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

TY - JOUR

T1 - Global Regulatory Differences for Gene- and Cell-Based Therapies

T2 - Consequences and Implications for Patient Access and Therapeutic Innovation

AU - Coppens, Delphi G M

AU - De Bruin, Marie L

AU - Leufkens, Hubert G M

AU - Hoekman, Jarno

N1 - © 2017 American Society for Clinical Pharmacology and Therapeutics.

PY - 2018/1

Y1 - 2018/1

N2 - 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.

AB - 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.

KW - Journal Article

KW - Review

U2 - 10.1002/cpt.894

DO - 10.1002/cpt.894

M3 - Review

C2 - 28983911

SN - 0009-9236

VL - 103

SP - 120

EP - 127

JO - Clinical Pharmacology and Therapeutics

JF - Clinical Pharmacology and Therapeutics

ER -