The BGTC seeks to overcome the current obstacles faced during the development of gene therapies by streamlining approaches in four different sectors:
Basic research
Clinical research
Manufacturing and production
Regulatory requirements
See below for an overview of the BGTC’s specific goals. [1]
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AAV accessibility | Make adeno-associated virus technology (AAV) more accessible to a broader range of diseases
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Streamlined testing | Streamline preclinical and product testing
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Standardized submissions | Facilitate scientific and regulatory advances that will ultimately benefit the entire field
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Timely therapy accessibility | Bring gene therapies to all affected populations sooner
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This playbook is a manifestation of “streamlined testing” and “standardized submissions,” taking a comprehensive and systematic approach to AAV gene therapy development. It covers all aspects of the development process up to IND submission and first-in-human studies.
Supplementing future iterations of the playbook, we plan to also include templates and minimum requirements (e.g., manufacturing, pre-clinical, etc.) to distill a more repeatable regulatory process for AAV gene therapies (see table below). The minimum requirements as they apply to the BGTC approach will get to the core of what is considered necessary and sufficient for a safety and efficacy evidence package in your regulatory submission to the FDA. This means reducing redundancies and leveraging available information to increase efficiencies in a typically complex and inefficient process.
The BGTC approach highlights best practices and recommendations to enable you to strike the right balance between meeting FDA data requirements and accelerating your submission as much as possible. You may refer to the Platform-based Approach for AAV Gene Therapies section for more information on how this approach further supports and defines what we collectively call “minimum requirements”.
Overview of BGTC Regulatory Playbook contents
Templates | Easy “plug-and-play” documents to check off submission requirements |
Illustrative callout boxes | Highlights of important information to consider along the journey |
Strategic insights | Based on FDA guidances, IQVIA expertise, and the consortium’s work |
Uniform regulatory requirements | Established minimum requirements to optimize likelihood of approval |
Sample outlines | Summaries / frameworks to construct and organize the sections |
Decision trees | Pathways / flowcharts to guide the process to submission efficiently |
Do’s and Don’ts | Tables summarizing What to include? and What not to include? |
User-friendly and interactive | Content annotated with icons and hyperlinks to examples and external sources |
This playbook helps you prepare for each stage of development, as well as answer the question “Am I ready?” each step of the way. At key milestones, we’ve provided Readiness Assessments and guidance for applying the BGTC approach in preparing for the INTERACT and Pre-IND meetings (see INTERACT Meeting and Pre-IND Meeting sections). This is intended to support you in reaching the optimal state of preparation at these milestones. We will guide you in applying one or more elements of the BGTC approach at critical timepoints throughout your regulatory journey.
Please read on to understand more about the BGTC approach as it relates to minimum pre-clinical and other requirements.