Key provisions of the bill are outlined below including an accelerated approval process as well as providing incentives to researchers to provide more focus on rare disease therapies. Another important provision will allow for an evaluation of patient tolerance to risks for certain therapies including greater patient feedback.
Praises Senate for Addressing Needs of Rare Disease Patients
May 22, 2012, WASHINGTON, DC--- The National Organization for Rare Disorders (NORD) applauds legislation passed today by the U.S. Senate -- S 3187, the Food and Drug Administration Safety & Innovation Act of 2012 (FDASIA) -- and says it contains the most comprehensive improvements to public policy for rare disease therapies since the landmark Orphan Drug Act of 1983.
“NORD has been working very hard over the past two years to ensure that the interests of the rare disease patient community are well represented in this important legislation,” said NORD President and CEO Peter L. Saltonstall. “We are thrilled that our nation’s leaders in Congress are working together and making substantive policy improvements to bring new therapies to patients who desperately need them.
“When this process began over two years ago,” he added, “our focus was on obtaining a commitment by the FDA to further incorporate the exceptional nature of rare diseases in the review of promising new therapies and medical devices. What we’ve been able to achieve since then, with our members and advocacy partners in the rare disease community, is nothing short of astonishing. Our sincerest thanks go to all who have helped to make this day possible.”
The bill contains numerous provisions that will enhance the regulatory process at FDA, incentivize further investment by innovators into rare disease therapies, and expand the scientific armamentarium needed to bring the most cutting-edge therapies to patients as quickly as possible.
Specific commitments by the FDA include:
- Expanding the Rare Disease Program at the Center for Drugs and extension of the program to the Center for Biologics
- A new initiative to further qualify and categorize biomarkers for use in clinical trial design, including under Accelerated Approval and similar mechanisms
- A new initiative to evaluate patient tolerance to risk of proposed therapies and to incorporate more patient input in the review process
- Codification and modernization of the Accelerated Approval process
- Improvements to policies governing conflicts of interest that will allow expert participation on FDA Advisory Committees
- Creation of a new category of "breakthrough" therapies that will encourage early public and private collaboration to aid in clinical trial design and review
- Expanded access to experts for rare diseases beyond the review process
- Expansion of devices intended for use in very small patient populations, including the possibility of profit for such devices when used in adult populations
Both the House and Senate bills are the culmination of a process conducted every five years to reauthorize the Prescription Drug User Fee Act (PDUFA) and related legislation that provides critical funding to allow FDA to review potential new therapies in a timely manner. As the voice of rare disease patients and their families in the U.S. since 1983, NORD has served as the primary representative of the rare disease patient community over the past two years as needs were prioritized and the legislation now being voted upon by the House and Senate was drafted.