From blockbusters commercialization to niche marketing: a qualitative study of the challenges in bringing orphan drugs to market

Purpose This study aims to investigate the distinctive characteristics of orphan drug markets and identifies marketing practices that facilitate the diffusion of these therapeutic innovations. Design/methodology/approach A total of 34 semi-structured interviews were conducted with managers and experts involved in the marketing of orphan drugs in France. Findings Experts highlighted that the unique nature of orphan drug markets requires a rethinking of traditional marketing strategies. Effective diffusion depends on strong scientific and medical positioning, patient-centered approaches and targeted key account management. The need for specific communication initiatives to address key barriers – ignorance, poor practices and non-compliance – was also emphasized. Originality/value This study addresses a gap in empirical research on marketing practices based on a systematic literature study conducted in May 2025 across Emerald, NIH National Library of Medicine, Science Direct and EBSCO Business Source Complete databases using the keywords “orphan drug” and “Orphan Drug Act.” While most literature on orphan drug management focuses on strategic and R&D issues, this study sheds light on the marketing practices that support the diffusion and adoption of these therapies. The results suggest a prevalence-based continuum between two marketing logics: mass market and niche. At one end lie “ultra-rare” diseases that call for highly targeted, personalized strategies; at the other end are more prevalent rare diseases that align more closely with conventional market approaches.