RDAC Position
Gene therapy holds tremendous potential to improve treatment and potentially cure many rare genetic diseases. The RDAC supports access to and coverage for gene therapy for Utah’s rare disease community and our healthcare system at large. Engagement with key stakeholders—including patients, providers, payers, manufacturers, and policymakers—is vital to equipping Utah’s healthcare system, and the many children and families it serves, to realize the tremendous potential of gene therapies for rare diseases.
The RDAC further encourages Utah’s Medicaid Program to explore innovative payment approaches, such as value-based payment arrangements, that could facilitate availability and utilization of life-changing treatments and, in some cases, cures for those with rare diseases.
Issue
Gene therapy is a new generation of medicine where a functioning gene is delivered to a patient to treat and potentially cure a genetic disease. Through gene therapy, the underlying cause of a genetic disease can be targeted and corrected, potentially with a one-time treatment.
Gene therapy holds tremendous promise for the estimated 1 in 10 Utahns, or an estimated 350,000 women, men and children in the state, who are afflicted by a rare disease. If you don’t yourself have a rare disease, you likely know a family member or friend with a rare disease—whether it is an immune deficiency, a rare cancer, or a genetic disorder. Rare diseases are often debilitating, life-threatening and lack alternative treatment options.
Unlike traditional medicines or therapies, which require frequent administration and focus on managing symptoms, gene therapy is designed as a potential one-time treatment aimed at targeting the underlying cause of a disease at the cellular level.
Gene therapy could deliver transformational improvement in health and quality of life for Utahns and their families and caregivers, as well as provide substantial value for Utah’s healthcare system and communities.
Utahns Living with Rare Diseases: Eliminate or reduce clinical burden of disease, improve quality of life and increase life expectancy.
Utah’s Families and Caregivers: Alleviate the emotional and physical burden of care, enabling work and socialization and facilitating family relationships.
Utah’s Healthcare System: Reduce the need for chronic treatment, lower occurrence of disease-related complications and decrease healthcare utilization.
Utah’s Communities: Contribute to longer, healthier and more productive lives, with increased contributions to schools, workplaces, our economy and other aspects of Utah’s local communities.
Utah’s Economy: Reduce direct and indirect costs of rare disease to Utah’s economy, including by decreasing chronic care costs and disability support, while increasing economic contributions from rare disease patients who are freed from symptoms. It’s estimated that the total economic burden of rare disease in the U.S. is over $966 billion.
While Utah may only comprise a portion of that economic burden, each year an estimated 150 Utahns born are diagnosed with a rare disease, resulting in significant healthcare utilization and economic impact. Gene therapies could benefit Utah’s healthcare system in transformative and sustainable ways, offering potential to avoid years—and potentially lifetimes—of burden associated with chronic disease management for severely debilitating and life-threatening diseases.
To illustrate, consider Duchenne muscular dystrophy (DMD) and hemophilia. In the U.S., the total annual burden of illness per DMD patient is approximately $121,000. Twenty-seven percent of caregivers said they reduced working hours or stopped working completely to care for a relative with DMD. For hemophilia, the overall lifetime cost of treating moderately severe to severe hemophilia ranges from $19 million to $23 million per patient. Underemployment or part-time employment as a result of American’s living with hemophilia is estimated to cause almost $4 million in societal losses per year.
Gene therapies that treat the underlying cause of DMD, hemophilia and other rare diseases could help Utah avoid these significant costs. Importantly, despite the upfront costs of gene therapies, projections indicate that they will not threaten health system financial sustainability. Despite an expected increase in approved therapies, projected 2030 gene therapy spending in the U.S. is only 0.2% of total healthcare spending.
Supporting Access to Gene Therapy
There are approximately 7,000 known rare diseases and 95% of them have limited or no treatment options. Fifty percent of people affected by rare diseases are children. With more than 500 gene therapies in clinical development, all Utahns could benefit from improved access to these potentially life-saving cures and treatments for their children and families.
Because Medicaid and Medicare cover a significant share of those with disability status, these payers are expected to be the prominent payers for gene therapies. As more and more innovative gene therapies are discovered for rare diseases, Utah’s Medicaid Program, along with Medicare, has an opportunity to develop innovative policy to ensure children with debilitating and life-threatening conditions can access these potentially transformative therapies.
One barrier for State Medicaid Programs, in particular, is the actuarial risk of paying for gene therapy treatments. Compared to traditional chronic therapies, gene therapy treatment comes with large upfront costs, which are counterbalanced by significant, potentially life-long reductions in healthcare utilization and increases in productivity. Anticipating the potential budget impact of such new therapies on Medicaid programs is therefore of utmost importance. Consideration should be given to the need for sufficient annual appropriations and the use of innovative payment approaches, such as annuity or pay for performance contracts with gene therapy manufacturers.
Stakeholders from across the healthcare continuum are exploring alternative, innovative approaches to paying for these upfront costs while realizing the long-term benefits of treatment with gene therapies. Currently, 12 state Medicaid programs have sought and gained approval from the Centers for Medicare and Medicaid Services to enter into innovative payment arrangements with gene therapy manufacturers. Utah is not one of them.
Citations
For more information about PBM reforms implemented in other states, see: https://insidesources.com/rein-in-profiteering-by-drug-industry-middlemen/ article by David Balto, April 11, 2022.
https://nhjournal.com/pezzillo-the-real-cause-of-the-high-price-of-prescription-drugs-pbms/ article by Rich Pezzillo, March 24, 2022.
https://www.pbmaccountability.org/ accessed on September 6, 2022
EveryLife Foundation. The National Burden of Rare Disease Study. February 25, 2021. https://everylifefoundation.org/burden-study/
EveryLife Foundation for Rare Diseases. Newborn Screening – Utah. https://everylifefoundation.org/states/utah/
Landfeldt, Erik, et al. The burden of Duchenne muscular dystrophy: an international, cross-sectional study. Neurology 2014;83(6):529-536
Rind et al. Valoctocogene Roxaparvovec and Emicizumab for Hemophilia A: Effectiveness and Value; Evidence Report. Institute for Clinical and Economic Review, October 16, 2020. https://icer.org/wp-content/uploads/2020/10/ICER_Hemophilia-A_Evidence-Report_102620.pdf. Accessed May 2022. Li et al. Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model. J Med Econ. 2021;24(1):363-7
Chen, S. Economic Costs of Hemophilia and the Impact of Prophylactic Treatment on Patient Management. American Journal of Managed Care. Am J Manage Care. 2016;22:S126-S133
Young, M., et all. Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues. https://doi.org/10.1016/j.drudis.2021.09.001.
