Regulatory Round-up - March 2024

See the latest regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA grants first approval via the new International Recognition Procedure in 30 days

Launched in January this year, the International Recognition Procedure (IRP) allows the MHRA to accelerate the assessment of new medicines by taking into account the expertise and decision-making of trusted regulatory partners in the authorisation process. As a result, medicines that have been approved in other countries with stringent regulators will get to UK patients without delay, resulting in a more rapid, efficient, and cost-effective process for life sciences companies. A new formulation for XGEVA (denosumab), a treatment used in adults to prevent serious bone-related complications caused by bone metastasis and to treat giant cell tumour of bone in adults and adolescents, is the first product to be authorised by the MHRA via the new IRP. The product was authorised in 30 days, providing UK patients with earlier access to this treatment thanks to international recognition.


European Commission (EC)

Air-Tightness of Cleanrooms & Containment Solutions: VDI 2083 Part 19 Updated

The DIN EN 15727 standard and the new version of the VDI guideline VDI 2083 Part 19 provide information on cleanroom airtightness and containment solutions. The latter specifies the procedure for testing and classifying the tightness of isolators, cleanrooms, and air ducts and contains instructions for creating tight cleanrooms. It applies to all rooms that are operated in positive or negative pressure compared to the environment, both for personal and product protection. Please find further information here.

Commission adoption of new measures for better lifecycle management of medicine authorisations

The Commission has carried out a review of the rules setting out the procedures for post-authorisation changes to a marketing authorisation for medicines for human use. The purpose of this review was to make the lifecycle management of medicines more efficient. Amended Annexes I, II, and III to Regulation (EC) No 1234/2008 Regulation as regards to the examination of variations to the terms of marketing authorisations for medicinal products for human use can be downloaded here.

Regulatory Affairs Professionals Society (RAPS)

EU patient groups say proposed definition of ‘unmet medical need’ is too restrictive

The overhaul of the EU pharmaceutical legislation, which includes the definition of unmet medical need, was adopted by the EC in April 2023. This definition carries significant implications as it shapes pharmaceutical companies' eligibility for regulatory data protection, conditional marketing authorisations, and enhanced regulatory and scientific support. Most notably, it has implications for the post-authorisation phase and health technology assessment (HTA). According to the European Patients' Forum (EPF) It is imperative that this definition is aligned with the true needs of patients. As the voice of patient organisations in Europe, the EPF calls for patient involvement in drawing up the definition.

EPF proposes a future framework to characterise "unmet medical need" as part of the revision of EU pharmaceutical legislation. According to the proposed framework it should:

  • Apply to medicinal products intended to treat, but also medicinal products intended to prevent or diagnose a disease, or to restore, correct or modify physiological functions as per the definition of medicinal products in the EU.
  • Acknowledge both immediate therapeutic needs perceived by patients that are unmet by current treatments and longer-term societal needs, including the impact on caregivers and potential public health concerns.
  • Embrace inclusivity, recognising that many chronic diseases, while not life-threatening, substantially burden patients, caregivers, and healthcare systems. It should take into account not only mortality and morbidity but also their broader impact on patients' lives and health-related quality of life.
  • Assess patients' therapeutic needs based on various criteria, encompassing the impact of the condition on life expectancy, the appropriateness of current standards of care, and patient experience data (PED), including their quality of life and any mental health implications.

Please find further information here.

Furthermore, in order to steer pharmaceutical investment into medicines for unmet medical needs the EC proposed the provision of targeted regulatory protection incentives and regulatory support for medicines addressing unmet medical need (UMN) and high unmet medical need (HUMN). Please find further details here.

European Medicines Agency (EMA)

DARWIN EU® continues expanding its capacity to deliver real-world data studies

The Data Analysis and Real World Interrogation Network DARWIN EU continues working towards higher capacity for real-world data (RWD) studies and aims to add ten new data partners this year. Currently, the network operates with 20 public or private institutions from 13 European countries. The data partners generate real-world evidence (RWE) from various sources such as hospitals, primary care, health insurance, registries and biobanks to support regulatory activities of EMA’s scientific committees and national regulators in the EU, enabling access to data from approximately 130 million patients across Europe. So far, 14 RWD studies have been completed and eleven are ongoing. The results from the completed studies have been shared with the relevant EMA committees and other stakeholders. Once the protocols and results are finalised, the protocols and results of all DARWIN studies are made publicly available in the HMA-EMA catalogue of RWD studies. Please find further information here.

Accelerating stakeholder collaboration to enhance the clinical trials environment in the EU

The Accelerating Clinical Trials in the EU (ACT EU) initiative has established a multi-stakeholder platform (MSP) aimed at improving the environment for clinical trials across the EU by providing a platform for stakeholders to exchange views on all aspects of clinical research. Topics for discussion include clinical trial design, conduct, statistical analysis, proposals for regulation optimisation, transparency of data, and patient engagement. Please find further information here.


