Thomson Reuters Life Sciences Consulting, The Johnson Building, 77 Hatton Garden, London EC1N 8JS, UK.
Drug Discov Today. 2012 Jul;17(13-14):660-4. doi: 10.1016/j.drudis.2012.02.005. Epub 2012 Feb 17.
Orphan drug incentives have stimulated research into diseases with significant unmet medical need. Although the targeting of orphan diseases is seen by industry as an attractive strategy, there are limited economic data available to support its use. In this paper we show that the revenue-generating potential of orphan drugs is as great as for non-orphan drugs, even though patient populations for rare diseases are significantly smaller. Moreover, we suggest that orphan drugs have greater profitability when considered in the full context of developmental drivers including government financial incentives, smaller clinical trial sizes, shorter clinical trial times and higher rates of regulatory success. The data support the targeting of rare diseases as an important component of a successful biopharma R&D strategy.
孤儿药激励措施刺激了对有重大未满足医疗需求的疾病的研究。虽然制药行业认为针对孤儿病是一种有吸引力的策略，但目前可用的经济数据有限，无法支持这种策略的应用。在本文中，我们表明，即使罕见病患者群体要小得多，孤儿药的创收潜力与非孤儿药一样大。此外，我们还表明，在包括政府财政激励、较小的临床试验规模、较短的临床试验时间和更高的监管成功率在内的发展驱动因素的全面背景下，孤儿药具有更高的盈利性。这些数据支持将罕见病作为成功的生物制药研发战略的重要组成部分。
