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Public Statements

Food and Drug Administration Safety and Innovation Act

Floor Speech

Location: Washington, DC


Mrs. HAGAN. First, Madam President, I do want to applaud the hard work of the Senate HELP Committee chairman Tom Harkin and the ranking member Senator Mike Enzi. This bill is truly one of the most bipartisan efforts I have had the opportunity to be a part of in the 3 years I have served in the Senate. It ought to be a reminder that, yes, when we work together across the aisle, the Senate can get things done.

I am particularly proud to support this bill because of what it will mean for patients who are suffering with diseases, who do not have access to adequate treatments, or who do not have access to any treatment at all. This bill we are voting on includes key provisions of the TREAT Act--the Transforming the Regulatory Environment to Accelerate Access to Treatments Act--which I introduced in February. These important provisions will expedite the review of treatments for serious or life-threatening diseases without compromising the FDA's already high standards for safety and effectiveness.

I introduced the TREAT Act after meeting with a family whose child suffered from spinal muscular atrophy or SMA. This is an incurable neuromuscular disease and is the leading genetic cause of infant deaths. Of course, that family was not alone. There are 30 million Americans suffering from rare diseases, and I have had the honor to meet a number of them. Their stories are both heartbreaking and inspiring.

When I visited the North Carolina Children's Hospital last month, I met with Megan and Jarrod Hendren of Lumberton, NC, whose 13-month-old twins Logan and Lucas suffer from Gaucher's disease. This disease is a painful and potentially debilitating metabolic disorder for which currently there is no cure.

I also met with 8-year-old Ashley Burnette from Raleigh, who is resilient and wise beyond her years, but who is suffering from neuroblastoma.

For the families and patients like these, suffering from these rare diseases for which there are no approved medications, medical advances cannot come fast enough. There are so many rare diseases, but fewer than 250 have FDA-approved therapies. The provisions of the TREAT Act that have been included in this bill take great steps toward resolving the problem.

There is currently a pathway at the FDA to expedite the review of drugs for illnesses that are serious or life-threatening and for which there is no adequate treatment. This is called the Accelerated approval pathway. Since the early 1990s, it has been successfully used to advance treatments for patients with HIV and cancer by leaps and bounds. However, it has not been applied regularly or consistently to the review of drugs to treat other diseases.

This inconsistency is why I introduced the TREAT Act. My bill will broaden the application of the accelerated approval pathway beyond HIV/AIDS and cancer to a wider range of diseases, with a particular focus on rare diseases. That is why my proposal enjoys broad support from patient advocates, including the National Organization of Rare Diseases, Us Against Alzheimers, Parkinson's Action Network, the Huntington's Disease Society of America, and many more.

By providing for consistent application, we will help the FDA implement these provisions, assist drug sponsors to navigate the approval process, and, hopefully, bring safe and effective treatments more rapidly to the patients who need them.

I am also proud to have played a critical role in the legislation that led to the negotiations of the first biosimilars user fee agreement, which is also included in the bill before us. Last Congress, we passed the Biologics Price Competition and Innovation Act to facilitate the introduction of lower cost alternatives to biologic drugs, while
ensuring continued research and development into innovative biologics which can save or improve the lives of millions of Americans.

The user fees negotiated by the industry and the FDA will provide the necessary funding for the review of these critical therapies. The biosimilars industry is in the earliest stages of development, and the biosimilars user fee agreement will help facilitate this industry's growth.

In addition, the FDA Safety and Innovation Act provides the necessary regulatory updates to keep pace with the rapid innovations of the biopharmaceutical industry. This is imperative for creating jobs in States such as mine--in North Carolina--and maintaining America's competitive edge in the global economy.

Companies with footprints in North Carolina are partnering with our world-class universities to improve the health of people all across the globe every day by researching, discovering, and developing lifesaving treatments for those suffering from these devastating diseases.

Passing the FDA Safety and Innovation Act for States such as North Carolina, and for our Nation, to remain global leaders is important. It is especially important if we are to help attract the jobs of the future.

The American public also expects the FDA to be the world's gold standard when it comes to ensuring the supply, the safety, and the integrity of our drug supply. By sending the FDA Safety and Innovation Act to the President's desk, we will establish a clear and effective pathway for turning ideas into cures and cures into treatments. And we will have shown the foresight and flexibility required to maintain our country's position at the top of the medical treatment and device industries.

I thank the Chair and I urge my colleagues to join in supporting the FDA Safety and Innovation Act.

I yield the floor.


Mrs. HAGAN. Section 901 of the managers' amendment to S. 3187, Enhancement of Accelerated Patient Access to New Medical Treatments states that an accelerated approval under section 506(b) of the Federal Food, Drug, and Cosmetic Act is subject to certain limitations, including the requirement that the sponsor conduct appropriate post- approval studies to verify and describe the predicted effect on irreversible morbidity or mortality or other clinical benefit. Does the lack of an explicit reference to postapproval validation of surrogate endpoints, as described in current law, in any way restrict the Secretary's existing authority to require such validation postapproval?


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