Wencker M, Teschler H, Vogelmeier C, Koczulla R
Ruhrlandklinik, Westdeutsches Lungenzentrum, Abteilung Pneumologie, Universitätsklinikum Essen.
Pneumologie. 2012 Jul;66(7):437-41. doi: 10.1055/s-0032-1309876. Epub 2012 Jun 12.
The importance of rare disease is appreciated by all parties and tremendous effort is made to increase the knowledge about the individual disorders and improve the care of affected patients. Political initiatives on a European level aim to improve the structure of medical care for patients with rare diseases in each member state. The provided incentives for the development of medicines for orphan diseases have led to increased research activities and numbers of licensed Orphan Drugs. Patients are organized nationally and internationally in various patient organizations and umbrella organizations. They are involved in health care policy, support the detection and research of rare diseases and offer support to affected patients and families with educational meetings and materials as well as options for discussions. Many experts are engaged in national and international networks and registries that generate and publish high quality research data on rare diseases. A well developed infrastructure is in place to support the search for qualified partners that can be of assistance with specific questions in a rare lung disease.
各方都认识到罕见病的重要性，并付出了巨大努力来增加对各种罕见病的了解，改善对患者的护理。欧洲层面的政治举措旨在改善每个成员国中罕见病患者的医疗结构。为罕见病药物研发提供的激励措施促使研究活动增加，获批的孤儿药数量增多。患者在国内和国际上组织成各种患者组织和伞状组织。他们参与医疗保健政策制定，支持罕见病的检测和研究，并通过教育会议、资料以及讨论渠道为患者及其家庭提供支持。许多专家参与了国家和国际网络及登记处的工作，这些机构生成并发布关于罕见病的高质量研究数据。已有完善的基础设施来支持寻找合格的合作伙伴，以协助解决罕见肺病的特定问题。
