Who will pay for these miraculous new medical therapies?

Abstract

Many high risk, high reward medical therapies that use cutting edge science are proving themselves in clinical trials. But are health services ready to pay for them—and how? Marianne Guenot reports On 16 September 2024 Jimi Olaghere became the first person with sickle cell disease to reach the summit of Mount Kilimanjaro. The 39 year old could hardly believe it as he contemplated his achievement from nearly 6000 m above sea level. “I was in shock. Five years ago, I couldn’t get out of bed,” Olaghere tells The BMJ . People with sickle cell disease are discouraged from venturing to areas above 3000 m, let alone to the top of Africa’s highest peak. Olaghere was one of the participants in Vertex Pharmaceuticals’ trial of exagamglogene autotemcel (exa-cel, brand name Casgevy), which works by editing a patient’s blood stem cells before they’re re-injected. In the phase 3 trial 29 of 30 (97%) participants with repeated vaso-occlusive crises—episodes when “sickled” red blood cells get stuck in blood vessels and cause pain—reported no crises and no related hospital admissions for at least a year.1 Exa-cel isn’t the only success story. BlueBio’s lovotibeglogene autotemcel (lovo-cel, brand name Lyfgenia) is also used to treat patients with sickle cell disease. Vertex and BlueBio are the first companies to produce therapies using the much hyped CRISPR-Cas 9 gene editing technology (see box). Exa-cel was approved by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) for sickle cell disease and thalassaemia, and for sickle cell disease in the UK.2 These approvals are “really exciting developments in the field,” says Uta Griesenbach, professor of molecular medicine at Imperial College London, adding that gene editing therapies have a “very strong potential to be transformative.” Exa-cel and lovo-cel are just two examples of advanced …