Looking for an MVP: The Urgent Need to Enhance Affordable Access to Novel Therapies

In a historic decision in December, the FDA approved the first gene therapy utilizing CRISPR, an innovative type of genome editing technology. This significant moment means that the novel therapy Casgevy is approved for the treatment of sickle cell disease—which affects 100,000 Americans—in patients 12 years of age and older. We at AMCP are truly excited about the possibilities that gene-editing treatments such as CRISPR can provide to patients across the world.

Yet, despite the incredible promise these groundbreaking therapies hold, they also can present a massive cost burden for patients. Casgevy alone is expected to cost $2.2 million for a single course of treatment per patient. But this challenge isn’t insurmountable; from a policy standpoint, there are certainly things we can do to alleviate this issue. In fact, one approach that comes to mind is the Medicaid VBPs for Patients Act (MVP Act).

The MVP Act is a top federal policy priority for AMCP. Introduced by US Representatives Brett Guthrie (R-KY), Anna Eshoo (D-CA), John Joyce (R-PA), Jake Auchincloss (D-MA), and Mariannette Miller-Meeks (R-IA), this important bipartisan bill will enhance Medicaid patient access to new, high-cost therapies by modernizing the framework for value-based purchasing arrangements. As we have seen in the past, these are a critical tool for getting expensive treatments to the patients who need them. As new but expensive therapies continue to emerge as the best methods to treat rare diseases and other difficult conditions like HIV, cardiovascular disease, and retinal disease, value-based agreements represent the mostly likely way that underserved and vulnerable populations—ones who are disproportionately at risk for many of these conditions—will be able to access these potentially life-saving treatments.

Our country is currently seeing more and more life-saving treatments emerge at an unprecedented pace. We must ensure that there are policies in place to keep up with this rapid change. AMCP is proud to be a champion for steps like the MVP Act that will enable patients to have affordable access to the life-altering therapies they deserve. We urge Congress to pass this vital bill and look forward to continuing to partner with our members to advocate for policies that have the ability to tangibly improve the lives of millions of Americans.

Susan A. Cantrell, MHL, RPh, CAE
Chief Executive Officer
Published on Nov. 17, 2023