Andrew Haskell, PhD, MLS, CT(ASCP)CM, MBCM, QLSCM
Member, ISCT North America LRA Committee
HAS Consulting, LLC
United States

Kevin Bosse, PhD, RAC-US, CABP(H)
Chair, ISCT North America LRA Committee
Nationwide Children's Hospital
United States

FDA Policy Updates to Align with Agency Priorities and New Technologies

New Approach Methodologies at the FDA

“New approach methodologies” (NAMs) is an umbrella term for research methods that, in terms of drug development and cell and gene therapy, can demonstrate human relevance in preclinical research. The rapid development of NAM technologies continues to provide exciting opportunities for the ISCT from basic research to clinical trials and every role supporting those involved in this research along the way. Our previous Watchdog issue covered a draft guidance from the FDA on using data from NAMs to reduce animal testing in preclinical efficacy studies. Additionally, there was draft guidance published by the FDA in April 2023 titled: “Acute Radiation Syndrome: Developing Drugs for Prevention and Treatment” which further permits for NAM data to replace one of two animals for approval under the Animal Rule. While it is important to clarify that in both of these instances the FDA considers NAM data to be “supportive” of existing data and that NAMs require qualification and validation with the FDA to ensure they produce reliable data, these rule changes demonstrate the FDA’s confidence in these technologies to streamline drug development and advance agency priorities without sacrificing safety. 

In fact, the FDA’s press release announcing the draft guidance in March included the following quotation from HHS Secretary Robert F. Kennedy Jr.: “This draft guidance advances our commitment to replace animal testing with human-relevant, scientifically rigorous methods. Clear validation expectations will help modern tools earn regulatory confidence and speed safer, more effective therapies to patients.” On April 20, 2026, the FDA announced that they had achieved their first-year animal reduction milestone while also expecting to “improve prediction of drug safety, accelerate development timelines, reduce costs” and continue working with industry partners.

Pediatric Clinical Trials

On April 9, 2026, the US FDA hosted a workshop titled “Advancing Pediatric Cell and Gene Therapy Clinical Trials: Scientific, Ethical, Regulatory, and Practical Considerations.” During the workshop, a panelist expressed frustration that the FDA continues to require drug developers to conduct adult clinical trials before pediatric clinical trials for many rare diseases with a direct benefit to pediatric patients. The resulting discussion (which begins with the panelist’s opening remarks at 57:23 in the recorded video) generated interest in the NA LRA Committee given the FDA’s recent trend toward updating longstanding policies to adapt to new technologies and align to the agency’s current priorities.

Given FDA’s increasing willingness to consider well-validated, fit-for-purpose NAM data, it is reasonable to ask whether these tools could support more flexible approaches to pediatric trial initiation, especially in rare disease development programs. In many rare diseases, particularly those that are pediatric-onset, rapidly progressive, or associated with irreversible loss of function, waiting for substantial adult clinical experience before opening pediatric enrollment may delay access for the very population most likely to benefit. Compelling NAM data comparing adult and pediatric biology, pharmacologic response, target engagement, or toxicity could help address some of the uncertainty that traditionally supports a more sequential adult-first approach. In this way, NAMs could contribute to a broader package of evidence supporting earlier pediatric enrollment, while preserving appropriate safeguards for safety, dose selection, and ethical review.

Given FDA’s increasing willingness to consider well-validated, fit-for-purpose NAM data, it is reasonable to ask whether these tools could support more flexible approaches to pediatric trial initiation, especially in rare disease development programs. In many rare diseases, particularly those that are pediatric-onset, rapidly progressive, or associated with irreversible loss of function, waiting for substantial adult clinical experience before opening pediatric enrollment may delay access for the very population most likely to benefit. Compelling NAM data comparing adult and pediatric biology, pharmacologic response, target engagement, or toxicity could help address some of the uncertainty that traditionally supports a more sequential adult-first approach. In this way, NAMs could contribute to a broader package of evidence supporting earlier pediatric enrollment, while preserving appropriate safeguards for safety, dose selection, and ethical review.

REFERENCES

1. FDA Guidance Leverages New Approach Methodologies to Spare Animal Research Requirements (NA LRA, 2026): https://www.isctglobal.org/telegrafthub/blogs/francheska-juliano/2026/04/08/north-america-lra-watchdog-april-2026
2. Acute Radiation Syndrome: Developing Drugs for Prevention and Treatment (Draft Guidance) (CDER, 2023): https://www.fda.gov/regulatory-information/search-fda-guidance-documents/acute-radiation-syndrome-developing-drugs-prevention-and-treatment
3. FDA Releases Draft Guidance on Alternatives to Animal Testing in Drug Development (Press Announcement) (FDA, 2026): https://www.fda.gov/news-events/press-announcements/fda-releases-draft-guidance-alternatives-animal-testing-drug-development
4. FDA Achieves Year 1 Goals in Reducing Animal Testing in Drug Development (Press Announcement) (FDA, 2026): https://www.fda.gov/news-events/press-announcements/fda-achieves-year-1-goals-reducing-animal-testing-drug-development
5. Advancing Pediatric Cell and Gene Therapy Clinical Trials: Scientific, Ethical, Regulatory, and Practical Considerations (Workshop Recording) (CBER, 2026): https://www.fda.gov/news-events/advancing-pediatric-cell-and-gene-therapy-clinical-trials-scientific-ethical-regulatory-and
6. FDA Announces Major Steps to Implement Real-Time Clinical Trials (Press Announcement) (FDA, 2026): https://www.fda.gov/news-events/press-announcements/fda-announces-major-steps-implement-real-time-clinical-trials