Giving a voice to rare disease patients – The Hill
Searching through my Google Alerts and I came across a great article in The Hill about the 21st Century Cures Act and how it is giving a voice to rare disease patients all over the US.
Rare Diseases affect 1 in 10 Americans – that’s over 30 million people who are directly impacted. Doesn’t seem so rare anymore? Despite this, 95% of rare diseases do not have an approved treatment. Despite significant legislation facilitating financial incentives to drug companies who are working on orphan drugs, there is still a huge need. As of now, drug manufacturers do not have a major incentive to repurpose mainstream treatments for rare disease patients, as each disease impacts a small population.
“The 21st Century Cures Act is a comprehensive legislative package that will help speed up the development, discovery, and delivery of lifesaving cures and treatments for patients in need. During the early stages of working on 21st Century Cures, I became especially inspired by those fighting rare diseases.” – Rep. Bilirakis, The Hill
Rep. Bilirakis along with the support of over 150 rare disease advocacy groups, and bipartisan support in Congress, has introduced the OPEN Act a provision to 21st Century Cures. This act supports the repurposing of hundreds of drugs for rare diseases, resulting in new therapies as well as new biotech jobs and investment.
The OPEN Act and 21st Century Cures passed the House by a wide margin. It is now being considered by the Senate. It is imperative that 21st Century Cures gets signed into law.
“We have the opportunity now to truly make a difference in people’s lives. Let’s get this legislation across the finish line, and give hope to millions of patients and their families.” – Rep. Bilirakis, The Hill
So to our new President-Elect – this is really an opportunity to make healthcare great again.
World Orphan Drug Congress USA 2017 is uniquely poised to be the first gathering of the orphan drug industry, where the impact of a new President will be felt and the 21st Century Cures Act will have been signed into law. Don’t miss it!