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. 2016 Nov 11:11:27-39.
doi: 10.1016/j.atg.2016.11.001. eCollection 2016 Dec.

Uncertainty and innovation: Understanding the role of cell-based manufacturing facilities in shaping regulatory and commercialization environments

Rosario Isasi  1 , Vasiliki Rahimzadeh  2 , Kathleen Charlebois  3
Affiliations

Uncertainty and innovation: Understanding the role of cell-based manufacturing facilities in shaping regulatory and commercialization environments

Rosario Isasi et al. Appl Transl Genom. .

Abstract

The purpose of this qualitative study is to elucidate stakeholder perceptions of, and institutional practices related to cell-based therapies and products (CTP) regulation and commercialization in Canada. The development of reproducible, safe and effective CTPs is predicated on regulatory and commercialization environments that enable innovation. Manufacturing processes constitute a critical step for CTP development in this regard. The road from CTP manufacturing to translation in the clinic, however, has yet to be paved. This study aims to fill an empirical gap in the literature by exploring how CTP manufacturing facilities navigate Canadian regulatory and commercialization environments, which together drive the translation of novel CTPs from bench to bedside. Using the multi-level model of practice-driven institutional change proposed by Smets et al., we demonstrate how CTP manufacturing practices are governed by established standards, yet meaningfully shape higher-order regulatory and commercial norms in CTP research and development. We identify four key themes that undergird such processes of innovation: 1) managing regulatory uncertainty, which stems from an inability to classify CTPs within existing regulatory categories for approval and commercialization purposes; 2) building a 'business case' whereby a CTP's market potential is determined in large part by proving its safety and effectiveness; 3) standardizing manufacturing procedures that mobilize CTPs from a research and development phase to a commercialization one; and 4) networking between researchers and regulators to develop responsible commercialization processes that reflect the uniqueness of CTPs as distinct from other biologics and medical devices.

Keywords: Canada; Cell-based therapies; Commercialization; Institutional change; Regenerative medicine; Regulation.

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Figures

Fig. 1
Fig. 1
Regulatory classification schemas for RM products in the European Union, United States and Canada.
Fig. 2
Fig. 2
Cross comparison of regulatory regimes and relevant statutes for CTPs in the European Union, United States and Canada.
Fig. 3
Fig. 3
Graphical schematic of the ‘Framework’ approach to qualitative data analysis for applied policy research adopted in the study.
See this image and copyright information in PMC

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