Recently, NBC News’ Today did a profile on one of the first pediatric patients in the world to receive a CAR-T cell therapy for the treatment of acute lymphoblastic leukemia (ALL). In April 2012, the patient was able to enroll in a pediatric phase I clinical trial to receive the CAR-T cell therapy, CTL019 or tisagenlecleucel. During the process, the patient’s T-cells were separated from the bloodstream and genetically engineered to express a chimeric antigen receptor (CAR) — an artificial receptor that allows T-cells to recognize cancerous cells. Once returned to the body, the re-engineered cells enabled the patient’s immune system to fight off the disease. Ten years after receiving the treatment, she now celebrates a decade of being cancer-free.
Decades ago, the idea of altering a gene or cell to treat, or even cure a disease, seemed like science fiction. Today, that concept is a reality. Across the biopharmaceutical industry, companies are working tirelessly to discover and bring novel cell and gene therapies to patients. Tisagenlecleucel was approved by the U.S. Food and Drug Administration (FDA) for the treatment of ALL in 2017 and, as of May 2022, the FDA has approved an additional five CAR-T cell therapies.
CAR-T cell therapies stem from the broader group of cell and gene therapies which represent the latest breakthroughs in scientific treatment advances. Compared to conventional medicines, which can be produced and stored well in advance before they are needed, many cell and gene therapies are tailored to a person’s genetic and biological makeup and must be manufactured on a per-patient basis. While this may present numerous logistical challenges, biopharmaceutical companies are working to harness advancements in technology to make the manufacturing and administration process more streamlined and efficient.
The innovative nature of cell and gene therapies offers the potential to reduce the burden of many complex diseases and improve the quality of life for patients. By targeting diseases at the source (the cellular or genetic level) these treatments can provide long-term or potentially curative benefits. In fact, several CAR-T cell therapies have been shown to cure some children and adolescents with advanced leukemia, sparing the short and long-term side effects of previous treatments. Further, our most recent report on gene therapies finds that these transformative treatments hold the promise to significantly reduce the burden of treatment for many blood disorders (e.g., sickle cell disease, hemophilia and beta thalassemia) and generate savings throughout the health care system.
Looking ahead, we must ensure that patients who need these treatments can access them and that our payment system keeps pace with innovation. Given that cell and gene therapies typically utilize one-time administration, payers and manufacturers are exploring new ways to finance them, including innovative contracts that help spread costs or tie payment to outcomes. These approaches can help payers manage budget impacts while increasing focus on patient outcomes and access. To ensure continued innovation and access, we encourage policymakers to consider new avenues to update federal rules to help facilitate the adoption of innovative payment arrangements and ensure patients have affordable access to these transformative therapies.
Lastly, new developments in medical and fundamental science — including platforms for vaccines and cell and gene therapies — hold the promise of treating debilitating diseases such as cancer, diabetes and many rare disorders. Fulfilling this promise depends on a modern regulatory framework, including modernization measures described in the Prescription Drug User Fee Act Commitment Letter, that can serve patients by providing timely, science-based regulatory decisions. It is critical that Congress reauthorizes the PDUFA program before its expiration in September 2022.
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