Regulatory News - January 2021

UK

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA Continue to Release and Update their Guidance for Industry and Organisations from 1 January 2021

On 1^st January, 2021 the Medicines and Healthcare products Regulatory Agency (MHRA) became the UK’s standalone medicines and medical devices regulator. As a result of the Northern Ireland Protocol, Northern Ireland will continue to have the MHRA as its’ National Competent Authority but will follow the EU regulatory framework, in essence, remaining part of the EU with respect to medicines regulation.

The MHRA website provides information on changes to the following, as well as how these will be dealt with in Northern Ireland:

• Clinical trials
• Devices
• Importing and exporting
• IT systems
• Legislation
• Licensing
• Pharmacovigilance
• Paediatrics

Full information on these changes are available here.

Additionally, the new MHRA submissions portal is now open for all UK submissions. The portal requires a simple online registration to use, and further information on how to do this is available here.

Human Tissue Authority (HTA)

New HTA office location

The HTA office has fully relocated to a new address, and now shares office space with four other public sector bodies: NICE, HFEA, HRA, and CQC. The new address is:

Human Tissue Authority, 2 Redman Place (2nd floor), Stratford, London, E20 1JQ

Further information on this can be found here.

UK BioIndustry Association (BIA)

BIA has Released a Paper on how Medicines for Orphan and Ultra‑Orphan Diseases Might be Prioritised by NICE in England

BIA has released a paper proposing new mechanisms to speed up market access for rare and very rare disease indications in England, to allow earlier and easier access to patients. The paper “A Rare Chance for Reform” is available to read here.

European Union

European Medicines Agency (EMA)

EMA have produced a guidance document on Assent / Informed Consent Guidance for Paediatric Clinical Trials with Medicinal Products in Europe

The guidance was developed by Working Group on Ethics of the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) to clarify recommendations for consent or assent in the case of paediatric clinical trials.

The document, Assent / Informed Consent Guidance for Paediatric Clinical Trials with Medicinal Products in Europe, is available here.

EMA have Updated their Brexit-Related Guidance for Companies

The guidance on the EMA website in relation to Brexit covers a variety of topics including the implications of the Northern Ireland Protocol, some of which are new or have been recently updated:

• Guidance on the Protocol on Ireland / Northern Ireland
• A Notice to Stakeholders After the End of the Transition Period
• Procedural and practical guidance regarding submission of changes and related fees
• European Commission notice to stakeholders on application of the EU pharmaceutical acquis in markets historically dependent on medicines supply from Great Britain (new)

Further information on Brexit-related changes in the EU can be found here or by clicking on the links above.

EMA Issues Draft Guidance for Parallel Article 58 “EU-M4all” (EU Medicines for All) Opinions

EMA have issued draft guidance on a new regulatory pathway which allows sponsors to pursue a parallel Article 58 (EU-M4all) opinion and centralised marketing authorisation application for medicines sponsors intending to market both in the EU and in countries outside the EU (so-called third countries). EMA’s Committee for Medicinal Products for Human Use (CHMP) would use this procedure to give opinions alongside the World Health Organization (WHO) on medicines intended to be used in low and middle income third countries in order to address public health priorities. The draft guidance is available here.

EMA has Updated the Innovation Task Force (ITF) Meeting Request Form

The updated ITF request form is available here, and should be used for all ITF meeting requests going forward.

EMA has Published a Report on Their Regulatory Highlights of 2020

EMA published a report on the human medicines highlights for 2020, which provides an overview on the authorisation and safety monitoring of medicines for human use. The report included the three ATMP products which were granted marketing authorisation in the EU in 2020. Links to the Public Assessment Reports (EPAR) are available in the summaries of these products below.

Libmeldy is a product containing autologous CD34+ cells encoding the arylsulfatase A (ARSA) gene used to treat children with the orphan indication metachromatic leukodystrophy (MLD). It is an ex vivo gene therapy using a lentiviral vector encoding the human ARSA gene. Marketing authorisation was granted in the EU on 17 December 2020 to Orchard Therapeutics.

Tecartus is a CAR-T therapy for the treatment of mantle cell lymphoma. Tecartus contains autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3zeta chimeric antigen receptor and cultured. Tecartus has been authorised in the EU as since 14 December 2020. Marketing authorisation was granted for Tecartus in the EU on 14 December 2020 to Kite Pharma EU B.V., Netherlands (a Gilead company).

