EU LRA Watchdog - August 2023

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Alejandro Barquero, MPharm, MSc
DanausGT Biotech Ltd.
Spain

Simona Stankeviciute, MD, MSc
Parexel
Lithuania

Committee of Advance Therapy (CAT)

Minutes of the meetings

Minutes of the meeting on 19-21 April 2023
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-19-21-april-2023_en.pdf

Minutes of the meeting on 22-23 March 2023
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-22-23-march-2023_en.pdf

Agendas

Agenda for the meeting on 12-14 July 2023
https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-12-14-july-2023_en.pdf

Draft agenda for the meeting on 15 June 2023
https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-15-june-2023_en.pdf

Other

CAT quarterly highlights and approved ATMPs, published May 30, 2023
https://www.ema.europa.eu/en/documents/report/cat-quarterly-highlights-approved-atmps-may-2023_en.pdf

Scientific Recommendations updates

CAT has convened on July 13 to discuss and align the quality, non-clinical and clinical parts of the guideline considering the comments received.
No updated guidelines have been published in the EU region since previous Watchdog report.

Updates and regulatory news

Summary from the Committee for Advanced Therapies (CAT) (16 May 2023)

CAT held a stakeholder meeting on Tuesday 16 May 2023 from 15:00 to 18:30 CET. The meeting was conducted in a hybrid mode, physical (at the EMA office in Amsterdam) and virtual attendance (via Webex). Interested parties from industry and academia were invited to submit topics for discussion.

Some key topics that were discussed during the meeting:

EU Commission: presented a proposal for a centralised GMO/ERA evaluation for clinical trial applications in the EU
European Medicines Agency (EMA): presented the initial work/findings done by a focus group comprising representatives from HMA, CHMP, PRAC, CAT, CMDh, Industry, and EMA on ‘predictability of marketing authorisation application submissions’
An update from National Competent Authority: Recommendation on a long-term follow-up of patients treated with high dose of adeno-associated viral vector products due to potential safety concerns
Committee for Advanced Therapies (CAT)
- a draft guideline from 2019 on ‘quality, non-clinical and clinical requirements for investigational ATMPs in clinical trials’ will be finalized soon. Expected to be published by the end of the year.
- The use of registries for regulatory decision-making using a case on Spinal Muscular Atrophy (SMA) for a pilot study of Real-World Evidence (RWE) initiatives.
- HTA Regulation recent experience and learnings from a bilateral agreement between EMA and EUnetHTA21 on ATMPs; optimisation of post-licensing evidence generation (PLEG) planning.

Opening procedures at EMA to non-EU authorities (OPEN) initiative

EMA piloted the OPEN initiative during the Coronavirus disease (COVID-19) public health emergency, to support international collaboration in the evaluation and supervision of COVID-19 medicines. EMA extended the scope of OPEN in June 2023 based on a review of the pilot's first year, to cover medicines:

intended to help combat Antimicrobial resistance;
designated under the PRIME: priority medicines scheme (temporarily excluding advanced therapy medicinal products), and other products that address a high unmet medical need;
intended to address public health threats and public health emergencies.

The following authorities with whom EMA has a permanent confidentiality arrangement in place: Therapeutic Goods Administration, Australia (TGA), Brazilian Health Regulatory Agency (ANVISA), Health Canada (HC), Ministry of Health, Labour and Welfare/Pharmaceuticals and Medical Devices Agency, Japan (MHLW/PMDA), Swiss Agency for Therapeutic Product (Swissmedic), and the World Health Organization (WHO).
https://www.ema.europa.eu/en/documents/other/questions-answers-open-framework_.pdf

Tenth industry stakeholder platform on research and development support

This was the tenth in a series of regular meetings between regulators and representatives of industry stakeholder organisations. It addresses all areas of evidence generation along the medicine’s life cycle and related to development support activities, such as scientific advice and qualification, as well as specifics for paediatric and orphan medicines. Presentations on the following topics are published in the link below:

progress with the implementation of the recommendations to further strengthen the PRIME scheme,
progress with the implementation of initiatives to optimise the application of the PIP framework,
cooperation at the HTA/regulatory interface,
development of complex generics,
update on latest developments in scientific advice along with reports from both the multi-stakeholder workshop on qualification of novel methodologies and from the Focus group to discuss opportunities for provision of scientific advice for medicinal product developments also comprising of devices and/or companion diagnostic.

https://www.ema.europa.eu/en/events/tenth-industry-stakeholder-platform-research-development-support

EMA annual activity report for 2022 has been published

https://www.ema.europa.eu/en/documents/report/annual-activity-report-2022_.pdf

Big Data Work Group at EMA annual plan updated

https://www.ema.europa.eu/en/documents/work-programme/workplan-2022-2025-hma/ema-joint-big-data-steering-group_en.pdf

EMA has held the fifth meeting of the Industry Stakeholder Group (ISG).

