NORD Petition Requests FDA Policy Statement on Orphan Drug Review Flexibility

By Kurt R. Karst –      

The National Organization for Rare Disorders (“NORD”) announced the submission of a Citizen Petition to FDA  requesting “that a documented policy be established regarding the review of potential treatments for people with rare diseases.”  The petition comes on the heels of a Report to Congress FDA issued earlier this year and required by Section 740 of the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act of 2010 (Public Law No. 111-80) (see our previous post here), in which the FDA reports on the findings and recommendations of two Agency groups to improve the current regulatory/scientific armamentarium to facilitate the development of products for rare and neglected diseases. 

In addition to the Report to Congress, Section 740 of the Appropriations Act also requires FDA to “issue, not later than 180 days after submission of the report to Congress . . . . guidance based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world,” and to “develop, not later than 180 days after submission of the report to Congress . . . internal review standards based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world.”  “Because the report was issued to Congress June 27, 2011, this guidance document is due to be issued no later than December 27, 2011,” says the NORD petition. 

NORD requests that FDA’s guidance explicitly include:

• Acknowledgement that the conduct of clinical trials for most orphan drugs is qualitatively and quantitatively different from the conduct of trials for drugs that treat common conditions.
• Acknowledgement that FDA review of marketing applications for most orphan drugs is accordingly qualitatively and quantitatively different from FDA review of applications for articles that treat common conditions.
• In recognition of the above, and notwithstanding the unchanged requirements that articles for rare diseases must demonstrate both efficacy and safety, we request a statement that it will now be FDA official policy to afford special flexibility to the regulatory review of submissions for all orphan drugs.

“This petition does not request any itemization of past actions – rather, through the language of forthcoming guidance, we request the establishment of a policy to direct future actions,” says NORD in the Citizen Petition.

In addition, NORD requests that FDA “incorporate mandatory training in this new policy and other matters related to orphan drug development for all full-time FDA review professionals,” and that this training become a requirement of FDA’s core curriculum.  As we recently reported, the Proposed PDUFA V Reauthorization Performance Goals and Procedures for Fiscal Years 2013 through 2017 includes an initiative, styled as “Advancing Development of Drugs for Rare Diseases,” that would require FDA to, among other things, “develop and implement staff training related to development, review, and approval of drugs for rare diseases.  The training will be provided to all CDER and CBER review staff, and will be part of the reviewer training core curriculum.”

In addition to the Report to Congress, NORD states in the petition that the organization’s requests are consistent with the recommendations of a recent finding of the Institute of Medicine, which released a report in October 2010, titled “Rare Diseases and Orphan Products: Accelerating Research and Development,” and with the testimony delivered by Hyman, Phelps & McNamara, P.C. Director and Chair of the Board of NORD Frank J. Sasinowski at a June 2010 FDA Part 15 hearing.