President Obama Signs FDA's User Fee Legislation

New legislation recently signed by the president promises to get novel therapies to patients in a more timely manner through the FDA’s Accelerated Approval process.

Patient advocacy groups in support of finding treatments for rare disease are celebrating President Obama’s recent move signing the Food and Drug Administration Safety and Innovation Act (FDASIA) into law. The act contains numerous provisions that will function to spur the delivery of lifesaving treatments to 30 million Americans suffering from rare diseases that are often overlooked.

There has been little additional support for the rare disease patient population since the Orphan Drug Act was passed more than 25 years ago, and there are currently fewer than 400 approved treatments for 7000 rare diseases.

Representatives Cliff Stearns (R, FL) and Ed Towns (D, NY) spearheaded the rare disease campaign, first by introducing the Unlocking Lifesaving Treatments for Rare Diseases Act (ULTRA), in which they asked the FDA to allow “surrogate endpoints” in clinical trials to assess a drug’s efficacy, and then by enhancing the tools to expedite review of therapies affecting rare disease patients in the Faster Access to Specialized Treatments (FAST) Act.

“We are thrilled the language to improve access to the FDA’s Accelerated Approval pathway for rare diseases has been included in FDASIA,” said Emil Kakkis, MD, president of EveryLife Foundation for Rare Diseases.

One hundred twenty-one organizations, such as the Breakthrough Cancer Coalition, the National Fabry Disease Foundation, and the United Pompe Foundation, signed a letter supporting the inclusion of the strongest rare disease provisions in the FDA user fee legislation.

"FDASIA will foster timely interactive communication with sponsors during the drug development phase as a core Agency activity to facilitate the conduct of efficient and effective drug development programs and help make safe and effective medicines available to Americans in a timely manner,” said Jim Greenwood, Biotechnology Industry Organization (BIO) president and CEO, in a statement. “Further, the enhanced Accelerated Approval pathway 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 standards for safety and effectiveness.”

Supporters of the legislation believe that investment and interest in the development of treatments for orphan diseases will rise once rare disease treatments have access to the Accelerated Approval pathway. This interest may also swell with the enactment of The Creating Hope Act, which aims to incentivize pharmaceutical companies to develop treatments for rare pediatric diseases.

A handful of proposed provisions failed to survive the legislative negotiations, however. The “track and trace” rule—a national traceability framework intended to identify and follow an individual drug through the pharmaceutical supply chain to protect against counterfeit drugs—was not included, despite strong support from BIO. Provisions outlining Risk Evaluation and Mitigation Strategies (“REMS”) and generic competition also did not make the cut.