Latest Regulatory Updates

June 2023

Food and Drug Administration (FDA)

Two draft guidances have been published for comment: Pediatric Drug Development Under the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA): Scientific Considerations  addressing selected clinical, scientific, and ethical issues regarding the development of drugs for paediatric use

and 

Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act intended to assist in compliance with the paediatric study requirements under PREA, and describing the process for qualifying for paediatric exclusivity and the protections that paediatric exclusivity offers under the BPCA.

The guidance documents revise and replace the previous Guidances for Industry (withdrawn in 2013).  Comments can be submitted until 17 July 2023.

FDA implemented the International Council for Harmonisation (ICH) Q9(R1) Quality Risk Management (QRM) guideline, providing principles and examples of tools for QRM, including development, manufacturing, distribution, and the inspection and submission/review processes throughout the lifecycle of drug substances, drug products, biological and biotechnological products. This final guidance replaces the draft guidance published in June 2022.

Final Guidance available for Testing High-Risk Drug Components for Diethylene Glycol (DEG) and Ethylene Glycol (EG). This guidance replaces the 2007 version and provides recommendations to manufacturers, re-packagers, other suppliers of high-risk drug components, and compounders to prevent the use of drug components contaminated with DEG and EG, which has led to more than 300 deaths.

The Center for Devices and Radiological Health (CDRH) now enables users to track medical device premarket submissions.  The online progress tracker has a dashboard that displays the status of FDA's progress in near real-time.

Also of note: Starting 01 October 2023, all 510(k) submissions (Class I, II, and III devices for which a Premarket Approval application is not required), must be submitted as electronic submissions using eSTAR unless exempted.

Center for Drug Evaluation and Research (CDER) published its first quarterly Regulatory Science Newsletter featuring new developments, opportunities, and initiatives in drug development and regulatory science, with the goal of advancing medical product development.

European Medicines Agency (EMA)

Clinical Trials Information System (CTIS) Multi-factor authentication (MFA) for user logins to CTIS will be launched on 1 June 2023. Instructions on setting up the MFA for EMA systems are available here.

EMA launched a Public consultation on the transparency rules for the operation of the Clinical Trials Regulation (CTR) and its CTIS. The consultation seeks views on the best possible approaches to balance transparency with confidentiality requirements, while also simplifying the modalities of use of CTIS. The consultation ends 28 June 2023.

See also: Interim guidance and its  Annex available on the protection of personal data and commercially confidential information while using CTIS.

Newsletter:  Human medicines highlights – Issue 169, May 2023

Primarily addressed to organisations representing patients, consumers and healthcare professionals, this newsletter provides a summary of key information relating to medicines for human use published in December.

EMA published its annual report 2022 outlining the agency’s most important highlights, key figures and milestones achieved last year.

Medicines and Healthcare products Regulatory Agency (MHRA)

Commercial clinical trials landscape: the Lord O’Shaughnessy review - final report on the state of clinical trials in the UK was published. The independent review sets out 27 recommendations where action should be taken by the government to address key challenges within the clinical trials system.

MHRA Inspectorate: A blog was published on the changes to the ICH E6 Good Clinical Practice (GCP) Guideline now that the updated version R3 has reached Step 2b and is available for public consultation. The consultation ends 31 August 2023.

MHRA Inspectorate: Manufacture of Investigational Medicinal Products – Frequently Asked Questions has been updated and now includes the most commonly received question (deciding if an activity should be considered as manufacture or reconstitution).

Notifying the MHRA of a clinical investigation of a medical device

The Agency has shared a validation checklist against which it will validate new submissions for a clinical investigation of a medical device.

Updated ‘Specials’ Guidance available for Supply of unlicensed medicinal products

An updated version of ‘MHRA guidance note 14 (Specials)’ with advice on manufacturing, importing, distributing and supplying specially manufactured or ordered products is now available.

MHRA announces new recognition routes to facilitate safe access to new medicines with seven international partners. The new approval recognition routes with Australia, Canada, the European Union, Japan, Switzerland, Singapore and the US are developed through the Access Consortium and Project Orbis and are planned to be in place by Q1 2024.

MHRA, the National Institute for Health and Care Excellence (NICE), Health Technology Wales (HTW) and Scottish Health Technology Group (SHTG) are preparing to launch the Innovative Devices Access Pathway (IDAP) later this year. This new, integrated support service for developers aims to bring innovative technologies and solutions to the forefront of the NHS and will include enhanced opportunities for engagement.

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

ICH guideline E19 on a selective approach to safety data collection in specific late-stage pre-approval or post-approval clinical trials. The guideline came into effect in March 2023 and details factors that can justify a reduced collection of certain data in a clinical trial and describes the types of data that may be appropriate for selective safety data collection, along with data that should generally be collected.