At DIA Europe 2025, one session that particularly stood out was titled “Overcoming global challenges for Advanced Therapy Medicinal Products (ATMPs) through collaboration, regulatory convergence and reliance.” The presentations, and the discussions that followed, addressed the pressing need for regulatory alignment to ensure equitable and efficient access to ATMPs across the globe.

Advanced Therapy Medicinal Products, which comprise gene therapies, cell therapies, and tissue-engineered products, are revolutionising the management of many diseases. However, their development and delivery are often hampered by fragmented regulatory environments, inconsistent definitions, and complex local and regional regulatory requirements.

The regulation of medicines has evolved to deal efficiently with medicinal products with broad applicability to large populations with the same disease to ensure the quality of the product and a favourable balance of risk and benefit throughout the lifecycle of the product. ATMPs are often tailored to an individual patient, and the treatment that the patient receives is unique to the subject being treated. Regulatory expectations and guidelines must be sufficiently flexible to adapt to the environment in which the nature of the treatment may vary from one patient to the next, and in which the treatment can only be delivered at a limited number of technically specialised centres.

The session highlighted how collaborative efforts can streamline regulation and improve patient access.

Key Challenges 

• Diverse Regulatory Frameworks

Definitions, classifications, and regulatory pathways for ATMPs vary significantly between jurisdictions, making global development and access inefficient and burdensome.

• In-country Testing Requirements

Some regions mandate additional testing upon import, further delaying clinical trials and approvals.

• Genetically modified organisms (GMOs)

The regulatory landscape is particularly fragmented for ATMPs containing GMOs:

• The EU has highly variable timelines for clinical trial approvals for studies that include a GMO.
• The US offers a more streamlined path with “categorical exclusions.”
• Many countries lack defined GMO regulatory procedures.

• Hospital Exemption Pathways

These provide a route for early access to ATMPs in exceptional circumstances. However, many countries do not offer this route, or apply it inconsistently. Japan stands out for applying Good Gene, Cellular, and Tissue-based Products Manufacturing Practice even under this exemption.

IFPMA’s Vision for Progress

The International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) has laid out a clear roadmap:

• Promote regulatory convergence by aligning definitions, requirements, and approval processes.

• Foster reliance models that allow countries to recognise or rely on regulatory decisions from trusted authorities.

• Streamline GMO requirements by encouraging adoption of harmonised environmental risk assessment (ERA) guidelines—such as those proposed by the EU.

• Explore unified digital labelling tools such as QR codes and 2D matrices.

• Support hospital exemption frameworks to allow compassionate use of unlicensed therapies where no alternatives exist.

Conclusion

ATMPs have already made a significant impact on the management of diseases, many of which are rare, for which there is an unmet medical need. The potential for ATMPs to treat hitherto untreatable conditions is immense, but unlocking their full impact requires global coordination. The IFPMA ATMP working group’s initiative—through mapping exercises, recommendations, and engagement with stakeholders—is a vital step toward a more coherent and patient-focused regulatory environment.

As a pharmacovigilance and regulatory consultancy, Sagittal Pharma Consulting supports these collaborative efforts and continues to monitor the evolution of ATMP regulations. We remain committed to guiding our clients through this dynamic landscape to ensure patients can access innovative therapies safely and efficiently.