Euro Roundup: EMA seeks feedback on plans to update good pharmacogenomic practice guideline

The European Medicines Agency (EMA) has posted a draft concept paper setting out its plans to update a guideline on good pharmacogenomic practices.

EMA published the first version of the guideline in 2018 to shape best practices, ensure consistency, and provide a clear framework for regulators and stakeholders. As expected in an emerging field, genomics has continued to evolve since the release of the guideline. Scientific progress, technological innovations, and emerging regulatory considerations have driven the evolution of the sector.

Seeking to reflect the developments, EMA is planning to incorporate new evidence, methodologies, and best practices into the guideline. The core objectives of the guideline remain the same, but EMA sees a need to update the details to ensure the framework supports high standards while fostering innovation.

EMA has identified pharmacogenomic methodology, interpretations and recommendations, reporting and nomenclature, and pharmacogenomic study design as the four high-level areas that need changing or adding in the new guideline. The draft concept paper outlines the changes EMA plans to make in each area.

Within pharmacogenomic methodology the agency plans to update its advice on sequencing technology to reflect third-generation long-read sequencing. As EMA explains in the concept paper, technologies can now generate longer reads, allowing for a more accurate distinction between complex gene structures. The progress has implications for identifying and interpreting pharmacogenetic variants.

EMA’s proposed changes to its advice on pharmacogenomic study design affect both interventional and noninterventional trials. The agency plans to provide recommendations on when and how to best use pharmacogenomics at all phases of interventional clinical development. To support noninterventional studies, EMA intends to include a section on the use of real-world data related to pharmacogenomics.

The draft concept paper is open for comment until 31 March 2026. EMA aims to finalize the paper in the second quarter of 2026 and adopt the text in the third quarter. The timeline positions the agency to post a draft guideline for consultation in 2027 and finalize the document in 2028.

Concept Paper

EMA finalizes guideline on developing and manufacturing synthetic peptides

EMA has published a final guideline on synthetic peptide development and manufacture. The text will take effect 1 June 2026.

The agency created the text to share information about the manufacturing process, characterization, specifications, and analytical control that are specific to synthetic peptides and not covered in broader documents about the chemistry of active substances. EMA also included information about conjugation, developing synthetic peptides using biological peptides as references, and other topics.

The final guideline has the same structure as the draft EMA released for consultation in 2023. Most of the document focuses on the active substance, with subsections addressing the manufacture, control, and stability of synthetic peptides.

EMA has made changes throughout the document in response to information learned since the draft was released for consultation. The updates include clarification of the scope of the guideline, which EMA said applies to marketing authorization applications and post-authorization procedures. While the text has a section on investigational products, EMA advises trial sponsors to liaise with national regulators.

The agency has revised the section on investigational products, which in the draft was focused on clinical trial applications. EMA’s changes clarify that the section only covers quality aspects that are specific to investigational medicinal products with synthetic peptides as the active substance. The agency intends the section to complement its guideline on all investigational medicinal products.

EMA’s Quality Working Party listed the synthetic peptide guideline among the documents it planned to publish in 2026. The working party’s schedule for 2026 also includes guidelines on synthetic oligonucleotides and guidance on co-processed excipients.

EMA Guideline

MedTech Europe calls for dedicated breakthrough, orphan, and pediatric device pathways

MedTech Europe has set out its vision for dedicated pathways for breakthrough, orphan, and pediatric medical devices.

The trade group shared its vision in a position paper. While welcoming initiatives such as the informal orphan device pathway established by European authorities, MedTech Europe wants to see stronger actions to accelerate access to certain classes of product. The pathways would apply to devices used in rare diseases and children, as well as to technologies that are particularly innovative.

MedTech Europe is urging European legislators to create a legal framework for the pathways. The trade group wants legislators to establish clear roles, procedures, and timelines for each pathway and support early dialogue between manufacturers and notified bodies. Other proposals include facilitating European market access for devices approved in other jurisdictions.

“Such a legal framework should be adaptive and subject to dedicated performance measurements to ensure that Europe becomes – and remains – competitive while ensuring the best patient outcomes,” the trade group said.

Press Release

EMA updates Q&A on biological medicines, adding advice on in-process controls

EMA has answered new questions about aspects of biological medicinal products that can be subject to different interpretations or require clarification.

In the latest update to the Q&A EMA added six questions and revised the responses to several existing questions. One new answer explains what details of unexpected deviations in safety-related critical in-process controls should be included in the dossier. EMA said that for controls of unprocessed bulk Module 3 should clearly state that a deviation from acceptance criteria would cause batch rejection.

Other new answers describe the expected sample burden for bioburden testing, how to reflect potency assignment in reference standard qualification protocols, and how to define the date of vaccine manufacture in the dossier.

EMA has revised its responses to existing questions about when to investigate low endotoxin recovery, what specification tests to use for routine control of antibody-dependent cell-mediated cytotoxicity, and what information to provide about excipients that have an intended biological effect or are made using recombinant technology.

EMA Q&A

HTA coordination group answers questions about joint clinical assessments

A European Union advisory group on health technology assessment (HTA) has answered questions about general methodological and procedural issues for joint clinical assessments (JCAs).

The Member State Coordination Group on HTA (HTACG) created the document based on questions it has received. The current version of the document features 12 questions, several of which relate to the PICO framework, an acronym of population, intervention, comparator, outcome. The PICO framework is the basis for how authorities will appraise evidence submitted by health technology developers (HTDs).

HTACG used the Q&A to advise HTDs that are unable to meet PICO requirements, for example because only data with slightly different subgroup definitions are available. In that situation, HTDs should provide relevant analyses that meet the requirements as closely as possible and explain and justify any deviation from PICO. Justifications should include appropriate literature searches as supportive arguments.

HTDs that enroll a study population that is wider than the PICO population should consider whether it is possible and scientifically valid to analyze a subpopulation of the trial. HTDs should explain and justify any subpopulations that they present in their dossiers.

HTACG Q&A

Other News:

The European Pharmacopoeia has published the first individual monoclonal antibody medicinal product monograph. The monograph covers golimumab, the TNF-alpha antagonist that Johnson & Johnson sells as Simponi and will come into effect on 1 April 2026. The monograph is part of a broader push to develop public standards for monoclonal antibodies. Press Release

Euro Roundup: EMA seeks feedback on plans to update good pharmacogenomic practice guideline

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