Access to Affordable Medicines: Obligations of Universities and Academic Medical Centers

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Gene Therapy

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Over the last decade, the development and approval of remarkably effective therapeutic interventions, many based on technologies directed at the human genome, have altered the treatment landscape for patients facing a range of serious and life-threatening conditions. These novel therapies include chimeric antigen receptor T cells (CAR-T) for lymphoma, childhood leukemia, and multiple myeloma; antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA); and gene therapy for SMA, Leber’s congenital amaurosis Type 2 (LCA, a rare form of inherited blindness), beta thalassemia major, and hemophilia B. Advances not directed at the genome include drugs targeting the cystic fibrosis transmembrane conductance protein, which have transformed the lives of most patients with that disease.

These therapies are effective but also expensive. For example, the U.S. list price of tisagenlecleucel CAR-T cell therapy for childhood leukemia is $475,000; the first year of ASO therapy for SMA is $750,000 ($375,000 for each subsequent year); and gene therapy ranges from $850,000 for LCA (both eyes) to $3.5 million for hemophilia B. High prices are not unique to cell and gene therapies. Combination therapy for cystic fibrosis runs approximately $300,000/year, and median inflation-adjusted launch prices for novel self-administered cancer drugs increased more than 20% from $188,842 to $232,788 per year of therapy between 2017 and 2021. Prices for many of these therapies exceed accepted cost-effectiveness thresholds. Furthermore, even when therapies are cost-effective, high prices prevent many patients from accessing necessary drugs, whether through payers’ decisions not to cover them or through unaffordable copayments, resulting in worsened health outcomes. Patients who receive these therapies can experience financial toxicity due to the substantial out-of-pocket costs that typically accompany receipt of costly drugs. Finally, high prices impose burdens on all members of society through rising health insurance premiums, taxes, and the opportunity costs of forgone investment in other societal priorities.

Recommended Citation

Allison M. Whelan, et al., Access to Affordable Medicines: Obligations of Universities and Academic Medical Centers, 30 Gene Therapy 753 (2023).







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