October 2024

Core Reference Webinar

Keep a placeholder for Wednesday 04 December 2024, 12:30 - 13.45 CEST (11.30 - 12.45 UK time)

Title: EMA Policy 0070 Real Life Experience – Voydeya

Presenters: Raquel Billiones, PhD and Alison McIntosh, PhD for the CORE Reference Team 

Register by clicking here.

This webinar will:

  • Provide a brief overview of the Policy 0070 Anonymisation Report and associated anonymisation methodology
  • Outline challenges presented by rare diseases as illustrated by the Voydeya submission
  • Outline challenges posed in maintaining data utility 
  • Review interactions with EMA including the pre-submission meeting
  • Discuss challenges and learnings from the Voydeya anonymisation process. 

Previous related presentation: The CORE Reference webinar on 7 June 2024 included a general introduction to the Policy 0070 Anonymisation Report (AnR) Template. For details, see https://www.core-reference.org/media/1088/core-reference-seminar-emwa-valencia-may-2024-and-webinar-07-june-2024-slides.pdf (refer to webinar slides 21 – 37 or presentation video at 30 – 58 minutes).

Core Reference Utility Survey

The team has now published the full results of the CORE Reference Utility Survey in the EMWA Medical Writing Journal as part of the transparency and disclosure September 2024 special issue of MEW (Medical Writing. 2024;33(3):38–45). The CORE Reference team would once again like to extend their thanks to all those who took time to complete the survey. 


Medicines and Vaccines

ICH

1. The ICH has published the findings of their study aimed at monitoring the adequacy of implementation and adherence to ICH Guidelines by regulatory authorities. This 2024 study aimed to monitor the adequacy of implementation and adherence to ICH Guidelines by regulatory authorities – continuing the assessment initiated in 2019 and repeated in 2021. The 2024 results will be used to support training needs as well as ICH-membership related activities.

2. ICH E8(R1) General Considerations for Clinical Studies now has an Introductory Training Video available.

CTR and CTIS

1. CTIS newsflash, dated 8 October 2024, included updates on a helpdesk for non-commercial sponsors, the expiration of trials, and system improvements (see latest system release).

2. CTIS walk-in clinic on 20 November 2024 (16:00 Amsterdam time) is open to all sponsor organisations, including pharmaceutical companies, contract research organisations, small and medium-sized enterprises and academic organisations. It will be a live broadcast; no registration is required for those wishing to follow the live broadcast on EMA’s website

UK and MHRA News

1. The Medicines and Healthcare products Regulatory Agency (MHRA), in partnership with the Health Research Authority (HRA), hosted a short webinar on 15 October 2024 16.00 BST to introduce joint plans to share draft guidance with stakeholders and to set out the anticipated timelines for implementing the new UK Clinical Trials regulations. The webinar will be recorded.

2. The HRA, in collaboration with the MHRA, has launched an informal consultation on their draft inclusion and diversity guidance which closes on 9 December 2024. The aim of the guidance is to help ensure research is designed to include people who could be impacted by the findings, and that people underserved by research are not overlooked.
They have also developed a set of questions and supporting guidance for researchers to consider when they design clinical trials.

FDA Guidance and News

1. The latest release notes for the Modernised PRS were issued 2 October 2024 and include enhancements to and remodelling of the record access list as well as the addition of numbering in the Outcome Measures Module. Two new demonstration videos have also been posted on Fast Forward from ClinicalTrials.gov.

2. The FDA has released the final guidance Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers. The guidance provides information for all stakeholders on the use of electronic systems, electronic records, and electronic signatures in clinical investigations of foods, medical products, tobacco products, and new animal drugs.

3. In October 2024, FDA issued final recommendations to sponsors for collection of a core set of patient-reported clinical outcomes in cancer clinical trials and related considerations for instrument selection and trial design.

4. The FDA and the National Institutes of Health (NIH) worked together to develop a draft glossary of clinical research terms related to innovative clinical trial design. The document helps bring clarity to terms that are inconsistently used within the scientific community. The glossary includes 37 clinical research terms related to innovative clinical study designs, including studies using real-world data (RWD) to generate real-world evidence (RWE), that support scientific, clinical, and/or regulatory decision-making. 

5. FDA will hold a virtual public webinar on Friday 8 November 2024 from 2:00 pm – 3:00 pm ET on “Informed Consent – More than Just Another Document to Sign?” The event is free and open to the public. However, registration is required and open until the meeting starts. Registration and details of the webinar are here. 

