organization), FDA, NIH, and the Duke Clinical Research Institute.)  Chairman of the NORD Board of Directors and Hyman, Phelps & McNamara, P.C. Director, Frank J. Sasinowski, authored the report and presented on it at the conference.  (A copy of Mr. Sasinowski’s conference presentation is available here.)

The NORD study was designed to examine how much flexibility (if any) FDA exercises in reviewing orphan drugs.  In other words, the study was intended to determine whether FDA requires that marketing applications for orphan drug provide the conventional or traditional effectiveness data that are ordinarily expected for most drugs for more prevalent diseases.  The report examines the quantum of effectiveness evidence that provided the basis for FDA’s approval of the 135 non-cancer orphan drug new chemical entities approved between the creation of the Orphan Drug Act in 1983 and June 30, 2010.  Of the 135 drug approvals studied, NORD concluded that 45 would have met traditional data requirements, 32 reflected “administrative flexibility” based on a previously documented FDA system, and 58 reflected flexibility applied on a case-by-case basis.

“This review of FDA actions concludes that two of every three orphan drugs approved show FDA’s historic flexibility in its review of effectiveness data on orphan drug therapies, ” Mr. Sasinowski said in a NORD press release.  “Therefore, FDA has demonstrated in its actions on orphan products that it recognizes the importance of therapies for persons with rare disorders.  It would be helpful for such flexibility and importance to be recognized in a formal FDA policy, and for FDA officials to incorporate and recognize that flexibility in a systematic way in their evaluations of each new therapy in development and under FDA review for Americans with any rare disease.”

In June 2010, FDA held a Part 15 hearing, titled “Considerations Regarding Food and Drug Administration Review and Regulation of Articles for the Treatment of Rare Diseases” (Docket No. FDA–2010–N–0218), at which Mr. Sasinowski, on behalf of NORD, testified and recommended that FDA “develop and issue a specific Statement of Policy on FDA’s role in regulating therapies for rare disorders, which includes an explanation and affirmation of the FDA’s historic position that FDA flexibly applies the standards of safety and effectiveness with respect to therapies for those with rare disorders.”  That hearing was followed by a NORD citizen petition requesting “that a documented policy be established regarding the review of potential treatments for people with rare diseases.”  (See our previous post here.)  

The NORD report follows both the issuance of the proposed PDUFA V Reauthorization Performance Goals and Procedures (Fiscal Years 2013 through 2017), which state that FDA “will develop and disseminate guidance and policy related to advancing and facilitating the development of drugs and biologics for rare diseases, including . . . . encouraging flexibility and scientific judgment, as appropriate, on the part of reviewers when evaluating investigational studies and marketing applications for drugs for rare diseases,” and an FDA Report to Congress from the Agency’s Rare Disease Group (“RDG”).  As we noted last month, in the report, the RDG “identified both strengths and constraints in the current paradigm for drug and device regulation.”  Among the key areas in need of additional efforts identified in the RDG report is for FDA to “[g]ain a thorough understanding of the regulatory history of orphan drug products to help identify effective development approaches, particularly for addressing the uncertainties of biomedical knowledge along the product development pathway, so that patterns can be adapted to future development programs.”  This includes performing “a detailed analysis of FDA’s 27-year history of orphan drug approvals to identify factors and methods that have led to successful drug development and approval or have impeded development and identify areas for improvement.”  The NORD report should certainly help out in this respect.