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CMS Granted Waiver is Duplicative, Costly and Steals Time From Those Without Time

For Immediate Release
January 13, 2021
Contact: Mike Eging
[email protected]

Washington, D.C. –

On January 8th, CMS granted a waiver to the State of Tennessee that will allow Tennessee to exclude drugs from its Medicaid formulary but continue to keep automatic Medicaid rebates.

One of the key elements of the waiver provides unprecedented discretion for the state to delay access to FDA approved therapies, particularly those approved via the FDA accelerated approval process.   In 2012, Congress passed the Food and Drug Administration Safety Innovations Act (FDASIA). Section 901 of FDASIA amends the Federal Food, Drug, and Cosmetic Act (FD&C Act) to allow the FDA to base accelerated approval for drugs for serious conditions that fill an unmet medical need on whether the drug has an effect on a surrogate or an intermediate clinical endpoint specifically allowing access to these products faster given the serious unmet medical need.

This subsequent state level review creates a duplication of process that can significantly delay access to therapeutics for patients with serious conditions with continued significant unmet medical need.  Often times, no other therapies are available for these rare diseases and by very nature of the FDA accelerated review process these products meet the FDA standards guided by statute and intended to speed access to innovative therapies to patients with serious conditions with little or no therapeutic options.

Rare Access Action Project (RAAP), Executive Director, Mike Eging released this statement in response to Tennessee’s approved waiver:

Many rare disease patients are seeing hope in recent developments in disease therapies because the data and innovative technology are so compelling that they can be granted accelerated review.   Other patients see progress through cell and gene therapy, where we can now change the course of a disease, but the therapy, which is based on patient disease progress is very time sensitive.  In either case, time is of the essence.

We have 7,000 plus identified rare diseases and less than 600 approved therapies.  Forcing patients to wait for a therapy that has been reviewed and approved by the FDA for a second State level approval is a duplicative, costly and grossly irresponsible move to deny access.  Patients don’t have the luxury of time for such state delays,” Eging concluded.


RAAP, the Rare Access Action Project, is a non-profit founded by a coalition of emerging and pre-commercial rare disease companies and patient organizations working together to develop solutions to access challenges with Rare Disease therapies and care. Find us at