6. In July, the FDA sent out a request for comments on proposals for new biosimilars product development guidance. The article by Hayes reports how “Biosimilars developers have strongly rejected the [FDA] proposal to develop product-specific or class-based guidance and are urging the agency to instead put its efforts toward updating its cornerstone guidance documents for the sector.”

7. The FDA has released the final guidance on Core Patient-Reported Outcomes in Cancer Clinical Trials. This guidance provides recommendations to sponsors for collection of a core set of patient-reported clinical outcomes in cancer clinical trials and related considerations for instrument selection and trial design.

EMA Guidance and News

1. The joint EC/HMA/EMA multi-stakeholder workshop on pharmacogenomics was held on 24 September 2024. The presentations and video recording are available here.

2. EMA now publishes a monthly European Regulatory Oncology Newsletter which is aimed at anyone with an interest in cancer medicines. Each month, they highlight the latest scientific opinions on oncology medicines in the EU, and share news about events, initiatives and activities from EU regulators in the field. Subscribe to receive the newsletter here. Read the October 2024 issue here.

3. The slides and recording of the ACT EU multi-stakeholder platform (MSP) annual meeting on 22 October 2024 can be found here. The MSP acts as a vehicle for clinical trials stakeholders and regulators to come together, voice their views and collaborate to improve the clinical trials environment for European patients and citizens.

4. An interim report is available on the European medicines regulatory network's experience with analysing patient-level clinical study data submitted voluntarily by marketing authorisation applicants and holders. This report offers preliminary insights into the regulatory benefits of including clinical study data in the marketing authorisation application dossier. It includes experience gained between September 2022 and December 2023.

Transparency and Disclosure Resources and News

1. The PHUSE Data Transparency Autumn Event took place from 17–19 September 2024. For those unable to attend presentations and event recordings can be accessed here.

2. The September edition of the EMWA  journal Medical Writing is dedicated to clinical trial transparency and disclosure (Volume 33, Issue 3, 2024). The whole journal is open access and feature articles can be downloaded from here.

3. A webinar to be held 7 Nov 2024 (5-5.30 pm GMT) entitled “Translation & Redaction - AI applied in Regulatory Content Preparation” will explore the impact of AI on regulatory content preparation; and specifically on document translation and information redaction. Register for the free event here.

4. Save the date: The Data Transparency Autumn Virtual Event will take place from 4–6 February 2025. Presentations will be delivered across the three days from 15:00-17:30(GMT) 10:00-12:30(EST). Call for Speakers is open.

5. Webinar: Thursday, 21 November 2024 (10am EST)  Best Practices for Clinical Data Anonymization Using the Quantitative Methodology. Register for free here.

6. The European Commission has launched a public consultation on the draft delegated act on access to online platform data for vetted researchers under the DigitalServicesAct (DSA). The new framework for access to data from very large online platforms and very large search engines is a key measure of the DSA to increase these platforms’ transparency and accountability. The consultation runs until 26 Nov 2024.

EMA Policy 0070 in Practice

  1. The  EMA is hosting a webinar on the next step in the resumption of the Clinical Data Publication (Policy 0070) entitled “Clinical Data Publication (Policy 0070) webinar - Step 2”.  Registration in advance is required. The webinar on Thursday 14 November 2024, (14:00 -15:00 [GMT]) will present EMA plans on the proposed expansion of activities (Step 2) related to Policy 0070 from April 2025 and the handling of legacy procedures, while also providing guidance for applicants and industry stakeholders. Participants will have the opportunity to ask questions and will need to register should they wish to participate. The agenda is available here.

Declaration of Helsinki

WMA Declaration of Helsinki – Ethical Principles for Medical Research Involving Human Participants was updated and a new version released on 21 October 2024. The updated version can be downloaded from here. 

According to the WMA substantive changes can be categorised in two areas:

  • Participant-centred inclusion, respect and protection
  • Research beneficence and value, including the pursuit of “individual and public health”, upholding scientific rigour and integrity, and considered distribution of benefits, risks and burdens.

Further, WMA states “The 2024 revision of the Declaration of Helsinki replaces ‘subjects’ with ‘participants' throughout out of respect for the rights, agency, and importance of those individuals.”

Development Strategy News

1. "Guidance on the validity of clinical studies" (for joint clinical assessments) from the Member State Coordination Group on HTA (HTACG) is on the EU Commission website. Download the 44-page V1.0 document dated 04 July 2024 here. It provides a high-level overview of Clinical study designs, including master protocols, RWD and real-world evidence and registries, within the scope of Health Technology Assessment.

