Issue 22: Pharmaceutical regulatory roundup

BY DR TIM SANDLE  | 27 June 2024


Catch up with the latest news from around the pharmaceutical industry with issue 22 of our regulatory review, curated by Dr Tim Sandle.



Pathogen safety


EMA has recently updated its position statement on Creutzfeldt-Jakob disease (CJD) and Plasma-Derived Medicinal Products (PDMPs), no longer classifying UK residence as a risk factor for transmission of variant CJD (vCJD) by PDMPs. In the revised statement, plasma from the UK, or from donors who have spent time in the UK (between 1980-1996) is "no longer considered to pose a health risk for recipients of plasma-derived medicinal products" based on the emerging clinical and epidemiological scientific evidence.

In addition, the statement recognises the PDMP manufacturing process as an important factor in the reduction of prion infectivity and PDMP safety. As part of continued precautionary measures, manufacturers are required to estimate the infectivity reduction capacity of the specific manufacturing processes using the flow diagram provided in the document.


Revision of the statement was facilitated by a joint presentation by PPTA and IPFA, with the chair of PPTA's Global Pathogen Safety Working Group (GPSWG) acting as one of the lead presenters.


For details, see: 




Dossier for sterile substances 


The EMA has issued a revision to “Content of the Dossier for Sterile Substances”, which is now out for public comment (fourth draft).


This document is intended for applicants as a guide for compiling a dossier to obtain a Certificate of Suitability (CEP) for a sterile substance.


This guideline should be read in conjunction with the:

  • Current EDQM policy “Content of the Dossier for Chemical Purity and Microbiological Quality”
    •  EMA Guideline on the sterilisation of the medicinal product, active substance, excipient and primary container (EMA/CHMP/CVMP/QWP/850374)
  • Ph. Eur., chapter 5.1.1 Methods of Preparation of Sterile Products
  • Ph. Eur., chapter 5.1.2. Biological Indicators and Related Microbial Preparations Used in the Manufacture of Sterile Products
  • Annex 1 of Eudralex Volume 4 EU Guidelines for Good Manufacturing Practice for Medicinal Products for Human and Veterinary Use


To view, see: 




Topical steroids: new labelling 


When prescribed and used appropriately, topical steroids are highly effective for the treatment of inflammatory skin conditions such as eczema and psoriasis. However, there can be risks especially with prolonged use at high potency.


As a result of regulatory action, topical steroid products will be labelled with information on their potency to simplify advice for patients. This will commence over the coming year.


The MHRA has issued new guidance on the labelling of these medicines. See: 




Guideline on the pharmaceutical quality of inhalation and nasal medicinal  products


The EMA has performed the first revision of the guideline on pharmaceutical quality of inhalation and nasal products (EMEA/CHMP/QWP/49313/2005 Corr).


The main aim of the first revision is to consolidate the information available in the previous guidance documents and the related published Q&A. It also takes into consideration recent advancements in the field, common practice and new regulations, including the medical device regulation.


Requirements for the demonstration of therapeutic equivalence for orally inhaled products (OIP) are included in the guidelines for asthma and chronic obstructive pulmonary disease (COPD). These should be read in conjunction with each other.


This is as a draft, for public comment.


For details see: 




Proposed framework for international recognition of medical devices


The MHRA has issued a statement of policy intent for the international recognition of medical devices.


It describes how the UK Government intends to recognise regulatory approvals from Australia, Canada, the European Union and the United States of America depending on device type, class and prior approval. The MHRA continues to review the list of comparable regulator countries and is in active discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) to explore the recognition of medical device approvals from Japan.


The MHRA’s statement of policy intent focuses on ensuring safe access to quality-assured medical devices and reducing the duplication of assessments by comparable regulators to enable resources to be focused on more innovative products for the benefit of patient health. This statement will also allow manufacturers to start considering whether their devices will be eligible for the proposed alternative routes to market.


To access see: 




Medical devices: new guidance for industry


The EMA has issued a Q&A document that clarifies the regulatory requirements for devices used in combination with medicines.


