Latest Regulatory Updates

May 2023

In this month's Regulatory Intelligence Digest we share the latest guidance and key updates from the regulatory agencies - FDA, EMA, MHRA, EC and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).

We are also pleased to share our latest free webinar on Early-Stage Drug Development: Challenges and Solutions - more on that later.

To discuss any of these updates, please contact a member of the Boyds Regulatory team who will be happy to help.
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European Commission (EC)

The Commission published its proposal to revise the EU's pharmaceutical legislation. The proposal amends and replaces the existing general pharmaceutical legislation (Regulation 726/2004 and Directive 2001/83) and the legislation on medicines for children and for rare diseases (Regulation 1901/2006 and Regulation 141/2000, respectively). 

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

ICH S12 Guideline on nonclinical biodistribution considerations for gene therapy products 

Both EMA and FDA highlight this guideline, which comes into effect on 30 September 2023 and provides harmonized recommendations for the conduct and overall design of nonclinical biodistribution studies for gene therapy (GT) products. The recommendations aim to facilitate the development of GT products while avoiding unnecessary use of animals.

Food and Drug Administration (FDA)

FDA publishes Final Question-and-Answer Guidance on a Risk-Based Approach to Monitoring Clinical Investigations. This guidance expands and complements the 2013 guidance for industry Oversight of Clinical Investigations - A Risk-Based Approach to Monitoring with additional recommendations to facilitate and encourage sponsors to implement risk-based monitoring.

Draft Guidance out for comment: Decentralized Clinical Trials (DCT) for Drugs, Biological Products, and Devices A DCT refers to a clinical trial where some or all of the trial-related activities occur at locations other than traditional clinical trial sites. The guidance provides recommendations including DCT design, sponsors and investigators roles and responsibilities, informed consent, safety monitoring  and the management of the investigational drugs, biological products, or devices in a non-centralized setting.
Submit Comments by 01 August 2023

Draft Guidance for comment: 
Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments (COA) into Endpoints for Regulatory Decision-Making. With a focus on COA issues associated with study endpoints, design, conduct and analysis, this guidance document is the fourth in a series intended to facilitate the advancement and use of systematic approaches to collect and use robust and meaningful patient and caregiver input that can more consistently inform medical product development and regulatory decision-making.
Submit Comments by 05 July 2023

Draft Guidance out for comment: Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients Under Section 506C of the FD&C Act This guidance document aims to help prevent and mitigate drug shortages by assisting applicants and manufacturers in providing FDA timely and informative notifications (“when, who, what and how”) about changes in the production of certain finished drugs, biologics and active pharmaceutical ingredients (API).
Submit Comments by 05 June 2023

European Medicines Agency (EMA)

Reflection paper: Establishing efficacy based on single arm trials submitted as pivotal evidence in a marketing authorisationEMA has opened a public consultation on the reflection paper, which aims to improve the design and conduct of single-arm trials and stimulate the scientific discussion around key concepts and challenges associated with these types of trials. Guidance is provided on the choice of endpoints, target populations, the role of external information and statistical principals.
The consultation ends on 30 September 2023.

EMA intends to gradually resume clinical data publication from September 2023 onwards, having previously suspended this activity for all products except treatments and vaccines for COVID-19. Initial marketing authorization applications (MAAs) for new active substances that receive a positive or negative CHMP opinion in September 2023 and onwards will be included in Step 1 of the restart. Withdrawn applications are also subject to publication.
SME Office
– Issue 58, April 2023. 
The bulletin highlights news, documents, and activities of interest to SMEs and their stakeholders.

Newsletter: Clinical Trials Highlights – Issue 14 April 2023.
Provides insights on clinical trials topics, including ACT EU and CTIS.

Newsletter:  Human medicines highlights – Issue 168, April 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 March.


Access Consortium: joint pipeline meetings
The MHRA, as part of the Access Consortium, along with the Therapeutic Goods Administration, Health Canada, Health Sciences Authority of Singapore and Swissmedic, are offering to host confidential pipeline meetings to exchange information on new developments and advise drug companies on their future product development plans.

Model Clinical Investigation Agreement (mCIA) and Clinical Research Organisation model Clinical Investigation Agreement (CRO-mCIA)
New model agreement templates have been issued. The updates bring the agreements in line with the guidance on the set-up of interventional research, and to support the delivery of research in a hub and spoke model. The (previous) September 2021 versions of these templates will only be accepted in IRAS submissions until the end of October 2023 after which the April 2023 templates will become mandatory.

Regulating medical devices in the UK
The MHRA updated its guidance to reflect the intended extension to acceptance of CE marked medical devices on the GB market beyond 30 June 2023. The updates include a summary of key requirements for placing a device on the GB market, GB registrations, UKCA marking, CE marking and Notified Bodies, labelling requirements plus regulation of medical devices in Northern Ireland reference guides.

Software and Artificial Intelligence (AI) as a Medical Device
MHRA has issued new guidance providing access to important Software Group outputs that might be of assistance to manufacturers and researchers. The Software Group works across the MHRA ensuring reasonable steps are taken to assure the safety of software (SaMD) and artificial intelligence (AIaMD) as a medical device

The Health Research Authority has become the latest organisation to join the new NHS Innovation Service.
The NHS Innovation Service, launched earlier this year, provides practical support and free expert advice and guidance for healthcare innovators. Industry experts, government bodies, the NHS and patient groups have collaborated to provide in-depth information from the very first stages of an idea, right through to their innovation being adopted in the NHS. Links to funding opportunities are also provided..


Early-Stage Drug Development: Challenges and Solutions
: Dr Nick Meyers, Vice President of Product Development at Boyds, shares his insights and solutions to help overcome or avoid some of the most common challenges faced in the very early phases of developing drugs, whether they be small molecules, traditional biologics or advanced therapies.

Established in 2005, Boyds provides a range of expertise and skills central to the development of pharmaceutical and biotechnology medicinal products.
Boyds has offices in Cambridge, Cheshire, Dublin and Pennsylvania.
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