The question of how to adequately capture the holistic value of orphan drugs (ODs) for rare diseases is the subject of much debate among industry stakeholders including manufacturers, clinical experts, health economists and payers. This is especially the case in markets using cost-effectiveness analysis (CEA) for their health technology assessments (HTA).
The problem is particularly relevant for manufacturers of ODs that may not be able to fulfil conventional assessment criteria but offer the intrinsic value of providing new treatments for rare and often severe diseases.
We set out to investigate how the value of disease rarity is incorporated into decision-making for ODs in England. In PMLiVE, we discuss our review of 47 non-oncology ODs that were approved by the European Medicines Agency (EMA) between 2006 and 2019. Reimbursement decisions and prices were collected or calculated from reports published by The National Institute for Health and Care Excellence (NICE) or National Health Service (NHS) in England. We focused specifically on exploring the price and access outcomes of orphan medications for chronic conditions. This type of drug often attracts greater attention from decision-makers due to the potential for significant and long-term budget impact.
The study focused on answering three key questions:
1. Is disease rarity a predictor of reimbursement?
2. Is there a relationship between disease rarity and the cost of treatment?
3. Are drugs for rare diseases disproportionately subject to commercial agreements?