Topics that were discussed in this meeting are now published, and they include among others high-level progress development updates of Pharma legislation review, ACT EU and CTIS implementation, new EMA Working Party governance and Quality Innovation Group.

https://www.ema.europa.eu/en/events/fifth-industry-standing-group-isg-meeting

EMA, the European Commission (EC) and the Heads of Medicines Agencies (HMA) are phasing out the extraordinary regulatory flexibilities for medicines put in place during the COVID-19 pandemic.

For already approved labelling flexibilities, e.g., the English-only labelling for COVID-19 vaccines, their application will be extended until the end of 2023, in order to ensure a smooth phase-out and avoid any supply difficulties or other disruptions due to a sudden change in applicable requirements. After 2023, the regular mechanisms foreseen in the legislation in relation to labelling exemptions should be followed. Concerning on-site GMP and GDP inspections, these have been restarted after being postponed or carried out remotely during the pandemic, however, a considerable number of postponed inspections still need to be carried out. The validity of GMP and GDP certificates has currently been extended until the end of 2023, and the GMDP Inspectors Working Group will issue in the coming months an update on the approach for 2024.

https://www.ema.europa.eu/en/news/phasing-out-extraordinary-covid-19-regulatory-flexibilities

Reform of the EU General Pharmaceutical Legislation
Joint Position Paper on the Hospital Exemption Scheme for ATMPs (ARM, ISCT, EFPIA, EuropaBio & EUCOPE)
https://www.efpia.eu/media/1wwnhpem/arm-efpia-europabio-eucope-isct-joint-paper-on-hospital-exemption-scheme-july-2023.pdf

Guidance Documents

European Medicines Agency Guidance for applicants seeking access to PRIME scheme

https://www.ema.europa.eu/en/documents/other/european-medicines-agency-guidance-applicants-seeking-access-prime-scheme_en-0.pdf

EMA pre-authorisation procedural advice for users of the centralised procedure Guidance updated

https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/european-medicines-agency-pre-authorisation-procedural-advice-users-centralised-procedure-document_en.pdf

Guidance for the transition of clinical trials from the Clinical Trials Directive to the Clinical Trials Regulation – 19 July 2023

https://health.ec.europa.eu/latest-updates/guidance-transition-clinical-trials-clinical-trials-directive-clinical-trials-regulation-2023-07-19_en

Clinical trials regulation (EU) No. 536/2014 – Questions & Answers July 2023

https://health.ec.europa.eu/system/files/2023-07/regulation5362014_qa_en.pdf

Open consultations

Reflection paper on establishing efficacy based on single arm trials submitted as pivotal evidence in a marketing authorisation under consultation.

https://www.ema.europa.eu/en/establishing-efficacy-based-single-arm-trials-submitted-pivotal-evidence-marketing-authorisation

Open from 21 April 2023 to 30 September 2023

Draft version of ICH E6(R3) Harmonised Guideline Good Clinical Practice has been endorsed on 19 May 2023 and it is currently under public consultation.

https://database.ich.org/sites/default/files/ICH_E6%28R3%29_DraftGuideline_2023_0519.pdf

Open from 19 May 2023 to 26 September 2023

EMA is seeking input on the use of Artificial Intelligence (AI) in the medicinal product lifecycle.

https://www.ema.europa.eu/en/documents/scientific-guideline/draft-reflection-paper-use-artificial-intelligence-ai-medicinal-product-lifecycle_en.pdf

Open from 19 July 2023 to 31 December 2023

Also, a joint Heads of Medicines Agencies (HMA)/European Medicines Agency (EMA) AI workshop is organised in November 2023. Registrations will open in September.

https://www.ema.europa.eu/en/events/joint-heads-medicines-agencies-hma-european-medicines-agency-ema-ai-workshop-smart-regulation

Concluded consultations

The European Pharmacopoeia (Ph. Eur.) has published the 59 texts (1 new general chapter, 5.1.13. Pyrogenicity, and 58 revised texts) concerned by the rabbit pyrogen test (RPT) replacement strategy for public consultation in Pharmeuropa 35.1

https://www.edqm.eu/en/-/public-consultation-on-ph.-eur.-rabbit-pyrogen-test-replacement-texts-in-pharmeuropa-35.1

Consultation closed on 31 March 2023

British Pharmacopoeia - Non-mandatory, best practice guidance for T cell and NK cell characterisation assays

https://www.pharmacopoeia.com/bp-consultations/guidance-on-t-cell-and-nk-cell-characterisation-assays

Consultation closed on 31 May 2023

EMA Reflection paper on the “Criteria to be considered for the evaluation of new active substance (NAS) status of biological substances” open consultation

https://www.ema.europa.eu/en/documents/scientific-guideline/reflection-paper-criteria-be-considered-evaluation-new-active-substance-nas-status-biological_en.pdf