21st Century Cures Discussion Document Released

The US House of Representatives Energy and Commerce Committee has released the 21st Century Cures discussion document. The 21st Century Cures initiative was created to investigate how the government can help “accelerate the discovery, development, and delivery of promising new treatments and cures for patients.”

This initiative is led by Rep. Fred Upton, who also chairs the Energy and Commerce Committee. Chairman Upton was one of the congressional guests at our 2014 Hope on the Hill event.

Accelerating Treatments

An estimated 70% of known diseases qualify as rare diseases, including spinal muscular atrophy. Fewer than 10% of those have an approved treatment. The cost and length of the drug approval process, combined with a high failure rate, can make it difficult to improve those statistics.

After a series of hearings, meetings, and roundtables, the committee identified several areas for improvement:

  • Incorporating patients into the regulatory process
  • Aiding young scientists who may help develop innovative cures
  • Streamlining the regulatory process
  • Modernizing clinical trials and medical product regulation

This discussion document summarizes their findings and proposed legislative solutions. As the title would suggest, the goal is to generate discussion that can further refine these proposals. Once this phase is complete, the committee will introduce formal legislation to the House of Representatives.

“These ideas represent an important milestone—a critical first step in a legislative process. Our solutions to boost cures and jobs are starting to take shape as we move from broad principles to legislative language,” Rep. Upton said in a statement released by the committee. “However, this document is far from the final product. Some things may be dropped, some items may be added, but everything is on the table as we hope to trigger a thoughtful discussion toward a more polished product. #Cures2015 is now underway. Together, we will get this done.”

How to Participate

Members of the SMA community who want to comment on the discussion document can send their feedback to [email protected].

Be sure to sign up for our e-news by entering your email in the “Sign up for the latest SMA information” box below. Once legislation is introduced, we’ll notify the community on critical next steps.

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