Alejandro Barquero, MPharm, MSc
DanausGT Biotech Ltd.
Spain
Simona Stankeviciute, MD, MSc
Parexel
Lithuania
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Committee of Advance Therapy (CAT) Minutes of the meetings:
Minutes of the meeting on 18-19 January 2023
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-18-19-january-2023_en.pdf
Minutes of the meeting on 15-16 February 2023
https://www.ema.europa.eu/en/documents/minutes/minutes-cat-meeting-15-16-february-2023_en.pdf
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Agendas:
Draft agenda for the meeting on 15-17 May 2023
https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-15-17-may-2023_en.pdf
Draft agenda for the meeting on 19-21 April 2023
https://www.ema.europa.eu/en/documents/agenda/agenda-cat-meeting-19-21-april-2023_en.pdf
Scientific Recommendations Updates:
https://www.ema.europa.eu/en/documents/report/scientific-recommendation-classification-advanced-therapy-medicinal-products-naturally-occurring_en.pdf
https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/advanced-therapies/advanced-therapy-classification/scientific-recommendations-classification-advanced-therapy-medicinal-products
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Updates and Regulatory News:
Reform of the EU Pharmaceutical Legislation
Commission adopted a proposal for a new Directive and a new Regulation, which revise and replace the existing general pharmaceutical legislation on 26 April 2026.
https://health.ec.europa.eu/medicinal-products/pharmaceutical-strategy-europe_en
EMA Mid-term Achievements of its Regulatory Science Strategy to 2025
The European Medicines Agency (EMA) has published a report summarising the mid-term achievements between March 2020 and December 2022 of its RSS to 2025.
https://www.ema.europa.eu/en/documents/report/emas-regulatory-science-strategy-2025-mid-point-achievements-end-2022_en.pdf
EMA Pilots Scientific Advice for Certain High-Risk Medical Devices
https://www.ema.europa.eu/en/news/ema-pilots-scientific-advice-certain-high-risk-medical-devices
EDQM released Microbiological Risk of Contamination Assessment tool (MiRCA)
to enhance safe use of substances of human origin (SoHo) to help users identify and classify potential risks for introducing microbiological contamination during the procurement or processing of tissues and cells and supports decision making to mitigate such risks.
CEP 2.0: New Certification of Suitability to the European Pharmacopoeia Monographs
EDQM have proposed extensive updates to the Certification of suitability to the European Pharmacopoeia monographs (CEP), calling the new version, CEP 2.0, which is a “new-look” CEP that will better meet the current needs of stakeholders and offer both enhanced user-friendliness and greater transparency of the information conveyed without, however, increasing the regulatory burden related to revisions of CEPs.
https://www.edqm.eu/en/what-is-the-cep-2.0#%7B%221508934%22:%5B0%5D%7D
Breyanzi: New Indication Licensed in the EU from 30 March 2023
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Breyanzi (Bristol-Myers Squibb Pharma EEIG). The CHMP adopted a new indication, high grade B cell lymphoma (HGBCL), for patients who relapsed within 12 months from completion of, or are refractory to, first-line chemoimmunotherapy.
https://www.ema.europa.eu/en/documents/smop/chmp-post-authorisation-summary-positive-opinion-breyanzi-ii-05_en.pdf
New Features Further Strengthen Priority Medicines Scheme (PRIME) (04 April 2023)
https://www.ema.europa.eu/en/news/new-features-further-strengthen-priority-medicines-scheme-prime
ACT EU Multi-Stakeholder Platform Kick-Off Workshop
Accelerating Clinical Trials in the EU (ACT EU) Priority Action 3 aims to establish a platform where all stakeholders involved in designing, regulating, performing, and participating in clinical trials can, through regular dialogue, identify relevant scientific, methodological, and technological advances to develop the clinical trials environment in the EU. A two-day kick-off workshop event will be held on from the 22nd to 23rd of June 2023
https://www.ema.europa.eu/en/events/act-eu-multi-stakeholder-platform-kick-workshop
Multi-stakeholder virtual workshop on ICH E6 R3 public consultation is being organised by ACT EU Priority Action 4 (Accelerating Clinical Trials in the EU - PA4).