2. FDA Final Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations - Questions and Answers. 01 Oct 2024.

3. Register now to join FDA on Friday 8 November 2024, from 14.00 -15.00 pm EST for a free virtual webinar: Informed Consent – More than Just Another Document to Sign? FDA leaders will:

  • Give an update on the FDA’s efforts to help improve informed consent materials so that they are more understandable for participants
  • Recommend how informed consent can be presented in a clear, comprehensible way
  • Discuss how revised consent can help individuals make an informed decision on whether to join a clinical trial.

Registrants can submit questions for speakers on the registration page and during the event.

4. An article by Hughes and Rodriguez-Chavez “The Evolution of Decentralised Clinical Trials: Blending Innovation and Regulation in the Digital Age” provides insights of the new patient-centric approach of clinical research using decentralised clinical trials (DCTs). This article discusses the benefits of DCTs, new technologies (including AI/ML) currently available to conduct a DCT, and the anticipation of DCTs in the foreseeable future.

5. The MRCT Center is organising the 2024 Annual Symposium on 14-15 November 2024. Participants can join this free event either on-site in Boston or virtually. Main events of the symposium include keynote addresses with Khair ElZarrad (Director of the Office of Medical Policy at the FDA) on the perspectives on current trends and future developments in clinical research and regulation and Steffen Thirstrup (CMO, European Medicines Agency) on the future of data sharing and the European Health Data Space. Register here.

6. The ACTEU (Accelerating Clinical Trials in the EU) initiative has established a multi-stakeholder platform to create a better environment for clinical trials in the EU. EMA is organising the annual meeting of the platform in Amsterdam on 22 October 2024. At this event, EMA:

  • Reviewed the key achievements of the ACT EU initiative and their impact on clinical research
  • Discussed the current clinical trials landscape
  • Looked to the future to visualise what success looks like for different stakeholder groups

The event slides are here and the video recording will be made available soon. 

7. The updated CIOMS Glossary of ICH terms and definitions, Version 7, is now free for download on the CIOMS website. New additions include definitions from the ICH guidelines E11A (Paediatric Extrapolation. Step 4 [final], 21 August 2024), M13A (Bioequivalence for Immediate-Release Solid Oral Dosage Forms. Step 4 [final], 23 July 2024), and Q4B(R1) (Evaluation and Recommendation of Pharmacopoeial Texts for Use in the ICH Regions. Final version, 5 June 2024).

8. A list of events or documents related to Clinical Trials or in Regulatory Affairs that are expected in November 2024 is compiled and presented here.

Artificial Intelligence/Machine Learning

1. The DIA Session on Leveraging AI to Transform Regulatory Medical Writing is free to attend:

Title: Leveraging AI to Transform Regulatory Medical Writing

Speaker: Axel Byrd Le Sage, YSEOP

When: 23 Oct, 2024 08:00 AM Eastern Time (US and Canada) 

In this session, Axel will explore how AI is revolutionising the medical writing landscape, particularly in clinical trials. He will also look at how AI-driven tools can automate key documentation tasks like Clinical Study Reports (CSRs) and clinical trial narratives, streamline workflows, and enhance compliance with regulatory requirements. He will also highlight real-world use cases and practical strategies for integrating AI into existing medical writing processes to improve efficiency and accuracy. Contact pooja.phogat@krystelis.com for the link to join the session.

2. In July 2024, Neyarapally GA et al published a paper titled Description and Validation of a Novel AI Tool, LabelComp, for the Identification of Adverse Event Changes in FDA Labeling. This describes AI to support postmarket drug safety research and discusses a novel AI tool created and validated by FDA that could enable researchers to efficiently identify and analyze safety-related drug labelling changes.

3. The EMA has published an updated Reflection paper on the use of AI in the medicinal product lifecycle, dated 9 September 2024. This paper reflects on principles relevant to the application of AI and machine learning (ML) at any step of a medicines' lifecycle, from drug discovery to the post-authorisation setting.

4. The article Influence of believed AI involvement on the perception of digital medical advice by Reis et at. looks at the public perception of AI-based tools. Their findings point toward an anti-AI bias when receiving digital medical advice, even when AI is supposedly supervised by physicians.

5. The paper Trust and Inclusion in Digital Health: The Need to Transform Consent by Brightwell et al looks at the interplay between data protection regulations and digital transformation initiatives such as health apps, telemedicine and the importance of patient consent.  “The consent interface in a health app can impact trust and inclusion in digital health for privacy-sensitive people.”

