#Cures2015 Legislation Will Help Patients Who Need It Most

The 21st Century Cures initiative continued today as the draft legislation was advanced through the Subcommittee on Health. The initiative has relied heavily on patients, advocates, physicians, innovators, and researchers from across the country to gain insight on how to get cures and treatments to patients faster. Over the past year, committee members hosted dozens of hearings and roundtables both in Washington, D.C., and across the country. Rep. Gus Bilirakis (R-FL) held two roundtables in Florida last summer to hear directly from folks in his district. His discussions with rare disease patients and families played a role in crafting his bipartisan OPEN Act, a part of the 21st Century Cures Act.

"Individually, each rare disease impacts less than 200,000 people. … When viewed collectively, however, 30 million Americans suffer from a rare disease. That’s about 1 in 10 people," Bilirakis writes in an op-ed featured in The Hill. As part of 21st Century Cures, the OPEN Act, "has the potential to help millions of people by incentivizing the testing of mainstream drugs – or repurposing them – to treat rare diseases and pediatric cancers."

Learn more about the 21st Century Cures initiative HERE. 

May 14, 2015

1 in 10

Individually, each rare disease impacts less than 200,000 people.  With such a small patient population, rare diseases hardly gain the attention that more widespread diseases such as cancer or heart disease garner.

When viewed collectively, however, 30 million Americans suffer from a rare disease. That’s about 1 in 10 people. That is more people than the entire population of Florida, which has nearly 20 million residents.

Rare diseases are not a rare problem and they cannot be overlooked. There are over 7,000 rare diseases, 95 percent of which have no FDA-approved cures or treatments. This leaves rare disease patients with little hope and few, if any, alternatives for treatments and cures.

The 21st Century Cures initiative is a nonpartisan undertaking by members of the Energy and Commerce Committee to help our healthcare innovation infrastructure thrive and deliver more hope for all patients. Led by Chairman Fred Upton (R-Mich.) and Rep. Diana DeGette (D-Colo.), we spent a year gathering ideas about how to find more and better cures and treatments faster and soon will continue in the legislative phase of this important cause. 

21st Century Cures has examined and seeks to accelerate the complete cycle of cures – from discovery to development to delivery and back again to discovery.

It is about finding new ways to drive innovation. People tell me: just increase funding at NIH. In the early 2000’s, the budget of NIH was doubled, but we were unable to double the cures.

In addition to adequate funding and resources we need to think critically about structural changes to streamline and modernize our health care system. We need to rethink what we have been doing and how we are doing it for the 21st Century.

This is what 21st Century Cures is giving us: an opportunity to address some of the structural barriers to new cures and promote new ways to incentivize developments.

Over the summer, I held two 21st Century Cures roundtables in my District to hear directly from those who know best – patients, patient advocates, physicians, and researchers. One of these rare disease patients, Ashleigh Pike, educated me on the importance of repurposing drugs.

Miss Pike, like other rare disease patients, is willing, even pleading, to take medication that has not been tested for her condition. Miss Pike and patients facing rare diseases deserve better. That is why I introduced the OPEN Act - Orphan Product Extensions Now ~ Accelerating Cures & Treatments. This policy is a key part of the 21st Century Cures initiative.

My bipartisan bill, introduced with fellow Energy and Commerce Committee member Rep. G.K. Butterfield, (D-N.C.), has the potential to help millions of people by incentivizing the testing of mainstream drugs – or repurposing them – to treat rare diseases and pediatric cancers.

…

Congress has a chance to come together to make a real difference in the lives of tens of millions of Americans who are desperate for treatments and cures.

We cannot let them down.

Read the full article online HERE.