FDASIA: A Step Forward for Patients

Earlier this year, Congress passed and President Obama signed the FDA Safety and Innovation Act (FDASIA) which included a re-authorization of the Prescription Drug User Fee Act (PDUFA). PDUFA provides essential funding to the FDA by authorizing the agency to charge user fees to companies seeking to have products reviewed. Originally enacted in 1992, it must be reauthorized every 5 years.

Sara Radcliffe, executive vice president of health at BIO, moderated a panel of experts and advocates entitled “FDA Safety & Innovation Act: A Step Forward for Patients” at the 2012 BIO Patient and Health Advocacy Summit. Panelists discussed what we learned from the latest re-authorization cycle and explained what needs to occur for successful implementation.

Start early. Finish early.

Almost everyone is pleased with FDASIA. How did that happen?

While past PDUFA re-authorizations were contentious at times, during this cycle stakeholders began their efforts two years prior to expiration. Advocates, patients, industry leaders and members of Congress collaborated to draft a popular bipartisan bill.

“This was an idea where people in the beginning said, ‘We’re starting already? We feel like we just finished!’” Janet Jenkins-Showalter, from Genentech, a Member of the Roche Group, said. “I think that really helped us. Not only did we start early, but we finished early.”

Ensuring successful implementation.

Even though FDASIA has been signed into law it is important for all stakeholders to remain involved in its implementation. The FDA has been proactive in releasing an implementation timeline that includes public meetings and release dates for draft guidance – opportunities to provide feedback.

“It’s really important for us to deliver messages about what is working as well as where our goals are getting blocked in a proactive manner,” said Mary Dwight, vice president of government affairs at the Cystic Fibrosis Foundation.

In addition, it is very important for all stakeholders to continue to work together in providing feedback throughout the entire implementation process.

“Don’t go it alone as an organization,” advised Jeff Allen, executive director with the Friends of Cancer Research. “No matter how big your organization, or how small it is, you’ll just get further working with your colleagues.”

For the rare disease community, FDASIA is the most important piece of legislation since the Orphan Drug Act.

“Since 1983 there have been about 400 orphan products that have been approved by the FDA,” explained Diane Dorman, vice president of public policy at the National Organization for Rare Disorders. “Unfortunately, they treat only about 250 of the nearly 7,000 rare diseases.”

Included in FDASIA is an Accelerated Approval pathway that will help expedite the development of modern, targeted, and personalized therapies for patients suffering from serious and life-threatening diseases while preserving the FDA’s robust safety standards.

“Accelerated approval has been used by the FDA to approve products for AIDS and for cancers, but rarely focused on other conditions,” Dorman said.

All the gains made in FDASIA hinge on funding for the FDA.

The FDA’s impact is far-reaching: It is responsible for the quality and safety of most of the food Americans eat, all of the medications they take, the cosmetics they use, the medical devices and diagnostic tests used in hospitals, and much more.

The Alliance for a Stronger FDA, of which BIO is a member, says that 25 cents of every consumer dollar spent in the United States goes to products that fall under FDA purview. To meet this challenge, Congress currently authorizes that the agency spend about $8 per year for each American. Just a minor increase would go a long way toward helping the FDA improve its operations.

“We really need to hear voices from the patient community about the importance of an appropriately funded FDA,” Dorman explained, “because, if they’re not funded properly, food will be less safe, you’ll get products at a later date, and companies won’t have their applications reviewed as quickly as possible.”

The BIO Patient and Health Advocacy Summit convened individuals from the patient advocacy community and biotechnology industry for two days of thought-provoking discussions on timely policy topics and issues of importance to nonprofit organizations. An inaugural event, the Summit featured presentations from key policy experts and national advocacy leaders, and provided a forum for nonprofit organizations to gather and exchange best practices to help advance their missions. 

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