Zolgensma (onasemnogene abeparvovec) is an adeno-associated viral vector serotype 9 (AAV9) containing the human SMN gene for the treatment of the orphan indication spinal muscular atrophy (SMA). Zolgensma is an in vivo gene therapy product. Marketing authorisation was granted in the EU on 18^th May, 2020 to AveXis, but in December 2020, AveXis EU Limited changed its’ name to Novartis Gene Therapies EU Limited.

Full document here: EMA Key Recommendations from 2020

European Commission (EC)

The European Commission has Launched a Consultation on the EU Legislation on Blood, Tissues, and Cells.

The European Commission (EC) have released a questionnaire to allow public consultation on the directives on blood and on tissues and cells. The EC will be reviewing these directives, taking into consideration lessons learned from the ongoing COVID-19 pandemic. Find out more on this consultation here. The consultation is open from 21 January 2021 to 15 April 2021.

European Commission Decides on Transition Period for the Decommissioning of Unique Identifiers on Medicines with Regard to Exports to the UK

The EC has agreed to waive the requirement to decommission the unique identifier (UI) on products shipped to the UK from the EU from 1 January 2021 to 31 December 2021. An amendment has been made to delegated Regulation 2016/161.

Previously, under delegated Regulation 2016/161, the UI for packs of (marketed) medicines exported to the UK would need to be verified and decommissioned prior to export to the UK. There would also have been an obligation for importers shipping products onward, e.g. to Cyprus, Ireland, Malta or Northern Ireland, to affix new UIs on products prior to their leaving the UK. More information on this is available here.

EDQM / Council of Europe

OCABR and NIBSC Sign Memorandum of Understanding on Human Vaccines and Medicinal Products Derived for Human Blood and Plasma

The National Institute of Biological Standards and Control (NIBSC, UK) have signed a Memorandum of Understanding (MOU) with the Official Control Authority Batch Release (OCABR) Network for human vaccines and medicinal products derived from human blood and plasma. This will renew the exchange and collaboration on common activities related to batch release of human vaccines and medicinal products derived from human blood and plasma which ceased on 1^st January 2021 due to Brexit. The MOU enters into force on 21 January 2021 and makes NIBSC an observer to the OCABR Network for human biologicals, as well as reinstating the sharing of information related to batch release activity in this field. More information is available here.

US

Food and Drug Administration (FDA)

FDA have Released Documents Updating Information with Regard to the COVID-19 Pandemic

FDA have released an update for establishments collecting human cells, tissues, or cellular or tissue-based products (HCT/Ps) from donors, in regard to the ongoing COVID-19 pandemic. FDA take the stance that respiratory viruses are generally not known to be transmitted through the use of HCT/Ps and confirm that there have been no cases reported of COVID-19 transmission through the use of these products. The HCT/P Responsible Person will continue to assess and document donor eligibility according to 21 CFR 1271.50. Further information is available here.

FDA has also released a related guidance document for licensed and investigational cell and gene therapies in relation to COVID-19. The scope covers recovery of donor source material, and onward processing of this material e.g., cell expansion in culture, viral reduction steps, formulation. The guidance document can be found here:

• Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency: Guidance for Industry

Public Consultations

EUROPEAN MEDICINES AGENCY (EMA)

EUROPEAN COMMISSION (EC)

The European Commission has launched a consultation on the EU legislation on blood, tissues and cells.

The directives on blood and on tissues and cells will be revised in conjunction with public consultation, taking into consideration the many learnings of the COVID-19 pandemic. Find out more on this consultation here. Consultation is open from 21 January 2021 to 15 April 2021.

EUROPEAN DIRECTORATE FOR THE QUALITY OF MEDICINES & HEALTHCARE (EDQM) / COUNCIL OF EUROPE (CoE)

Publication of Version 33.1 of Pharmeuropa – European Pharmacopoeia Revisions for Public Consultation

The 33.1 edition of Pharmeuropa has been published for public consultation. Pharmeuropa publishes new and revised texts of the European Pharmacopoeia (Ph. Eur.) for public consultation prior to adoption into the Ph. Eur. The deadline for comments on Pharmeuropa 33.1 is 31 March, 2021. Pharmeuropa requires registration for access (free of charge) and is accessible here.

UNITED STATES FOOD AND DRUG ADMINISTRATION (FDA)