Policy & Advocacy   |   Policy Summit

Policy Summit’s Sickle Cell Disease Session Emphasizes Patient Access

Emily Nettesheim - October 05, 2020

The Policy Summit session on sickle cell disease focused on patient education and the policy reforms necessary to bring curative gene therapy treatments to the millions of patients suffering from SCD around the world.

Sickle cell disease (SCD) presents in patients as severe anemia, pain, and eventual organ damage. The simple genetic origins of this disease, a single mutation at the sixth position of the beta-globin chain of hemoglobin, make it a great target for gene therapy. However, there is much more to a successful gene therapy treatment than just the science used to develop it. The 2020 ASGCT Policy Summit session on SCD focused on patient education and the policy reforms necessary to bring curative gene therapy treatments to the millions of patients suffering from SCD around the world.

John Tisdale, MD., Senior Investigator in the Cellular and Molecular Therapeutics Branch of the NHLBI set the stage regarding how SCD is currently treated in the clinic. With an estimated 100,000 patients in the U.S., up to 90% of whom are African American, it is the most commonly inherited blood disorder in the country. Currently, the only curative treatment available is an allogeneic bone marrow transplant, which has shown to be highly effective in both children and adults. Patients who either do not qualify for a bone marrow transplant or elect against this treatment are left only with the treatment options of red blood cell transfusions and treatment of symptoms, or open clinical trials if they meet eligibility criteria. Tisdale noted that the number of specialty providers available to SCD patients is minimal, with some states not having any listed. The lack of availability of specialized care leads to high community costs as SCD patients are forced to go the emergency room when they have bouts of pain, and often receive unnecessary tests during the diagnostic process. Clinical trial enrollment also suffers, as primary care physicians may not inform their patients that they have the option to participate.

One organization working to engage the sickle cell community through education is the Sickle Cell Disease Association of America (SCDAA), a non-profit organization that has served SCD patients for more than 40 years. Beverley Francis-Gibson, President and Chief Executive Officer of SCADD, echoed Tisdale’s comments about the lack of easily available specialized care, adding that a lack of patient education about gene therapy treatments and patient distrust in the medical system are also notable factors for low clinical trial enrollment. While most of the patients that Francis-Gibson speaks with are excited about the possibility for a cure, they are also skeptical and would often prefer to live with the disease they have had all their lives due to concern over the potential of being used as a “guinea pig” in clinical trials. This distrust in the medical system is rooted in historic mistreatment of the African American population at the hands of medical and research professionals. Therefore, a primary message from Francis-Gibson to developers working on gene therapy for SCD and clinicians providing trials was to engage with patients early in the process in order to build trust and understanding through transparency.

The panel also included Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, and Nick Leschly, President and Chief Executive Officer of bluebird bio. Both CRISPR Therapeutics and bluebird bio are among the companies that have developed potentially curative gene therapy treatments for sickle cell disease that are currently in clinicals trials, with promising results. Leschly mentioned that bluebird bio could be filing for a biologics license application as early as the end of next year. However, with promising clinical data come the questions on every excited patient’s mind: how much will it cost, and will my insurance cover it? Both CEOs acknowledged that the current health care system is not set up to handle the upfront cost of gene therapy for a large population of patients, especially for public payers (Medicaid and Medicare), as these therapies will be quite expensive—although less so than a lifetime of emergency room visits, blood transfusions, and current standard-of-care treatments. Leschly proposed a value-based payment-over-time system, which would allow insurers to pay for therapies over five years if the therapy continues to be effective. In this payment model, a patient would receive the therapy, and the payer would pay a certain amount per year until the cost of the therapy is paid off. However, if at annual points the patient does not meet predetermined outcomes, the insurer would stop paying for the therapy. This model makes the high upfront cost of gene therapy a smaller barrier for patients than a lump sum payment, and ties payment to the value/outcomes of the treatment. Challenges include that public payer systems are currently based on annual budgets, and there are regulatory and legal barriers to these novel payment models that are currently being evaluated.

Kulkarni and Leschly both called for continued discussion around policy corrections to encourage updated Medicaid and Medicare policies, ideally before the therapies are approved. Clarity and open communication between companies, regulators, and the health care system will be necessary to increase the speed at which gene therapy treatments for SCD will be accessible to the patients who need them most.

To learn more about sickle cell disease and how gene therapy can help, check out the new resources available from ASGCT’s Patient Education program.

Emily Nettesheim is a member of the ASGCT Communications Committee.

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