Lang J, Wood S C
R&D Clinical Development Department, Pasteur Mérieux Connaught, Lyon, France.
Emerg Infect Dis. 1999 Nov-Dec;5(6):749-56. doi: 10.3201/eid0506.990602.
The development of vaccines against rare emerging infectious diseases is hampered by many disincentives. In the face of growing in-house expenditures associated with research and development projects in a complex legal and regulatory environment, most pharmaceutical companies prioritize their projects and streamline their product portfolio. Nevertheless, for humanitarian reasons, there is a need to develop niche vaccines for rare diseases not preventable or curable by other means. The U.S. Orphan Drug Act of 1983 and a similar proposal from the European Commission (currently under legislative approval) provide financial and practical incentives for the research and development of drugs to treat rare diseases. In addition, updated epidemiologic information from experts in the field of emerging diseases; increased disease awareness among health professionals, patients, and the general public; a list of priority vaccines; emergence of a dedicated organization with strong leadership; and the long-term pharmacoeconomic viability of orphan products will be key factors in overcoming the complexity of orphan status and the limited need for vaccine.
针对罕见新发传染病的疫苗研发受到诸多不利因素的阻碍。在复杂的法律和监管环境下，面对与研发项目相关的内部支出不断增加，大多数制药公司会对其项目进行优先级排序并精简产品组合。然而，出于人道主义原因，有必要开发针对那些无法通过其他手段预防或治愈的罕见疾病的小众疫苗。1983年美国的《孤儿药法案》以及欧盟委员会的一项类似提案（目前正在立法审批中）为治疗罕见疾病药物的研发提供了资金和实际激励。此外，来自新发疾病领域专家的最新流行病学信息；卫生专业人员、患者和公众对疾病认识的提高；优先疫苗清单；一个拥有强大领导力的专门组织的出现；以及孤儿产品的长期药物经济学可行性，都将是克服孤儿药地位的复杂性和疫苗需求有限这一问题的关键因素。
