Updated 25/05/2026
The Danish Medicines Agency has published new guidance on the Investigator’s Brochure (IB) for clinical trials involving advanced therapy medicinal products (ATMPs). This guidance establishes requirements for documenting safety, efficacy, and manufacturing data before human studies begin.
Documentation areas covered by Danish Medicines Agency guidance
Essential sections for ATMP Investigator’s Brochures
Source: Danish Medicines Agency, 2026 | Georgian Medical Journal News
Background on ATMP clinical trials
Advanced therapy medicinal products—encompassing cell therapies, gene therapies, and tissue-engineered medicines—represent developments in precision medicine. The Danish Medicines Agency’s new IB guidance provides documentation expectations for sponsors.
The IB serves as the technical dossier used by clinical trial ethics committees and competent authorities to evaluate whether a proposed human study is scientifically sound and ethically justified. For ATMPs, detailed documentation is required before dosing participants.
What must be included in an ATMP Investigator’s Brochure
According to the Danish Medicines Agency guidance, comprehensive reporting is required across multiple domains. Non-clinical data and manufacturing information must include preclinical efficacy studies, toxicology profiles, and quality control specifications. Clinical sections must synthesize existing human data along with risk-benefit assessment tailored to the proposed patient population.
The guidance places emphasis on characterization and potency assays. Cell and gene therapies exhibit variability based on source material, expansion methods, and genetic modification approaches. Sponsors must provide detailed analytical characterization demonstrating batch-to-batch consistency and establishing acceptance criteria before clinical manufacture begins.
The guidance also addresses post-manufacturing safety surveillance, with attention to off-target effects, insertional mutagenesis (for gene therapies), and immunological reactions.
Regulatory implications
By publishing this dedicated ATMP guidance, the Danish Medicines Agency provides documentation standards for advanced therapies. The framework aims to reduce sponsor uncertainty during IB preparation and provide ethics committees with guidance for review.
For academic and commercial researchers planning cell or gene therapy trials, the Danish Medicines Agency guidance offers clarity on documentation, quality standards, and risk reporting requirements.
Key takeaways
- The Danish Medicines Agency has published new guidance for Investigator’s Brochures in advanced therapy clinical trials.
- ATMP documentation must include detailed non-clinical data, manufacturing quality specifications, clinical safety and efficacy summaries, and risk-benefit assessments.
- The guidance emphasizes analytical characterization and batch-to-batch consistency for cell and gene therapies.
- The framework provides standardized requirements for sponsors and ethics committees.
Frequently asked questions
Why is an Investigator’s Brochure specifically needed for ATMPs?
Cell and gene therapies exhibit biological variability. An ATMP IB must document this variability through detailed characterization, potency assays, and manufacturing consistency data to establish that the therapy is sufficiently reproducible and safe for human use.
How does this Danish guidance differ from previous IB standards?
The new guidance introduces ATMP-specific requirements around manufacturing characterization, off-target risk assessment, and long-term durability data.
When should sponsors begin preparing the ATMP IB?
Preparation should begin during preclinical development, ideally in parallel with manufacturing scale-up and quality control validation. Early engagement with regulatory authorities can help clarify documentation expectations.
The Danish Medicines Agency’s new guidance establishes documentation requirements for sponsors, ethics committees, and national authorities involved in ATMP trial initiation.
Source: For Clinical Trials with ATMP: Guidance to the content of the Investigator’s Brochure published
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Medically reviewed by Prof. Giorgi Pkhakadze, MD, MPH, PhD. Spotted an error? Contact the editorial team.
