How does rare disease prevalence impact drug pricing in England?

February 4, 2022
Pharma production

This article was originally published in PharmaPhorum.

In this second article of a three-part series exploring pricing in Europe, CRA’s Life Sciences Practice team discusses the role that disease rarity plays in reimbursement decisions among payers in England, comparing assessments by NICE versus the NHS.

Decision makers in the healthcare industry including payers, especially those who rely on the use of cost-effectiveness analysis (CEA) to evaluate new drugs, are often criticised for failing to capture the holistic value of drugs in their health technology assessments (HTAs). The criticism is fair, as conventional assessment criteria for reimbursement and pricing decisions do not typically include comprehensive analysis of a drug’s total value beyond its clinical benefits. This presents several challenges for manufacturers of rare and orphan disease therapies specifically, given they often cannot fulfil conventional assessment criteria due to unique factors generally associated with these drugs, such as small target populations, limited safety and efficacy data, and lack of clinical trial comparators.

Our team at CRA conducted a recent analysis to determine how the value of disease rarity is captured in HTAs in England, whether led by The National Institute for Health and Care Excellence (NICE) or the National Health Service (NHS). We focused on orphan drugs for chronic conditions as their potential for significant and long-term budget impact often attracts greater attention among decision makers.

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