US policy aiming to expand access to gene and cell therapies for SCD
Pricing model to support 'quality, affordable' care for patients on Medicaid
Starting in 2025, the U.S. Department of Health and Human Services (HHS) will introduce a pricing model that aims to make new cell and gene therapies more accessible for people with sickle cell disease who are on Medicaid, the health program for lower income residents jointly funded by the federal and state governments.
“HHS is using every tool available to us to increase access to high-quality, affordable health care and lower health care costs,” Xavier Becerra, HHS secretary, said in an agency press release.
An estimated 100,000 people live with sickle cell disease in the U.S. The disorder disproportionately affects Black Americans, and the HHS estimates that at least half of all U.S. residents with sickle cell are insured by Medicaid.
A gene therapy and cell therapy for sickle cell were approved in late 2023
“While medical advancements bring us closer to cures, too many individuals with sickle cell disease and their loved ones still face challenges obtaining the care they need,” Becerra said.
The U.S. Food and Drug Administration approved two groundbreaking treatments for sickle cell late last year: the gene therapy Lyfgenia (lovotibeglogene autotemcel) and the gene-editing therapy Casgevy (exagamglogene autotemcel). HHS did not specifically state if Lyfgenia, Casgevy, or both are expected to be included in the policy’s pricing model.
Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is indicated for patients ages 12 and older with recurrent vaso-occlusive crises (VOCs), which are painful crises that occur due to blood vessel blockage. Lyfgenia, from Bluebird Bio, is approved for patients ages 12 and older who have a history of vaso-occlusive events (VOEs), such as VOCs, acute chest syndrome, and other sickle cell-related complications. Both therapies have been shown in clinical trials to substantially reduce the risk of VOCs and VOEs.
“Gene therapies for sickle cell disease have the potential to treat this devastating condition and transform people’s lives, offering them a chance to live healthier and potentially avoid associated health issues,” said Chiquita Brooks-LaSure, CMS administrator. The Centers for Medicare and Medicaid Services, or CMS, is the agency that oversees Medicaid.
“Increasing access to these promising therapies will not only help keep people healthy, but it can also lead to savings for states and taxpayers as the long-term costs of treating sickle cell disease may be avoided,” Brooks-LaSure said.
Policy based on agreements linking treatment payment to patient benefits
Under the policy, CMS will work with participating states and manufacturers to set up outcomes-based agreements (OBAs) for the groundbreaking new therapies.
Under an OBA, the price of a cell or gene therapy is determined on an individual basis based on how well it works for a specific patient. It’s akin to a money-back guarantee — the basic understanding is that Medicaid will only be paying full price for patients who experience substantial benefits from treatment, predetermined in the agreement.
The policy is a piece of the Biden administration’s Cell and Gene Therapy Access Model, a part of the administration’s broader effort to bring down treatment costs.
“The goal of the Cell and Gene Therapy Access Model is to increase access to innovative cell and gene therapies for people with Medicaid by making it easier for states to pay for these therapies,” said Liz Fowler, CMS deputy administrator and director of the CMS Innovation Center. “By negotiating with manufacturers on behalf of states, CMS can ease the administrative burden on state Medicaid programs so they can focus on improving access and health outcomes for people with sickle cell disease.”