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

Evidence from laboratory and animal studies provides groundwork for understanding mechanisms and potential before human studies continue.

• Level A · Stronger Clinical Evidence
• Level B · Emerging clinical evidence with positive signals
• Level C · Early human research exploring benefits
• Level D · Scientific groundwork from lab and animal studies
• Emerging · Emerging topic under active research

Abstract (original English)

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

Evidence level

Evidence from laboratory and animal studies provides groundwork for understanding mechanisms and potential before human studies continue.

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