Dooms Marc M
University Hospitals Leuven, Leuven, Belgium.
Orphanet J Rare Dis. 2016 Mar 3;11:22. doi: 10.1186/s13023-016-0393-3.
Regulatory and economic frameworks stimulated the research and development of orphan drugs, but very little has been done for devices necessary for the in-vivo diagnosis, prevention and treatment of life-threatening conditions with a low prevalence/incidence.
A general public consultation in Europe has shown a positive attitude towards an "orphan device" directive. The United States of America have a Humanitarian Use Device exemption, but Europe is still waiting for such a stimulating framework. Post-marketing surveillance ("materio-vigilance") will be necessary for follow-up, patient-reported outcome measures (quality of life versus survival) needed and off-label use data available for patient-safety reasons. The marketing period for devices is shorter than for medicinal products. Incentives are necessary to stimulate research and development of such "orphan devices" especially when surgical intervention is the only option.
监管和经济框架刺激了孤儿药的研发，但对于危及生命的罕见疾病的体内诊断、预防和治疗所需的设备，所做的工作却很少。
欧洲的一次公众咨询显示，人们对“孤儿设备”指令持积极态度。美国有人道主义使用设备豁免，但欧洲仍在等待这样一个激励性框架。出于患者安全原因，上市后监测（“物料警戒”）对于随访、所需的患者报告结局指标（生活质量与生存率）以及可用的标签外使用数据来说是必要的。设备的上市期比药品短。有必要采取激励措施来刺激此类“孤儿设备”的研发，尤其是在手术干预是唯一选择的情况下。
