Final Inflation Reduction Act Guidance Risks Stifling Innovation for Rare Disease Community

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Today, the Centers for Medicare and Medicaid Services (CMS) finalized a key component of the Inflation Reduction Act’s Medicare Drug Price Negotiation Program (MDPNP).

Unfortunately, today’s guidance threatens vital innovation for the thousands of rare diseases that lack FDA-approved therapies. CMS has missed a critical opportunity to assert that orphan drugs remain excluded from drug price negotiation until research into a second disease actually leads to a second FDA approval. As a result, NORD is deeply concerned drug companies may not study their drugs for additional rare diseases in an effort to prevent those drugs from becoming negotiation eligible. It is critical this research and development continues, given that more than 95% of rare diseases lack an FDA approved treatment.

“The hope of millions in our country depends on continued research into new and better therapies,” said NORD’s President and CEO, Peter L. Saltonstall. “History tells us without the right incentives, rare disease therapies are not developed, which is why the Orphan Drug Act was created 40 years ago,” adds Saltonstall.

“We don’t want to lose the tremendous progress that’s been made for rare disease patients. NORD looks forward to working with Congress to protect these critical orphan drug incentives. We will also continue to work with the Biden Administration to ensure the Inflation Reduction Act can best support people living with a rare disease,” commits Saltonstall.

A bright spot in today’s guidance is that CMS established clearer opportunities for meaningful patient engagement in the negotiation process by committing to organize patient listening sessions. This has been a long-standing priority for NORD, as patients are critical stakeholders and bring a unique perspective to the negotiation table.  Frequently, there is a dearth of published data about rare diseases, so patients and their caregivers are often the best and only experts to gather information related to rare disease treatments.  NORD applauds the aspect of CMS’ guidance as a critical component to ensuring the final negotiated price reflects the value a therapy brings to a person living with a rare disease and their caregivers.

While certain aspects of today’s guidance are deeply disappointing, NORD is committed to ensuring the Inflation Reduction Act helps meet the complex needs of the more than 25 million Americans living with a rare disease.