Breakthrough Care Comes at a Cost

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Gene therapy has captivated the oncology treatment community for years. After promising results in clinical trials, the first generation of gene therapies are being introduced to the market following Food and Drug Administration (FDA) approval. The FDA recently approved Kymriah (tisagenlecleucel, Novartis), a chimeric antigen receptor T (CAR-T) therapy designed to treat young adults diagnosed with relapsed or refractory acute lymphoblastic leukemia.1 Clinical trial results showed an overall remission rate of 83% within 3 months of treatment with Kymriah for this vulnerable patient population.2

Although much excitement has surrounded the FDA’s decision to approve the first CAR-T therapy, many questions remain. The one-time therapy carries a price tag of $475,000, which has caused some analysts to suggest that the market might not be ready for the payments required by these drugs. In a blog post titled “Gene Therapy Holds Great Promise, but Big Price,”3 Steve Miller, MD, MBA, chief medical officer of Express Scripts (St. Louis, MO), wrote that “gene therapies will require payment and patient care systems which are as novel as the medications themselves. … As these life-saving and revolutionary treatments continue to be developed, it is up to payers, pharma companies, and policymakers to unite and ensure they reach patients.”

Journal of Clinical Pathways spoke with Dr Miller about his blog post, and how he sees future payment models adapting to an influx of costly new therapies as they enter the marker.

Your blog post alluded to the FDA’s first major gene therapy approval as a major challenge for the current health care system. What are some of those financial challenges?

The new CAR-T therapy for childhood leukemia obviously carries a big price tag of $475,000. This will not be the biggest challenge, but it is just the beginning of gene and cell therapy coming to the marketplace at what are going to be some extraordinary high prices. In this particular case, you have a very lethal condition that is associated with large spending already, because what we spend for chemotherapy and stem cell transplants for these children is often up to $400,000, and the survival rate is still below 50%. 

In this particular case, you are going to have a product that is priced at a premium, but it is a well-deserved premium because the survival rates go up to about 80%. And so, it is an exciting breakthrough—a movement in the right direction to curing more kids—but when you see prices of products that are upwards of $500,000, it gives the payers a great pause.



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