Food and Drug Administration (FDA)

BLA approval: Lenmeldy

The FDA has approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic (blood) stem cells (HSCs), which have been genetically modified to include functional copies of the ARSA gene.Please find further information here.

Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products

The FDA has issued a draft guidance which provides recommendations to sponsors who are considering submitting a non-interventional/observational study proposal to the FDA. This draft guidance was developed in response to stakeholders’ growing interest in the potential use of non-interventional/ observational studies to contribute to a demonstration of the effectiveness or safety of a drug.

Artificial Intelligence and Machine Learning for Biological and Other Products Regulated by CBER

Since 2016, CBER has identified an increasing use of artificial intelligence and machine learning (AI/ML) in IND submissions of vaccines, cellular products, and gene therapies. Currently, there are 70 IND applications with AI/ML, used in several disciplines (e.g., clinical, CMC, pharmacovigilance) which involve prediction, classification, clustering, and anomaly detection. CBER supports the validated use of AI/ML throughout the product life cycle to expedite product development and approval, and support effective product oversight. CBER coordinates its AI/ML activities through the Artificial Intelligence Coordinating Committee (AICC). The AICC is executing a strategy focusing on staff education, communication, facilitating review of AI/ML in submissions, and project tracking and submission landscaping. Please find further information here.

Select Updates for the Premarket Cybersecurity Guidance: Section 524B of the FD&C Act

The FDA has developed a draft guidance to propose select updates to the FDA guidance document "Cybersecurity in Medical Devices: Quality System Considerations and Content of Premarket Submissions” (hereafter referred to as the “Premarket Cybersecurity Guidance"). FDA is proposing to add a Section VII. to the Premarket Cybersecurity Guidance to address new considerations for cyber devices. The new section identifies the cybersecurity information FDA considers to be needed to support obligations under section 524B of the FD&C Act.

FDA Works to Make Informed Consent Easier to Understand

It is the research community’s ethical obligation to ensure that individuals who participate in research understand the purpose and the potential risks and benefits of the research before agreeing to participate. Too often, however, informed consent forms are difficult for potential research participants to understand. To improve this informed consent process, the FDA and the Office for Human Research Protections have published a draft guidance titled “Key Information and Facilitating Understanding in Informed Consent.” This guidance is intended to assist institutional review boards (IRBs), investigators, and sponsors engaged in or responsible for oversight of human subject research subject to FDA and/or Health and Human Services (HHS) regulations with the development of consent information that would comply with 45 CFR 46.116(a)(5) and FDA’s proposed revisions to 21 CFR 50.20(e), if finalized as proposed. Electronic and written comments can be submitted until 1st of May 2024.


International Conference on Harmonisation (ICH)

ICH Q12 IWG “Regulatory and Technical Considerations for Pharmaceutical Product Lifecycle Management"

Following the publication of the ICH Q12 Module 8 in February 2024, ICH Q12 “Regulatory and Technical Considerations for Pharmaceutical Product Lifecycle Management Introductory Video” has been made available on the Quality Guidelines webpage.

Regulatory Affairs Professionals Society (RAPS)

Asia-Pacific Roundup: Australia’s TGA consults on updated draft companion diagnostics guidance

Australia’s Therapeutic Goods Administration (TGA) is seeking feedback on its plans to revise the current guidance on companion diagnostics (CDx), to help companies determine if companion testing is needed and introducing a concept to recognize the diagnostic evaluations carried out as part of a medicine application. The proposed updates aim to provide sponsors and manufacturers of medicines, biologicals and in vitro diagnostic (IVD) medical devices further clarification by providing the following additional sections:

  • A CDx testing identification guide to assist them in identifying whether their medicine or biological indication requires companion testing,
  • An introduction to the concept of the ‘companion testing plan’ to recognise the CDx component evaluations undertaken as part of the medicine application.
  • Improved clarity on clinical and analytical performance requirements for CDx, and
  • Case studies to assist sponsors of medicines and devices on the regulatory process and the technical documentation required for an IVD CDx.

The draft guidance is open for comment until 17 June. The draft document and information on how to submit comments can be found here.

Public consultations

European Medicines Agency (EMA)

Food and Drug Administration (FDA)


Consultation Period



Advanced Manufacturing Technologies Designation Program

Guidance for Industry

End date: Open

Draft guidance


Master Protocols for Drug and BiologicalProduct Development

Guidance for Industry

End date: Open

Draft guidance


Potency Assurance for Cellular and GeneTherapy Products

End date: 27th March 2024

Draft guidance


Use of Data Monitoring Committees in Clinical Trials
Guidance for Industry

End date: 16th April 2024

Draft guidance


Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients Under Section 506C of the FD&C Act

End date: Open

Draft Guidance


Select Updates for the Medical Device User Fee Small Business Qualification and Certification Guidance

Draft Guidance for Industry and Food and Drug Administration Staff

End date: 23rd April 2024

Draft Guidance


Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products Guidance for Industry

End date: 19th June 2024

Draft Guidance

Click here to download the PDF