Current State-Of-Play of the EU Advanced Therapy Medicinal Product (ATMP) Field, With an Emphasis on Belgian Human Cell and Tissue Products.

IF 3.4 3区 医学 Q2 CELL BIOLOGY
Gilbert Verbeken, Lieke Convents, Nicolas Delmotte, Jean-Pierre Draye, Serge Jennes, Alain Vanderkelen, Griet Nijs, Philippe Lewalle, Etienne Baudoux, Olivier Cornu, Ineke Vanlaere, Anne Pierlot, Thomas Rose, Jean-Paul Pirnay
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Abstract

The late 1980s saw the emergence of experimental therapies based on human cell and tissue products (HCTPs) within academic and hospital settings, several of them wound healing related. In 2008, the European Commission introduced the Regulation on advanced therapy medicinal products (ATMPs), defining many of these HCTPs as ATMPs, and more specifically as somatic cell therapy medicinal products (sCTMPs) or tissue-engineered products (TEPs). In 2013, we predicted that the ATMP regulation would adversely impact Member States' health care systems and would threaten the sustainability of many HCTPs provided by public health institutions. To assess the current ATMP state of play and investigate whether these predictions ultimately came true, we consulted relevant scientific and trade literature and official competent authority reports and surveyed the former Belgian HCTP producers. We found that the ATMP Regulation produced 19 authorised ATMPs, with 16 of them (84.2%) belonging to the gene therapy medicinal product (GTMP) class and only 3 HCTPs (15.8%), 2 TEPs and 1 sCTMP. List prices varied according to the ATMP class, with public health insurances struggling to reimburse ATMPs, especially the exuberantly priced GTMPs. This led to marketing authorization withdrawals, and crowd funding approaches and lotteries to determine who would receive lifesaving treatments. A hospital exemption (HE) scheme was enacted to protect ATMPs not intended for commercial exploitation. Whilst limited financial resources generally hampered HE utilisation by public actors, stringent regulatory policies made it virtually impossible in Belgium, resulting in meaningful HCTPs no longer being available to surgeons and their patients.

Abstract Image

欧盟先进治疗药物产品(ATMP)领域的现状,重点是比利时人类细胞和组织产品。
20世纪80年代末,在学术和医院环境中出现了基于人类细胞和组织产品(hctp)的实验性疗法,其中一些与伤口愈合有关。2008年,欧盟委员会引入了先进治疗药物产品(atmp)法规,将许多hctp定义为atmp,更具体地说是体细胞治疗药物产品(sctmp)或组织工程产品(TEPs)。2013年,我们预测ATMP法规将对成员国的卫生保健系统产生不利影响,并将威胁到公共卫生机构提供的许多HCTPs的可持续性。为了评估目前的ATMP状态并调查这些预测是否最终成为现实,我们查阅了相关的科学和贸易文献以及官方主管当局的报告,并调查了前比利时HCTP生产商。我们发现,ATMP法规生产了19种获批的ATMP,其中16种(84.2%)属于基因治疗药品(GTMP)类,只有3种hctp(15.8%), 2种TEPs和1种sCTMP。清单价格因ATMP类别而异,公共健康保险难以偿还ATMP,尤其是定价过高的gtmp。这导致了市场许可的撤销,以及众筹方式和彩票来决定谁将获得挽救生命的治疗。颁布了一项医院豁免(HE)计划,以保护不打算用于商业利用的atmp。虽然有限的财政资源通常阻碍了公共行为者对HE的利用,但严格的监管政策使其在比利时几乎不可能实现,导致外科医生及其患者不再能够获得有意义的HCTPs。
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来源期刊
Wound Repair and Regeneration
Wound Repair and Regeneration 医学-皮肤病学
CiteScore
5.90
自引率
3.40%
发文量
71
审稿时长
6-12 weeks
期刊介绍: Wound Repair and Regeneration provides extensive international coverage of cellular and molecular biology, connective tissue, and biological mediator studies in the field of tissue repair and regeneration and serves a diverse audience of surgeons, plastic surgeons, dermatologists, biochemists, cell biologists, and others. Wound Repair and Regeneration is the official journal of The Wound Healing Society, The European Tissue Repair Society, The Japanese Society for Wound Healing, and The Australian Wound Management Association.
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