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Report from Rare Disease Week in D.C.

It has been an amazing week to come together with rare disease advocates from 48 states representing over 160 rare disease to advocate for policies supportive of our communities. We are so strong together – over 30 million Americans are affected by a rare disease – 50% of them children! 80% are genetic. And less than 5% have effective treatments.

Rare Disease Day at NIH underscored the promise of research – and the critical role patient organization need to continue playing in advocating for research to bring answers to our children.

There was universal acknowledgement that active advocacy and involvement by affected families was one of the most vital factors behind the successful passage of the 21st Century Cures Act last December. That historic legislation holds real promise to accelerate research and innovation in the treatment of rare diseases.

But much of the earlier progress could be dramatically reversed by the current Congress. Regardless of your personal politics, it is a fact that public policies made by the US Congress can either help or impede progress in research and new drug development. And proposals being debated to replace the Affordable Care Act present a major risk of a return to high risk pools for our children with exorbitant premiums, limited coverage, annual and lifetime caps, and even denial of any coverage.

Today was Rare Disease Lobby Day on Capitol Hill and as we visited over 230 Congressional offices, we had four “ASKS”

  1. Preserve protections for rare disease patients in any rewrite of the Affordable Care Act

  2. Fully fund the NIH and FDA – and protect them from across the board budget cuts and hiring freezes.

  3. Reauthorize the OPEN Act to provide incentives for drug companies to re-purpose existing drugs for treatments for rare diseases

  4. Join the Rare Disease Caucus to stay informed about emerging issues and concerns in the rare disease communities.

For anyone interested in learning where their state or member of Congress stands in addressing the vital needs of the rare disease communities – our children – check out the following:

NORD’s State Report Cards

Everylife Foundation’s Congressional Scorecard

Get involved! Speak up!

Your voice is needed!

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