Regulatory Round-up - April 2022

EUROPE

European Directorate for the Quality of Medicines (EDQM)

Pharmeuropa 34.2 released

All new European Pharmacopoeia (Ph. Eur.) texts, and texts that have undergone technical revisions are published in Pharmeuropa for public consultation. The deadline for comments on Pharmeuropa 34.2 is 30 June 2022. Two tests of interest to the community that have been updated include:

• 2.6.30. Monocyte activation test
• 2.6.7. Mycoplasmas

Note: to view the updated texts and comment, individuals must have the appropriate EDQM account.

New general text on comparability of alternative analytical procedures: European Pharmacopoeia launches public consultation

The European Pharmacopoeia (Ph. Eur.) at the European Directorate for the Quality of Medicines & HealthCare (EDQM) is seeking feedback on its new general text on the comparability of alternative analytical procedures (5.27).

The new general text is published in Pharmeuropa 34.2 for public consultation from April until the end of June 2022.

Note: to view the updated texts and comment, individuals must have the appropriate EDQM account.

EDQM helping increase tissue donation thanks to a better understanding of post-mortem blood testing practices

The EDQM/Council of Europe, within the framework of a Grant Agreement with the European Commission, has led a project to better understand screening practices in Europe of blood samples obtained post-mortem. A group of leading experts in the field was set up to benchmark practices among European tissue establishments and develop recommendations to improve post-mortem donor testing. The results of this work has been published. The ultimate goal of the project is to support future policy decisions that will make it possible to increase the number of tissue donors while maintaining the required safety levels for the detection of infectious diseases.

EDQM sticker no longer to appear on Certificates of suitability

As of the 1 May 2022, the European Directorate for the Quality of Medicines & HealthCare (EDQM) sticker will no longer appear on Certificates of suitability to the European Pharmacopoeia monographs (CEPs). This adhesive sticker (see picture here) will no longer be pasted on CEPs next to the wet signature.

Any certificate granted from this date on the specific EDQM paper and without the EDQM sticker shall be considered to be a valid certificate.

European Medicines Agency (EMA)

Yescarta receives positive CHMP opinion for extension of indication

On 22 April 2022, 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 Yescarta. The marketing authorisation holder for this medicinal product is Kite Pharma EU B.V.

The CHMP adopted a new indication for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy.

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR) after a decision on this change to the marketing authorisation has been granted by the European Commission.

EMA and the EUnetHTA 21 consortium set priorities for their collaboration

The European Medicines Agency (EMA) and the European Network for Health Technology Assessment (EUnetHTA) 21 consortium have published a joint work plan until 2023. The focus of the plan is to prepare, from a methodological and operational perspective, for the coming into application of the Regulation on Health Technology Assessment (EU) 2021/228 in January 2025 after a 3-year implementation period.

More information is available through the press release.

EMA releases adjusted fees for 2022

General, non-pharmacovigilance fees payable to EMA by applicants and marketing-authorisation holders are increasing by 0.3% and 5.3% as of 1 April 2022, to reflect the inflation rate adjustments of 2020 and 2021 respectively.

Full details of the new fee levels are available in Commission Regulation (EU) 2022/510 amending Council Regulation (EC) No 297/95, its implementing rules, and the corresponding explanatory note on fees, published on 29 March 2022. These documents include the new fees for all types of procedures handled by the Agency, except for pharmacovigilance procedures.

United Kingdom

Medicines and Healthcare products Regulatory Agency (MHRA)

Early Access to Medicines Scheme (EAMS) integrated into UK Legislation

The Early Access to Medicines Scheme (EAMS) has now been integrated into UK Statutory Instruments (2022 No.352) and came into force on 14 Apr 2022. The Human Medicines (Amendments Relating to the Early Access to Medicines Scheme) Regulations 2022 detail the amendments to the Human Medicines Regulation.

Additional guidance and a revamp of the MHRA EAMS page are currently under development and further information will be provided once available.

Public consultation on a new Code of Practice for the Expert Advisory Committees

Stakeholders and the UK public are invited to have their say on how the Medicines and Healthcare products Regulatory Agency (MHRA) manages the conflicts of interest for independent experts and how patients can be more involved in expert committee meetings to ensure consistency and transparency.

A six week consultation was launched on the 12 Apr 2022 and closes on 24 May 2022 and the results will impact all expert groups including:

• The Commission on Human Medicines (CHM), which advises MHRA on the safety, efficacy and quality of medicinal products;
• The Devices Expert Advisory Committee (DEAC), which provides MHRA with advice on a wide range of aspects relating to the introduction and safe use of medical devices;
• The British Pharmacopoeia Commission (BPC), which provides official standards for pharmaceutical substances and medicinal products;
• UK Stem Cell Bank Steering Committee (UKSCBSC), which oversees the activities of the UK Stem Cell Bank and UK research involving established human embryonic stem cell lines, whether obtained from the bank or from elsewhere.

