Innovative regenerative medicines in the EU: a better future in evidence?
Innovative regenerative medicines in the EU: a better future in evidence?
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DOI：
10.1186/s12916-017-0818-4
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发表时间：
2017-03-08
期刊：
影响因子：
9.3
通讯作者：
Woolacott N
中科院分区：
文献类型：
--
作者：
Corbett MS;Webster A;Hawkins R;Woolacott N
尽管有源源不断的头条新闻暗示高科技再生药物将改变医疗保健的未来，但到目前为止，患者很难获得这种药物，在过去的7年里，只有8种药物获得了欧盟营销许可证。在这里，我们概述了一些历史原因，为什么缺乏获得许可的创新再生药物。我们讨论了为加快这一复杂和快速变化的医学领域的发展而需要克服的挑战，以及对未来更加乐观的可能理由。临床实践中缺乏尖端再生药物的原因有几个。这些问题包括计划如何开发个人疗法、将其生产到商业水平并最终成功地提供给患者所涉及的巨大费用和困难。在评估这些疗法的安全性、有效性和成本效益时，也存在特定的挑战。此外，许多治疗方法是在没有欧洲药品管理局许可证的情况下使用的，根据欧共体立法的“医院豁免”。对于获得许可的产品，可能需要医疗保健提供者的替代融资方法，因为许多疗法将具有巨大的前期成本，但从长远来看，好处和危害是不确定的。然而，越来越多的政治兴趣和更灵活的治疗许可和融资机制现在已经很明显；这可能是再生医学在临床实践中未来增长和发展的关键。最近监管过程的发展，加上政治兴趣的增加，可能会为改善从实验室到尖端细胞或组织疗法市场的漫长且往往困难的路线带来一些希望。公共资助的研究人员和制药业之间的合作可能是再生医学在临床实践中未来发展的关键；这种合作也可能为制药业的创新危机提供一种可能的解药。
Despite a steady stream of headlines suggesting they will transform the future of healthcare, high-tech regenerative medicines have, to date, been quite inaccessible to patients, with only eight having been granted an EU marketing licence in the last 7 years. Here, we outline some of the historical reasons for this paucity of licensed innovative regenerative medicines. We discuss the challenges to be overcome to expedite the development of this complex and rapidly changing area of medicine, together with possible reasons to be more optimistic for the future. Several factors have contributed to the scarcity of cutting-edge regenerative medicines in clinical practice. These include the great expense and difficulties involved in planning how individual therapies will be developed, manufactured to commercial levels and ultimately successfully delivered to patients. Specific challenges also exist when evaluating the safety, efficacy and cost-effectiveness of these therapies. Furthermore, many treatments are used without a licence from the European Medicines Agency, under “Hospital Exemption” from the EC legislation. For products which are licensed, alternative financing approaches by healthcare providers may be needed, since many therapies will have significant up-front costs but uncertain benefits and harms in the long-term. However, increasing political interest and more flexible mechanisms for licensing and financing of therapies are now evident; these could be key to the future growth and development of regenerative medicine in clinical practice. Recent developments in regulatory processes, coupled with increasing political interest, may offer some hope for improvements to the long and often difficult routes from laboratory to marketplace for leading-edge cell or tissue therapies. Collaboration between publicly-funded researchers and the pharmaceutical industry could be key to the future development of regenerative medicine in clinical practice; such collaborations might also offer a possible antidote to the innovation crisis in the pharmaceutical industry.