According to the CDC, sickle cell disease affects 90,000 to 100,000 people in the United States. The disease occurs in about one of every 500 Black or African American births and one of every 36,000 Hispanic American births.[1] In December 2023, the FDA approved two novel gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease. These potentially life-changing therapies come with a hefty price tag of $2.2 million and $3.1 million. According to CMS, between 50% and 60% of people living with sickle cell disease are on Medicaid and the high price of these treatments may strain state Medicaid agencies’ budgets.[2]
The federal government has created a program called the Cell and Gene Therapy Access Model, the goal of which is to provide people with Medicaid who have rare and severe diseases access to potentially transformative treatments. The federal government will negotiate with the gene therapy manufacturers and then each state can decide whether they would like to participate and accept the negotiated, outcomes-based agreement. As part of accepting the negotiated rate, the state must agree to make it available to its Medicaid population.
The CGT Access Model is a multi-year, voluntary model for both states and manufacturers which will initially focus on gene therapy for sickle cell disease. Per CMS, they anticipate states to begin with a rolling start in January 2025. CMS released a Request for Application (RFA) to manufacturers on March 8, 2024 and anticipates releasing an RFA and Notice of Funding Opportunity (NOFO) to states in summer 2024. States may choose to participate in the model under a participation agreement with CMS by responding to the state RFA. States may apply for model funding by applying to the NOFO, but they are not required to respond to the NOFO to participate in the model. States may begin participation anytime between January 2025 and January 2026.[3] For more information, please view the CMS fact sheet or visit https://www.cms.gov/priorities/innovation/innovation-models/cgt.
Article by Kathy Clark, RN, BSN, CMCN, Vice President, Director of Managed Care. For more information about how this may affect your plan, please contact your Summit ReSources care specialist. The following sources were used as reference material for this article:
[1] What is SCD? CDC.gov. Accessed 3/14/2024. https://www.cdc.gov/ncbddd/sicklecell/documents/SCD-factsheet_What-is-SCD.pdf
[2]CGT Access Model Frequently Asked Questions. CMS.gov. Accessed 3/14/2024. https://www.cms.gov/cgt-access-model-frequently-asked-questions
[3] Cell and Gene Therapy Access Model. CMS.gov. Accessed 3/14/2024. https://www.cms.gov/priorities/innovation/innovation-models/cgt