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.  

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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. ButterfieldGeorge (G.K.) Kenneth ButterfieldConyers resigns amid sexual misconduct allegations Government study shows lack of diversity in tech Black lawmakers give tech sector low marks amid Silicon Valley trip MORE, (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. 

Another patient I met with, Candace, used Rituxan, a drug manufactured primarily to treat rheumatoid arthritis, off-label to treat her rare disease: Immune Thrombocytopenia.

She is currently in remission.

The OPEN Act would unlock a new world of potential treatments – it would put them “on-label.” Imagine if Rituxan was an FDA-approved treatment for Immune Thrombocytopenia.  

Candace would have the option of taking a drug with the comfort of knowing that the FDA has approved it to treat the disease that she is facing.   The OPEN Act also has the potential to unlock countless cures for other patient suffering from rare diseases.  

My legislation opens the door to the development of hundreds of safe and effective treatments for rare disease patients, which could potentially be reimbursed through insurance coverage.

In addition to the hope that the OPEN Act could provide to patients, there would also be an accompanying surge in biotechnology jobs and investment.

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.

Bilirakis represents Florida’s 12th Congressional District and has served in the House since 2007. He sits on the Veterans’ Affairs Committee and on the Energy and Commerce Subcommittee on Health.