Evaluating the Cell and Gene Therapy Access Model
Highlights
- Under a new CMS model, people with sickle cell disease can gain access to the latest, but otherwise too expensive, treatments.
- New cell and gene therapies can cure children and young adults with sickle cell disease of the deadly illness.
- As the evaluation lead of the model, Abt will help determine whether CMS-led negotiations of outcome-based agreements with drug manufacturers can manage long-term costs for CMS and State Medicaid Agencies while improving equitable access to care and the h
Modern cell and gene therapy treatments offer tremendous hope to those with chronic and rare diseases, in some cases curing the illnesses outright. However, these new treatments come at high upfront costs to which the current healthcare system is unaccustomed. The Centers for Medicare & Medicaid Services (CMS), via its Innovation Center, created the Cell and Gene Therapy Access Model. It allows CMS to negotiate with manufacturers on behalf of states to make high-cost specialty drugs available and affordable. It also holds the manufacturers accountable for delivering treatments that improve beneficiaries’ quality of life.
CMS contracted with Abt Global to evaluate the model at the beneficiary, state, and federal levels. Abt will lead the collection and analysis of primary data on CMS, State Medicaid Agency, and patient experiences and the acquisition and analysis of secondary data on costs and patient outcomes. To participate, patients must have a diagnosis for sickle cell disease, be eligible for cell and gene therapy, and be enrolled in Medicaid or their state’s Children’s Health Insurance Program (CHIP). The Innovation Center estimates that as many as 2,400 Medicaid and CHIP beneficiaries nationally could receive a gene therapy for SCD in the first six years of the Model.
Abt will conduct an evaluation that will help CMS determine whether their negotiations with manufacturers lowered cost and helped states better organize their response for taking on a new therapy. The Abt team will also look at what factors led to states’ and manufacturers’ willingness to participate in the model and evaluate the results of grants made to states to facilitate their participation in the model and promote equitable access to cell and gene therapy. Finally, Abt will look at the impacts of the model on access, spending, quality of care, and health outcomes. In this way, CMS will be able to guide future policy with respect to cell-based and gene therapy treatments.