Dates: 13 July 2023 to 14 July 2023
https://www.ema.europa.eu/en/events/act-eu-pa04-multi-stakeholder-workshop-ich-e6-r3-public-consultation
MHRA to Streamline Clinical Trial Approvals in Biggest Overhaul of Trial Regulation in 20 years
A series of new measures will be introduced by the Medicines and Healthcare products Regulatory Agency (MHRA) with support from partners to make it faster and easier to gain approval and to run clinical trials in the UK. Under the new framework, clinical trials application processes in the UK will be more proportionate, streamlined, and flexible without compromising on safety, helping to cement the UK as an attractive destination for trials, including global “multi-site” trials.
https://www.gov.uk/government/news/mhra-to-streamline-clinical-trial-approvals-in-biggest-overhaul-of-trial-regulation-in-20-years
MHRA - Manufacture of Investigational Medicinal Products – FAQs – When it is considered reconstitution?
According to the MHRAs blog on the manufacture and supply of Investigational Medicinal Products (IMPs) post-Brexit, the most frequently asked question relates to deciding if an activity should be considered manufacturing or reconstitution.
EU GMP Annex 13 states that reconstitution should be undertaken “as soon as practicable before administration”. The MHRA´s interpretation of this is ideally at the bedside, however it may be acceptable for the activities to be performed in the clinic’s pharmacy e.g., where IMP reconstitution is required to be performed in a clean area such as a laminar air flow cabinet. Preparation of IMP to be subsequently stored for use at a later date would not be considered reconstitution.
https://mhrainspectorate.blog.gov.uk/2023/02/03/manufacture-of-investigational-medicinal-products-frequently-asked-questions/
MHRA Announces New Recognition Routes to Facilitate Safe Access to New Medicines with Seven International Partners on 26 May 2023
MHRA new regulatory recognition routes for medicines will be established using approvals from Australia, Canada, the European Union, Japan, Switzerland, Singapore, and the United States.
https://www.gov.uk/government/news/mhra-announces-new-recognition-routes-to-facilitate-safe-access-to-new-medicines-with-seven-international-partners?s=08
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Guidance Documents:
EMA - Guideline on Computerized Systems and Electronic Data in Clinical Trials Adopted from 07 March 2023
https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-computerised-systems-electronic-data-clinical-trials_en.pdf
Companion Diagnostic Initial Consultation Timetable - ATMP
Update 26 April 2023
https://www.ema.europa.eu/en/documents/other/timetable-companion-diagnostic-initial-consultation-atmp_en.pdf
NICE - Life-Changing Gene Therapy for Children with Ultra-Rare Genetic Disorder in Final Guidance Published on 19-Apr-2023
Children with the condition, called aromatic L-amino acid decarboxylase (AADC) deficiency, in the UK, may be now eligible for treatment with eladocagene exuparvovec.
https://www.nice.org.uk/guidance/hst26/resources/eladocagene-exuparvovec-for-treating-aromatic-lamino-acid-decarboxylase-deficiency-pdf-50216322673861
ICH Adopts M7(R2) Guideline And Addendum To The Guideline On Mutagenic Impurities To Limit Carcinogenic Risk.
M7 guideline aims to harmonize the framework for assessing and controlling DNA mutagenic impurities in pharmaceuticals as well as the addendum listing the 21 impurities that should be tested along with their daily permissible limits (PDEs).
ICH adopts S12 Guideline on Nonclinical Biodistribution Considerations for Gene Therapy Products
https://database.ich.org/sites/default/files/ICH_S12_Step4_Guideline_2023_0314.pdf
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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) 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
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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
Consultation closed on 31 May 2023
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