The Centers for Medicare and Medicaid Services plans to begin testing whether outcomes-based agreements might improve access to two new gene therapies approved for sickle cell disease.
Cell and gene therapies have struggled to gain traction commercially, given their million-dollar price tags and small patient populations.
The pilot program, known as the Cell and Gene Therapy Access Model, is expected to run from 2025 through 2035. It will initially focus on Vertex Pharmaceuticals and CRISPR Therapeutics’ sickle cell gene therapy Casgevy, which costs $2.2 million per dose, and bluebird bio’s gene therapy Lyfgenia, priced at $3.1 million per dose.
The model aims to help states better negotiate and implement outcomes-based agreements in which companies have to pay rebates if certain outcomes aren’t met. The pilot program could potentially open up treatment options in states that wouldn’t otherwise seek access. CMS estimates that about 50% to 60% of people with sickle cell disease are enrolled in Medicaid, and the total annual nationwide cost of sickle cell is about $2.98 billion per year.
“Overall, the model aims to reduce the burden to states and manufacturers of operating an OBA [outcomes-based agreements], while maximizing access to novel and transformative therapies for beneficiaries,” CMS said.
The agency on Friday launched a call for states to begin submitting applications to participate in the model.
As part of that announcement, CMS is requiring biopharma companies to financially support “a defined scope of fertility preservation services at no cost to beneficiaries who receive treatment within the model or to other payers,” CMS said. Cell and gene therapies can sometimes cause fertility issues.
That requirement is meant to test whether manufacturer payments, rather than beneficiary or Medicaid payments, for fertility preservation services would improve health outcomes.
“Specifically, manufacturer payment for fertility preservation services may yield learning that could inform state Medicaid agencies’ future decision-making regarding coverage for fertility preservation services in connection with gene therapy and the potential for contracting arrangements with manufacturers to fund the cost of treating adverse outcomes related to use of the manufacturer’s therapy,” CMS said.
Some states say they are not using outcomes-based models for gene therapies because of the complexity of negotiating with manufacturers, according to CMS, in addition to a lack of leverage stemming from few alternative treatments, coverage obligations, the burden of data collection and need to evaluate the therapies over multiple years.
CMS says it will take a central role in data collection and monitoring to facilitate the adoption and implementation of the outcomes-based agreements and related monitoring.