May 2026 included several regulatory updates relevant to clinical trial oversight, safety reporting, submission standards, rare disease development, and advanced therapeutic technologies. FDA issued final guidances on postapproval pregnancy safety studies, continuous glucose monitoring data in clinical trials, CMC flexibilities for cell and gene therapy products., and developing drugs for treatment for tuberculosis and CD infection. Additionally, FDA issued multiple draft guidances on the development of opioid and non-opioid drugs. China’s NMPA/CDE released multiple final and draft documents affecting clinical research governance, rare disease development, pediatric trial design, patient-reported outcomes, bioequivalence studies, and electronic submission standards.
U.S. Food and Drug Administration (FDA)
Final updates
Postapproval Pregnancy Safety Studies; Guidance for Industry
FDA issued final guidance providing recommendations to sponsors and investigators on designing postapproval pregnancy safety studies for drug and biological products. This update is relevant to Phase IV/postapproval safety evidence generation and pregnancy exposure risk assessment.
Submitting Continuous Glucose Monitoring Data in Clinical Trials; Guidance for Industry
FDA issued final guidance describing technical specifications for submitting continuous glucose monitoring (CGM) data from clinical trials to support marketing applications for drug and biological products. This affects clinical trial data standards and submission planning where CGM endpoints or data streams are used.
FDA issued final guidance describing how chemistry, manufacturing, and controls (CMC) flexibilities may apply to human cellular and gene therapy products developed for biologics license applications. While primarily CMC-focused, it is indirectly relevant to early clinical development and development planning for cell and gene therapy programs.
Pulmonary Tuberculosis: Developing Drugs for Treatment
This guidance is to assist sponsors in the clinical development of investigational drugs for the treatment of pulmonary tuberculosis (TB). This guidance provides the FDA’s current recommendations regarding the overall development program for a new investigational drug or drugs to be used in combination with drugs already approved for treatment of pulmonary TB or for other indications or as a new treatment regimen that includes one or more investigational drugs to support an indication for the treatment of pulmonary TB.
This guidance outlines the FDA’s current thinking regarding the clinical development of drugs to support an indication of treatment, reduction of recurrence, or prevention of Clostridioides difficile infection (CDI).
Draft Updates
Opioid Analgesic Drugs: Considerations for Benefit-Risk Assessment Framework
This draft guidance describes the benefit-risk assessment framework that the FDA uses in evaluating whether applications for opioid analgesic drugs meet the standard for approval under section 505 of the Federal Food, Drug, and Cosmetic Act. This guidance summarizes the information that should be included in a new drug application for an opioid analgesic drug to facilitate the Agency’s benefit-risk assessment.
Development of Non-Opioid Analgesics for Acute Pain
This guidance addresses FDA’s current thinking on three specific topics: development of non-opioid analgesic products for acute pain, labeling claims, and expedited programs as they pertain to this purpose.
Development of Local Anesthetic Drug Products with Prolonged Duration of Effect
This guidance is developed to assist sponsors that are developing local anesthetic drug products to produce postoperative analgesia for a prolonged duration, for which a new drug application (NDA) is appropriate. This guidance includes information related to clinical trial design.
Stimulant Use Disorders: Developing Drugs for Treatment
This guidance is developed to assist sponsors in the clinical development of drugs for the treatment of stimulant use disorders.
Development of Non-Opioid Analgesics for Chronic Pain
This guidance is developed to assist sponsors in the clinical development of non-opioid drugs for the treatment of chronic pain.
European Medicines Agency (EMA)
Final updates
Model-informed drug development scientific advice and protocol assistance: Pilot procedure
EMA is running a pilot procedure providing scientific advice and protocol assistance on model‑informed drug development (MIDD). Model‑informed drug development refers to the use of quantitative modelling and simulation approaches to generate evidence informing drug development and regulatory decisions. These approaches integrate non-clinical data, clinical data, and prior knowledge such as drug- and disease-specific information. The pilot is intended for drug development programmes in which MIDD evidence is expected to play a key role in regulatory decision-making. It enables in-depth interaction between drug developers and regulators for development programmes with a high model impact. EMA launched this pilot in May 2026
Record of data processing activity for Modelling and Simulation Pilot – Use of Clinical Study Data for Scientific Advice (public)
On 07 May 2026 EMA published this document dated 30 March 2026 the on their website, which describes the processing operation of clinical study data employed to provide the best possible scientific advice on the evaluation of the safety and efficacy of medicinal products for human use.
National Medical Products Administration (NMPA – China)
Final Updates
Effective 1 May 2026, the State Council regulation establishes a governance framework for clinical research and translational application of new biomedical technologies, including high-risk areas such as gene editing and cell therapy. While not a traditional Phase I–IV drug clinical trial regulation, it is indirectly relevant to advanced therapeutic technology development in China because it may affect clinical research governance, institutional responsibilities, risk management, and translational application pathways.
Rare Disease Innovative Drug Development Encouragement Pilot Program (“Care Plan – Extension”)
CDE issued the finalized pilot program to strengthen early communication and lifecycle regulatory support for innovative rare disease drug development. The program is relevant to clinical development planning for rare disease products, particularly before pivotal-stage development.
CDE issued final documents defining major deficiency scenarios for chemical generic drug pharmaceutical studies and bioequivalence studies. The bioequivalence component is directly relevant to clinical pharmacology and BE study conduct, including issues related to study quality, data integrity, and clinically meaningful differences in absorption parameters.
Draft Updates
Draft Technical Guideline for Clinical Trial Design in Adolescent Weight Management Indications
CDE opened consultation on a draft guideline addressing clinical trial design for adolescent weight management indications. The draft is directly relevant to pediatric/adolescent development strategy, endpoint selection, and study design.
CDE opened consultation on a draft guideline for applying PRO measures in pediatric drug clinical trials. The document is relevant to endpoint development, outcome assessment, and patient-centered evidence generation in pediatric studies.
CDE opened consultation on the draft eCTD v4.0 guideline to support the transition from eCTD v3.2.2 to v4.0 in China. Although not phase-specific, the document may affect electronic submission standards for clinical trial and registration dossiers.
Therapeutic Goods Administration (TGA – Australia)
Final Updates
No new TGA final updates directly affecting Phase I–IV drug or biologic clinical trial design were identified during May 2026.
Draft Updates
No new TGA draft consultations specific to Phase I–IV drug or biologic clinical trials were identified during May 2026.
Health Canada
Final updates
No new Health Canada final guidance specific to Phase I–IV drug or biologic clinical trials was identified during May 2026.
Draft updates
No new Health Canada draft guidance or consultation documents specific to Phase I–IV drug or biologic clinical trials were identified during May 2026.
International Council for Harmonisation (ICH)
Final updates
ICH M11: The Clinical electronic Structured Harmonised Protocol
The ICH M11 Expert Working Group issued a final overview presentation (at Step 4 of the ICH harmonisation process) for ICH M11: The Clinical electronic Structured Harmonised Protocol (CeSHarP), a guideline adopted in November 2025.
ICH M11 is a comprehensive guideline that actually consists of three interconnected documents that together standardise clinical trial protocol format, content and exchange: a harmonised guideline; a clinical trial protocol template; and a technical specification to enable the electronic exchange of protocol contents.
These three documents apply to interventional clinical trials of medicinal products across all phases and therapeutic areas, covering pharmaceuticals, biologics, vaccines, drug-device combinations and cell or gene therapy products.
Prior to ICH M11, there was no globally harmonised standard for clinical trial protocol format and content, leading to significant variability across trial sponsors and regions.
Draft updates
No new ICH draft clinical trial guideline publications were identified during May 2026.