Issue 17: Pharmaceutical regulatory roundup

BY DR TIM SANDLE  | 24 January 2024



Catch up with the latest news from around the pharmaceutical industry with issue 17 of our regulatory review, curated by Dr Tim Sandle.


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WHO Guidance on waste and wastewater management in pharmaceutical manufacturing with emphasis on antibiotic production


As pharmaceuticals are biologically active, often highly potent molecules with conserved targets across species, environmental emissions may cause unwanted effects on other organisms. While the overall largest volume of Active Pharmaceutical Ingredients (APIs) that reach the environment most likely originates from patient use, the highest environmental concentrations are the result of pollution from manufacturing, localised in certain industrial areas.


Pollution with antimicrobials provides a case of special concern. In addition to direct ecological effects, this pollution may also contribute to the development of resistance, in both non-pathogenic and pathogenic microbes. This therefore threatens the effectiveness of antimicrobials as therapeutic agents in humans, as well as farmed and domestic animals and crops. 


Such effects are not restricted to the site of the emissions, as microorganisms have the ability to propagate and eventually spread world-wide. 


There is a recognised need for international evidence-based guidance and tools on the management of industrial waste containing antimicrobials. To aid in this process, the World Health Organisation has issued a draft document titled “WHO Guidance on waste and wastewater management in pharmaceutical manufacturing with emphasis on antibiotic production.”


The document aims to establish an independent, scientifically derived framework for applying targets for managing discharges from antibiotic manufacturing facilities, with the intent to limit antibiotic resistance development and ecological effects caused by discharges of antibacterial agents into the environment.


The document can be accessed here: 




Accelerating phage therapy


The European Medicines Agency (EMA) has issued a new document titled “Concept paper on the establishment of a Guideline on the development and manufacture of human medicinal products specifically designed for phage therapy.”


This concept paper proposes to establish a scientific guideline for the pharmaceutical development and manufacture of bacteriophage medicinal products intended for the therapeutic treatment or prophylaxis of one or more specific bacterial infection(s) or infectious disease(s) in humans. 


There is currently no appropriate regulatory guidance for medicinal products for human use in the EU and the concept paper seeks to remedy this.


For details see: 




Cell and Gene Therapy


The US FDA has issued guidance that provides recommendations for developing a science and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product.


The document is titled “Potency Assurance for Cellular and Gene Therapy Products”.


A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect.


The draft can be found here: 




Ophthalmic drug products 


A new US FDA draft guidance discusses certain quality considerations for ophthalmic drug products (i.e., gels, ointments, creams and liquid formulations such as solutions, suspensions, and emulsions) intended for topical delivery in and around the eye. Specifically, the guidance discusses microbiological considerations.


The draft guidance is titled “Quality Considerations for Topical Ophthalmic Drug Products”.


The guidance also includes approaches to evaluating visible particulate matter, extractables and leachables, and impurities and degradation products. Also contained within the guidance are recommendations for design, delivery and dispensing features of Container Closure Systems (CCSs).


The draft guidance can be accessed here: 




Regulatory Roadmap provides new measures to support safe access to medical technology: AI and diagnostics


The MHRA has set out new plans to protect patient safety and enable prompt access to innovative medical technologies for UK patients. This is in the form of a new regulatory roadmap, intended to enhance the UK’s ability to benefit from rapidly advancing medical technology, offering significant new opportunities for patients and healthcare.


Transformative technologies such as new implantable devices, healthcare AI and software, and diagnostics for early detection and prevention of disease, all demand a new regulatory framework.


The MHRA’s roadmap sets out a route to deliver enabling regulation via a series of new Statutory Instruments (SIs). Priority measures to protect patient safety will be put in place this year, with core elements of the new framework intended to be in place by 2025.


The planned regulations are also designed to deliver greater international harmonisation, with more patient-centred, proportionate requirements for medical devices which are responsive to technological advances.


For details, see: 



Artificial Intelligence workplan to guide use of AI in medicines regulation


The European Medicines Agency (EMA) has published an Artificial Intelligence (AI) workplan to 2028, setting out a collaborative and coordinated strategy to maximise the benefits of AI to stakeholders while managing the risks.


Pharmaceutical companies increasingly use AI-powered tools in research, development and the monitoring of medicines. National competent authorities are responding to the new opportunities and challenges by starting to use and develop AI tools (as with details of the MHRA activities above).


One of the aims is to identify and provide frameworks across the network to use AI tools to increase efficiency, enhance understanding and analysis of data and support decision-making. Full compliance with data protection legislation will be ensured.


The workplan can be accessed here: 




Clinical development of vaccines 


The EMA has issued a concept paper on the development of an addendum to the guideline on clinical development of vaccines on clinical trials for vaccines for immunocompromised individuals.


The guideline addresses the clinical evaluation of vaccines intended for the prevention of infectious diseases. It includes considerations for trials intended to document the safety, immunogenicity and efficacy of new candidate vaccines and to support changes in the prescribing information of licensed vaccines. It also considers the need for and use of vaccine effectiveness studies.






Guidance on good manufacturing practice and good distribution practice: Questions and answers


The EMA has updated its Q&A in relation to the interpretation of aspects of EU GMP. This is in the form of frequently asked questions on good manufacturing practice (GMP) and good distribution practice (GDP), as discussed and agreed by the GMP/GDP Inspectors Working Group.


To access see: 




Inquiries into medical device falsification


The Committee of Experts on Minimising Public Health Risks Posed by Falsification of Medical Products and Similar Crimes (CD-P-PH/CMED) has carried out a survey on how authorities in Council of Europe member states perceive and address the issue of the falsification of medical devices. The results are now available in a report providing a selection of questions, answers and comments, along with the key findings.


The survey responses confirmed the existence of the issue of falsified medical devices. However, few investigations (at criminal level, in particular) are instigated, and there are even fewer prosecutions. Data collection is practically inexistent - hence very little is available.


To access, see:,very%20little%20data%20is%20available




Q&A on implementation of Ph. Eur. Medicinal Product Monograph


The EMA has updated its guidance on putting in place medicinal product monographs. This is to assist with demonstrating compliance with Ph. Eur. Medicinal Product Monographs (MPM).


To access, see: 




Benzene warning in relation to drug products


The US FDA has issued a new guidance document titled “Reformulating Drug Products That Contain Carbomers Manufactured With Benzene”.


The guidance provides recommendations for applicants and manufacturers on what tests should be performed and what documentation should be submitted or available to the FDA to support the reformulation of drug products that use carbomers manufactured with benzene. 


Certain United States Pharmacopeia (USP) carbomer monographs currently allow for unacceptable levels of benzene, which raises safety concerns. Hence, the FDA has requested that the USP omit (or remove) these monographs. Applicants and manufacturers may need to reformulate their drug products to avoid using these carbomers. 


The guidance provides recommendations for testing and documentation related to reformulation, taking into consideration the various routes of administration and dosage forms of affected drug products. 


The guidance can be accessed here: 



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