Regulatory Round-up - July 2022

EUROPE

European Commission (EC)

Draft legislative proposal for a new EU Regulation on Blood, Tissues, and Cells

On 14^th July 2022, the European Union (EU) Commission issued its draft legislative proposal for a new EU Regulation on Blood, Tissues, and Cells (BTC), in which the EU Commission proposes to leverage on the technical standards and proven expertise of the European Centre for Disease Prevention and Control (ECDC) and the European Directorate for the Quality of Medicines & HealthCare (EDQM) of the Council of Europe as expert bodies. This proposal is the result of a comprehensive revision of the existing EU legislation, which has included a wide consultation of BTC stakeholders and international organisations active in the field, such as the Council of Europe/EDQM. The press release can be found here.

European Directorate for the Quality of Medicines (EDQM)

CTIS & EudraVigilance - Update for Sponsors of Clinical Trials

The EMA launched the enhanced EudraVigilance system in November 2017 to support the reporting and analysis of suspected adverse reactions (SARs) from clinical trials and the post-approval phase of medicines. Based on a recommendation from the Pharmacovigilance Risk Assessment Committee (PRAC), the mandatory use of the International Standard on "Individual Case Safety Report" (ICSR) revision 3 (ICH-E2B(R3)) for all submissions to EudraVigilance will be implemented starting June 30, 2022. The use of the ICH E2B (R2) format will therefore be phased out. For more information, see Change management for the EudraVigilance system.

List of Reference Standards Updated

The list of available reference standards is revised by the EDQM at regular intervals and published on their website. In April 2022, the list, which includes more than 3,000 substances, was updated again .

In the most recent list, 20 reference standards were replaced by new batches, and some substances have been removed from the list of reference substances or will be deleted (as of July 2022). For all these substances, a transitional period of one year has been granted until they are completely removed from the list, and they are still available for purchase for a certain defined period.

The complete list of all available reference standards and the substances affected by the updates can be found on the EDQM website.

European Medicines Agency (EMA)

EMA grants conditional marketing authorisation for Roctavian

The CHMP have adopted a positive opinion recommending the granting of a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec). Roctavian has been developed for the treatment of severe haemophilia A in adults who do not have factor VIII inhibitors (auto-antibodies produced by the immune system which make factor VIII medicines less effective) and no antibodies to adeno-associated virus serotype 5 (AAV5). Roctavian is the first gene therapy to treat haemophilia A and comprises an AAV gene therapy product containing the gene for factor VIII. The full article from the EMA can be read here.

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

Horizon Scanning Case Study: Point of Care Manufacture

Horizon scanning at the MHRA has led to a proposed new framework to enable the supply and increase in the availability of innovative new medicinal products made at the point of care to patients. The MHRA identified a clear need to amend The Human Medicine Regulations 2012 and The Medicines for Human Use (Clinical Trials) Regulations 2004. This new framework will accommodate the needs of medicines products manufactured at POC but will also provide control measures equivalent to those currently in place for medicinal products manufactured in factory-based locations, this will ensure that POC products have appropriate quality, safety, and efficacy attributes. This is particularly applicable to those product types with particularly short shelf lives such as ATMPs. The full article can be read here.

British Pharmacopoeia

AQbD guidance is now live on BP online

The Analytical Quality by Design (AQbD) supplementary chapter provides best practice guidance on how to enhance method understanding by successfully adopting AQbD concepts in analytical methods.

BP scientists have worked together with MHRA assessors and inspectors as well as the Therapeutic Goods Administration of Australia (TGA), multinational biopharmaceutical manufacturers, the generics manufacturing industry and from the field of metrology to provide this guidance. Available now in the British Pharmacopoeia (BP) 2022 edition, this guidance will help you ensure that the analytical methods used are robust and fit for purpose. This will enable a better assessment of product quality and ultimately provide greater assurance to safeguard patient safety.

More information can be found out here (requires log-in credentials).

USA

Food and Drug Administration (FDA)

CBER Workshop for Patient Advocates - recording available to view

The FDA hosted a workshop back in May – the Annual Patient Engagement & Regenerative Medicine Meeting 2022: An FDA CBER Workshop for Patient Advocates. The recording of this workshop is now available online for viewing.

FDA issues draft guidance on labelling for dosing based on weight or body surface area

The FDA has issued a draft guidance on Human Prescription Drug and Biological Products–Labeling for Dosing Based on Weight or Body Surface Area for Ready-to-Use Containers– “Dose Banding”. This guidance provides recommendations to assist applicants in incorporating information into proposed human prescription drug labeling when:

• dosing for the drug product is based on weight or body surface area (BSA),
• the drug product is available in a range of strengths in ready-to-use containers,
• the entire drug content of the ready-to-use container(s) is intended to be administered to a patient.

This practice is referred to as dose banding. This guidance applies to proposed labeling in a new drug application (NDA) submitted under section 505(b) of the FD&C Act; a biologics license application (BLA) submitted under section 351(a) of the PHS Act; or a supplement to one of these approved applications. The FDA welcomes comments on the guidance.

Public consultations

Food and Drug Administration (FDA)

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