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Op-Ed by Michael Astrue: The FDA Returns to the Dark Ages

In subsequent years the FDA continued to use the accelerated drug regulations for some cancer indications, but quietly retreated for most other diseases. Accelerated approval is broadly popular among patient groups, and Congress has tried to push the FDA back on the right track multiple times since the promulgation of the 1992 regulations. In 1997 and 2012 Congress codified and expanded FDA’s accelerated approval authority, but the agency has not fully implemented those statutes.

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Senator Al Franken & Minnesota Governor Mark Dayton Team Up to Urge NIH to Re-institute Connective Tissue Study

Late last year, the National Institute on Aging, which is part of NIH, halted a long-running observational study of fibromuscular dysplasia and four other rare diseases, saying it was no longer collecting data or enrolling patients, and that the study’s goals had been met. Those affected by this decision argued that fibromuscular dysplasia’s cause or cure still isn’t known, and mobilized in an effort to get a clearer understanding of why the study had been ended. One of the affected participants, Kari Ulrich, set up an online petition asking the NIH to fully address the closure of the study.  This petition quickly gained popularity, and when printed out was a phonebook-size document containing names and comments of about 11,000 people. Their efforts were even documented in an article in The Wall St. Journal. Read More


Senator Elizabeth Warren calls for more FDA & NIH funding

EWarren massbio

During a speech at the Massachusetts Biotechnology Council, Senator Elizabeth Warren has called for more National Institute of Health funding. Warren also said the FDA should be given the funding resources to hire more qualified scientists to work with their industry counterparts in the drug approval process.

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Take Action

Sign-on Letter to CMS to Protect Incentives for Rare Disease Drug Development

Please consider adding your organization to the letter to Centers for Medicare and Medicaid Services (CMS) being circulated by Global Genes Project, encouraging the agency to revise its proposed rule on Medicaid “line extension” rebates and exclude orphan drugs from

Protect Medicare Part B funding

We are circulating a letter that thanks Californian congressmen and calls for their continued leadership in the effort to protect Medicare Part B funding.

If you are an organization that has ties to or is based in California please read the letter …

Sign on to protect the Orphan Drug Tax Credit

Stand with NORD and the entire Rare Disease Community to urge congress to reconsider the proposal to repeal the Orphan Drug Tax Credit.

“Repeal of this provision would squelch medical research and innovation. Repeal would remove one of the major …

HR 2725 – FDA SOS Act of 2013

The FDA Safety over Sequestration Act is bipartisan legislation that would exempt FDA user fees from sequestration.  The bill amends the Budget Control Act.  To sign onto the letter to support FDA SOS, contact Jenny Carey by Friday Dec. …

Alliance for a Stronger FDA

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