The associated press release and public consultation are linked for reference.

Clinical trials for medicines: authorisation assessment performance

The MHRA published its latest transparency data relating to clinical trials for medicines: authorisation assessment performance on 13 Apr 2022. This data includes the number of Advanced Therapy Investigational Medicinal Product (ATIMP) CTA submissions (page 8).

Human Tissue Authority (HTA)

HTA website project passes its final assessment

The HTA launched it’s new website on the 20 Apr 2022. The old website, previously accessed from archive.hta.gov.uk will be decommissioned, and no longer accessible. Users will be able to continue to access older snapshots of the website through the National Archive. For full information on the new website, please see the press release.

USA

Food and Drug Administration (FDA)

FDA issues final Nanomaterials Guidance for Industry

The FDA has issued (21^st April 2022) the finalised version of the document Guidance for Industry: Drug Products, Including Biological Products, that Contain Nanomaterials. This document provides guidance on the development of human drug products, including thosethat are biological products, in which a nanomaterial (as explained in the guidance) is present in the finished dosage form.

FDA issues draft Guidance for Industry: Diversity Plans to Improve Enrollment of Participants From Underrepresented Racial and Ethnic Populations in Clinical Trials

The FDA issued (14^th April 2022) the draft guidance, “Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Subgroups in Clinical Trials,” which recommends that sponsors of medical products develop and submit a Race and Ethnicity Diversity Plan to the agency early in clinical development, based on a framework outlined in the guidance.

Industry is encouraged to submit either electronic or written comments on the draft guidance by June 13, 2022 to ensure that the Agency considers any comments prior to commencing work on the final versions.

Annual Patient Engagement & Regenerative Medicine Meeting 2022: An FDA CBER Workshop for Patient Advocates

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) is hosting a public workshop on Tuesday, May 24, 2022, from 11:00 a.m. - 3:00 p.m. ET. Registration for the event can be found here.

The meeting will bring together patients, caregivers, advocates, and other important stakeholders to discuss the purpose and importance of natural history studies. FDA staff and panelists will explore how natural history studies contribute to improved understanding of diseases and drug development, including regenerative medicine treatments such as gene and cell therapies.

INTERNATIONAL

International Conference on Harmonisation (ICH)

The ICH E11A draft Guideline reaches Step 2 of the ICH process

The ICH E11A draft Guideline on Pediatric Extrapolation reached Step 2 of the ICH process on 4 April 2022 and now enters the public consultation period. The European deadline for comments is 6 August 2022.

This new ICH Guideline aims to promote international harmonisation of methodologies and strategies to incorporate paediatric extrapolation into overall drug development plans and to improve the speed of access to new drugs for paediatric patients, while limiting the number of children required for enrolment in clinical trials.

The ICH Revision of Q2(R2) draft Guideline reaches Step 2 of the ICH process

The ICH Revision of Q2(R2) draft Guideline on Validation of Analytical Procedures reached Step 2 of the ICH process on 24 March 2022 and now enters the public consultation period.

The scope of the revision of ICH Q2(R1) will include validation principles that cover analytical use of spectroscopic or spectrometry data (e.g., NIR, Raman, NMR or MS) some of which often require multivariate statistical analyses. The guideline will continue to provide a general framework for the principles of analytical procedure validation applicable to products mostly in the scope of Q6A and Q6B. The proposed guidelines Q2(R2) and Q14 are intended to complement ICH Q8 to Q12 Guidelines, as well as on-going ICH Q13 for Continuous Manufacturing, with a view to potentially combine both documents into one, for simplification and clarity.

The ICH Q14 draft Guideline reaches Step 2 of the ICH process

The ICH Q14 draft Guideline on Analytical Procedure Development reached Step 2 of the ICH process on 24 March 2022 and now enters the public consultation period.

The new guideline is proposed to harmonise the scientific approaches of Analytical Procedure Development, and to provide the principles relating to the description of Analytical Procedure Development process. This new guideline is intended to improve regulatory communication between industry and regulators and facilitate more efficient, sound scientific and risk-based approval as well as post-approval change management of analytical procedures. The proposed guidelines Q2(R2) and Q14 are intended to complement ICH Q8 to Q12 Guidelines, as well as on-going ICH Q13 for Continuous Manufacturing, with a view to potentially combine both documents into one, for simplification and clarity.

Public consultations

Medicines and Health products Regulatory Agency (MHRA)

Food and Drug Administration (FDA)

Internation Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)