What’s New in Clinical Development Practices & Regulations Quarter 4 – 2023

Our Industry Update Newsletter returns for the final update of 2023.

As always, this quarter’s update has been expertly compiled by Christina Hägglund, QA Manager.


Clinical Trial Information System (CTIS) – Transparency Rule and Updated Sponsor Handbook

There is a revised rule for the publication of information on clinical trials submitted via CTIS. It is likely to become effective mid-2024, following implementation of the necessary technical aspects. Stakeholders, including patients and healthcare professions, will then experience faster and more efficient access to clinical trial information. EMA revised transparency rules for CTIS – S-cubed Global (s- cubed-global.com)

An updated version of the CTIS Handbook for Sponsors was issued in November by the EMA. clinical- trial-information-system-ctis-sponsor-handbook_en.pdf (europa.eu)

The ‘COMBINE’ Project

The European Commission (EC) and competent authorities of EU Member States (MS) have published information on the interface between the Regulations on Clinical Trials of Medicinal Products, Medical Devices and In vitro Diagnostics. Combined studies (europa.eu)

Accelerating Clinical Trials in the European Union (ACT EU)

A website has been launched for the ACT EU initiative and contains updates on the progress of priority actions, as well as news and events. Homepage (europa.eu)

EMA Guidance – Post-Authorisation Procedural Advice for Users of the Centralised Procedure

This guidance document, issued in November, addresses a number of questions which Marketing Authorisation Holders (MAHs) may have on post-authorisation procedures. european-medicines- agency-post-authorisation-procedural-advice-users-centralised-procedure_en.pdf (europa.eu)

Addressing Critical Shortages in Medicines in the EU

In October, the EMA published details of a new voluntary system developed by the EMA Medicines Shortages Steering Group (MSSG). The new system allows EU MS to support each other when facing critical medicines shortages. mssg-solidarity-mechanism_en.pdf (europa.eu)

EMA and Health Technology Assessment (HTA) Bodies Form a Permanent Collaboration

The Regulation on Health Technology Assessment ((EU) 2021/228) came into force in January 2022 and will apply from January 2025. It aims to harmonise the clinical and scientific aspects of HTA.
The EMA and HTA bodies have formed a permanent collaboration and will work together on clinical assessments, scientific consultations and identification of emerging health technologies. Towards a permanent collaboration framework for EMA and Health Technology Assessment bodies | European Medicines Agency (europa.eu)

Phasing out Covid-19 Regulatory Flexibilities

With immediate effect, the EMA, EC and the Heads of Medicines Agencies (HMA) have announced that the regulatory flexibilities introduced during the pandemic, may no longer be granted. Phasing out of extraordinary COVID-19 regulatory flexibilities | European Medicines Agency (europa.eu)

FDA Pilot Programme for Rare Diseases

At the end of September, the FDA revealed plans to help further accelerate the development of novel drug and biological products for rare diseases, via a pilot programme. Federal Register :: Support for Clinical Trials Advancing Rare Disease Therapeutics Pilot Program; Program Announcement


01 January 2025 – New Requirements for UK Medicine Supply

Windsor Framework will ensure medicines can be approved and licensed on a UK-wide basis by the Medicines and Healthcare products Regulatory Agency (MHRA) and that the same packaging and labelling can be used across the UK. Manufacturers may supply medicines in legacy EU packaging until 31 December 2024, and packs already on the UK market and within the supply chain may remain until the date of their expiry.
For measures commencing 01 January 2025 Windsor Framework medicines announcement – GOV.UK (www.gov.uk) and for further information Implementation of the Windsor Framework – S-cubed Global (s-cubed-global.com)

International Recognition Route for Authorised Medicines in the UK

As mentioned in the Q3 edition of this Newsletter, the MHRA has created a new international recognition route for medicines, utilising pre-existing approvals from Australia, Canada, the EU, Japan, Switzerland, Singapore and the USA. The new framework will support patients in the UK with medicines approved by trusted regulatory partners. International Recognition Procedure – GOV.UK (www.gov.uk). The MHRA has now also launched an online Eligibility Checker Tool. Eligibility Checker and submitting your Marketing Authorisation application – GOV.UK (www.gov.uk)

