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Rare Patients Left Out of IRA?


NORD goes on record that IRA’s MDPNP will lead to stifling of innovation for rare disease community.

Closeup of the documents of the Inflation Reduction Act of 2022. The Unites States Senate passed the Inflation Reduction Act, the climate, tax and healthcare legislation, on Sunday, August 7, 2022. Image Credit: Adobe Stock Images/Tada Images

Image Credit: Adobe Stock Images/Tada Images

With the Centers for Medicare and Medicaid Services (CMS) releasing its revised guidance1 detailing the requirements and parameters of the Inflation Reduction Act's Medicare Drug Price Negotiation Program (MDPNP), NORD released its statement against the guidance.

In the statement, it said, “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.”

Specifically, NORD's President and CEO, Peter L. Saltonstall, pointed to the progress that has been made for rare disease patients since the Orphan Drug Act was created 40 years ago. "History tells us without the right incentives, rare disease therapies are not developed,” he said in the release, and NORD will continue working with Congress and the Administration to support those rare disease patients.

The CMS guidance does include a provision to include “additional opportunities for drug companies and members of the public to engage with CMS during the negotiation process on the selected drugs (e.g., through patient-focused listening sessions).”2

NORD noted its appreciation for this provision in its statement, noting that “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.”


1. Final Inflation Reduction Act Guidance Risks Stifling Innovation for Rare Disease Community. Quincy, Mass., June 30, 2023, PRNewswire.

2. CMS Releases Revised Guidance for Historic Medicare Drug Price Negotiation Program,Jun 30, 2023. CMS.gov

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