Increased Patient Participarticipation in the Fight Against Rare Disease

Today the Institute of Medicine (IOM) issued a new report: “Rare Diseases and Orphan Products: Accelerating Research and Development”. The report received some press by Amy Dockser Marcus on the Wall Street Journal Health Blog, which features some statements by Thomas Boat, the chairman of the IOM committee that wrote the report, and Timothy Coté, director of the FDA’s orphan products development office. Marcus says the report calls for more aggressive efforts to tackle R&D for rare diseases, asking the question “Is it time to declare a national ‘war on rare diseases’ comparable to the famous ‘war on cancer’ that began in the 1970s?”

Certainly policymakers and Federal agencies such as FDA and NIH have ramped up efforts, from public hearings and a newly formed Congressional Caucus, but recent campaigns suggest that perhaps the biggest impact will be lead by YOU: patients, caregivers, and family members. While we need Federal support to create opportunities we might not otherwise realize, the rare disease community is not waiting around for something to happen – they are getting out and making a difference every day.

The IOM report calls for a strategy that would include seven key elements:

  1. Active involvement and collaboration by a wide range of public and private interests, including government agencies, commercial companies, academic institutions and investigators, and advocacy groups.
  2. Timely application of advances in science and technology that can make rare diseases research and product development faster, easier, and less expensive.
  3. Appropriate use and further development of trial design and analytic methods tailored to the special challenges of conducting research on small populations.
  4. Creative strategies for sharing research resources and infrastructure to make good and efficient use of scarce funding, expertise, data, biological specimens, and participarticipation in research by people with rare conditions.
  5. Reasonable rewards and incentives for private-sector innovation and prudent use of public resources for product development when the latter appears to be a faster or less costly way to respond to important unmet needs.
  6. Adequate organization and resources, including staff with expertise on rare diseases research and product development, for the public agencies that fund biomedical research on rare diseases and regulate drugs and medical devices.
  7. Mechanisms for weighing priorities for rare diseases research and product development, establishing collaborative as well as organization-specific goals, and assessing progress toward these goals.

That’s right; you read that correctly (I cut/pasted directly from the html version of the report) – at least until they update the error, the report calls for participarticipation! Let’s read it again:

Creative strategies for sharing research resources and infrastructure to make good and efficient use of scarce funding, expertise, data, biological specimens, and participarticipation in research by people with rare conditions.

Well, you are certainly participating. Let’s have a quick round-up of efforts in September (and early October) alone:

  • Vote 4 Hope Campaign to support The Global Genes Project in the Pepsi Refresh Contest finished 14th, with the potential to continue the fight for $250,000 by finishing in one of the top two spots again in October.
  • Rare diseases represented in the recent Health 2.0 conference in San Francisco by The Children’s Rare Disease Network alongside some other tremendous patient advocates such as Regina Holliday and e-Patient Dave. A note on Health 2.0 for those who are curious: Health 2.0’s original tag line of ‘user-generated healthcare’ contains the germ of a compelling idea—patients are using new tools to guide their own care. And now those tools are starting to integrate with the health care system. Doctors, patients, and health care organizations are all starting to use a new generation of online and mobile technologies which are fundamentally changing the way health care works.
  • The Change.org petition to “Tell Congress: Help Find Cures for Rare and Neglected Diseases”, urging our members of Congress to become part of the Rare and Neglected Disease Caucus.

YES! It’s safe to say that many of you, patients and caregivers, are driving change – your voice is being heard, so don’t give up. I urge you to continue to look for opportunities for participarticipation.

How?

  • Continue to track and support the efforts mentioned above. As discussed, Vote 4 Hope may continue in October and the Change.org petition needs your signature now! Moreover, the IOM report calls for Secretary Sebelius of DHHS to establish a national task force on accelerating rare diseases research and product development. We need to make sure that that happens.
  • Join the Society for Participatory Medicine (co-founded by e-Patient Dave, mentioned above).  The Society for Participatory Medicine is a 501(c)3 public charity devoted to promoting the concept of participatory medicine by and among patients, caregivers and their medical teams and to promote clinical transparency among patients and their physicians through the exchange of information, via conferences, as well through the distribution of correspondence and other written materials. Participatory Medicine is a cooperative model of health care that encourages and expects active involvement by all connected parties (patients, caregivers, healthcare professionals, etc.) as integral to the full continuum of care.
  • Support the launch of my non-profit, MarbleRoad, (I know, I know, shameless plug) which will provide support for people with complex illness. Proceeds from our launch event will be used to provide financial assistance for rare disease patients and community health center patients who seek specialty care services not offered by their primary care providers.
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~ by marbleroad on October 4, 2010.

2 Responses to “Increased Patient Participarticipation in the Fight Against Rare Disease”

  1. Howard – Although V4H will not continue into October, we raised a lot of awareness and had a tremendous outpouring of support. One more way to stay in touch with the rare community is to attend conferences, like Blog World Expo, where e-Patient Dave will be the keynote. The all day Social Health track will focus on Healthcare Communications and Social Media.

  2. Janis – Thanks for the update on V4H and Blog World Expo. There are so many amazing ways for everyone to get engaged right now, I love it!

    PS, for those interested, Janis is an incredible blogger and you can find her blog in the sidebar: “A Rare Mom – Sneak Peek at Janis”.

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