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Regulatory Framework, Challenges, and Initial Strategic Planning for Advanced Therapy Products (PTAs) Development in
Flávia Nathiely Silveira Fachel1,2, Roselena Silvestri Schuh3,4, Patrícia Bencke Grudzinski3
1Células, Tecidos e Genes (CTG), Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, R. Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil. flavia.fachel@ufrgs.br.
Therapeutic Innovation & Regulatory Science
|October 10, 2023
Summary
This study outlines regulatory pathways for advanced therapies, including cell, tissue, and gene therapies, focusing on Brazilian guidelines. It proposes a strategic framework for pre-clinical studies to navigate manufacturing and clinical challenges.
Area of Science:
- Biotechnology and Pharmaceutical Sciences
- Regulatory Science
- Regenerative Medicine
Background:
- Advanced therapies, including cell, tissue, and gene therapies, offer novel treatments for complex diseases with limited alternatives.
- Translating basic research into approved medicinal products requires adherence to stringent regulatory requirements.
- Current regulatory landscapes present challenges in manufacturing, product characterization, and pre-clinical development.
Purpose of the Study:
- To review and analyze key regulatory recommendations for advanced therapies, with a specific focus on Brazilian regulations.
- To identify critical challenges in the development pathway of advanced therapies, from manufacturing to clinical endpoints.
- To propose a strategic framework for planning pre-clinical studies to facilitate regulatory compliance.
Main Methods:
- Comprehensive review of existing regulatory guidelines and recommendations for advanced therapies.
- Analysis of critical development stages: manufacturing, product characterization, non-clinical trials, and endpoint definition.
- Development of a strategic planning framework for pre-clinical studies, illustrated with a gene editing product example.
Main Results:
- Identified significant hurdles in advanced therapy development, including manufacturing complexity, product characterization difficulties, and inadequate animal models.
- Highlighted the absence of validated therapeutic endpoints as a major challenge for clinical translation.
- Presented a proposed framework to guide strategic pre-clinical study planning for advanced therapies.
Conclusions:
- Navigating the regulatory pathway for advanced therapies requires careful strategic planning, particularly for pre-clinical studies.
- The proposed framework offers a structured approach to address manufacturing, characterization, and clinical validation challenges.
- The principles outlined are applicable to various advanced therapy modalities, including gene editing products.
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