Regulatory Round-up - December 2022

EUROPE

European Commission (EC)

EU Guideline on orphan applications (for designation and transfer) - 2022/C 440/02

The EC have published a guideline providing supplementary advice on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another (2022/C 440/02). It should be read in conjunction with the existing information and guidance on the format of applications, available on the European Medicines Agency (EMA) website.

The Accelerate Clinical Trials in the European Union (ACT EU) initiative

The EC, the Heads of Medicines Agencies (HMA) and the EMA have published recommendations that aim to facilitate the conduct of decentralised clinical trials (DCTs) while safeguarding the rights and well-being of participants as well as the robustness and reliability of the data collected. The recommendations include an overview of national provisions for specific decentralized clinical trial elements to be used in clinical trials. These recommendations under ACT EU are a first and important step towards clarifying the use of decentralised clinical trials in the EU/EEA by the European medicines regulatory network. The recommendation paper on decentralized elements in clinical trials can be found here.

European Medicines Agency’s (EMA) fee system - Impact assessment and Commission proposal

On 13 December 2022, the Commission adopted a Proposal for a Regulation of the European Parliament and of the Council on fees and charges payable to the European Medicines Agency, amending Regulation (EU) No 2017/745 of the European Parliament and of the Council and repealing Council Regulation (EC) No 297/95 and Regulation (EU) No 658/2014 of the European Parliament and of the Council.

European Directorate for the Quality of Medicines (EDQM)

EDQM releases new edition of the Tissue and Cells Guide, providing state-of-the-art guidance for healthcare professionals

The European Directorate for the Quality of Medicines & HealthCare (EDQM) has published the 5th edition of the Guide to the Quality and Safety of Tissues and Cells for Human Application. The updated guide provides an overview of the most recent advances in the field, as well as technical guidance on ensuring the quality and safety of human tissues and cells applied to patients. It is intended for healthcare professionals involved in the relevant activities, from identifying potential donors to the clinical application in patients and their follow-up. The guideline can be downloaded here.

Revision of Ph. Eur. General chapter Chromatographic separation techniques (2.3.46) – Signal-to-noise ratio

The revised general chapter Chromatographic separation techniques (2.2.46) was published in the 11^th Edition of the European Pharmacopoeia (Ph. Eur.) in the framework of international harmonisation. This new version (implemented on 1 January 2023) includes many important changes, one of these being the sensitivity requirement (signal-to-noise ratio). The revised chapter can be found on the EDQM website.

European Medicines Agency (EMA)

Clinical Trials Information System (CTIS) training session

The EMA and the European Medicines Regulatory Network are holding training events and information sessions to support Clinical Trails Information System (CTIS) users. Sessions will be held in February, May and June 2023. A list of the upcoming events and recordings from previous training sessions can be found on the EMA’s website.

Priority Medicines (PRIME): List of products granted eligibility

During its December 2022 meeting, the CHMP reviewed 3 recommendations for ATMPs with respect to their eligibility to PRIME: 1 was granted and 2 were denied. More information can be found here.

Hemgenix receives positive CHMP opinion.

The Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorisation for the advanced therapy medicinal product (ATMP), Hemgenix* (etranacogene dezaparvovec), the first gene therapy for the treatment of severe and moderately severe Haemophilia B.

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA to receive nearly £1m BEIS funding to unlock digital, data and scientific regulatory innovation

The MHRA has received a total of £970,688 from BEIS’ Regulators’ Pioneer Fund for three projects that aim to unlock cutting edge regulatory innovation, to drive real world benefits for patients, the healthcare sector and clinical research. More information can be found here.

USA

Food and Drug Administration (FDA)

FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer

On the 16^th of December 2022, the U.S. Food and Drug Administration granted approval of Adstiladrin to Ferring Pharmaceuticals A/S. Adstiladrin (nadofaragene firadenovec-vncg) is a non-replicating (cannot multiply in human cells) adenoviral vector based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

Public meeting: FDA Rare Disease Day 2023

FDA will host Rare Disease Day, a virtual public meeting, on February 27, 2023, 9:00 am – 4:45 pm ET. On this patient focused event, participants will have an opportunity to hear directly from the FDA on ATMP development for rare diseases as well as patients, carers and family members will be engaged to provide their perspectives. Information on how to register for this event can be found here.

FDA guidance proposes devices be deemed adulterated for refusing inspections

The US Food and Drug Administration’s (FDA) has issued a draft guidance proposing that devices to be regarded as adulterated and refused entry to the US market if companies refuse to allow inspections of their facilities without adequate justification. According to the current FDA policy, only drugs can be considered adulterated if FDA inspectors are refused to carry out inspections.

The draft guidance defines the types of actions that constitute unreasonable conditions for delaying denying or limiting an inspection.It aligns with an update in the law under the FDA Reauthorization Act (FDARA), which requires devices also be deemed adulterated and refused entry for staff that block FDA inspections.

The deadline for submitting comments is 14 February 2023.

National Institutes of Health (NIH)

NIH’s All of Us Research Program returns genetic health-related results to participants

The National Institutes of Health's All of Us Research Program has begun returning personalized health-related DNA results to more than 155,000 participants. The aim of this research programme is to accelerate health research and medical breakthroughs, enabling individualized prevention, treatment, and care. Please find more details of this programme here.

Artificial intelligence tools help scientists decode genomic disorders and communicate genomic risks

In order to advance the field of genomics, more sophisticated technologies are required to diagnose, monitor and treat genetic conditions. Artificial intelligence tools, such as Machine learning, which mimic human intelligence to solve problems, are found to be useful to tackle these complex tasks. Please find more details on studies exploring how artificial intelligence tools can help clinicians identify genetic conditions here.

Public consultations

EUROPEAN MEDICINES AGENCY (EMA)

Medicines and Healthcare products Regulatory Agency (MHRA)

Food and Drug Administration (FDA)

International Conference on Harmonisation (ICH)

Click here to download the PDF