MDUFA, PDUFA, FDASIA head out to Pennsylvania Avenue

After a long and collaborative effort between the FDA and Industry, the Food and Drug Administration Safety and Innovation Act (FDASIA) including reauthorization of MDUFA and PDUFA head to the White House for signature into law.

It’s been a long journey full of twists and turns but the job got done in Washington on Tuesday with a major step forward for health and healthcare innovations. While the nation waits for the announcement of the Supreme Court ruling on the Affordable Care Act, other key legislation moved forward and now is on its way to the President’s desk.

The Senate voted 92-4, to pass S. 3187 – the Food and Drug Administration Safety and Innovation Act (FDASIA) which included reauthorization of both critical user fee programs as MDUFA III and PDUFA V well before the September expiration deadline. (The House had passed the Bill by a unanimous voice vote last Friday.)

“FDASIA reflects enhancements to PDUFA that were widely and strongly supported by stakeholders. The legislation will enhance the development and review of innovative new therapies through increased transparency and scientific dialogue, advancements in regulatory science and strengthened post-market review” shared Biotechnology Industry Organization (BIO) President and CEO Jim Greenwood. “It will also increase the Food and Drug Administration’s (FDA) access to external expertise to improve the drug review process.

“The final bill will enhance the efficiency of FDA’s human drug review program, and will help bring safe, effective, and innovative medicines to patients in a timely manner” shared Pharmaceutical Research and Manufacturers of America (PhRMA) President and CEO John J. Castellani. “The result of this process is evident in the permanent reauthorization of the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act, which will improve medical care for children by driving research to create innovative medicines for use in pediatric patients.”

“It’s been a long, yet productive process since FDA first began meeting with industry and other public stakeholders on the reauthorization of user fees nearly two years ago” shared Margaret Hamburg, M.D. Commissioner of the U. S. Food and Drug Administration. “What has emerged, I believe, will truly result in a stronger and better FDA for everybody.”

“This legislation will help improve an already robust review process by giving FDA the additional tools and resources it needs to improve its timeliness and consistency,” said Stephen J. Ubl, AdvaMed president and CEO. “That means more American patients will have access to safe and effective treatments and diagnostics sooner, which is the shared goal of both FDA and industry.”

The passage of 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. Here are some of the key features:

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.
The permanent reauthorization of the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act in FDASIA will encourage continued investment in pediatric research and help ensure that new drugs and biologics can be used safely and appropriately in pediatric patients.
Two new User Fee programs for Generic Drugs and BioSimilars.
Mechanisms for greater patient input in both the approval and prioritization process.
The legislation includes a series of strong, measureable performance goals and additional funding that should help reverse the decline in performance FDA has experienced in recent years.
- Reduce total review times, measured for the first time from the time of submission to the time FDA makes a final decision on a PMA or a 510(k), and significantly improve review performance relative to current FDA performance;
- Leave “no submission behind” by requiring FDA to meet with companies if a performance goal on a PMA or 510(k) is missed and work out a plan for completing the review;
- Provide a substantive interaction with applicants halfway through the targeted time for completion of a review; and
- Implement an analysis of FDA’s management of the review process by an independent consulting organization, coupled with an FDA corrective action plan to address opportunities for improvement.
Congress also made significant policy changes that will help alleviate drug shortages and improve the integrity of the pharmaceutical supply chain.

Passage of FDASIA ushers in a new era of closer and more effective collaboration between industry and the FDA. No organiztion can be truly effective with out both sufficient funding and actionable performance metrics that it can measure and be held to. With this new legislation, the FDA has been given just that.

“We are thankful for the efforts of our leaders in both houses of Congress, the FDA, and all of the industry leaders who have worked together over the last two years to make this happen. AZBio Members traveled to Washington D.C. to share their stories with their elected leaders, wrote letters and shared comments with regulators, and participated in numerous committees to help bring us to this point” shared Arizona Bioindustry Association President and CEO Joan Koerber-Walker. “This may be the end of one journey, but a new collaborative journey now begins, one that has at its outcome more life saving, life sustaining, and life improving innovations for patients. At the end of the day, that’s what this new law, the FDA, and our industry is all about.”

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