advanced therapy medicinal products; hospital exemption; human cell and tissue products; keratinocytes; transplantation; Humans; Belgium; European Union; Cell- and Tissue-Based Therapy/economics; Tissue Engineering/legislation & jurisprudence; Tissue Engineering/economics; Genetic Therapy; Wound Healing
Abstract :
[en] 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.
Disciplines :
Public health, health care sciences & services
Author, co-author :
Verbeken, Gilbert ; Laboratory for Molecular and Cellular Technology, Queen Astrid Military Hospital, Brussel, Belgium
Convents, Lieke; Laboratory for Molecular and Cellular Technology, Queen Astrid Military Hospital, Brussel, Belgium
Delmotte, Nicolas; Laboratory for Molecular and Cellular Technology, Queen Astrid Military Hospital, Brussel, Belgium
Draye, Jean-Pierre; Laboratory for Molecular and Cellular Technology, Queen Astrid Military Hospital, Brussel, Belgium
Jennes, Serge; Burn Wound Centre, Grand Hôpital de Charleroi, Gilly, Belgium
Vanderkelen, Alain; Burn Wound Centre, Queen Astrid Military Hospital, Brussel, Belgium
Nijs, Griet; Centre for Gene Therapy and Regenerative Medicine, University Hospital Antwerp, Edegem, Belgium
Baudoux, Etienne ; Centre Hospitalier Universitaire de Liège - CHU > > Laboratoire de thérapie cellulaire et génique (LTCG)
Cornu, Olivier; Musculoskeletal Tissue and Cell Therapy Unit, University Hospital Saint-Luc, UCLouvain, Brussels, Belgium ; Neuro-Musculoskeletal Laboratory (NMSK), Institute of Experimental and Clinical Research (IREC), UCLouvain, Brussels, Belgium
Vanlaere, Ineke; Belgian Association for Tissue and Cell Banks (BVWB-ABBT), Edegem, Belgium
Pierlot, Anne; Laboratory for Molecular and Cellular Technology, Queen Astrid Military Hospital, Brussel, Belgium ; Burn Wound Centre, Queen Astrid Military Hospital, Brussel, Belgium
Rose, Thomas; Laboratory for Molecular and Cellular Technology, Queen Astrid Military Hospital, Brussel, Belgium ; Burn Wound Centre, Queen Astrid Military Hospital, Brussel, Belgium
Pirnay, Jean-Paul; Laboratory for Molecular and Cellular Technology, Queen Astrid Military Hospital, Brussel, Belgium
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