MACPAC'S MISSION IS TO ENSURE ACCESS. NOT DENY IT TO OUR MOST VULNERABLE!

RAAP Set to Fight MACPAC Efforts to Restrict Access to Rare Medicines in Medicaid

WASHINGTON, Jan. 31, 2023 /PRNewswire/ -- On Friday, January 27^th, the Medicaid and CHIP Payment and Access Commission (MACPAC) voted to recommend that Congress permit states to restrict and delay access to therapies approved under the accelerated approval pathway. The U.S. Congress already mandated, as part of the Medicaid rebate agreement, that patients must have access to therapies at FDA approval. This includes medications under the accelerated approval pathway.

MACPAC's recommendation completely undermines clear congressional intent that protects patient access in exchange for manufacturer discounts. RAAP reminds MACPAC that its mission is to advise the Congress on payment structures to ensure access to care for covered beneficiaries, not make recommendations that hamper that access.

"Currently, there are already significant delays in access to rare disease therapies in Medicaid," explains Michael Eging, Executive Director of the Rare Access Action Project, a non-profit focused on rare patient access. "A rare therapy can face significant delays due to state Medicaid reviews that already range between 90 days to 24 months or more. MACPACs recommendation would continue this trend of delay at the expense of rare patients who rely on the program for their treatment and care."

As was noted by commissioners during the debate over the provision, the recommendation would harm patient access to therapies. This MACPAC approach simply does not align with the intent of the Accelerated Approval Program.

Accelerated approval therapies are critical to rare patients because they often rely on new targets for clinical research, and smaller patient populations require innovative thinking by researchers, and the Agency. However, the accelerated approval pathway is not an experimental approval, as asserted by MACPAC in their recommendation. It is a rigorous approval that requires the FDA to find a drug is safe and effective for its intended use. Accelerated approval simply permits FDA to accept a different type of data from traditional drug approval.

RAAP will continue our efforts to educate stakeholders as well as directly engage MACPAC and Congress on the critical importance of both Medicaid providing access to rare therapies at FDA approval, as well as implementing the accelerated approval pathway.

RAAP, the Rare Access Action Project, is a coalition of life sciences and patient stakeholders that explore creative policy solutions to address structural issues in access and coverage. Our priority is to help ensure rare disease patients have access to the care and treatments they need. RAAP is a registered 501 c4 non-profit organization.

SOURCE Rare Access Action Project (RAAP)