FDA To Offer Reviewers Rare Disease Training; Patient Groups Want Separate ODE
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FDA will offer training next year for reviewers to better understand rare diseases and their associated product applications that could help speed the approval process, although it appears to be optional. The training fits into Commissioner Margaret Hamburg’s push to improve regulatory science, which includes an increased emphasis on orphan products as more manufacturers and developers enter the space. Because rare diseases affect small populations and are not well understood, drugs and biologics in the category can be difficult to understand and review. FDA said clinical trial design and endpoint selection are tough. There also are ethical considerations, since many rare diseases affect children or are life-threatening. FDA disclosed the upcoming training during an Oct. 22 Prescription Drug User Fee Act reauthorization meeting with stakeholders after a representative of the Alliance for Regenerative Medicine suggested reviewers undergo more rare disease training, according to meeting minutes. The training would help update the application review staff on the most current science and issues associated with rare diseases. Reviewers would learn about navigating “the many complexities [rare disease] applications present,” during the course, the agency said in an e-mailed statement. Any reviewer willing to take the course is eligible, the agency said. Classes are scheduled to start in February. The training is expected to be similar to a workshop for rare disease developers and investigators organized by the National Institutes of Health, National Organization for Rare Disorders, Duke University Medical Center and FDA. The agency said more than 100 researchers attended. Reviewers In Several Offices Want Training Among the agency’s 12 proposals for improvement of PDUFA is a new program to create regulatory policy, procedures and guidance specifically for rare diseases. That includes conducting workshops on rare disease drug development issues. Previous negotiations with industry indicated the agency expects more orphan product development based on increases in orphan designations and interest among large pharmaceutical companies (Also see "User Fee Discussions Include Funding Parts Of FDA Regulatory Science Plan" - Pink Sheet, 25 Oct, 2010.). FDA also wants to work with rare disease advocates and other stakeholders to push for more drug development in the area. Appropriations legislation includes increased money for orphan product development grants and additional funding for the Office of the Associate Director for Rare Diseases in CDER (Also see "FDA Funding Bill In Senate Gives Generic Drugs More, But Commissioner Less" - Pink Sheet, 2 Aug, 2010.). Agency staff in CDER, CBER and the Office of Orphan Products within the Commissioner’s Office already have indicated interest in the training. The agency said it did not know how many will attend. It is unclear whether all review staff will eventually take the course. All the review divisions in the CDER Office of New Drugs receive rare disease applications, although rare disease advocates want that changed. They already have called for FDA to create a new Office of Drug Evaluation for Genetic and Biochemical Diseases and a separate review team to handle rare disease applications (Also see "Effectiveness Of Priority Review Voucher, Orphan Status Part Of Tropical Disease Hearing" - Pink Sheet, 20 Jul, 2010.). All rare disease expertise would be centralized there and alleviate confusion about approval requirements. The idea was proposed again during the PDUFA meeting. Advocates want the agency to be more flexible in some evidentiary requirements for rare disease applications. Some have argued the classic double-blind, placebo-controlled clinical trial may not be the only trial design the agency should accept. During a public hearing this summer, groups also asked the agency to approve more biomarkers and surrogate endpoints to help expand the development pipeline (Also see "Orphan Drug Reviews: Industry Wants FDA To Move Out Of Its Comfort Zone" - Pink Sheet, 5 Jul, 2010.). |