title: The path to successful commercialization of cell and gene therapies: empowering patient advocates.
abstract: Often, novel gene and cell therapies provide hope for many people living with incurable diseases. To facilitate and accelerate a successful regulatory approval and commercialization path for effective, safe and affordable cell and gene therapies, the involvement of patient advocacy groups (PAGs) should be considered early in the development process. This report provides a thorough overview of the various roles PAGs play in the clinical translation of cell and gene therapies and how they can bring about positive changes in the regulatory process, infrastructure improvements and market stability.
authors.affiliation: University of California Davis, Institute for Regenerative Cures, Sacramento, California, USA.
authors.forename: Gerhard
authors.initials: G
authors.lastname: Bauer
authors.affiliation: Berlin-Brandenburg Center for Regenerative Therapies, Charité-Universitätsmedizin Berlin, Berlin, Germany. Electronic address: mohamed.abou-el-enein@charite.de.
authors.forename: Mohamed
authors.initials: M
authors.lastname: Abou-El-Enein
authors.affiliation: Genetic Alliance UK, London, United Kingdom.
authors.forename: Alastair
authors.initials: A
authors.lastname: Kent
authors.affiliation: International Society for Cellular Therapy, Vancouver, Canada.
authors.forename: Brian
authors.initials: B
authors.lastname: Poole
authors.affiliation: International Society for Cellular Therapy, Vancouver, Canada; TxCell SA, Valbonne-Sophia Antipolis, France.
authors.forename: Miguel
authors.initials: M
authors.lastname: Forte
mesh_terms: D064987:Cell- and Tissue-Based Therapy / Q000191:economics* / Q000941:ethics; D003132:Commerce / Q000379:methods / Q000639:trends; D015316:Genetic Therapy / Q000191:economics* / Q000941:ethics / Q000331:legislation & jurisprudence; D006295:Health Resources / Q000191:economics / Q000600:supply & distribution; D006297:Health Services Accessibility / Q000191:economics; D006801:Humans; D010344:Patient Advocacy / Q000191:economics; D010358:Patient Participation / Q000191:economics*; D011209:Power, Psychological; D035703:Therapies, Investigational; D057170:Translational Research, Biomedical
keywords: affordability; cell and gene therapies; clinical trials commercialization; patient advocacy; regulation; reimbursement