ATMP regulatory requirements are among the most complex in European medicines. Whether you’re developing a cell therapy, gene therapy or tissue-engineered product, the pathway to authorisation differs significantly from standard biological or small-molecule development.
At Pharma Design, we support you with specialist ATMP regulatory consulting across the EU, UK, and Switzerland. We can help you navigate classification, accelerated designations, clinical trial applications, and the full route to marketing authorisation.
What Are Advanced Therapy Medicinal Products?
Advanced therapy medicinal products are defined under EU Regulation 1394/2007 and regulated centrally by the EMA’s Committee for Advanced Therapies (CAT). They cover three product types:
- Gene therapy – medicines that introduce, alter or replace a genetic sequence to treat or prevent disease.
- Somatic cell therapy – medicines based on cells or tissues that have been substantially manipulated or used for a non-homologous function.
- Tissue-engineered products – products containing engineered cells or tissues intended to regenerate, repair or replace human tissue.
Products combining more than one category are classified as combined ATMPs. Some products used in a single hospital setting on a non-routine basis may qualify for a hospital exemption, though this route does not result in a marketing authorisation.
Why ATMP Regulatory Strategy Requires a Specialist Approach
ATMP development is high-risk and capital-intensive. For companies with limited internal regulatory resources, specialist support makes a great difference across the areas that matter most:
- Post–authorisation commitments – PASS/PAES obligations are common for ATMPs and need to be anticipated well before submission.
- Product classification – incorrect classification early in development leads to misaligned plans and delays at submission. CAT classification opinions should be sought early.
- Manufacturing – ATMPs carry GMP requirements beyond standard biologics, with specific expectations around starting materials, donor screening, and traceability.
- Clinical trial applications – CTA strategy for ATMPs must account for gene modification, donor eligibility, and long–term follow-up from the outset.
- Accelerated pathways – ATMPs frequently qualify for PRIME (EU) and ILAP (UK), but accessing them effectively requires preparation and eligibility planning.
ATMP Support
1. ATMP Classification and Early Regulatory Strategy
Early classification is not optional – it determines everything that follows, from your development plan to your dossier structure. Our ATMP consultants can support you with:
- CAT classification opinion preparation and submission
- Scientific advice strategy aligned to ATMP product type
- Regulatory pathway mapping across EMA, MHRA, and Swissmedic
- Early identification of combined ATMP considerations
- Hospital exemption assessment (EU), where applicable
2. PRIME and ILAP Designation for ATMPs
ATMPs addressing serious or life-threatening conditions frequently qualify for EMA’s PRIME scheme or MHRA’s Innovative Licensing and Access Pathway (ILAP). We help you access these pathways effectively with:
- PRIME eligibility assessment and application preparation
- ILAP and PIM designation support in the UK
- Early scientific advice coordination with EMA and MHRA
- Integrated regulatory and HTA strategy for designated products
3. Clinical Trial Applications (CTA) for ATMPs
Clinical trial applications for ATMPs carry additional requirements beyond a standard CTA. We manage the complexity so your team can focus on the science. This includes:
- ATMP-specific CTA strategy and preparation across the EU, UK, and Switzerland
- CTIS submission and correspondence management
- Substantial amendment strategy for evolving protocols
- Coordination with ethics committees and competent authorities
- Long-term follow-up protocol planning
4. Paediatric Development and Orphan Designation
Many ATMPs target rare diseases, particularly in oncology and genetic conditions, where paediatric populations are central to the development programme. Our work includes:
- Alignment of orphan and paediatric status with HTA requirements
- Paediatric Investigation Plan (PIP) strategy and PDCO interaction
- Orphan designation applications for rare disease ATMPs
- Orphan maintenance strategy through to MAA
5. MAA Preparation and Submission for ATMPs
The ATMP MAA dossier requires specialist structuring that reflects the unique requirements of the CAT review process alongside the standard CHMP assessment. This includes:
- Post-authorisation commitment planning and PASS/PAES strategy
- Full MAA preparation and submission management for ATMPs
- CAT and CHMP interaction and response management
- Risk management plan (RMP) development with ATMP–specific safety considerations
Why Pharma Design?
ATMP regulatory consulting requires a depth of experience that most small and mid-sized biotechs can’t create internally. Our consultants bring hands-on expertise across cell therapy, gene therapy, and tissue-engineered products – so you have specialist support without the overhead of a large internal regulatory function.
Working with Pharma Design on your ATMP programme means:
- Integrated rare disease and paediatric strategy where applicable
- Accurate classification and pathway mapping from day one
- Accelerated access to PRIME and ILAP where you qualify
- CTA strategies built around long-term follow-up requirements
- MAA dossiers structured to meet CAT expectations
- Integrated rare disease and paediatric strategy where applicable
A trusted partner across the full ATMP lifecycle
We work as an extension of your team across the full regulatory lifecycle – from initial classification through to post-authorisation commitments. For companies without European infrastructure, we can also provide regulatory representation through our own established EU and UK entities, already registered with EMA, MHRA, and Swissmedic. We can also support you with market access and medical writing services.
Speak to an ATMP Regulatory Consultant
If you’re developing a cell therapy, gene therapy, or tissue-engineered product and need specialist regulatory support across the EU, UK, or Switzerland, we can help.
Our Values
The value we bring to pharmaceutical companies falls into four main areas:
- Objective, impartial advice tailored to your needs
- Help cut your development costs
- Help reduce your time to market
- Keep your drug on the market