Today, the Centers for Medicare and Medicaid Services (CMS) finalized a key component of the Inflation Reduction Act\u2019s Medicare Drug Price Negotiation Program (MDPNP).<\/p>\n
Unfortunately, today\u2019s 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.<\/p>\n
\u201cThe hope of millions in our country depends on continued research into new and better therapies,\u201d said NORD\u2019s President and CEO, Peter L. Saltonstall. \u201cHistory tells us without the right incentives, rare disease therapies are not developed, which is why the Orphan Drug Act was created 40 years ago,\u201d adds Saltonstall.<\/p>\n
\u201cWe don\u2019t want to lose the tremendous progress that\u2019s 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,\u201d commits Saltonstall.<\/p>\n
A bright spot in today\u2019s 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.\u00a0 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.\u00a0 NORD applauds the aspect of CMS\u2019 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.<\/p>\n
While certain aspects of today\u2019s 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.<\/p>\n","protected":false},"excerpt":{"rendered":"
Today, the Centers for Medicare and Medicaid Services (CMS) finalized a key component of the Inflation Reduction Act\u2019s Medicare Drug Price Negotiation Program (MDPNP). Unfortunately, today\u2019s guidance threatens vital innovation … <\/p>\n