FDA pilots program to encourage new drug development tools
The US Food and Drug Administration (FDA) on Monday announced it will pilot a new program it hopes will spur the development of novel drug development tools (DDTs) that do not fit within the agency’s existing DDT qualification programs.The qualification process for DDTs was revamped under the 21st Century Cures Act and FDA currently offers specific qualification programs for biomarkers, clinical outcome assessments (COAs) and animal models for use under the Animal Rule, which allows for the approval of drugs based on data from animal studies when human efficacy studies are not ethical or feasible.
Under the agency’s new Innovative Science and Technology Approaches for New Drugs (ISTAND) program, FDA says it will offer developers early feedback on proposed DDTs that are “out of scope for existing DDT qualification programs” and provide a path to qualification or other outcomes when qualification is not appropriate.
Examples of potential DDTs that might be considered for the ISTAND program include clinician-reported outcomes (ClinROs), patient-performed digital photography and the development of novel nonclinical pharmacology/toxicology assays. Other potential DDTs could include the use of “tissue chips” to assess safety or efficacy questions and the use of AI to evaluate patients, develop novel endpoints or inform study design.
With the goal of the program being to qualify new types of DDTs and encourage novel approaches to drug development, DDTs that are deemed appropriate for qualification will go through a three-step qualification process before being qualified for use by other drug development programs within the qualified context of use.
“Although ISTAND’s goal is to qualify novel drug development tools for use in drug development programs, for some novel DDTs, qualification may not be the optimal path forward,” FDA writes, explaining that it will work with developers to find other avenues for DDTs that are not appropriate for qualification but may still offer value to drug development programs.
Those options include additional meetings with FDA staff, a public meeting to gather broader input on a particular approach, a white paper to discuss considerations for implementing a novel DDT or new guidance from the agency.
While FDA does not say how long it expects the pilot program to last, the agency says it expects to receive 2-4 submissions each year, which in turn will be triaged
The pilot comes just days after FDA finalized its guidance on the qualification process for DDTs. (FDA finalizes guidances on complex generics meetings, drug development tools, Regulatory Focus 25 November 2020).
FDA