New Streamlined Notification Scheme for Lowest-Risk Clinical Trials

The MHRA has announced a new streamlined notification scheme for the lowest-risk clinical trials, enabling them to be processed by the MHRA within 14 days. New streamlined notification scheme for lowest-risk clinical trials marks start of MHRA overhaul of regulation – GOV.UK (www.gov.uk) and Clinical Trial Legislation in the UK – S-cubed Global (s-cubed-global.com)

Decentralised Clinical Trials

The Health Research Authority (HRA) has published a position statement to confirm the current UK position on the use of decentralised trial methods. Decentralised trial methods position statement – Health Research Authority (hra.nhs.uk)

MHRA Announces Three New UK Approved Bodies to Certify Medical Devices

Following this announcement, the UK’s capacity for certifying safe and effective medical devices for healthcare professionals and the public has nearly doubled. Three new UK Approved Bodies.

MHRA Addressing Clinical Trial Application (CTA) Backlog and Assessment Delays

The MHRA has issued performance metrics relating to the CTA backlog CT-Performance-Metrics_2208- 2307.pdf (publishing.service.gov.uk). The Agency has implemented several crisis measures to improve delays in assessment of clinical trials and established medicines MHRA performance data for assessment of clinical trials and established medicines – GOV.UK (www.gov.uk) and confirms these measures have had a positive impact CT-Performance-Metrics_2210_2309.pdf (publishing.service.gov.uk)


Call for Comprehensive Reform to EU Regulatory Framework for Medical Technologies

In September, representatives from the medical technology industries across the EU (plus Switzerland) sent an open letter to the European Commissioner for Health emphasising the importance of a regulatory system that ensures medical technologies reach healthcare systems in a timely manner. The letter implies the Medical Devices Regulation (MDR) and the In Vitro Diagnostic Medical Devices Regulation have not fully achieved their intended objectives. medtech-europe_open-letter- kyriakides.pdf (medtecheurope.org)

Guidance ‘Extension of the MDR Transitional Period and Removal of ‘Sell off’ Periods’

Published in July, the EC revised the above guidance which now includes new questions and footnotes. mdr_proposal_extension-q-n-a.pdf (europa.eu)

Update Manual from the Medical Device Coordination Group (MDCG)

In September, the MDCG updated the manual on borderline and classification in the community regulatory framework for medical devices. md_borderline_manual_en.pdf (europa.eu)

Guidance on Harmonisation of Personalised Medical Devices

In September, the voluntary group ‘International Medical Device Regulators Forum’ (IMDRF) issued updated guidance (Edition 2) entitled ‘Personalized Medical Devices – Regulatory Pathways’. IMDRF_PMD WG_N58 FINAL_2023 (Edition 2)_0.pdf

Guiding Principles for Machine Learning-Enabled Medical Devices (MLMD)

In October, the MHRA, FDA and Health Canada issued guiding principles for predetermined change control plans (PCCPs) for MLMDs, aiming to remove the regulatory burden for developers. MHRA and international partners publish five guiding principles for machine learning-enabled medical devices – GOV.UK (www.gov.uk)

Guidance Document ‘Off-the-shelf Software Use in Medical Devices’

In August, the FDA issued the above Guidance for Industry. Off-The-Shelf-Guidance_0.pdf (fda.gov)


Withdrawal of Consent in Oncology Clinical Trials

An article published in JAMA Oncology evaluates the numbers and types of participants at high risk of withdrawing from oncology clinical trials. Rates of and Factors Associated With Patient Withdrawal of Consent in Cancer Clinical Trials | Oncology | JAMA Oncology | JAMA Network

European Federation of Pharmaceutical Industries and Associations (EFPIA) White Paper

A White Paper was issued by EFPIA in September entitled ‘Improving the Understanding, Acceptance and Use of Oncology-Relevant Endpoints in HTA Body/Payer Decision-Making’. improving-the- understanding-acceptance-and-use-of-oncology-relevant-endpoints.pdf (efpia.eu)


Final FDA Guidance Documents:

Draft FDA Guidance Documents:

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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