FDA Updated Guidance

August 2026

The FDA’s Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products provides recommendations for formal interactions between FDA and sponsors developing human drugs and biologics regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). The guidance outlines the various meeting types available throughout product development, including Type A, B, B(EOP), C, D, and INTERACT meetings, and describes the procedures for requesting meetings, preparing meeting packages, conducting meetings, obtaining FDA feedback, and documenting outcomes. The goal is to promote timely, consistent, and effective communication between FDA and sponsors at key development milestones.

The guidance is particularly important for sponsor-investigators and academic researchers developing investigational drugs, biologics, cell and gene therapies, and other FDA-regulated products. It emphasizes the value of early FDA engagement through mechanisms such as pre-IND and INTERACT meetings, helping sponsors obtain regulatory input on clinical, nonclinical, manufacturing, and product development issues before submitting major regulatory applications. The guidance also establishes procedural timelines and expectations that can help researchers efficiently navigate FDA interactions and advance product development programs.

Source: FDA Guidance for Industry, Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products (August 2026)

August 2026

This guidance provides FDA’s current recommendations and answers to common questions that arise during the development of cellular and gene therapy (CGT) products. The guidance covers the full development lifecycle, including IND submissions and FDA interactions, chemistry, manufacturing, and controls (CMC), nonclinical testing, clinical trial design, expedited programs, and preparation for biologics license applications (BLAs). Its goal is to help sponsors develop safe, effective, and high-quality CGT products while better understanding FDA’s expectations and review processes.

For clinical researchers and sponsors, the guidance is particularly useful because it consolidates FDA’s recommendations on topics such as product characterization, manufacturing changes, proof-of-concept and toxicology studies, biodistribution testing, endpoint selection, and long-term safety monitoring. While focused on CGT products, it provides practical insight into regulatory expectations that can inform investigator-sponsored and industry-sponsored development programs alike. Source Guidance: FDA, Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products (Guidance for Industry, August 2026)

August 19, 2026

This document provides FDA recommendations for designing, validating, and evaluating potency assays used to assess active immunotherapy products (ACTIMPs). These products work by inducing, stimulating, or modulating immune responses against an existing disease and include certain peptide-, protein-, vector-, and cell-based immunotherapies. The guidance focuses on developing potency-testing strategies that demonstrate product quality and consistency throughout development and manufacturing.

For the UAB research community, this guidance is particularly relevant to investigators developing cancer immunotherapies, therapeutic vaccines, personalized neoantigen therapies, cell-based immunotherapies, and other immune-modulating biologic products under FDA oversight. It may affect researchers holding INDs, working with GMP-manufactured immunotherapy products, or planning translational studies that could ultimately support product licensure. Researchers conducting conventional drug trials, observational studies, or non-immunotherapy research would be less directly affected.

Bottom line for UAB: This guidance is highly relevant to UAB's immunology, oncology, cell therapy, gene therapy, and translational medicine programs because it outlines FDA's expectations for demonstrating potency, a critical component of product quality and regulatory approval for active immunotherapies.

Source Document: Potency Assessment of Active Immunotherapy Products (Draft Guidance for Industry, August 19, 2026)

August 3, 2026

Applying Human Factors and Usability Engineering to Medical Devices explains FDA’s expectations for incorporating human factors and usability engineering into the design and development of medical devices. The guidance is intended to help manufacturers and device developers identify, evaluate, and mitigate use-related risks by considering how intended users interact with a device, its labeling, software, controls, displays, training materials, and use environment. FDA emphasizes that device design should minimize use errors that could result in patient harm or compromised medical care and recommends a risk-based approach that includes user research, task analyses, identification of critical tasks, usability testing, and human factors validation testing.

The guidance is most relevant to organizations developing or modifying medical devices for FDA submission, including products regulated through IDE, 510(k), De Novo, or PMA pathways. FDA recommends that human factors activities be integrated into the device development and risk management process and that human factors validation testing be used to demonstrate that intended users can safely and effectively use the device under expected real-world conditions. While the guidance is nonbinding, it reflects FDA’s current thinking and is frequently referenced during device review.

