Rare Disease Community Brings Knowledge, Passion and Ideas to #RarePOV Tweetchat

Let’s Continue the Conversation on Rare Diseases

10.18.13 | By Stephanie Fischer

The cultural anthropologist Margaret Mead once said, “Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has.”

The #RarePOV Tweetchat yesterday was PhRMA’s first, a bit of an experiment.  As a rare disease patient, I was moved by the passion and energy of everyone who participated. The Tweetchat, which we expected to last for 30 minutes, went on for more than an hour due to the overwhelming and enthusiastic engagement.

My personal goal was to have #RarePOV trending on Twitter, to elevate the importance and awareness of rare disease outside of the small but committed band of advocacy organizations, patients and caretakers. We succeeded! #RarePOV was briefly a trending topic nationally during the chat, with 730 tweets between 4 p.m. and 6:30 p.m. And I am pleased to see the conversation continue, as participants reach out to each other to follow-up on comments.

A few takeaways and observations:

  1. Ensuring that both patients and doctors are informed and empowered would shorten the average time for diagnosis. @RemRunner shared an infographic on narcolepsy that depicts symptoms, helping to raise awareness that can lead to an accurate diagnosis more quickly, and @RareDiseases gave an example of a physician awareness campaign.
  2. There is some disagreement about whether identifying a disease as “rare” makes it harder to get the attention of the public and the biopharmaceutical industry. The key, as @AfternoonNapper noted, is to convey that “Collectively we’re not rare”.  Taken together, rare diseases affect 1 in 10 Americans.

    The “rare” designation is important to encourage the development of new treatments for rare disease, thanks to the incentives provided by the Orphan Drug Act of 1983.  PhRMA’s recent report on the 452 Medicines and Vaccines in Development for Rare Diseases demonstrates that biopharmaceutical research companies are not shying away from rare diseases, even though they can be more complex than more common diseases.

  3. Collaboration is the key to long-term success. Everyone has an important role to play, from individual patients and caregivers, to researchers, policymakers and payers. As @TargetCancer said, “Patient advocates/foundations must collaborate and leverage existing resources to accelerate #raredisease treatments”.  This reinforces the call to action from Sharon Terry of Genetic Alliance on PhRMA’s Conversations Forum for advocacy organizations to collaborate rather than compete by sharing infrastructure, research findings and best practices.

What did you take away from the conversation and how can we build on it? If you didn’t have a chance to participate, I encourage you to review #RarePOV on Twitter or Storify. It’s not too late to join the discussion – reTweet, reply, engage!

And feel free to add a comment to our “Conversations” forum about the questions that we should ask to raise awareness and drive action.

Yesterday’s discussion reflects the eagerness to share ideas to achieve the shared goal of having approved treatment options for rare disease patients.

As @RemRunner said during the chat, “We may be RARE, but we’ve got ROAR.”

Comments

Hide Comments

More On PhRMA — powered by PhRMApedia