A major issue with gene and cell therapies is the upfront cost. While these treatments can be life changing, they are expensive and costs can be as high as $4.25 million for treating one patient.
So far, this has limited access to these therapies, particularly in older or resource poor groups eligible for Medicare or Medicaid in the US.
The Cell and Gene Therapy Access Model, run by the Centers for Medicare & Medicaid Services (CMS), was first mentioned as part of Biden’s executive order “Lowering Prescription Drug Costs for Americans” in February 2023. Following on from this, the Biden-Harris Administration announced early this year that sickle cell disease would be the first focus of the model.
The idea of the model is to allow people with sickle cell disease to be treated with advanced therapies who would otherwise not be able to access them due to the cost. If the model works for sickle cell, then more such therapies and disease areas may be added to the scheme going forward.
Companies and US states can decide whether or not they want to participate in the model. The costs will be lower for providers through this scheme, but companies will have the chance to treat more patients with their therapies overall.
This week it was announced that Bluebird Bio and Vertex Pharmaceuticals, both developers and manufacturers of gene therapies for sickle cell disease, have agreed to participate in the scheme.
The Model will launch in early 2025 with US states having a year to decide whether to join the scheme. Government funding is available to help offset the cost of the therapies to the providers and companies that join the scheme have to agree to a ‘pay for performance’ type model where payments are linked to patient outcomes.
“Cell and gene therapies hold significant potential to improve patient outcomes and transform lives, ultimately reducing long-term health care spending,” said Deputy Administrator and Director of the CMS Innovation Center Liz Fowler, in a press statement.
“However, due to the high costs, these therapies can pose challenges to state budgets. This model will afford state Medicaid agencies more budget predictability while helping improve access to these innovative therapies for people with Medicaid with sickle cell disease.”
Tackling sickle cell disease with advanced therapies
Sickle cell disease is a relatively common genetic condition impacting the red blood cells that is very painful and can lead to many health complications including stroke, difficulty breathing, acute chest syndrome, or chronic organ damage. It mostly affects Black and Hispanic people and until recently treatment options were fairly limited.