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A Horizon Scan of Novel, Unique, and Innovative Science and Technology Approaches for Use as Tools in New Drug Development: A Project with the ISTAND Pilot Program

Authors:
Poster Author(s)
Lucas Zhou, FDA/CDER (Summer Fellow); Ovya Ganesan, FDA/CDER (Summer Fellow); Margaret VanHeusen, FDA/CDER (Fellow, Peer Mentor); Steve Berman, FDA/CDER (Mentor)
Center:
Contributing Office
Center for Drug Evaluation and Research

Abstract

Poster Abstract

Background

Within the Food and Drug Administration (FDA), paths to drug development tool (DDT) qualification already exist for biomarkers, clinical outcomes assessments (COA), and certain animal models for use under the Animal Rule. However, until recently, there was no clear path for those with novel or unique technologies outside of these traditionally defined types to get formal feedback throughout their tool development or potential qualification. Now, the Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program seeks to fill this role by providing a new regulatory mechanism, which supports the development and qualification of these innovative technologies. Some examples of innovations that have shown promise for DDT use include artificial intelligence-enabled imaging and microphysiological systems.  Due to the novel nature of the submissions to ISTAND, it is challenging to predict which DDTs may be proposed. Thus, it is unclear how product development, regulatory review, and ISTAND program implementation will be influenced. Identifying trends in scientific innovation will allow ISTAND to appropriately allocate its resources and will strengthen the ISTAND program engagement process for parties developing innovative technology outside of FDA. This engagement may take the form of public meetings, engagement with experts at FDA, publication of white papers, development of guidance documents, and others. These forms of engagement can happen during early innovation or at any point until the submission of qualification packages for novel DDTs. Engagement will streamline the qualification process, allowing for DDTs to be made available more rapidly. In turn, this will benefit those who later utilize qualified DDTs during product development. Advanced knowledge of innovation trends will also improve the qualification and ISTAND participation experience for FDA regulators when assisting in expert review of these novel DDTs and when reviewing future drug approvals utilizing these DDTs. Ultimately, ISTAND will allow for thoughtful and efficient integration of these novel technologies into the drug development process and will benefit regulatory agencies, tool developers, and pharmaceutical industries.  

Final Project Objective

To identify areas of emerging technology and scientific innovation, with the potential for use as DDTs, that demonstrate value to drug development and fit into the scope of ISTAND.

Methods

We performed a horizon scan to systematically explore existing and novel DDTs. A horizon scan, as compared to a traditional systematic review, can be used to assess emerging trends in an industry by giving researchers room to explore a wide range of sources, including traditional literature, white papers, news, articles, and blogs. For this scan, we created a search strategy using terms related to drug development and novel technologies. We applied this strategy to databases such as MEDLINE (via PubMed) and Embase. This search strategy was modified to investigate lay press/grey literature. We screened the titles and abstracts of scientific articles, and screened titles and website previews of lay press/grey literature.  The general inclusion criteria included literature from a specified publication date range. General exclusion criteria included biomarkers, animal models for use in Animal Rule applications, or clinical outcome assessments. If a source did not indicate the use of a tool as a DDT, we manually assessed these tools for their potential as a DDT. A data collection form with criteria including drug development tool characteristics, potential use cases, population and use of DDT, and categorization of DDT was created to extract data from sources.  

Findings

Preliminary results are presented in our poster.  

Conclusion and Relevance

Through this research, we aim to curate a list of areas of innovation that may become relevant to drug development and FDA regulation in the near future. The identification of these areas will allow for more focused and efficient allocation of resources by the ISTAND program, support the development of these novel technologies, and facilitate evaluation of their use in drug development. This will catalyze the drug development and approval processes for medical products entering the market. Ultimately, this will improve access to efficacious treatments for patients.

Future Steps

We will complete the screening, collection, and analysis of our data. The final data analysis will allow for the identification of trends and areas of emerging innovation and technological advances that ISTAND may review in coming years. We plan to present our findings according to the identified trends.

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