Muscle & Nerve — Comparison of the United Kingdom and United States approaches to approval of new neuromuscular therapies

A review of the different approaches the US regulatory body, the US Food & Drug Administration (FDA), pursues approving new drugs to treat rare neuromuscular diseases versus the UK regulatory body. The US requires less evidence of clinical trial efficacy for approving drugs for rare diseases versus drugs for common diseases. Also, the FDA does not set prices once the drug is approved. In contrast, the UK requires the same level of evidence for drugs for rare diseases as for common diseases and negotiates the price as part of the approval process. These differences mean that more drugs are approved for rare diseases in the US than in the UK. For drugs that are approved in the UK, they come onto the market later than in the US because the regulatory body needs additional time to negotiate the drug price. Drug prices are generally much higher in the US.