Fda Reauthorization Bill
Mr. President, I wish to speak on the importance of maintaining a strong Food and Drug Administration. Today we approved the user fee reauthorizations for the FDA. We have done the important work of passing these essential user fee…
Mr. President, I wish to speak on the importance of maintaining a strong Food and Drug Administration. Today we approved the user fee reauthorizations for the FDA. We have done the important work of passing these essential user fee agreements out of the committee and have now debated and passed them on the Senate floor.
The HELP Committee is filled with strong personalities. These personalities reflect the passion and diversity of opinion of millions across our nation today. While we may disagree on certain policies, most of us can agree that funding the drug, device, and biologic centers of the FDA is essential.
Our future scientific endeavors require a strong FDA that communicates openly with the industry that it regulates, and this agreement sets up protocols to achieve that goal. A strong FDA also requires clear steps for product review, and only through such deliberative actions can we bring more competition and clarity to our drugs, devices, and biologic products.
I have championed multiple provisions in this bill, but there are two I would like to highlight today. First, there is the counterfeit and diverted drug language. This language makes importation neither harder nor easier. In fact, it doesn't change importation laws at all. Rather, it protects and strengthens the drug supply chain by
simply increasing penalties for criminals that choose to divert drugs into the United States or sell counterfeit drugs.
Current penalties for illegally diverting drugs in the United States change arbitrarily based on the location where the drugs are manufactured. Our bill addresses this disparity by enforcing the same penalties for diverting drugs made outside the United States as for those made inside the United States. To ensure public health and to enhance consumer confidence, it is critical that Congress eliminate these differing penalties for certain types of diversion and counterfeiting.
The second provision I wish to call attention to is a bipartisan proposal from Senators Bennet, Burr, and Casey. These fine Senators have joined together to address how clinical trials are designed early on in their development. By offering guidance on how to include the intended patient population, especially those with rare diseases, drug sponsors can craft trials that generate useful data for health professionals and patients to review.
This bill builds upon the success of other expanded access provisions that put the patient at the heart of the healthcare system. FDA does consummate work when reviewing products for market, but including a wider patient mix, when appropriate, will enable phase I, II, and III trials to be more complex. I strongly believe that accurately portraying the intended patient population in a clinical trial is key to ensuring that drugs are both safe and effective.
I support this bill, but I also feel compelled to speak for a moment on the OPEN ACT. While not included in the package being debated today, the provisions of the Orphan Product Extension Now Accelerating Cures and Treatments Act--a bill I introduced this Congress with Senator Menendez and last Congress with Senator Klobuchar--would promote new therapies for rare diseases.
New therapies are essential to help the nearly 30 million Americans suffering from a rare disease or condition. Because complex rare diseases with small patient populations have limited market potential, there are few economic incentives to develop new drugs targeting those diseases. While there are 7,000 rare diseases that impact millions of Americans, 95 percent of these diseases have no treatment. All too often, misconceptions about the dangers of exclusivities keep bipartisan measures from being introduced. We must remain focused, however, and remember that, each day we delay in getting treatments to the rare disease community, patients and their families suffer.
Drug companies possess considerable scientific knowledge on drugs that have already been approved for common diseases. Some of these drugs could be repurposed for the treatment of rare diseases. Repurposing drugs is faster, less expensive, and generally less risky than traditional drug development.
The OPEN ACT would encourage such repurposing by providing an additional 6 months of market exclusivity to drugs that are repurposed and approved by FDA for a rare disease or condition.
Finding legislative ways help medical innovators treat rare diseases has been among my top priorities for over 30 years, since I first championed the bipartisan, bicameral Orphan rug Act in 1983. The OPEN ACT is a natural next step in expanding that effort to close the gap for rare diseases for which we have yet to develop treatments. In addition to increasing the number of rare disease therapies, this legislation will boost innovation and provide safer options for rare disease patients using drugs off-label. My bill enjoys enormous support with the backing of over 225 rare disease organizations and patient advocacy groups, not to mention overwhelming support from academic medical and research centers.
Although this provision is not in the bill before us, I have had assurances from Chairman Alexander that he will continue working with me and the cosponsors of this bill to see it become law. I have spoken to Ranking Member Murray in the past about it, and I remain optimistic that my colleagues share my concern for the rare disease community and are willing to advance this legislation in the future. I would like to thank the chairman and ranking member for their dedication to children and families in need.
I wish to conclude by reminding my colleagues that many of the debates that have led to the bill before us today are the culmination of years of experience. When I led the effort to pass what became Hatch-Waxman, the true impact of that law dwarfed even our loftiest hopes. Hatch-Waxman was a resounding success because Senators and Congressmen worked together to improve our country's situation and reduce barriers to market entry. This bill is vital to continuing that goal, and I am pleased to see where the negotiations have landed.