Standing Up for Patients with Rare Cancers: Why CCF Is Speaking Out

At the Cholangiocarcinoma Foundation (CCF), advocacy isn’t just about raising awareness, it’s about taking action to protect access to care and treatment for all those affected by this rare and aggressive cancer.

Recently, CCF joined other members of the Protecting Innovation in Rare Cancers (PIRC) coalition in signing two letters addressed to Dr. Mehmet Oz, Administrator of the Centers for Medicare & Medicaid Services (CMS). These letters express serious concerns about how proposed changes to the Medicare Drug Price Negotiation Program (MDPNP) could unintentionally harm patients living with rare cancers like cholangiocarcinoma (bile duct cancer).

Why It Matters

The MDPNP, introduced through the Inflation Reduction Act, is intended to make medications more affordable. However, the initial guidance for implementation—scheduled for the 2028 applicability year—raises several red flags for our community. The PIRC letters, which CCF strongly supports, call for CMS to consider the unique needs of rare cancer patients by:

• Protecting Research & Development
  Ensuring that the new incentive structures do not discourage innovation or the repurposing of existing drugs for rare cancers.
• Increasing Agency Oversight
  Urging CMS to make sure Medicare plans cover all necessary medications and that any utilization restrictions are grounded in sound clinical evidence.
• Clarifying Coverage Guidelines
  Calling for transparent rules around Medicare Part D drug coverage, formulary tiers, and how utilization management tools are applied.
• Monitoring the Program’s Impact
  Encouraging proactive evaluation of how the MDPNP and related redesign efforts affect drug access and prescribing patterns.
• Preventing Access Barriers
  Identifying and eliminating any delays or denials in access to critical medications for rare cancer patients.
• Creating Clear Communication Channels
  Establishing a formal process for patients and advocacy organizations to share real-time feedback about access barriers directly with CMS.

Our Commitment to You

Cholangiocarcinoma is already a difficult disease to diagnose and treat. The last thing patients need is a system that makes it harder to access the medications they depend on. By signing on to these letters, CCF is ensuring your voice is heard in critical national policy discussions that impact the future of rare cancer care.

We will continue to work alongside partners, advocates, and policy leaders to make sure the needs of our community are not overlooked. Because every patient deserves timely, evidence-based treatment, and every voice deserves to be heard.