At the time of Pre-IND, you may not have clinical data for your product yet. In this case, your goal for this section is to provide the FDA with relevant clinical data from analogues and a brief overview of the clinical investigational plan for your product. You may be able to leverage similar data based on serology, capsid type, disease, biomarker, endpoint, route of administration, etc. (refer to the Platform-based Approach section). Please remember to keep your data and summaries accurate, concise, and relevant to your product.
To ensure you are providing the FDA with the key information needed, consider breaking this section into the following sub-sections while filing your Pre-IND meeting information package.
Prior data from analogues
Please note that analogues aren’t always available. Therefore, please consider this section only in the case when they are available and you are confident in the comparison between the analogues and your product, which should be highlighted in this section.
What is an analogue?
The use of analogues, therapies that work in a similar way or target similar diseases, can help strengthen our understanding of how well an upcoming therapies will work over a period of time. Analogues help define specific parameters including safety profile, dose range, and tissue tropism, which is the viral vector type (e.g., AAV, LV, Ad, etc.) and their subcategories (e.g., AAV serotype/capsid). In cases where you have used novel capsids or modified an existing one enough that it may generate variations in the expected parameters, new studies will be required by the FDA. [4, 9],
Under this sub-section, your goal is to provide the FDA with a hypothesis of what to expect from clinical studies. This section may be limited if your investigational therapy is unique and novel. However, there is typically precedence for similar therapeutics in clinical trials. It may be helpful to present a summary of this research and any learnings from such studies. If analogues are available, it will be helpful to summarize why you consider them analogues (i.e., vector features, indication, etc.), aspects of the analogues’ clinical studies that may apply to your study design as well as in any safety or efficacy results, key findings and learnings from the analogue cases and draw a hypothesis of what the clinical experience with your product may look like. Sources of publicly available data include peer-reviewed publications, clinicaltrials.gov, product labeling and Summary Basis of Approval issued by the FDA. If other sponsor data is intended to be relied on to support the safety of your product, you will be required to obtain a Right of Reference Letter from that sponsor and include this information in the IND.
General Investigational Plan
Here, your goal is to provide the FDA with an overview of the clinical investigational plan for your drug. Consider including the following topics discussed in the Planned/Upcoming clinical studies sub-section including the goal and status around the clinical development of your product.