Our Industry Update Newsletter returns for the first update of 2025.
As always, this quarter’s update has been expertly compiled by Christina Hägglund, QA Manager.
WORLDWIDE INITIATIVES FOR CLINICAL TRIALS
US Initiatives
The need for more dedicated paediatric research was highlighted in a research letter published in December 2024 in JAMA Pediatrics. The researchers recommend further assessments of potential gaps in the current regulatory processes to encourage paediatric drug development, in particular for non- orphan indications. FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023 | Pediatrics | JAMA Pediatrics | JAMA Network
EU Initiatives
The COMBINE programme is a cross-sector initiative to streamline combined studies of medicines and medical devices, including diagnostics. The programme will be rolled-out over the coming years. For further information: Combined studies – European Commission
In January 2025, three European pharmaceutical industry associations proposed a phased roll-out of electronic Product Information (ePI). Currently, PI is usually available as a PDF on EU regulatory websites (as well as on printed package leaflets in the medicine box). The proposed move to ePI follows a successful pilot project. iatf-position-papers-on-epi.pdf
The European Shortages Monitoring Platform (ESMP) is now fully operational. The ESMP enables Marketing Authorisation Holders (MAHs) and National Competent Authorities (NCAs) to directly report information on supply, demand and availability of nationally and centrally authorised medicines during public health emergencies and major events. European Shortages Monitoring Platform fully operational for monitoring of shortages in the EU | European Medicines Agency (EMA)
UK Initiatives
On 30 January 2025, the Medicines and Healthcare products Regulatory Agency (MHRA) announced the availability of the refreshed Innovative Licencing and Access Pathway (ILAP). This is a UK-wide initiative that will offer a clearer, more streamlined and integrated process for developers to help get transformative new medicines to patients in the National Health Service (NHS) in the shortest time possible. UK patients enabled access to transformative new medicines in shortest time possible via new, integrated Innovative Licensing and Access Pathway – GOV.UK
The MHRA’s Data Strategy promotes data-driven innovation and early access to innovative products through Real World Evidence (RWE) and proactive approaches to safety surveillance. To support this, the MHRA has launched a pilot RWE Scientific Dialogue Programme. This initiative is designed to help innovators refine their evidence generation strategies while providing clear guidance on regulatory expectations. MHRA Real-World Evidence Scientific Dialogue Programme – GOV.UK
REGULATION & ICH GxP GUIDELINES
Major Overhaul of UK Clinical Trial Regulation
A new legislation was laid before UK parliament in December 2024. The legislation aims to:
- Reduce unnecessary administrative burdens on trial sponsors without compromising patient safety
- Remove duplicative requirements
- Streamline processes
The current Combined Review and Notification scheme will be embedded into the legislation, as applicable. Once the new legislation comes into force, a 12-month implementation period will ensure readiness. Patients, the NHS and the Life Sciences sector set to benefit from new clinical trials framework being laid in parliament today – GOV.UK ABPI statement: Enhancing the role of UK medicine regulation
Health Technology Assessment (HTA) Regulation (2021/2282) and European Commission (EC) Joint Scientific Consultations
In the EU, a centrally authorised medicine is accessible to patients after regulatory assessment and authorisation by the European Medicines Agency (EMA) and following evaluation by HTA bodies who help EU member states make decisions about the use, price and reimbursement level of a new health technology.
On 12 January 2025, the HTA Regulation came into force. One of the key aspects is joint scientific consultations on medicinal products for human use. This means close interaction between regulators, HTA bodies and other relevant parties which is critical to enable patient access to new treatments.
The EC adopted an implementing regulation setting out the rules for joint scientific consultations under the HTA Regulation. New EU rules for hta become effective | EMA and HTA – Commission adopts rules
China – Reform of Drug and Medical Devices Regulation
On 06 January 2025, the Chinese government issued a document, stating that by 2027, their legal and regulatory frameworks for drug and medical device oversight will be significantly enhanced and the quality and efficiency of review and approval processes for innovative drugs and devices will be much improved. China deepens comprehensive reform to strengthen drug, medical device regulation
ICH E6(R3) Good Clinical Practice (GCP) Guideline
ICH E6(R3) Principles and Annex 1 has been adopted by the EMA and becomes effective in Europe on the 23 July 2025. E6(R3) encourages a fit for purpose approach focusing on the clinical trial’s Critical to Quality (CtQ) factors. The CtQ factors need to be identified, managed proactively and adjusted if necessary.
- CtQ factors are factors that have a meaningful impact on participants rights, safety and wellbeing (and may therefore encompass scientific as well as other factors).
