High prices for new sickle cell gene therapies present a challenge despite their breakthrough potential.

• Officials are working to ensure that gene therapies for sickle cell disease are accessible to all.
• An estimated 100,000 Americans, predominantly Black people or people of color, are affected by a disease that can cause debilitating pain.
• The Biden administration will discuss price reductions for Medicaid treatments with Vertex Pharmaceuticals and Bluebird Bio.

The approval of two gene therapies for sickle cell disease has given hope to patients who suffer from the disease, which disproportionately affects Black people and people of color.

Officials must now determine how to make expensive treatments accessible fairly to all.

Michael Goodwin's life is unpredictable due to the crippling episodes of pain from his genetic blood disorder, sickle cell. This condition has forced him to leave his job and at times taken him away from his family.

"Sometimes, I spend 20 days in the hospital each month, which is a lot more than I used to when I was younger," said Goodwin, who is now 36 and has a son and a spouse.

He is reluctant to undergo the new one-time gene therapies because they necessitate extensive medical preparation, including chemotherapy, to prepare patients' bone marrow stem cells for extraction and editing.

While Goodwin is concerned about the cost of gene therapy, Casgevy lists for $2.2 million, and Lyfgenia lists for $3.1 million.

"Although I have insurance, I am still facing medical bills," he stated.

The approval of therapies in December was met with excitement as a solution to treat the disease. However, concerns about equitable access and the lack of infrastructure to administer the treatments have raised doubts about how many people will actually benefit from them.

Dr. Julie Kanter, director of the Adult Sickle Clinic at the University of Alabama at Birmingham, was not surprised by Goodwin's reluctance to seek treatment.

According to Kanter, who is also the president of the National Alliance of Sickle Cell Centers, even if we allowed everyone access to this therapy, only 10% of those affected by sickle cell would want it, which would still be too much for us to handle at the moment.

According to the Centers for Disease Control and Prevention, over 100,000 Americans have sickle cell disease, and between 50% and 60% of them are covered by Medicaid.

It will take time to increase capacity and establish facilities nationwide to treat patients in large numbers, as stated by Kanter.

"We hope that the establishment of the National Alliance of Sickle Cell Centers will enable us to enhance our centers' capabilities to provide better care for individuals with this disease, which we have been unable to do due to financial constraints," she stated.

High cost brings a new payment model

Officials are struggling to determine how to make costly new treatments accessible to thousands of Medicaid patients as they work to increase treatment capacity.

Kate McEvoy, executive director of the National Association of Medicaid Directors, stated that the new treatment is providing an opportunity to address medical conditions for which there have been unsatisfactory treatments. However, she emphasized that the high cost of the treatment is a concern, and state budgets cannot manage it on their own.

The University of Washington study determined that gene therapy treatments for acute sickle cell patients, priced at $2 million or less, would provide an acceptable value, outweighing the long-term medical and quality-of-life expenses. These patients often need multiple hospitalizations and blood transfusions, which can prevent them from working.

Greater access could be ensured by a lower price, concluded the researchers, which was closer to $1 million.

The Biden administration is set to commence negotiations with Vertex and Bluebird Bio in the upcoming weeks to secure discounts for state Medicaid plans, with payments tied to patient health outcomes. This move is part of the Centers for Medicare & Medicaid Services’ Cell and Gene Therapy Access Model, aimed at making new high-priced treatments more accessible. The administration has accelerated the implementation of the new payment demonstration program, starting in January 2025, following the approval of sickle cell treatments.

McEvoy stated that there are approximately 100 therapies in the FDA's pipeline that are in the advanced stage of application, making it a priority to develop strategies that will support Medicaid programs in covering these treatments.

The direct talks with sickle cell drugmakers come as large pharmaceutical firms like Johnson & Johnson and others are suing the Biden administration over the Inflation Reduction Act Medicare price negotiations, which began in February. These negotiations could result in significantly lower price offers on the first 10 drugs selected for negotiation.

Executives on Vertex's quarterly earnings call expressed confidence about the negotiation process for sickle cell disease treatment. They stated that discussions with individual state Medicaid agencies would help ensure wide access and address long-standing inequities of care in the sickle cell disease community.

Steve Arbuckle, Vertex executive vice president and chief operating officer, informed analysts that they are not waiting for the demo before granting access to patients with Medicaid coverage. The profile of Casgevy is so strong that they are discussing an outcomes-based agreement that focuses on whether a small number of patients may not respond.

Employers are taking note

Workplace health programs are facing challenges in funding the rising number of expensive novel treatments, according to Dan Mendelson, CEO of Morgan Health.

Employers are aware of the value of cell and gene therapies, but for smaller companies, the cost of one case can exceed the cost of insuring an entire population, and they may not know if the employee will continue using the therapy.

Small- and medium-sized businesses may benefit from new risk-sharing payment models being explored by Morgan Health to cover the increasing costs of specialty treatments.

Goodwin is insured under his wife's employer health plan. Although he's aware of the new sickle cell treatments, he hasn't examined the coverage provided by her plan because he's uncertain if they're suitable for him.

"If they could assure me that I wouldn't have sickle cell, I would do it immediately," he stated.

Vertex and Bluebird Bio are also focusing on educating doctors and patient communities about the advantages of their new treatments, aside from payment discussions.

Bluebird expects its first patient to begin treatment with Lyfgenia in the first quarter.