Global Regulatory Differences for Gene- and Cell-Based Therapies: Consequences and Implications for Patient Access and Therapeutic Innovation

Delphi G M Coppens; Marie L De Bruin; Hubert G M Leufkens; Jarno Hoekman

doi:10.1002/cpt.894

Global Regulatory Differences for Gene- and Cell-Based Therapies: Consequences and Implications for Patient Access and Therapeutic Innovation

Clin Pharmacol Ther. 2018 Jan;103(1):120-127. doi: 10.1002/cpt.894. Epub 2017 Nov 6.

Authors

Delphi G M Coppens¹, Marie L De Bruin^{1

2}, Hubert G M Leufkens¹, Jarno Hoekman^{1

3}

Affiliations

¹ Utrecht/WHO Collaborating Centre for Pharmaceutical Policy and Regulation, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
² Copenhagen Centre for Regulatory Science (CORS), Department of Pharmacy, University of Copenhagen, Copenhagen, Denmark.
³ Innovation Studies Group, Copernicus Institute for Sustainable Development, Utrecht University, Utrecht, The Netherlands.

PMID: 28983911
DOI: 10.1002/cpt.894

Abstract

Gene- and cell-based therapies (GCTs) offer potential new treatment options for unmet medical needs. However, the use of conventional regulatory requirements for medicinal products to approve GCTs may impede patient access and therapeutic innovation. Furthermore, requirements differ between jurisdictions, complicating the global regulatory landscape. We provide a comparative overview of regulatory requirements for GCT approval in five jurisdictions and hypothesize on the consequences of the observed global differences on patient access and therapeutic innovation.

Publication types

Review

MeSH terms

Cell- and Tissue-Based Therapy / methods*
Drug Approval* / methods
Drug Approval* / organization & administration
Drug and Narcotic Control / organization & administration*
Genetic Therapy / methods*
Global Health
Health Services Accessibility
Humans