6. FDA has published a special communication entitled “Perspective on the Regulation of Artificial Intelligence in Health Care and Biomedicine.” In this publication the authors review the history of the US FDA regulation of AI; present potential uses of AI in medical product development, clinical research, and clinical care; and present concepts that merit consideration as the regulatory system adapts to AI’s unique challenges.

7. An article by Heta Mehta  entitled “The evolving role of contact-free AI devices in clinical trials” has been published in RF Quarterly. 2024;4(3):34-41 and can be downloaded for free from here.

8. EMA and Heads of Medicines Agencies (HMA) are hosting a virtual workshop on AI on 5 November 2024 (08:00 - 17:00). Register in advance here. 

The workshop will provide updates on:

  • the state-of-the-art of AI developments with keynote speakers;
  • policy and legislative environment and the revision of the AI reflection paper, and discuss their impact;
  • HMA/EMA activities conducted under the multi-annual AI workplan.

9. The open access book Artificial Intelligence and Machine Learning in Health Care and Medical Sciences by Simon and Aliferis can be downloaded for free as pdf or epub here. The book focuses on the transparent, ethical and unbiased use of AI in healthcare. “It underscores that while AI can truly revolutionize healthcare, responsible implementation is essential.”

News from Asia Regulators

1. PMDA Japan has a Youtube channel “PMDA Channel” in which the health authority creates various content about regulations of drugs and medical devices in Japan, including content in English. A recent video released on the channel, “Regulatory Approach to Software as a Medical Device (SaMD) in Japan”, provides definition, classification, and general review points of SaMD in Japan.

2. The Medical Device Administrative Control System (MDACS) is an administrative control system in Hong Kong which is run by the Medical Device Division office under the Department of Health and tasked to oversee the listing system for medical devices and adverse events reporting system. The latest Guidance Notes for Overview of the Medical Device Administrative Control System Ed. 7, revised on 30 September 2024, contains updated sections on the Validity of Listing Approval and Application Renewal.











Medical Devices


This subsection covers transparency in relation to medical devices, and the emerging intersection of the regulatory medical devices and the regulatory drugs spaces. Devices information or regulations that impact the reporting of device studies are also covered. Contextualisation is provided, where possible, to help readers understand how the information could be used. 


General Updates and News


1. BioMed Alliance, an organisation representing biomedical societies in Europe has issued a press release urging decision-makers to allow more time for a comprehensive evaluation of the EU Medical Device Regulation (MDR).  The alliance believes that plans made in haste to change MDR risk further neglecting the needs of patients who rely on medical technologies.


2. Check out this free white paper on Regulatory Compliance for AI-Enabled Medical Devices, covering both EU and US requirements.


3. The MDCG guidance on borderline between medical devices and medicinal products under Regulation (EU) 2017/745 on medical devices (MDCG 2022-5 Rev.1) has been updated. Healthcare products are normally regulated as medicinal product or as medical device but not both. Borderline products are those whose regulatory classifications are unclear. The guidance aims to provide clarity.


4. The European Parliament has passed a resolution highlighting the urgent need to revise the EU MDR and EU IVDR to avoid shortages, delays, and disparities. The key points raised are patient access & health equity, fast-track approvals to facilitate innovation, and transparency & harmonisation.


5. TGA Australia has updated the guidance on Understanding personalised medical devices rules (including 3D-printed devices). Check out the TGA Fact Sheet on personalised devices here.


6. The International Medical Device Regulation Forum (IMDRF) is a voluntary group of medical device regulators from around the world who have come together to build on the strong foundational work of the Global Harmonization Task Force on Medical Devices (GHTF) and aims to accelerate international medical device regulatory harmonization and convergence. IMDRF has just opened the public consultation on Considerations for the Selection of IMDRF Adverse Event Terminology. The IMDRF AE Terms are used to code AEs for medical devices (equivalent to MedDRA in medicinal products). The purpose of the document is to provide further guidance on the correct application and consistent use of the terminology, including general coding principles on reporting incidents using IMDRF codes and providing examples to address common coding challenges.


In addition, IMDRF has also released the Technical document on Common Data Set for Adverse Event Data Exchange between IMDRF Regulators, aimed to provide a framework to standardize the exchange of AE data between regulators.


7. The UK MHRA has released a new statutory instrument (SI) The Medical Devices (Post-market Surveillance Requirements) (Amendment) (Great Britain) Regulations 2024. The new legislation aims to introduce clearer and risk-proportionate requirements that improve the safety of medical devices across Great Britain and provide certainty for manufacturers ahead of introducing the wider future regulatory regime.

	
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