For more information: FDA, Applying Human Factors and Usability Engineering to Medical Devices: Guidance for Industry and Food and Drug Administration Staff (originally issued February 3, 2016; current version updated August 3, 2026)

July 2026

The FDA Oncology Center of Excellence (OCE), Center for Drug Evaluation and Research (CDER), and Center for Biologics Evaluation and Research (CBER) issued the final guidance, Cancer Clinical Trial Eligibility Criteria: Laboratory Values. The guidance recommends that laboratory-based eligibility criteria in cancer clinical trials be scientifically justified and tailored to the investigational product and study population, rather than relying on historically accepted thresholds. FDA notes that overly restrictive laboratory requirements may unnecessarily exclude patients, slow enrollment, and limit the generalizability of trial results.

The guidance encourages sponsors to routinely reassess laboratory exclusion criteria, consider expected variations in laboratory values, and use evidence-based thresholds that appropriately balance patient safety with broader trial participation. The recommendations are intended to support more representative enrollment while maintaining adequate safety protections, particularly in later-phase oncology trials.

Relevance to UAB: This guidance is relevant to investigators and sponsors conducting oncology clinical trials, particularly those designing or reviewing eligibility criteria for IND studies. It may also be useful to IRBs, protocol development teams, and regulatory personnel involved in cancer clinical trial design.

July 2026

This is a technical FDA guidance that describes how sponsors should submit next-generation sequencing (NGS) protocols, datasets, and analyses to support antiviral drug resistance assessments during drug development. The guidance specifies expectations for NGS methods, raw sequencing data, bioinformatics analyses, resistance reporting, and data formats so that FDA can independently evaluate antiviral resistance findings.

For the UAB research community, this guidance is most relevant to investigators conducting antiviral drug development, including studies involving HIV, hepatitis viruses, respiratory viruses, emerging infectious diseases, or other viral pathogens where genomic sequencing is used to evaluate resistance. It may also be important for researchers holding INDs, collaborating with industry sponsors, or generating sequencing data intended to support FDA regulatory submissions. Researchers using NGS solely for basic science, observational research, diagnostics, or non-antiviral clinical studies would generally be less affected.

Source Document: FDA, Submitting Next-Generation Sequencing Data to the Division of Antiviral Products (July 2026)

FDA Guidance Page: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/submitting-next-generation-sequencing-data-division-antiviral-products

July 28, 2026

The FDA announced the availability of the final guidance, Cancer Clinical Trial Eligibility Criteria: Performance Status. The guidance recommends that sponsors carefully evaluate whether traditional performance status (PS) restrictions are scientifically necessary and encourages consideration of including patients with a broader range of performance status when appropriate. FDA notes that unnecessarily restrictive PS criteria can limit trial access, slow enrollment, and reduce the applicability of study results to the patients who will ultimately use the therapy in clinical practice.

The guidance encourages the use of evidence-based eligibility criteria, consideration of alternative trial designs, and additional assessments of functional status where appropriate. The goal is to improve the representativeness and generalizability of oncology clinical trial populations while maintaining participant safety and adequate evaluation of benefit-risk.

Relevance to UAB: This guidance is relevant to investigators, sponsors, protocol development teams, and IRBs involved in oncology clinical trials. It may affect the design and justification of eligibility criteria for cancer studies conducted under INDs and encourages enrollment practices that better reflect real-world patient populations.

July 28, 2026

The FDA issued the final guidance, Cancer Clinical Trial Eligibility Criteria: Washout Periods and Concomitant Medications. The guidance recommends that washout periods and restrictions on concomitant medications be scientifically justified and tailored to the investigational product, rather than relying on standardized or historically accepted requirements. FDA notes that unnecessarily long washout periods and overly restrictive medication exclusions may delay enrollment, limit patient participation, and reduce the representativeness of trial populations.

The guidance encourages sponsors to use risk-based approaches when determining washout requirements and to allow concomitant medications whenever they do not meaningfully affect patient safety or trial interpretation. The recommendations are intended to broaden access to oncology clinical trials while maintaining participant safety and data integrity, supporting enrollment of patient populations that more closely reflect real-world clinical practice.

