On September 15, 2023, the FDA announced expanded features of the Veterinary Innovation Program as part of the agency’s Animal and Veterinary Innovation Agenda. VIP Plus includes new tools that will assist product development and continually improve the efficiency of the FDA’s review process of VIP products by tailoring data requirements to answer risk questions specific to the product. The agency will also provide advanced computational tools that allow reviewers and developers to analyze complex genomic data. The agenda also includes plans to continue investment in data modernization, to re-imagine CVM’s vision for its science program, and scan for emerging technologies and scientific developments that may eventually lead to new kinds of animal and veterinary products.
What’s is VIP Sci-Assist?
VIP Sci-Assist brings new tools to regulation and development of VIP-eligible IGAs and ACTPs. The tools are intended to address specific product development and regulatory needs and enhance CVM’s regulatory efficiency. The tools we are currently developing are described below. We will add to this list in the future:
- Standardized molecular characterization and evaluation of genome editing. FDA-funded research in collaboration with the National Institute of Standards and Technology is underway with two primary goals.
- Generate standardized measurements for characterizing IGAs in cattle and swine that are developed using genome editing. This is a tool that developers and any regulators, including those in other agencies or countries, can use to evaluate whether the genome editing process accomplishes the intended edits, whether there are any off-target edits, and what those off-target edits are, where applicable. When this work concludes, we will make these measurements available to IGA developers.
- Provide access to suitable comparator or reference materials. This will increase confidence in molecular characterization data by providing well-understood controls that can be used to validate a developer’s findings.
- Computational Power Boosts. The FDA has unique, advanced computational tools that offer developers boosts to their development process and FDA unparalleled ability to analyze complex genomic data.
- PrecisionFDA is an advanced web-based platform that offers developers a secure pipeline for transmitting raw next-generation sequencing (NGS) data and other large datasets to the FDA as part of their product submission. PrecisionFDA also includes access to point and click bioinformatics analysis that allows developers to analyze their data using pre-built pipelines without needing to know computer programming or coding, saving them time and money.
- High Performance Computing (HPC) allows for rapid processing of large datasets and complex analyses using supercomputers or computer clusters, such as the analysis of NGS data. CVM has access to HPC resources at its sister Center, the Center for Devices and Radiological Health, and is consequently able to perform complex DNA sequencing analysis that allows us to conduct an independent analysis of the developer’s data confirming the characterization of the IGAs, including identify unintended alterations in the genome that would otherwise be difficult to find. This is helpful both to FDA regulators and to developers of VIP products who may need help in confirming that the alteration they have produced is as intended. For example, upon request, CVM may assist developers with the assessment of sequencing data in support of product characterization, including the identification of unintended alterations that a developer may wish to remove from their production herd.
- Validated Tests for Detecting Disease Agents in Animal Donors of ACTPs. FDA is conducting research at its Office of Applied Science to provide developers with an important tool: the development of validated test methods to ensure ACTP donors are free of relevant disease agents and will not transmit disease to humans and animals. FDA will share the test methods with developers so they can readily test their ACTP donors. This will ease the burden on them by facilitating an effective screening and it will help FDA regulators in their ACTP reviews. Currently, the research is in horses and for domestic disease agents.
What is VIP Fast-Step?
VIP Fast-Step facilitates development of VIP products by shortening review times for the critical early steps of product development occurring under a Veterinary Master File. CVM is committed to significantly accelerating these early review steps, thereby enabling developers to embark on their initial research and development without undue delay. The types of submissions and accelerated review times are as follows:
|VMF Submission Type||VIP Fast-Step Review Time||Non-VIP Review Time|
|Establishing a File (A-0000)||100 days||180 days|
|New Facility/Product||100 days||180 days|
|Shipment Notice||50 days||180 days|
|Reporting a New Study||50 days||180 days|
|Annual Report||100 days||180 days|
What VIP Research is Underway?
CVM is committed to pursuing research projects that will both support its review work of VIP products and provide new insights and tools to developers that will facilitate their production of new, beneficial products. Currently, CVM is conducting research, initially as pilot studies slated to be completed by the end of 2024, at its Office of Applied Science laboratories to:
- Generate data to support aspects of the evaluation and approval process that are common across categories of IGA products, such as IGAs in different species, made using different types of edits, and for varied uses, including as a source of organs, tissues, or other medical products to be used in humans or animals. Potential goals include generating data demonstrating the consistency of inherited IGAs across generations, or data comparing the efficiency and risk for unintended alterations when using different editing methodologies (e.g., different delivery methods, nucleases [standard Cas9, high-efficiency versions, TALENs]). CVM has funded and is starting a pilot study comparing the fidelity of multiple nucleases.
Understanding when, why, and how often unintended effects result from genomic alterations may allow developers and CVM to better predict and mitigate the risks from unintended effects, facilitating the development of safe and effective products derived from FDA-regulated IGAs and potentially decreasing data requirements for developers of FDA-regulated products with better-understood risks. CVM will strengthen its partnership with other FDA components like the Center for Biologics Evaluation and Research to boost CVM’s ability to leverage converging expertise on unintended effects, and to explore how to adapt and evolve regulatory processes and endpoints to manage this potential proportionate to the risk posed by regulated products. CVM recognizes that consumer acceptance and trust of novel products is critical to ensure the benefits of new technologies are realized, making it essential to mitigate the risks of unintended effects in FDA-regulated products.
- Identify characteristics of ACTPs that are critical to potency. Understanding the characteristics and quality attributes that impact potency of ACTPs is key to development and approval of a quality product that consistently produces the intended effect. Potential goals of this research include developing functional bioassays and analytical tests for potency assessment based on the secretome, genome, and proteome of ACTPs from different cell and tissue sources, donors, and processing methods. This research is aimed at facilitating development of appropriate potency assays. This can support development of quality products, facilitate CVM review, and ease the burden on developers of FDA-regulated products for demonstrating comparability through the product lifecycle. CVM is starting a pilot study evaluating the secretome of canine adipose-derived mesenchymal stem cells.