The Biden administration’s access model offers hope for gene therapies

In the field of cell and gene therapy, science continues to outpace commercialization. Take, for example, the first two gene therapies for sickle cell disease approved by the Food and Drug Administration, Vertex’s Casgevy (exagamglogen autotemcel) and bluebird bio’s Lyfgenia (lovotibeglogene autotemcel). Marketing authorization was granted for both in December 2023. But not until August this year 20 patients had started Casgevy treatment and, according to him, only four patients had started Lyfgenia Lead care manager.

The Biden administration announced this week that the manufacturers of Lyfgenia and Casgevy have entered into agreements with the Centers for Medicare and Medicaid Services to participate in the Cell and Gene Therapy Access Model, which allows CMS to negotiate outcomes-based agreements on behalf of state Medicaid . cell and gene therapy programs, starting with treatments for sickle cell disease. If successful, it could serve as a blueprint for other cell and gene therapies that have faced significant barriers to patient access.

Sickle cell disease is a group of congenital disorders of the red blood cells, called sickle cell disease because of their crescent-shaped shape. The disease changes the structure of hemoglobin, the molecule in red blood cells that delivers oxygen to organs and tissues throughout the body. As a result, it causes severe pain, anemia, organ damage and infections. People with the disease have a shorter life expectancy, on average more than 20 years. The most common type of sickle cell disease is sickle cell anemia.

The condition affects millions of people around the world. About 100,000 people in the United States live with the disease, which mainly affects people of sub-Saharan African descent.

In addition to painkillers to relieve symptoms and antibiotics to treat infections, hydroxyurea, a bone marrow suppressant that reduces red blood cell production, can be used to reduce the frequency of painful episodes. It has been in use since the 1980s. The FDA has approved several new therapies over the past decade, but none are as promising as Lyfgenia and Casgevy. These two new therapies can do just that reduce or possibly eliminate pain crises in patients. Nevertheless, it is difficult to get access to the Casgevy or Lyfgenia, which are worth $2.2 and $3.1 million respectively.

It’s not just gene therapies for sickle cell disease that face a huge range of barriers. All cell and gene therapy manufacturers face a challenging environment. The regulatory hurdles are enormous to begin with, but the post-approval manufacturing challenges are also significant. In addition, patient preparation, side effects, and side effect profiles can be intolerable. This can deter patients from signing up to start treatment. And then there is not much money to be made from the often small numbers of patients who qualify for treatment. So while the price of each therapy is eye-watering (some cost more than $1.5 million per treatment), there aren’t many patients using the therapies. This implies that generating income is not easy. In addition, payers concerned about high per-unit costs often impose coverage limitationsas the Tufts Center for the Evaluation of Value and Risk in Health describes.

Nevertheless, gene therapies in particular hold the promise of breakthrough improvements in health outcomes across multiple disease areas. Therefore, overcoming barriers to optimal patient access is critical.

In the case of sickle cell disease treatments, outcomes-based agreements are a promising avenue for patient access. Given that Medicaid is a dominant payer – 50% to 60% – in the field of sickle cell disease, it is critical to reach agreements between manufacturers and state Medicaid agencies.

The voluntary modelled by the Center for Medicare and Medicaid Innovation, aims to test outcomes-based agreements for cell and gene therapies. CMS says the model will increase access for patients, which in turn will likely improve their health outcomes, especially in areas of unmet need such as sickle cell disease. CMS Administrator Chiquita Brooks-LaSure said that “this is a new frontier in providing people with sickle cell disease access to potentially transformative treatments.”

The outcomes-based agreements for Lyfgenia and Casgevy will tie payments to whether each therapy improves health outcomes. Sickle cell disease could serve as one reasonable test case for OBAsbecause it is a disease whose outcomes can be monitored and measured relatively efficiently.

CMS plans to move the process forward by engaging all states participating in the Medicaid Drug Rebate Program to help them decide whether to participate in the model. Deputy Administrator and Director of the CMS Innovation Center Liz Fowler asserted that the “model will provide state Medicaid agencies with greater budget predictability.” The risk-sharing element through pay for performance is essential in light of the potential for significant expenditure if a significant number of patients choose to undergo the new treatments.

The model will be launched next month. Pending whether the next Trump administration decides to keep the model intact, states can choose to begin participating anytime between January 2025 and January 2026. After launch in 2025, this model could be expanded to include other cell and gene therapies.