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WASHINGTON—Congressman Paul D. Tonko today led a powerful conversation on his bipartisan HEART Act with local advocates and parents whose children are living with rare disease.

Tonko’s Helping Experts Accelerate Rare Treatments (HEART) Act—introduced alongside Congressman David McKinley (R-WV)—strengthens the U.S. Food and Drug Administration (FDA) review process for drugs that treat rare and ultra-rare diseases by directly involving scientists and doctors with expertise throughout the review process. He was inspired to create this bill because of the incredible advocacy of local constituents, several of whom participated in today’s event.

Participants shared the experiences they and their loved ones have living with rare disease and highlighted the importance of advancing Tonko’s legislation to ensure scientists, doctors and patients all have a voice in the FDA review process.

Watch the full recording of the discussion HERE.

Also, see below some remarks from Congressman Tonko on the HEART Act:

The estimated 25 million Americans living with rare diseases have long been overlooked or underserved by our healthcare system. Many of those affected have struggled to find effective, affordable treatments or cures for their conditions.

While the individual communities of people affected by any individual rare disease are small by definition, the combined impact is vast, with some seven thousand different rare diseases impacting millions of American families.

The commonsense steps outlined in my HEART Act will vastly expand treatment access for patients living with rare diseases and help save lives today. While there has long been an urgency to get this done, with COVID and the effect on research and those living with rare diseases, we must act now and pass this bill into law.

We can and must do better—for our friends and our neighbors who are still searching for answers and all who are struggling to find care.

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