Issue 23: Pharmaceutical regulatory roundup

BY DR TIM SANDLE  | 30 July 2024

 

Catch up with the latest news from around the pharmaceutical industry with issue 23 of our regulatory review, curated by Dr Tim Sandle.

 

  

Environmental risk assessment of medicinal products for human use - scientific guideline

 

The European Medicines Agency (EMA) has issued a new guidance for conducting environmental risk assessments for pharmaceuticals.

 

It is mandatory for a Marketing Authorisation Application (MAA) for a medicinal product for human use (HMP) to include an Environmental Risk Assessment (ERA). This ERA is based on the use of the product and the physico-chemical, ecotoxicological and fate properties of its active substance(s). 

This guideline describes how to perform an ERA and how to evaluate potential risks to the environment, with the aim of protecting aquatic and terrestrial ecosystems including surface water, groundwater and soil. The guidance also identifies and describes the potential hazards of the active substance of a medicinal product. 

 

The guideline includes consideration of potential precautionary and risk mitigation measures and provides guidance on how to report the findings in an Environmental Risk Assessment Report.

 

For details, see: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-environmental-risk-assessment-medicinal-products-human-use-revision-1_en.pdf 

 

 

 

Labelling

 

The United States Pharmacopeia (USP) chapter for labelling - 〈7〉 ‘Labeling’ is being revised.

 

This proposal is based on the version of the chapter official as of September 1, 2023. The Nomenclature and Labeling Expert Committee is proposing the following revisions:

 

  • Apply a new format for epinephrine in multi-entity anesthetic drug products and remove ratio expressions of strength
  • Add text that includes flexible containers for potassium chloride for injection concentrate
  • Clarify alphanumeric expiration date formats
  • Update labelling for unit-dose cups
  • Add a new section entitled ‘Dietary Supplements’
  • Change the section entitled ‘Products That Contain Vitamins’ to ‘Products That Contain Vitamins and Minerals’ and add:
    • New labelling requirements for dietary supplements containing iron
    • New labelling requirements for dietary supplements containing potassium
    • New labelling requirements for dietary supplements containing calcium
  • Add labelling for products containing oral liquid dosing tools
  • Add a new section addressing the labelling of radiopharmaceuticals. [Note—A delayed implementation is being considered.

 

Other changes for clarity or to update terminology have been incorporated throughout the chapter.

 

The draft has been published in the Pharmacopeial Forum (for which registration is required).

 

 

 

Container Content for Injections

 

The United States Pharmacopeia chapter 〈697〉 ‘Container Content for Injections’ is being revised.

 

This proposal is based on the version of the chapter official as of May 1, 2015. The United States Pharmacopeia is the coordinating pharmacopeia for the international harmonisation of the compendial standards for this chapter, as part of the process of international harmonisation of monographs and general analytical methods of the European, Japanese and United States pharmacopeias. The following chapter, which represents the Revision 1, OFFICIAL INQUIRY STAGE 2 document, includes the following changes:

 

  • Single-dose containers:
    • Revise to not specify a container number that must be tested. The focus should be on individual decisions based on process variability and lowest possible fill volume
    • Revise to allow any suitable syringe to be used
    • Revise to allow the testing of extractable volume on a per-container basis for small-volume parenterals ≤2 mL
    • Revise so that containers ≥10 mL can be tested as per labelling instructions
  • Injections in cartridges or prefilled syringes:

    • Revise to not specify a container number that must be tested. The focus should be on individual decisions based on process variability and lowest possible fill volume
  • Large-volume intravenous solutions:

    • Revise to not specify a container number that must be tested. The focus should be on individual decisions based on process variability and lowest possible fill volume

 

The draft has been published in the Pharmacopeial Forum (for which registration is required).

 

 

 

Filters and membranes

 

The USP is proposing to include a new chapter entitled 〈1002〉 ‘Filters and Membranes’. 

 

It is proposed to add this new chapter with recommendations on the selection and characterisation of filters and membranes used in analytical procedures. This chapter does not address the use of filters and membranes in in-process, manufacturing or compounding processes.

 

The draft has been published in the Pharmacopeial Forum (for which registration is required).

 

 

 

Biological assays

 

The USP will be updating its general chapter 〈1030〉 ‘Biological Assay Chapters—Overview and Glossary’.

