Sponsorship and Funding for Gene Therapy Trials in the United States

March 3, 2020, Vol 323, No. 9, Pages 809-902


Research Letter
Sponsorship and Funding for Gene Therapy Trials in the United States
Zachary Kassir, BA; Ameet Sarpatwari, PhD, JD; Brian Kocak, BS; et al.
March 3, 2020
JAMA. 2020;323(9):890-891. doi:10.1001/jama.2019.22214
Since 2017, the US Food and Drug Administration (FDA) has approved 4 gene therapies: tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta) for cancer, voretigene neparvovec-rzyl (Luxturna) for a genetic form of blindness, and onasemnogene abeparvovec-xioi (Zolgensma) for spinal muscular atrophy. Although clinically transformative, they carry list prices of $475 000, $373 000, $425 000, and $2.1 million, respectively, for 1-time infusions. High development costs and the need to incentivize innovation are common justifications for such prices. However, public funding played an important role in each product’s invention and clinical testing.15