Following a recent study on non-oncology orphan drug pricing in France, CRA’s Cécile Matthews was interviewed by reporter Francesca Bruce. The interview was included in an article in Pink Sheet, “Unlocking Orphan Pricing In France.” In the article, Matthews is quoted saying, “While unmet needs play an important role in the assessment, manufacturers should come with the best evidence possible and clear outline of benefits to patients otherwise they risk being turned down for reimbursement.”
The original study titled, “How Eligible Patient Numbers Affect Price in France for Non-Oncology Orphan Drugs,” looked at 22 non-oncology orphans approved in the EU between July 2012 and the end of 2019. It focused on non-curative treatments for chronic diseases, for which payers were more likely to scrutinize annual treatment costs (ATCs). ATCs were based on publicly available ex-manufacturer list prices at the time of launch.
In the conclusion of the article, Matthews is quoted stating, “We can expect the CT [Transparency Committee] to potentially restrict and even provide negative assessments where value is not clearly demonstrated versus competitors.”