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Nov
19

Sign-on to Support the OPEN ACT: Encourage Companies to Repurpose Drugs for Rare Diseases!

Take action to support the OPEN Act (HR 5750), which helps remove drug development roadblocks and incentivizes the development of novel treatments for the benefit of all rare disease patients. Biopharmaceutical companies possess a wealth of scientific knowledge from drugs that have already been approved for common diseases, some of which may have the ability to treat rare diseases.

However, there are inadequate incentives in place for companies to study potential impact for rare disease patients. Some patients may elect to take an approved drug for off-label use, in the hopes that it could treat a rare disease. Off-label usage, however, is typically not studied or tracked for clinical effectiveness. In addition, off-label use is not reimbursed by insurance plans, creating undue and unnecessary financial burdens on patients.

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Nov
8

Rare Disease Caucus Election Results

During the November 2014 Midterm Elections, members of the Rare Disease Congressional Caucus lost 2 elections to the House:

  • Bruce Braley (D) IA-1
  • Carol Shea-Porter (D) NH-1

8 members did not seek re-election to the House:

  • Spencer Bachus (R) AL-6
  • Phil Gingrey (R) GA-11
  • Tim Griffin (R) AR-2
  • Rush Holt (D) NJ-12
  • Tom Latham (R) IA-3
  • Jon Runyan (R) NJ-3
  • Henry Waxman (D) CA-33
  • Frank Wolf (R)

To see complete election results of all Caucus Members & incoming Representatives read more

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Nov
4

Nov. 13 Caucus Briefing

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Rare Disease Legislative Advocates and EB Research Partnership in coordination with Rare Disease Congressional Caucus Co-Chairs
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing on

“Creating Economic Incentives to Spur the Development of Treatments for Ebola and Other Life-Threatening Rare Diseases”

Thursday, November 13, 2014
12:00 noon – 1:30 pm
Rayburn House Office Building, Room: B-318

Take Action

Sign-on to Support the OPEN ACT: Encourage Companies to Repurpose Drugs for Rare Diseases!

Take action to support the OPEN Act (HR 5750), which helps remove drug development roadblocks and incentivizes the development of novel treatments for the benefit of all rare disease patients. Biopharmaceutical companies possess a wealth of scientific knowledge from drugs …

Action Needed: Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care

Rare Disease Patients and their families will be dramatically impacted by the Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care recently released for comment by the Office of Human Research Protections. We are writing to …

Public Comment needed on two model insurance regulations

Recently, the NAIC circulated for public comment two model insurance regulations – one relating to the individual market (both on and off exchanges) and the second relating to small group coverage. Each draft model proposes guidance on a range of

Huntington’s Disease Action Alert

The Huntington’s Disease Society of America is asking advocates to contact their Members of Congress to expedite Social Security Disability Insurance for patients upon diagnosis.

Huntington’s Disease is a rare, fatal, genetic neurodegenerative disease that progressively causes total physical and …

Alliance for a Stronger FDA

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