Relevance to UAB: This guidance is relevant to oncology investigators, sponsors, protocol development teams, and IRBs involved in cancer clinical trials. The recommendations may affect protocol design, eligibility criteria, and regulatory review of oncology studies conducted under INDs by encouraging evidence-based justification for washout periods and medication exclusions.

June 2026

The FDA has issued an important revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, which modernizes the agency's flexibility in evaluating drug and biologic efficacy. While the traditional regulatory benchmark historically required two independent, well-controlled clinical trials, this updated framework formalizes the pathway for a single, highly persuasive clinical investigation supported by robust confirmatory evidence.

This update is highly advantageous for UAB sponsor-investigators, particularly those working in rare diseases or specialized oncology pipelines, as it explicitly outlines how researchers can leverage external data sources, such as institutional natural history studies, real-world evidence (RWE), and data from related disease mechanisms, to meet FDA approval standards with a more streamlined trial blueprint.

Because relying on a single trial requires exceptional study power, generalizability, and minimal missing data, the FDA strongly emphasizes that academic sponsors must engage the agency early in the design process, no later than the End-of-Phase 2 meeting. UAB investigators planning upcoming clinical drug or biologic protocols are highly encouraged to review the new standards to optimize their translation timelines and regulatory strategies.

June 2026

The FDA draft guidance Master Protocols for Drug and Biological Product Development (June 2026) provides recommendations on the design, conduct, statistical analysis, and regulatory submission requirements for clinical trials conducted under a master protocol, where multiple treatments, diseases, or patient populations are studied within a single overarching trial structure. FDA's focus is on randomized trials intended to generate evidence of safety and effectiveness to support regulatory decision-making.

For UAB researchers, the guidance is most applicable to investigators conducting complex oncology, precision medicine, rare disease, and other multi-site clinical trials, especially those sponsored through cooperative groups, NIH networks, or investigator-held INDs. It may influence trial design, statistical planning, informed consent approaches, data monitoring, and FDA interactions.

Source Document: FDA, Master Protocols for Drug and Biological Product Development (Draft Guidance for Industry, June 2026)

June 2026

This update provides FDA recommendations on using quantitative systems pharmacology (QSP) modeling to determine safe starting doses for first-in-human studies. The guidance focuses on products where the MABEL approach is appropriate, particularly novel drugs and biologics that may produce potent biological effects such as cytokine release, T-cell activation, or other significant pharmacologic responses.

For the UAB research community, this guidance is most relevant to investigators conducting early-phase translational research, especially those developing biologics, immunotherapies, cell and gene therapies, or other novel therapeutics under investigator-sponsored INDs.

Bottom line for UAB: This guidance is especially relevant to translational scientists and IND sponsors developing innovative therapeutics, as it describes how QSP modeling can support the selection of safe and scientifically justified starting doses in first-in-human trials.

Source Document: Quantitative Systems Pharmacology (QSP)-Based Dose Selection for Minimum Anticipated Biological Effect Level (MABEL) in First-in-Human (FIH) Trials (Draft Guidance for Industry, June 2026)

June 3, 2026

The FDA issued the final ICH M15: General Principles for Model-Informed Drug Development guidance, establishing a harmonized framework for the planning, evaluation, documentation, and regulatory use of model-informed drug development (MIDD) approaches.

The guidance is relevant to clinical and translational research programs that utilize pharmacokinetic/pharmacodynamic modeling, exposure-response analyses, disease progression models, clinical trial simulations, or other quantitative methods to inform study design, dose selection, and evidence generation.

May 2026

The FDA announced two final guidance documents and several draft and revised draft product-specific guidances on establishing bioequivalence (BE) for drug products. Together, these documents update FDA’s current recommendations on statistical methods, pharmacokinetic study design, and product-specific approaches for demonstrating BE in support of INDs, NDAs, and ANDAs.

May 29, 2026

The FDA released the draft guidance Oncology Pharmaceuticals which proposes approaches to reduce or eliminate unnecessary animal testing in the nonclinical safety assessment of certain oncology biologics and conjugated products, while maintaining the information needed to support investigational and marketing applications.

The draft guidance describes circumstances in which animal studies may be reduced, conducted in a single relevant species, or supplemented by weight-of-evidence assessments and other new approach methodologies.