Innovative regenerative medicines in the EU: a better future in evidence?

BMC Medicine

Debate

Mark S. Corbett¹  Nerys Woolacott¹  Robert Hawkins²  Andrew Webster³ 

[1] Centre for Reviews and Dissemination, University of York, YO10 5DD, Heslington, York, UK;Medical Oncology, The Christie Hospital and University of Manchester, Wilmslow Road, M20 4BX, Manchester, UK;Science and Technology Studies Unit, Department of Sociology, University of York, YO10 5DD, Heslington, York, UK;

关键词: Regenerative medicine;    European Medicines Agency;    Cell therapy;    Gene therapy;   

DOI  :  10.1186/s12916-017-0818-4

 received in 2016-10-19, accepted in 2017-02-14,  发布年份 2017

来源: Springer

【 摘 要 】

BackgroundDespite 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.DiscussionSeveral 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.ConclusionsRecent 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.

【 授权许可】

CC BY   
© The Author(s). 2017