title: Hurdles in clinical implementation of academic advanced therapy medicinal products: A national evaluation.
abstract: Since the implementation of the European Union (EU) regulation for advanced therapy medicinal products (ATMPs) in 2009, only six ATMPs achieved marketing authorization approval in the EU. Recognizing the major developments in the ATMP field, starting mostly in academic institutions, we investigated which hurdles were experienced in the whole pathway of ATMP development towards clinical care.
  
  
  
  Quality interviews were executed with different stakeholders in The Netherlands involved in the ATMP development field, e.g. academic research groups, national authorities and patient organizations. Based on the hurdles mentioned in the interviews, questionnaires were subsequently sent to the academic principal investigators (PIs) and ATMP good manufacturing practice (GMP) facility managers to quantify these hurdles.
  
  
  
  Besides the familiar regulatory routes of marketing authorization (MA) and hospital exemption (HE), a part of the academic PIs perceived that ATMPs should become available by the Tissues and Cells Directive or did not anticipate on the next development steps towards implementation of their ATMP towards regular clinical care. The main hurdles identified were: inadequate financial support, rapidly evolving field, study-related problems, lacking regulatory knowledge, lack of collaborations and responsibility issues.
  
  
  
  Creating an academic environment stimulating and planning ATMP development and licensing as well as investing in expanding the relevant regulatory knowledge in academic institutions seems a prerequisite to develop ATMPs from bench to patient.
authors.affiliation: Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Sofieke
authors.initials: S
authors.lastname: de Wilde
authors.affiliation: Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Louise
authors.initials: L
authors.lastname: Veltrop-Duits
authors.affiliation: Dutch Cancer Society (KWF Kankerbestrijding), Amsterdam, The Netherlands.
authors.forename: Merel
authors.initials: M
authors.lastname: Hoozemans-Strik
authors.affiliation: Netherlands Organisation for Health Research and Development (ZonMw), The Hague, The Netherlands.
authors.forename: Thirza
authors.initials: T
authors.lastname: Ras
authors.affiliation: Netherlands Organisation for Health Research and Development (ZonMw), The Hague, The Netherlands.
authors.forename: Janine
authors.initials: J
authors.lastname: Blom-Veenman
authors.affiliation: Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Henk-Jan
authors.initials: HJ
authors.lastname: Guchelaar
authors.affiliation: Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Maarten
authors.initials: M
authors.lastname: Zandvliet
authors.affiliation: Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands. Electronic address: p.meij@lumc.nl.
authors.forename: Pauline
authors.initials: P
authors.lastname: Meij
mesh_terms: D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D003132:Commerce; D055808:Drug Discovery / Q000331:legislation & jurisprudence*; D005062:European Union; D006801:Humans; D011795:Surveys and Questionnaires; D035703:Therapies, Investigational / Q000379:methods*; D023822:Tissue Engineering; D057170:Translational Research, Biomedical / Q000379:methods*
keywords: Biomedical research; Cell- and tissue-based therapy; Clinical trials as topic; Genetic therapy; Investigational; Therapies; Tissue engineering; Translational medical research