CMS negotiating sickle cell disease treatment prices for states

Sickle cell disease will be the first focus of a federal model designed to help state Medicaid agencies pay for expensive cell and gene therapy treatments.

The Centers for Medicare and Medicaid Services announced Tuesday that it will negotiate with the manufacturers of sickle cell disease treatments via the Cell and Gene Therapy Access Model, which the agency unveiled last February.

Related: Blue Cross first to open sickle cell gene therapy floodgates

Although CMS did not name the manufacturers, BlueBird Bio—whose $3.1 million list-price Lyfgenia gene therapy treatment for sickle cell disease was approved by the Food and Drug Administration in December—said in a news release earlier this month that it was in discussions with the CMS Innovation Center regarding the CGT model.

The FDA in December also approved Vertex Pharmaceuticals' $2.2 million Casgevy sickle cell disease treatment.

The treatments are intended as cures for the more than 100,000 people who suffer from sickle cell disease, the majority of whom are Black and on Medicaid. But drugmakers' high price tags have raised questions about the therapies' long-term fiscal impacts, particularly for financially strapped state Medicaid agencies. CMS estimated that hospitalizations and other care related to sickle cell disease cost the health industry almost $3 billion annually. 

"Gene therapies for sickle cell disease have the potential to treat this devastating condition and transform people's lives, offering them the chance to live healthier and potentially avoid associated health issues," CMS Administrator Chiquita Brooks-LaSure said in a news release.

"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.

Under the CGT model, regulators seek to balance therapies' upfront costs with their long-term benefits by working with drugmakers to create outcomes-based agreements, which will tie treatment price to patient health outcomes. BlueBird Bio inked a similar, three-year agreement with Blue Cross plans' Synergie Medication Collective contracting agency in December. The agreement links Lyfgenia's cost to patients' hospitalizations and emergency department visits post-treatment. 

CMS said its agreements with drugmakers will include additional pricing rebates, a standardized access policy and fertility preservation services. Side effects from sickle cell gene therapy treatments can include hair loss, infertility and lowered immunity.

State Medicaid agencies can decide in 2025 and 2026 if they want to sign on to CMS' deal with drug manufacturers, the news release said. The federal government will offer additional funding for states that opt into the model, that promote equitable access to these sickle cell gene therapies and that create care management programs for patients. 

"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," Liz Fowler, CMS deputy administrator and director of the CMS Innovation Center, said in a news release.