Acute Respiratory Distress Syndrome (ARDS) is a critical condition characterised by a sudden and severe impairment of lung function, primarily marked by the inability of the lungs to adequately oxygenate the blood and - to a lesser extent - eliminate carbon dioxide.
The European Medicines Agency (EMA) has issued a new guidance document on the development of medicinal products for the treatment of ARDS and/or preventing disease progression. This is the first revision of the guidance on clinical investigation of medicinal products in patients with ARDS issued in 2006.
The key requirements are described in terms of study population, (co)primary and secondary efficacy endpoints. Specific issues, including biomarker and/or (sub)phenotype defined drug development and preparedness are addressed for a potential future pandemic due to a viral pathogen that causes ARDS.
The document is titled ‘Guideline on the Clinical Investigation of Medicinal Products in the Treatment of Patients with Acute Respiratory Distress Syndrome’.
The document can be accessed here: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-investigation-medicinal-products-treatment-patients-acute-respiratory-distress-syndrome-revision-2_en.pdf
The U.S. Food and Drug Administration (FDA) has issued a guidance document titled ‘Sponsor Responsibilities - Safety Reporting Requirements and Safety Assessment for IND and Bioavailability/Bioequivalence Studies’.
The guidance provides recommendations for sponsors and sponsor-investigators to comply with the requirements of Investigational New Drug (IND) application and Bioavailability (BA) safety reporting, as well as Bioequivalence (BE) studies. This guidance provides interpretations of terms used for safety reporting, makes recommendations on when and how to submit a safety report and provides information on other safety reporting issues raised by sponsors.
To facilitate appropriate IND safety reporting practices, this guidance provides recommendations related to the two IND safety reporting provisions that require assessment of aggregate data.
The document can be accessed here: https://www.fda.gov/media/150356/download
Patient Preference Studies (PPS) aim to assess the relative desirability / acceptability of actual or potential health interventions, or their characteristics and outcomes. PPS can generate structured insights about the relative importance of characteristics - also referred to as attributes - that are considered by patients when making decisions about drugs. These attributes may include efficacy or safety outcomes or any other potentially relevant characteristics.
This harmonised guideline outlines general considerations about the use, design, conduct, analysis and submission of PPS aimed at informing drug development, regulatory submission and evaluation, drug approvals and maintenance of such approvals.
Note: The new International Council for Harmonisation (ICH) guideline addresses PPS and the value that patients place on characteristics of drugs - it does not focus on patient reported outcome measures.
The guidance can be found here: https://www.ema.europa.eu/en/documents/scientific-guideline/ich-e22-guideline-general-considerations-patient-preference-studies-step-2b_en.pdf
A third revision of the European Medicines Agency’s (EMA’s) stability testing guidelines has been issued. This document provides general guidance on the stability data which need to be generated to support a variation to a marketing authorisation. It provides general guidance on stability testing for type IA and type IB variations and addresses the data requirements for common type II variations.
The document can be accessed here: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-stability-testing-applications-variations-marketing-authorisation-revision-3_en.pdf
The FDA has issued a document titled ‘Investigator Responsibilities – Safety Reporting for Investigational Drugs and Devices’.
The guidance is intended to help clinical investigators comply with the safety reporting requirements for Investigational New Drug (IND) application studies and Investigational Device Exemption (IDE) studies. Recommendations are provided in this guidance to help investigators identify safety information that needs to be reported to sponsors and institutional review boards.
The document can be accessed here: https://www.fda.gov/media/152530/download
Leachables are chemical entities that migrate from manufacturing components/systems, packaging or delivery device components into a drug product under the established manufacturing and labelled storage conditions.
Extractables are chemical entities that are intentionally extracted from manufacturing components/systems, packaging or delivery device components under specified laboratory test conditions and thus are potential leachables.
A new ICH draft guidance presents a holistic framework and process for the assessment and control of leachable impurities to further expand the existing ICH guidelines on impurities, including impurities in new drug substances (ICH Q3A), new drug products (ICH Q3B), residual solvents (ICH Q3C), elemental impurities (ICH Q3D), as well as DNA reactive (mutagenic) impurities (ICH M7).
The framework of this guideline follows the principles of risk management as described in ICH Q9. While the guideline includes material characterisation and process understanding, its primary purpose is to protect patient safety and product quality through assessment and control of leachables in the drug product.
Due to rapid advances in materials engineering, device innovations, new manufacturing paradigms and novel therapeutic modalities, this guideline aims to provide principles and concepts that are forward looking within the scientific and regulatory landscape.