The document covers regulatory and procedural guidance for:

  • Integral drug-device combinations (medical devices that form an integral product with a medicine, such as pre-filled syringes) and their lifecycle management
  • Medicinal products that include a medical device in their packaging (referred to as co-packaged) and how these should be labelled
  • The consultation procedure for medical devices with an ancillary medicinal substance (a substance that supports the proper functioning of the device)
  • The consultation procedure for companion diagnostics, diagnostic tests that are essential for the correct use of a specific medicine


To review, see: 




Post-authorisation procedural advice for users of the centralised procedure


The EMA has updated its post-authorisation procedural advice for  users of the centralised procedure (EMEA-H-19984/03 Rev. 107). This comes in the form of a question-and-answer document.


The following sections of this Q&A have been updated for 2024:


  • 1 Type IA Variations - Q1.3
  • 4. Extension of marketing authorisation – Q4.12
  • 13. Post Authorisation Safety Study (PASS) – Q13,2
  • 19. Transfer of Marketing Authorisation – Qs 19.1 19.2 19.5, 19,8, 19.13-15


This is a detailed document running to 303 pages. 


To access: 




Revision of the guideline on risk assessment of medicinal products on human reproduction and lactation


The EMA has issued a “Concept paper on revision of the Guideline on Risk Assessment of Medicinal Products on Human Reproduction and Lactation: from Data to Labelling”.


The concept paper outlines the areas in this guideline which are planned to be updated. This has been developed by a drafting group involving members from CHMP, PRAC, Non-clinical Working Party (NcWP), 3Rs Working Party (3RsWP), Quality Review of Documents Working Party (QRD) and Healthcare Professionals Working Party (HCPWP). 


Representatives from the Patient and Consumers Working Party (PCWP) have been invited and intend to contribute at a later stage of the guideline revision. The current guideline provides guidance on the integration processes of clinical and non-clinical data for the assessment of the risk of adverse maternal, foetal or child effects in humans, as well as effects 
on fertility. 


Further, there is guidance on how to communicate the potential or identified risk through the Summary of Product Characteristics (SmPC). A decision scheme to determine whether a medicinal product should be contraindicated during pregnancy is also included. 


The concept paper can be accessed here and public consultation is currently open: 




European Pharmacopeia and phage therapy


A new general chapter for the European Pharmacopoeia on phage therapy medicinal products (5.31) has been adopted and pre-published on the EDQM website.


Rising levels of antimicrobial resistance has been identified as one of the leading threats to global public health and development. This has prompted renewed interest in the use of phage therapy for the treatment of bacterial infections. 


This led the European Pharmacopoeia Commission (EPC) to make the elaboration of Ph. Eur. Texts, with standardising the requirements for phage therapy as one of its top priorities.


The new general chapter provides a framework of requirements for the production and control of phage therapy products and allows a degree of flexibility with the complex approaches currently employed.


For details, see: 




Draft Guideline on stability testing for applications for variations to a marketing authorisation for veterinary medicinal products


The EMA is intending to update its guideline on stability testing for applications for variations to a marketing authorisation for veterinary medicinal products.


The guideline provides general guidance on stability testing for variations not requiring assessment (VNRA) and variations requiring assessment (VRA).


The draft can be accessed here: 




Gene therapy


A new European Pharmacopoeia Commission approach to gene therapy has been set out. This will be as a general monograph “Gene Therapy Medicinal Products for Human Use” (1386) and an accompanying general chapter titled “Additional Information on Gene Therapy Medicinal Products for Human Use” (5.34), which will replace general chapter 5.14.


General monograph 1386 contains three individual sections describing additional requirements for the classes of gene therapy medicinal products (GTMPs) currently approved in Europe:


  • Genetically modified human autologous cells modified by integrating retroviral or lentiviral vectors
  • Adeno-associated virus vectors for human use
  • Oncolytic herpes simplex virus for human use


The accompanying general chapter 5.34 includes recommendations on product classes that are not yet on the European market to assist users and contains:


  • Plasmid vectors for human use
  • Adenovirus vectors for human use
  • Poxvirus vectors for human use
  • Retroviridae-derived vectors for human use
  • A newly drafted section on genetically modified bacterial cells for human use


In addition, general chapter 5.2.12. “Raw Material of Biological Origin for the Production of 
Bell-based and Gene Therapy Medicinal Products” has also been revised to align with this new approach.


