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Journal Review: Comparison of the United Kingdom and United States approaches to approval of new neuromuscular therapies
Journal Review: Comparison of the United Kingdom and United States approaches to approval of new neuromuscular therapies

Many novel therapies are now available for rare neuromuscular conditions that were previously untreatable. Hereditary transthyretin amyloidosis and spinal muscular atrophy are two examples of diseases with new medications that have transformed our field. The United States and the United Kingdom have taken disparate approaches to the approval and coverage of medications, despite both providing incentives to develop therapies targeting rare diseases. The US requires less evidence for approval when compared with medications for common diseases and does not have a mechanism to ensure or even encourage cost-effectiveness. The Institute of Clinical and Economic Review provides in-depth cost-effectiveness analyses in the US, but does not have the authority to negotiate drug costs. In contrast, the UK has maintained a similar scientific threshold for approval of all therapies, while requiring negotiation with National Institute for Health and Care Excellence to ensure that medications are cost-effective for rare diseases. These differences have led to approval of medications for rare diseases in the US that have less evidence than required for common diseases. Importantly, these medications have not been approved in the UK. Even when medications meet traditional scientific thresholds, they uniformly arrive with high list prices in the US, whereas they are available at cost-effective prices in the UK. The main downsides to the UK approach are that cost-effective medications are often available months later than in the US, and some medications remain unavailable.

1) Understand the differences between the US and the UK in the approval process for drugs for rare diseases;
2) Understand and be able to explain to patients some of the reasons for high drug costs for new drugs in the US;
3)Be able to understand costs as well as benefits for new drugs for neuromuscular diseases, and to balance costs and benefits when prescribing these medications. 

The AANEM is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

The AANEM designates this enduring material for a maximum of 1 AMA PRA Category 1 Credits TM.  Physicians should claim only the credit commensurate with the extent of their participation in the activity. Credit expires 11/15/2024.

No one involved in the planning of this CME activity had any relevant financial relationships to disclose. 

Gary W. Gallagher, MD, Dustin Nowacek, MD, Olivia Gutgsell, MD, Brian C. Callaghan MD, MS.
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Expires on Nov 15, 2024
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