The document – ‘Q3E Guideline for Extractables and Leachables’- can be accessed here: https://www.fda.gov/media/189890/download
US Pharmacopeia (USP) chapter <81>, relating to antimicrobial assays, went live on 1 December 2025. The activity (potency) of antibiotics can be demonstrated by their inhibitory effect on microorganisms under suitable conditions. A reduction in antimicrobial activity may not be adequately demonstrated by chemical methods.
The chapter summarises procedures for the antibiotics recognised in the USP for which the microbiological assay is the standard analytical method.
Two general techniques are employed:
The chapter is accessible via the USP.
The FDA has issued a draft guidance titled ‘Medical Gases - Current Good Manufacturing Practice’.
The draft document can be accessed here: https://www.fda.gov/media/70973/download
The USP chapter <771> ‘Ophthalmic Products - Quality Tests’ is undergoing a revision. The current draft is available to view via the USP website.
〈1771〉 ‘Ophthalmic Products - Performance Tests’ in the USP is also under review.
USP chapter <621> on chromatography is set to become live on 1 June 2026.
Chromatographic separation techniques are multistage separation procedures in which the components of a sample are distributed between two phases, one of which is stationary while the other is mobile. The stationary phase may be a solid or a liquid supported on a solid or a gel. The stationary phase may be packed in a column, spread as a layer, or distributed as a film, etc. The mobile phase may be gaseous, liquid or supercritical fluid. The separation may be based on adsorption, mass distribution (partition), ion exchange, etc., or may be based on differences in the physicochemical properties of the molecules such as size, mass, volume, etc.
The chapter is accessible via the USP.
Radiopharmaceuticals are used for both diagnostic and therapeutic purposes. They are typically administered only once (or on a limited number of occasions) and contain small amounts of active substances with a radionuclide attached to them. This enables graphic imaging, measurement of biodistribution or therapeutic treatment.
The EMA is updating its guideline, which is currently in draft form and expected to take effect in April 2026. The document outlines the specific additional information that must be submitted for radiopharmaceuticals in the context of applications for marketing authorisations or variations to authorised medicinal products.
The draft document can be found here: https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-quality-radiopharmaceuticals-revision-2_en.pdf
In addition, USP chapter 〈825〉 Radiopharmaceuticals - Preparation, Compounding, Dispensing and Repackaging, is undergoing revision. The latest draft is accessible via the USP website.
EMA has updated the document ‘Abbreviations used in EMA scientific committees & CMD documents and in relation to EMA’s regulatory activities’. This is an ongoing collection of official abbreviations.
The document can be found here: https://www.ema.europa.eu/en/documents/other/abbreviations-used-ema-scientific-committees-coordination-group-mutual-recognition-decentralised-procedures-cmd-documents-relation-emas-regulatory-activities_en.pdf
USP chapter 〈1225〉 ‘Validation of Compendial Procedures’, is undergoing revision. The draft provides enhanced clarity and direction on critical aspects of analytical procedure validation, particularly those conducted within Stage 2 of 〈1220〉 ‘Analytical Procedure Life Cycle’. This revision adapts the chapter to its common usage for validation of both compendial and non-compendial analytical procedures and provides connectivity to related USP chapters.
In addition, the revision introduces expanded discussions on key concepts, including fitness for purpose, reportable results and replication strategy, highlighting how they relate to analytical procedure validation. Ensuring the quality of reportable results is emphasised as the primary goal of these validation activities.
The draft chapter offers guidance on the use of statistical intervals for assessing performance characteristics such as accuracy and precision. It includes best practices for designing validation studies that align with specific study objectives. Additionally, this chapter introduces approaches for determining acceptance criteria for analytical procedures and technology-driven considerations to support analytical procedures validation.
The purpose of this revision is to provide a science-based framework for validating analytical procedures as part of the broader analytical procedure life cycle, offering guidance for both the minimal (also known as traditional) and enhanced approaches to ensure successful validation.
This revision is appearing along with a new chapter, 〈1221〉 ‘Ongoing Procedure Performance Verification’, which serves as the capstone in the life cycle series and should be considered with this chapter as a basis for monitoring and improving the performance characteristics of a procedure.
The draft chapter is accessible via the USP website.
The European Pharmacopeia (Ph. Eur.) is updating its chapter on rubber closures.
This is in relation to:
The draft chapter is accessible via Pharmeuropa (requires registration).
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