1 in 10 Americans have a rare disease. 95% of rare diseases don’t have a single FDA-approved therapy
While individual diseases may affect only relatively small numbers of people, the total number of people with a rare disease is large. The total number of Americans living with a rare disease is estimated at between 25-30 million. (1) What’s even worse is that ninety-five percent of the 7,000 rare diseases have no FDA-approved treatment. Finding treatments for these diseases is difficult, but our guest has proven that it is possible.
To learn more about what can be done, and to share his personal story, I spoke with Dr. David Fajgenbaum. David is a physician, scientist, patient, author, speaker, husband, father and more. He’s also a pioneer in changing the way we go about developing drugs to treat people with rare diseases, and, in so doing, he’s managed to put his own condition into remission, and help many others as well.
David has a really powerful story and he’s on a mission, but frankly this cause needs more support. I want to ask you for your support in helping David and thousands of others be victorious over their rare diseases.
How can you do that? There are several ways, and we put together a variety of resources in the show notes including a link to a PDF that has a lot more information. Please grab the link to that in the show notes below.
In the PDF, you’ll find links to learn more about the Open Act that needs to be voted on by the US congress – David and I discuss that in the program. The Open Act is bipartisan legislation supported by 268 patient organizations including Genetic Alliance, Global Genes, National MPS Society, National Organization for Rare Disorders, and the Pediatric Cancer Foundation.
It passed the House in July of 2015, but was not signed into law – largely because of the political stigma associated with anything that could be perceived as benefiting big pharma – when in reality this is needed to help thousands of people. The bill was reintroduced in the House in February 2017 as HR 1223 and in the Senate in June 2017 as S 1509 but we need to put some pressure on to get this voted on. In the PDf, you’ll find links on how to find your senators and representatives, and I encourage you to call and write to them to encourage them to vote on, and pass the Open Act.
Another way you can help is by raising awareness of this. February 29 is Rare Disease day so we will be helping to spread the word of the importance of finding treatments for Rare Diseases. I’m also sure that this is something that could benefit from higher awareness in the mainstream media. This is covered a little bit but needs more attention. If you’re listening and you can help connect David with big names that can help raise awareness – people like Oprah Winfrey, Ellen, Michelle Obama, or others – please get in touch with me or David and let’s get him connected.
Huge thanks to Michelle Longmire and the team at Medable for giving me the opportunity to meet David at the Medable event in San Francisco.
Links and Resources:
- Connect with David Fajgenbaum: Web | LinkedIn | Twitter
- David’s Blog about Innovation for Rare Diseases
- David’s talk at Exponential Medicine Nov 2019 (video)
- David on Good Morning America (video)
- Link to David’s book: Chasing My Cure
- Read more and donate to Castleman Disease Collaborative Network (CDCN.org) | Twitter | Instagram | Facebook
ABOUT THE HOST
As an engineer and business leader, I've worked to develop, launch and scale innovative products and solutions that impact health and wellness. As an entrepreneur, I know firsthand what it takes to start a business, build a team, and compete in the global marketplace.
On Digital Health Today, I leverage my experiences to help great leaders and innovators tell their stories and connect to users, investors and other stakeholders.
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