What We Learned at the Energy and Commerce Committee's Hearing on User Fees

Yesterday, the House Energy and Commerce Committee's Subcommittee on Health held its second hearing of the year on the FDA's user fee programs, including PDUFA. While the bulk of the hearing actually focused on other public health issues such as drug counterfeiting and the pharmaceutical supply chain, we still heard some good discussion about the many benefits of PDUFA to patients and the FDA.

Janet_Woodcock_3.pngThe hearing's first witness was Dr. Janet Woodcock, Director of the FDA's Center for Drug Evaluation and Research (CDER). In her testimony, Dr. Woodcock stated that PDUFA "has really assured that the US is the leader in developing and introducing new and important drugs to the public, so that Americans have access to that cutting-edge science and to drugs that will treat life-threatening conditions." Witness John Maraganore, CEO of Alnyam Pharmaceuticals, echoed this in his own testimony, saying, "In a time when the U.S medical innovation ecosystem is facing severe strains and increased global competition, it is imperative that FDA's policies and practices find the right balance... to ensure we are able to deliver the next generation of breakthrough treatments and therapies." PDUFA V will help the FDA strike that balance between safety and innovation to ensure that patients receive effective and lifesaving drugs, while maintaining the United States' global leadership in biomedical innovation.

The need for a wider variety of treatment options for patients with rare diseases was a popular topic during the hearing. As we've previously discussed, PDUFA V includes a Rare Disease Program Initiative that will advance scientific knowledge about rare diseases and give the FDA additional resources to train its staff and new drug sponsors on the unique challenges of the development and review processes for medicines for rare diseases

We especially appreciated that Congressman Henry Waxman drew attention to the possibility that adding additional legislation to PDUFA has the potential to slow down its reauthorization, ultimately causing delays in the FDA's review process and in the agency's ability to deliver safe, effective medicines to patients in a timely manner. Congressman Waxman:

For us to do a responsible job on these proposals, we need time and we need bipartisan agreement. We should not rush this work; we should prioritize getting it right, not just getting it done, and if we're able to come to a bipartisan agreement in the time available, it makes sense to move them along with the other bills. Otherwise, I hope we can all agree it will be better to wait so that we don't jeopardize the passage of the underlying user fee bills.

There's no doubt that issues like counterfeit drugs and drug shortages are important; however, it would be impossible and irresponsible for Congress to tack a long list of controversial additions onto the PDUFA reauthorization, if it impedes passage. Instead, we hope to see Congress pass a PDUFA based on the technical letter already endorsed by FDA, patients, healthcare professionals, and the pharmaceutical industry.

We enjoyed the great discussion on Facebook and on Twitter about PDUFA and the hearing, and we'd like to thank you for engaging with us. We encourage you to visit our Facebook page and let us know what you would tell Congress about the need for safe, innovative medicines and a clean PDUFA.