At The Life Raft Group we are thankful to have a strong and motivated member community.  Despite dealing with their own diagnoses, ongoing care and side effects, members often tell us, “I want to get more involved. What can I do to help?” Although there are always plenty of opportunities to assist with Life Raft Group operations (see our Volunteer Page: another important way to contribute is to be an advocate for the rights of patients and caregivers.

Advocacy can be an intimidating word that often mistakenly implies a level of education, polish and public speaking skills that can lead people to count themselves out. Don’t! There are so many ways to be an advocate, and as someone who only a year ago was a complete newbie herself (and has worked with brand new advocates since) I can tell you that absolutely anyone can do it.  The only requirement is a passion for bringing about change. As a GIST patient or caregiver, you are already more invested than you realize and possess the greatest weapon an advocate has—your story.

One great entry into the world of advocacy is the LRG’s annual trip to Washington, DC with the One Voice Against Cancer (OVAC) Lobby Day. See our website for a detailed article: ( OVAC provides comprehensive training and a supportive group atmosphere in which to meet with your state’s legislators and to let them know that as their constituent, supporting federal funding for cancer research is important to you.  Be on the lookout for dates for the next trip taking place in the summer of 2016.

Until then, take a moment to familiarize yourself with some of the currently proposed legislation of special importance to the rare disease community:

Name: The Patient Focused Impact Assessment Act  (PFIA) (S.1597)

Sponsoring Senators:

Roger Wicker (R-MS), Amy Klobuchar (D-MN), Michael Bennett (D-CO), Susan Collins (R-ME), Al Franken (D-MN), Johnny Isakson (R-GA)

What’s it About? – Strengthening the patient voice in the medical product development process.  The PFIA will require the development of a patient engagement assessment tool whose results would be included within the publicly disclosed data package of any approved drug.  Topics would include benefit/risk data, patient-preference data and the views of patients and other external experts on the application.  PFIA aims to keep the patient voice at the heart of the FDA’s review and development processes.

Name: The OPEN Act (S.1421)

Sponsoring Senators:

Orrin Hatch (R-UT), Amy Klobuchar (D-MN)

What’s it About? – The OPEN Act establishes an “Orphan Product Exclusivity Extension” which would provide an additional six months of market exclusivity for any drug when the sponsor company establishes that the repurposed therapy is designated to treat a rare disease.  95% of rare diseases still have no FDA-approved treatment and biopharmaceutical companies seldom consider repurposing already approved therapies to treat rare diseases because there is no economic incentive for them to do so.  The OPEN Act hopes to give them this incentive.

Name: 21st Century Cures Act (HR 6)


Chairman – Fred Upton (114th Congress), passed the House by a vote of 344-77 on July 10, 2015.

Next Stop:

On to the Senate, for a vote this fall.

What’s it About?

  • Removing barriers to increased research collaboration
  • Incorporating the patient perspective into the drug development and regulatory review process
  • Measuring success and identifying diseases earlier through personalized medicine
  • Modernizing clinical trials
  • Removing regulatory uncertainty for the development of new medical apps

You can write to your senators and representatives, expressing your support for this legislation. We will be happy to help you with suggested wording for your correspondence.

Another way to advocate is to raise awareness in your local media outlets. LRG members have written letters to the editor and have told their GIST stories as a way of advocating for support for GIST and other rare diseases.

To get the latest updates on GIST advocacy news, event and focus group opportunities, join the LRG’s advocacy mailing list. To subscribe, email Mildred Menos at