 

This proposal is based on the version of the chapter official as of December 1, 2013. The Statistics Expert Committee proposes to update and expand the scope of the current chapter by changing the title to ‘An Introduction to the Biological Assay Chapters—Overview and Glossary’ and adding the following new sections:

  • Introduction to Biological Assays
  • Some Important Characteristics of Bioassays
  • Life Cycle Concepts and Framework for the Bioassay Chapters
  • Categories and Levels of Variability Throughout the Bioassay Life Cycle
  • Brief Description of the Bioassay Chapters
  • Bioassay Glossary

 

This major revision will assist bioassay professionals by starting with the more technical chapters ‘Design and Development of Biological Assays’ 〈1032〉, ‘Biological Assay Validation’ 〈1033〉, and ‘Analysis of Biological Assays’ 〈1034〉.

 

Additionally, minor editorial changes have been made to update the chapter to current USP style.

 

The draft has been published in the Pharmacopeial Forum (for which registration is required).

 

 

 

Addressing Misinformation About Medical Devices and Prescription Drugs

 

The U.S. FDA has issued a draft guidance for industry, ‘Addressing Misinformation About Medical Devices and Prescription Drugs: Questions and Answers,’ which, when finalised, will describe the agency’s current thinking on common questions companies may have when voluntarily addressing misinformation related to their approved/cleared medical products.

 

This revised draft guidance would only apply to approved/cleared medical products, which include medical devices for human use (including biological products), prescription human drugs (including biological products) and prescription animal drugs.

 

This document revises the previous draft guidance for industry entitled ‘Internet/Social Media Platforms: Correcting Independent Third-Party Misinformation About Prescription Drugs and Medical Devices,’ issued in June 2014.

 

This revised guidance will help the public get the accurate, up-to-date, science-based information they need to inform their decisions about medical products to maintain and improve their health.

 

See: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/addressing-misinformation-about-medical-devices-and-prescription-drugs-questions-and-answers?utm_medium=email&utm_source=govdelivery 

 

 

 

Processes and Practices Applicable to Bioresearch Monitoring Inspections

 

The US FDA has issued a draft guidance for industry entitled ‘Processes and Practices Applicable to Bioresearch Monitoring Inspections.’ The draft guidance is being issued to comply with the Food and Drug Omnibus Reform Act of 2022, which directs the Agency to issue guidance describing the processes and practices applicable to inspections of sites and facilities under FDA’s Bioresearch Monitoring inspection program, to the extent not specified in existing publicly available FDA guides and manuals.

 

The draft guidance is intended to cover: 

  • The types of records and information required
  • Best practices for communication between FDA and industry in advance of / during an inspection or request for records / other information
  • Other inspections-related conduct

 

To access, see: https://www.fda.gov/media/179027/download 

 

 

 

When is a biological product interchangeable with a reference product?

 

The US FDA has issued a draft guidance for industry entitled ‘Considerations for Demonstrating Interchangeability with a Reference Product: Update.’ This draft guidance describes considerations regarding a switching study or studies intended to support a demonstration that a biological product is interchangeable with a reference product.

 

FDA issued the guidance for industry ‘Considerations in Demonstrating Interchangeability With a Reference Product’ (May 2019) (Interchangeability Guidance) before receiving and reviewing any biologics license applications (BLAs) submitted under section 351(k) of the Public Health Service Act (PHS Act) for a proposed interchangeable biosimilar. 

 

Since publication of the Interchangeability Guidance, experience has shown that for the products approved as biosimilars to date, the risk in terms of safety or diminished efficacy is insignificant following single or multiple switches between a reference product and a biosimilar product.

 

Accordingly, FDA’s scientific approach to when a switching study or studies may be needed to support a demonstration of interchangeability has evolved. 

 

For details see: https://www.fda.gov/media/179456/download 

 

 

 

Demonstrating Bioequivalence for Type A Medicated Articles Containing Active Pharmaceutical Ingredient(s)

 

The US FDA has issued guidance that describes an approach to satisfy the requirements for the completion of the bioequivalence technical section for generic type A medicated articles (TAMAs) containing poorly soluble, locally acting, active pharmaceutical ingredients (APIs) that have little to no systemic absorption, and for which blood level studies are not considered appropriate to demonstrate product bioequivalence.