To read more., see: 




High-throughput Sequencing for the detection of viral extraneous agents


High-Throughput Sequencing (HTS - also known as Next-Generation Sequencing) is a sensitive, state-of-the-art molecular biology technology, increasingly used to test for viral extraneous agents in biological products. In contrast to conventional tests, this advanced technology can detect a broad range of viruses, both known and unknown. HTS can also offer advantages over conventional methods.


A new general chapter has been proposed for the European Pharmacopeia. The chapter describes HTS methodologies used for the detection of viral extraneous agents in biological products. It outlines the different steps in the HTS workflow, the design of the method, analysis approaches and the controls used in the routine test. Importantly, it provides guidelines for HTS method validation, including recommendations for the selection of the spiking material for validation and the evaluation of the relevant performance characteristics for HTS, as well as product-specific validation.


The draft general chapter has now been published in Pharmeuropa 36.2 (requires registration to access).




Cell-based preparations


In response to the need for a text covering the quality of cell-based preparations in the rapidly evolving field of advanced therapy medicinal products (ATMPs), the European Pharmacopoeia Commission is proposing a new general chapter “Cell-based preparations for human use (5.32)”.


The chapter provides a framework of requirements for the production and control of cell-based preparations and comprises a definition, an extensive set of general requirements common to all cell-based preparations followed by four detailed individual sections describing additional specific requirements (for human haematopoietic stem/progenitor cells, human chondrocytes, human limbal stem cells and human mesenchymal stromal cells, respectively).


The draft general chapter has now been published in Pharmeuropa 36.2 (requires registration to access).




New general texts on mRNA vaccines


The European Pharmacopoeia is seeking stakeholder feedback on a series of three new general texts on mRNA vaccines.


The mRNA vaccine field has developed rapidly in the past few years and continues to evolve into an increasingly important technology tailored to fight different infectious diseases. The development and authorisation of COVID-19 mRNA vaccines provided an important impetus and is paving the way for the emergence of novel vaccine coding for a range of specific antigen(s). This has underscored the need for common quality standards for mRNA vaccines across Europe and beyond.


To help reach this goal, the Ph. Eur. has developed three new general texts addressing key aspects related to the production and control of mRNA vaccines and their components, with the aim of supporting developers, manufacturers, regulatory agencies and national control laboratories worldwide:


  • mRNA vaccines for human use (5.36), covering mRNA packaged in lipid nanoparticles, i.e. mRNA-LNP medicinal products
  • mRNA substances for the production of mRNA vaccines for human use (5.39), covering mRNA active substances used in the manufacture of mRNA vaccines
  • DNA template for the preparation of mRNA substances (5.40), covering the linear DNA template used as the starting material for the preparation of mRNA substances


The draft general chapter has now been published in Pharmeuropa 36.2 (requires registration to access).




World Health Organisation GMP


World Health Organisation (WHO) Technical Report Series 1052, 2024 has been issued. The following new guidance texts were adopted and recommended for use:


  • WHO good manufacturing practices for excipients used in pharmaceutical products (revision)
  • IAEA/WHO good manufacturing practices for in-house cold kits for radiopharmaceutical preparations (new)
  • WHO good practices for pharmaceutical quality control laboratories (revision).
  • WHO/UNFPA female condom generic specification (new).
  • WHO Biowaiver List: proposal to waive in vivo bioequivalence requirements for WHO Model List of Essential Medicines immediate-release (updated), solid oral dosage forms.
  • WHO guideline on Biopharmaceutics Classification System-based biowaivers (revision).
  • Multisource (generic) pharmaceutical products:  guidelines on registration requirements to  establish interchangeability (republished). 


Each of the above are included in the report and are recommended by the WHO for implementation.


The details can be found here: 


Interested in getting our monthly roundup delivered directly to your inbox?

Sign up using the button above


Need to contact us? Get help or ask us a question