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#apaperaday: Spending on Targeted Therapies for Duchenne Muscular Dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled:  Spending on Targeted Therapies for Duchenne Muscular Dystrophy

Today’s pick is a letter to the editor from @JAMA_current by Bendicksen et al on the expenditure on approved dystrophin restoring drugs in the USA (elevidys, eteplirsen, casimersen, golodirsen and viltolarsen). DOI: 10.1001/jama.2024.2776

Authors point out that all approved dystrophin restoring treatments were approved based on surrogate endpoints, via the accelerated approval mechanism and that confirmatory data are not yet in because of delays with the confirmatory trials.

Authors here wanted to assess the costs of these drugs. They could not do this for Elevidys (too recent approval), and for viltolarsen as the company trading the drug is private so there is no public data available. So left are eteplirsen, golodirsen and casimersen.

Authors outline that the sales for these three ASOs went up from 7 million in 2016 to 879 million in 2022, with a total of 3.1 billion $ over 7 years. For medicaid and medicare the expenditure was 1.1 billion and 104 million respectively.

The majority of spending was for eteplirsen (2.6 billion). Authors discuss that these numbers are high and make an impact on the healthcare system, especially taking into account that efficacy has not yet been confirmed.

They point out >50% of treatments approved via accelerated approval from 2012-2021, do not yet have confirmatory data. This is of course not a good development as we do not want to expose patients to treatments that may not work or the healthcare systems to high unnecessary costs.

It is OK to have the accelerated approval pathway for rare diseases with unmet need, but there needs to be a better way to enforce timely presentation of confirmatory data. Note: we do not know if these treatments work or not – but confirmatory studies are close to completion.