 

The suggested approach described in this guidance uses a combination of in vitro and in vivo data to support a determination of bioequivalence to address the unique challenges associated with demonstrating bioequivalence of TAMAs. 

 

To read the guidance, see: https://www.fda.gov/media/168684/download 

 

 

 


Improving diversity in clinical trials

 

The US FDA has issued draft guidance about the form, content and manner of diversity action plans. This includes the applicable medical products and clinical studies for which a diversity action plan is required, the timing and process for submitting diversity action plans, and the criteria and process by which FDA will evaluate sponsors’ requests for waivers.

 

This replaces the draft guidance for industry entitled ‘Diversity Plans to Improve Enrollment of Participants From Underrepresented Racial and Ethnic Populations in Clinical Trials,’ published April 14, 2022.

 

FDA is issuing this guidance as mandated under section 3602 of FDORA, which requires that FDA update or issue guidance relating to the format and content of diversity action plans required by sections 505(z) and 520(g) of the FD&C Act (21 U.S.C. 355(z) and 360j(g) as amended by section 3601 of FDORA.

 

To view the contents, see: https://www.fda.gov/media/179593/download 

 

 

 

Drug delivery systems

 

New FDA guidance addresses key aspects of drug delivery performance information for devices and combination products that include device constituent parts (intended for delivery of a human drug, including a biological product). The guidance describes FDA’s recommendations related to the device design outputs that are essential for establishing and assessing drug delivery performance.

 

The guidance includes recommendations for the information and data to submit in investigational, marketing and post-market change applications.

 

For details: https://www.fda.gov/media/179545/download 

 

 

 

End of rabbit pyrogen testing in Europe

 

Pyrogen detection is essential for ensuring the safety of parenteral medicines. For decades, the rabbit pyrogen test (RPT) has been the traditional method. The RPT involves measuring the rise in body temperature in rabbits following intravenous injection of the substance to be examined.

 

At its 179th session in June 2024, as the outcome of a broad exercise aiming at the complete removal of the RPT from the European Pharmacopoeia (Ph. Eur.), the Ph. Eur. Commission adopted 57 revised texts from which the RPT has been deleted, together with a new general chapter on Pyrogenicity (5.1.13), marking the end of the RPT era in the Ph. Eur. 

 

This is a major achievement for animal welfare and for the advancement of modern in vitro approaches for pyrogenicity testing. As a result, the use of the RPT will no longer be required in any text of the Ph. Eur. and it will be the responsibility of medicine developers to select a suitable in vitro test (e.g. the monocyte-activation test) to control the pyrogenicity of their product, based on a risk assessment as described in the new general chapter.

 

The revised texts omitting the RPT and Pyrogenicity (5.1.13) will be published in Supplement 11.8 of the Ph. Eur., with an implementation date of 1 July 2025.

 

 

 

Pharmaceutical grade water

 

The European Pharmacopoeia (Ph. Eur.) is seeking stakeholder feedback on the following revised texts, published in Pharmeuropa (36.3) for comment:

  • Water for injections (0169)
  • Purified water (0008)
  • Total organic carbon in water for pharmaceutical use (2.2.44)

 

The focus of the revision is the replacement of the test for oxidisable substances (OS) by the test for total organic carbon (TOC) in the section on ‘Sterilised water for injections’ (SWFI) in the monograph on ‘Water for Injections’ (0169).

 

The TOC test is a state-of-the-art quantitative method that is more sensitive and non-selective than the OS test for the control of organic impurities present in pharmaceutical waters. If adopted, the revision constitutes a major step towards a global quality standard for SWFI, the most widely used excipient in the pharmaceutical industry, with obvious benefits for manufacturers worldwide.

 

Furthermore, in the proposed revision of general chapter 2.2.44, the reagents sucrose R and 1,4-benzoquinone R have been replaced by chemical reference substances (CRSs) to streamline the application of the TOC test.

 

The Ph. Eur. would like to encourage all stakeholders to provide their comments on these important texts via the appropriate channel. The commenting period runs until 30 September 2024.

 

To review see Pharmeuropa (requires registration): https://www.edqm.eu/en/comment-on-drafts-pharmeuropa- 

 

 

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