Daniel Michael G, Pawlik Timothy M, Fader Amanda N, Esnaola Nestor F, Makary Martin A
Departments of *Surgery †Surgery and Oncology ‡Obstetrics and Gynecology, Johns Hopkins University School of Medicine ∥Department of Surgery and Health Policy & Management, Johns Hopkins University School of Medicine, Johns Hopkins University Bloomberg School of Public Health, Baltimore, MD §Department of Surgery, Fox Chase Cancer Center Temple Health, Philadelphia, PA.
Am J Clin Oncol. 2016 Apr;39(2):210-3. doi: 10.1097/COC.0000000000000251.
The Orphan Drug Act has fostered drug development for patients with rare cancers and other diseases; however, current data suggest that companies are gaming the system to use the law for mainstream drugs. We identify a pattern of pharmaceutical companies submitting drugs to the Food and Drug Administration (FDA) as orphan drugs but once approved, the drugs are used broadly off-label with the lucrative orphan drug protections and exclusivity benefits. Since the law was passed, the proportion of new FDA-approved drugs that were submitted as orphan drugs has increased with a peak last year of 41% of all FDA-approved drugs approved as orphan drugs. On the basis of the current data, we suggest that patients with rare cancers and other diseases may suffer due to dilution of the incentives and benefits. We propose reform to increase submission scrutiny, decrease benefits based on off-label use, and increase price transparency.
《孤儿药法案》推动了针对罕见癌症及其他疾病患者的药物研发；然而，当前数据表明，企业正在钻制度的空子，利用该法律来开发主流药物。我们发现一种模式，即制药公司将药物作为孤儿药提交给美国食品药品监督管理局（FDA），但一旦获批，这些药物就会在广泛的非标签使用中享受利润丰厚的孤儿药保护和独占权福利。自该法律通过以来，作为孤儿药提交的新获批FDA药物的比例有所增加，去年达到峰值，占所有获批FDA药物的41%。基于目前的数据，我们认为，罕见癌症及其他疾病患者可能会因激励措施和福利的稀释而受到影响。我们提议进行改革，以加强提交审查、减少基于非标签使用的福利并提高价格透明度。
