Mukoviszidose Institut gGmbH, Bonn, Germany.
J Cyst Fibros. 2011 Jun;10 Suppl 2:S103-9. doi: 10.1016/S1569-1993(11)60015-5.
Small and middle-sized enterprises (SMEs) can make important contributions to medical progress through the development of new safe and effective drugs that address the greatest unmet needs of patients. Regulatory inconsistencies across agencies in various countries, however, remain major challenges in cystic fibrosis (CF) drug development. Clear and consistent treatment guidelines, well educated clinical trial sites, a patient registry and grant funding for early development programs are important success-factors for an efficient development process. SMEs developing products for CF need partners in the CF community to assist with disease education and awareness for ongoing clinical trials. SMEs should collaborate and communicate with the CF community in a legally compliant way to take a patient-centric approach to drug design, development and administration. Furthermore, they can help to develop educational tools and fund medical education activities to increase the understanding of the underlying defects and mechanisms of CF disease.
中小企业（SMEs）可以通过开发新的安全有效的药物为医学进步做出重要贡献，这些药物可以满足患者最大的未满足需求。然而，各国各机构之间的监管不一致仍然是囊性纤维化（CF）药物开发的主要挑战。明确一致的治疗指南、受过良好教育的临床试验场所、患者登记册以及为早期开发计划提供的赠款资金，是提高开发效率的重要成功因素。为 CF 开发产品的中小企业需要在 CF 社区找到合作伙伴，以帮助开展正在进行的临床试验的疾病教育和宣传。中小企业应依法与 CF 社区合作和沟通，以患者为中心的方式进行药物设计、开发和管理。此外，它们还可以帮助开发教育工具并资助医学教育活动，以提高对 CF 疾病潜在缺陷和机制的认识。
