Gibson Shannon, Raziee Hamid R, Lemmens Trudo
World Med Health Policy. 2015 Mar;7(1):3-27. doi: 10.1002/wmh3.131.
Pharmaceutical research and development is increasingly focused on niche markets, most notably treatments for rare diseases and "personalized" medicine. Drawing on the results of a qualitative study of 34 key Canadian stakeholders (including drug regulators, funders, scientists, policy experts, pharmaceutical industry representatives, and patient advocates), we explore the major trends that are reportedly contributing to the growing interest of the pharmaceutical industry in niche markets. Informed by both these key informant interviews and a review of the relevant literature, our paper provides a critical analysis of the many different-and sometimes conflicting-views on the reasons for and extent of the shift toward niche markets. We consider some of the potential advantages to industry, as well the important implications and risks that arise from the increasing pursuit of niche markets and pharmacogenomics. While there are many potential benefits associated with targeted therapies and drug development for historically neglected rare diseases, niche market therapies also present evidentiary challenges (e.g., smaller clinical trials and enrichment strategies) that can make approval decisions difficult, and uncertainties remain around the true benefits of many therapies.
制药研发越来越关注细分市场，最显著的是针对罕见疾病的治疗和“个性化”药物。基于对34位加拿大关键利益相关者（包括药品监管机构、资助者、科学家、政策专家、制药行业代表和患者权益倡导者）的定性研究结果，我们探讨了据报道促使制药行业对细分市场兴趣日益浓厚的主要趋势。基于这些关键信息提供者访谈以及对相关文献的回顾，我们的论文对关于向细分市场转变的原因和程度的许多不同且有时相互冲突的观点进行了批判性分析。我们考虑了对行业的一些潜在优势，以及因越来越多地追求细分市场和药物基因组学而产生的重要影响和风险。虽然针对历史上被忽视的罕见疾病的靶向治疗和药物开发有许多潜在益处，但细分市场疗法也带来了证据方面的挑战（例如，规模较小的临床试验和富集策略），这可能使批准决策变得困难，而且许多疗法的真正益处仍存在不确定性。
