Aaron S. Kesselheim (
Benjamin N. Rome is a resident physician at Brigham and Women's Hospital.
Health Aff (Millwood). 2017 Feb 1;36(2):362-370. doi: 10.1377/hlthaff.2016.1340. Epub 2017 Jan 18.
To incentivize pharmaceutical manufacturers to invest in areas of unmet medical need, policy makers frequently propose extending the market exclusivity period of desired drugs. Some such proposals are modeled after the pediatric exclusivity patent extension program, which since 1997 has provided six additional months of market exclusivity for drugs studied in children. The most recent proposal would encourage rare disease research by providing six months of extended exclusivity for any existing drug that is granted subsequent FDA approval for a new rare disease indication. Yet the economic impact of such proposals is rarely addressed. We found that for the thirteen FDA-approved drugs that gained supplemental approval for a rare disease indication from 2005 through 2010, the median projected cost of clinical trials leading to approval was $29.8 million. If the exclusivity extension had been in place, the median discounted financial gain to manufacturers would have been $94.6 million. Median net returns would have been $82.4 million, with higher returns for drugs with higher annual sales. Extending market exclusivity would provide substantial compensation to many manufacturers, particularly for top-selling products, far in excess of the cost of conducting these trials. Alternative strategies to incentivize the study of approved drugs for rare diseases may offer similar benefits at a lower cost.
为了激励制药商在未满足医疗需求的领域进行投资，政策制定者经常提议延长所需药物的市场独占期。一些此类提议是仿照儿科独占性专利延期计划提出的，该计划自 1997 年以来为在儿童中进行研究的药物提供了额外的六个月市场独占期。最近的一项提议将通过为任何获得后续 FDA 批准用于新罕见疾病适应症的现有药物提供六个月的延长独占期来鼓励罕见病研究。然而，此类提议的经济影响很少被提及。我们发现，对于 2005 年至 2010 年间因罕见疾病适应症而获得补充批准的十三种 FDA 批准药物，批准前临床试验的预计成本中位数为 2980 万美元。如果独占期延长，制造商的中位数贴现财务收益将达到 9460 万美元。中位数净回报为 8240 万美元，销售额较高的药物回报率更高。延长市场独占权将为许多制造商提供大量补偿，尤其是对畅销产品而言，远远超过进行这些试验的成本。激励研究已批准用于罕见病药物的替代策略可能以更低的成本提供类似的好处。
