The Food and Drug Administration Safety and Innovation Act, signed into law on July 9, 2012, includes important measures for rare disease patients and families. Part of the reauthorization of the Prescription Drug User Fee Act (commonly known as PDUFA), the Safety & Innovation Act contains groundbreaking provisions for the rare disease community. It is rewarding to see that patients and caregivers will benefit from the newly signed FDA act.
As a board member of the National Organization for Rare Disorders, I am proud of NORD's efforts to help ensure the following were included:
- Accelerated patient access to new medical treatments
- Resolution of conflict-of-interest provisions introduced in the previous PDUFA reauthorization
- Accelerated development of "breakthrough therapies" that show early promise
- Enhanced FDA consultation with rare disease medical experts
- A rare pediatric disease priority review voucher incentive program
- The development of Humanitarian Use Devices (medical devices for small patient populations)
These provisions reflect in many cases years of advocacy, education and relationship-building by NORD and its member organizations on behalf of the rare disease community. Kudos to the many organizations and individuals who worked so hard to make this legislation a reality.
A combination of several bills
In Congress, several bills that initially were introduced separately were later rolled into PDUFA V. Those of particular interest to rare disease patients, and supported by NORD, included:
- Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act
- Faster Access to Specialized Treatments (FAST) Act
- Advancing Breakthrough Therapies for Patients Act
- Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act
- Creating Hope Act
- Humanitarian Use Devices (HUDS)
Of the 7,000 rare diseases, only about 250 have FDA-approved therapies. These numbers are indicative of the challenges facing the rare disease community and the critical need to develop more therapies. The Safety & Innovation Act is a big step in the right direction, and I look forward to future positive developments.
For more information on orphan drugs and rare diseases, check out the World Orphan Drug Congress USA. Siren Interactive is a silver sponsor.
For more on this topic, take a look at the 4 key impacts of the FDA Safety and Innovation Act here
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