Invited Review: Comparison of the United Kingdom and United States approaches to approval of new neuromuscular therapies-Article

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This review article compares the approaches of the United Kingdom (UK) and the United States (US) in approving and providing coverage for new neuromuscular therapies for rare diseases. The US has a lower scientific threshold for approval of medications for rare diseases compared to common diseases and does not consider cost-effectiveness in its approval process. In contrast, the UK maintains a similar scientific standard for approving all therapies and requires negotiation with the National Institute for Health and Care Excellence (NICE) to ensure cost-effectiveness for rare diseases. The differences in approach have led to the approval of medications for rare diseases in the US with less evidence compared to the UK. Additionally, the US lacks mechanisms to control the costs of therapies, which has resulted in high list prices for approved medications. In the UK, medications for rare diseases are made available at cost-effective prices through negotiations between pharmaceutical companies and NICE. However, the UK approach can lead to delays in the availability of medications compared to the US and may result in some medications remaining unavailable. The article provides examples of specific neuromuscular therapies, such as those for hereditary transthyretin amyloidosis, spinal muscular atrophy, Duchenne muscular dystrophy, amyotrophic lateral sclerosis, myasthenia gravis, and Lambert-Eaton myasthenic syndrome, to illustrate how the different approaches have affected the approval, cost-effectiveness, and availability of these medications in the two countries.

Keywords

review article

United Kingdom

United States

neuromuscular therapies

rare diseases

scientific threshold

cost-effectiveness

NICE

medications

availability