ICH E6(R3) final guideline document: ICH_E6(R3)_Step4_FinalGuideline_2025_0106.pdf and ICH training: ICH Official web site : ICH
Annex 2 is for clinical trials with decentralised elements and is currently under public consultation. ICH_E6(R3)_Annex 2_Step2_DraftGuideline_2024_1024_0.pdf
MHRA – Exceptions and Modifications to the EU Guidance on Good Pharmacovigilance Practice (GVP)
On 07 February 2025, the guidance note on GVP was updated and applies to UK MAHs and the licensing authority. Modifications to the EU guidance on good pharmacovigilance practices that apply to UK marketing authorisation holders
MHRA – ICH M15 ‘General Principles for Model-Informed Drug Development’
The ICH M15 guideline is open for public consultation, including the countries/regions detailed below:
- EU – ICH M15 Guideline on general principles for model-informed drug development_Step 2b
- UK – Consultation on the International Council for Harmonisation ICH M15 Guideline for Model Informed Drug Development (MIDD) – GOV.UK
- US – M15 General Principles for Model-Informed Drug Development
DATA TRANSPARENCY
EU Data Transparency
On 18 December 2024, the EMA and Heads of Medicines Agency (HMA) adopted updated principles on the identification and disclosure of Commercially Confidential Information (CCI) and Personal Data (PD). The final document represents a comprehensive overhaul of the previous EU guidance, by specifically addressing the identification of CCI and PD within the structure of the Marketing Authorisation Application (MAA) dossier. HMA/EMA guidance document
Health Research Authority (HRA) Data Transparency Wording Template
To help ensure clinical trial participants have all the information they need to make an informed decision about the use of their data, the HRA have developed a General Data Protection Regulation (GDPR) template, recommended for use by all sponsors from 01 April 2025. Transparency wording for all sponsors – Health Research Authority
MEDICAL DEVICES
EC Targeted Evaluation of Regulations 2017/745 and 2017/746 on Medical Devices and In Vitro Diagnostic
The EC has launched a public consultation and a call for evidence on the EU’s legislation on medical devices and in vitro diagnostic medical devices, as part of the targeted evaluation of these rules.
The consultation is open until 21 March 2025. EU rules on medical devices and in vitro diagnostics – targeted evaluation
EMA Scientific Advice for High-Risk Medical Devices
The EMA, in close collaboration with the EC, has established a standard procedure for manufacturers of certain high-risk medical devices to request scientific advice on their intended clinical development strategy and proposals for clinical investigation. EMA establishes regular procedure for scientific advice on certain high-risk medical devices | European Medicines Agency (EMA)
MHRA Manufacturers’ Guidance
This guidance is for medical device manufacturers to help prepare for the new Post-Market Surveillance (PMS) Regulation, which becomes effective 16 June 2025.
- Guidance: Medical devices: post-market surveillance – GOV.UK
- Regulation: The Medical Devices (Post-market Surveillance Requirements) (Amendment) (Great Britain) Regulations 2024
MHRA Digital Mental Health Technology (DMHT) Guidance
This forms part of a series of guidance documents, relating to the regulation and evaluation of DMHT. Some DMHTs qualify as ‘software as a medical device’ (SaMD) and must comply with relevant medical device regulations. This guidance explains how to determine this and how UKCA / CE certification shows compliance with these regulations. 2025.02.10_MHRA_guidance_on_DMHT_-.pdf
GUIDANCE DOCUMENTS
Final Guidance
- December 2024: FDA ‘Standardized Format for Electronic Submission of NDA and BLA Content for the Planning of Bioresearch Monitoring (BIMO) Inspections for CDER Submissions’ E Submission of NDA and BLA
- December 2024: FDA ‘Advance Manufacturing Technologies Designation Program’ Advanced Manufacturing Technologies
- December 2024: EMA – Two IRIS documents have been updated IRIS guide v2.16 and iris-guide- applicants.pdf
Draft Guidance
- January 2025: FDA ‘Study of Sex Differences in the Clinical Evaluation of Medical Products’ Sex Differences
- January 2025: FDA ‘Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Product’ Artificial Intelligence
- January 2025: FDA ‘Artificial Intelligence-Enabled Device Software Functions: Lifecycle Management and Marketing Submission Recommendations’ Artificial Intelligence
- January 2025: FDA ‘Considerations for Including Tissue Biopsies in Clinical Trials’ Tissue Biopsies
- January 2025: FDA ‘Accelerated Approvals and Considerations for Determining Whether a Confirmatory Trial is Underway’ Guidance for Industry
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Thank you for taking the time to read this Industry Update from S-cubed
Prepared by: Christina Hägglund, QA Manager, S-cubed Ltd Email: christina.hagglund@s-cubed.co.uk