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.
