A ‘fair’ price for rare disease cures: Payers, physicians, and patients weigh in

June 18, 2020
Various alphabetized medical records

This Pink Sheet / Informa Pharma Intelligence article discusses findings from research by Charles River Associates that explores diverse perceptions about pricing for high-cost therapies – including curative cell and gene therapy – among various stakeholder groups. The research finds that there is a wide disparity in the prices that US payers, physicians, and patients consider to be “fair” for one-time curative therapies and therapies used to treat rare diseases.

On average, physicians report that a price of $852,000 is fair for curative treatments, while payers believe an average of $693,000 is fair and patients point to a price of $381,000 as fair for these therapies. Findings from the survey, reflect responses from 50 pharmacy and medical directors from large insurers, health plans and pharmacy benefit managers, 164 physicians who treat rare diseases and cancer, and 149 patients and family members or caregivers of patients with rare diseases and cancers.

In the article, Andrew Parece and Matthew Majewski note that the survey “breaks new ground by providing an understanding of the key factors that drive stakeholders’ perceptions of whether prices are considered fair” for high-cost innovative drugs. The survey also enables a comparison across the research cohorts – payers, physicians and patients – regarding which factors are of particular importance to each group. The authors note that “perceptions of fairness in general are not currently considered by many stakeholders when deciding how to manage or utilize a product but may become more relevant as the concept is brought to light.”