Rodriguez-Monguio R, Spargo T, Seoane-Vazquez E
Department of Health Promotion and Policy, School of Public Health and Health Sciences, University of Massachusetts, 322 Arnold House. 715 North Pleasant Street, Amherst, MA, 01003-9304, USA.
Department of Health Promotion and Policy, School of Public Health and Health Sciences, University of Massachusetts, Amherst, 325 Arnold House. 715 North Pleasant Street, Amherst, MA, 01003-9304, USA.
Orphanet J Rare Dis. 2017 Jan 5;12(1):1. doi: 10.1186/s13023-016-0551-7.
More than 6,800 rare diseases and conditions have been identified in the US, which affect 25-30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing of drugs to treat rare diseases and conditions. This study analyzed all orphan designations and FDA approvals since 1983 through 2015, discussed the effectiveness of incentives for the development of treatments for rare diseases, and reflected on the ethical imperatives for timely access to orphan drugs.
Study data were derived from the Food and Drug Administration (FDA) Orange Book and the Office of Orphan Drugs Development. A search was conducted to assess literature on the ethical principles and economic incentives for the development of orphan drugs.
In the period 1983-2015, the FDA granted 3,647 orphan drug designations and 554 orphan drug approvals. The orphan drug approvals corresponded to 438 different brand names. Cancer was the therapeutic area with the highest number of approvals. The increased number of patients with rare diseases and the growth in the cost of orphan drugs pose a significant economic burden for patients, public programs and private third party payers. Regulatory differences to qualify for orphan designation and various population thresholds employed by the FDA and the European Medicines Agency lead to further unmet health needs for patients with rare diseases and aggravate health inequities. There is no societal consensus on the population and economic thresholds, the drug effectiveness indicator(s), or the societal value to be placed for the approval and reimbursement of orphan drugs.
Orphan drug development and marketing in the US concentrate in few therapeutic areas. Despite the increase in the number of FDA approved orphan drugs, the unmet needs of patients with rare diseases evidence that the current incentives are not efficiently stimulating orphan drug development. There is need to balance economic incentives to stimulate the development and marketing of orphan drugs without jeopardizing patients' access to treatment. Thus, aligning pharmaceutical companies' incentives with societal budgetary constraints is necessary and the ethical imperatives of timely access to orphan drugs need to be agreed upon.
在美国，已确认有超过6800种罕见疾病和病症，影响着2500万至3000万美国人。1983年，美国国会颁布了《孤儿药法案》（ODA），以鼓励开发和销售用于治疗罕见疾病和病症的药物。本研究分析了1983年至2015年期间所有的孤儿药认定和美国食品药品监督管理局（FDA）的批准情况，讨论了激励措施对罕见病治疗药物开发的有效性，并思考了及时获取孤儿药的伦理必要性。
研究数据来源于FDA橙皮书和孤儿药开发办公室。进行了一项检索，以评估有关孤儿药开发的伦理原则和经济激励措施的文献。
在1983年至2015年期间，FDA授予了3647个孤儿药认定和554个孤儿药批准。孤儿药批准对应438个不同的品牌名称。癌症是获批数量最多的治疗领域。罕见病患者数量的增加以及孤儿药成本的增长给患者、公共项目和私人第三方支付者带来了巨大的经济负担。FDA和欧洲药品管理局在孤儿药认定资格方面的监管差异以及所采用的各种人群阈值，导致罕见病患者的健康需求进一步未得到满足，并加剧了健康不平等。对于孤儿药批准和报销的人群和经济阈值、药物有效性指标或社会价值，社会尚未达成共识。
美国的孤儿药开发和销售集中在少数治疗领域。尽管FDA批准的孤儿药数量有所增加，但罕见病患者未得到满足的需求表明，当前的激励措施并未有效刺激孤儿药的开发。需要在刺激孤儿药开发和销售的经济激励措施与不危及患者获得治疗的机会之间取得平衡。因此，使制药公司的激励措施与社会预算限制保持一致是必要的，并且需要就及时获取孤儿药的伦理必要性达成共识。
