Patient-Centric ATMP Specifications
Austin Chuang • April 10, 2026
Navigate the regulatory shift towards patient-centric quality attributes for autologous cell and gene therapies with expert insights.
Patient-Centric ATMP Specifications
Executive Summary
The ECA ATMP Group has highlighted a critical regulatory and scientific challenge: traditional pharmaceutical development models, which rely on highly controlled processes and reproducible materials, fall short for ATMPs. Because starting materials (like a patient's own cells) are inherently variable, adhering to overly strict, process-based specification limits risks unnecessary out-of-specification (OOS) results. The newly proposed patient-centric framework prioritizes attributes relevant to patient safety and therapeutic efficacy over simple manufacturing reproducibility.
Starting cells derived directly from patients vary widely due to age, disease status, and tumor environments, making conventional industrial control difficult.
Complex, patient-specific mechanisms of action, coupled with small batch sizes, make standardizing reference materials and in vitro bioactivity assays highly challenging.
A paradigm shift towards patient-centric specifications ensures that quality limits reflect true efficacy and safety rather than arbitrary manufacturability constraints, preventing the need for repeat biopsies.
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18.03.2026
ECA ATMP Group publishes position paper on specifications considering patient-centricity
The position paper "Reflections on specifications for ATMPs, considering patient-centricity" by the ECA ATMP Group addresses a key regulatory and scientific challenge: The development of appropriate product specifications for cell and gene therapies, particularly for autologous cell therapies.
In the classic pharmaceutical development and manufacturing model, specifications – i.e. quality attributes, test methods and acceptance criteria – are defined on the basis of controlled manufacturing processes and reproducible starting materials.
This concept is enshrined in regulatory guidelines such as the ICH Guidelines and works well for conventional medicinal products or biotechnologically produced proteins.
However, this approach has its limitations when it comes to ATMPs.
The main reason for this is the high variability of the starting material.
In many ATMPs, especially autologous cell therapies, the starting cells come directly from the patient and are naturally very different in their properties.
Factors such as age, disease status, previous therapies, inflammatory processes or tumour environment can significantly influence the quality and function of the cells.
This makes the manufacture of ATMPs with these starting materials much more difficult to control than in classic industrial production processes.
Another problem is that the mechanisms of action of many ATMPs are complex and often not yet fully understood.
In particular, the development of suitable potency or bioactivity assays presents a major challenge.
In some cases, the therapeutic effects are patient-specific, for example due to individual antigens.
This makes it difficult to establish standardised reference materials or clear in vitro tests.
At the same time, small batch sizes and short shelf lives make it difficult to comprehensively characterise the products.
Against this background, the paper raises the question of whether the current regulatory approaches – which focus heavily on process-based specifications and statistical evaluation of batch data – are sufficiently suitable for ATMPs.
In particular, there is a risk that overly narrow specification limits will lead to frequent out-of-specification results.
In the case of autologous therapies, this would have direct consequences for patients: A non-approved product could necessitate another tissue sample or biopsy, which would mean additional risks and stress for patients.
As a possible solution, the position paper discusses the concept of patient-centric specifications.
This concept focuses more on which quality attributes are actually relevant for patient safety and efficacy, rather than solely reflecting the manufacturability of the process.
The aim is to design specifications in such a way that they take into account the inherent variability of ATMPs and enable safe and effective therapy.
The document calls for a rethink in the regulatory and scientific assessment of ATMP quality requirements: Away from purely process-based criteria towards a more patient-oriented understanding of quality that better reflects the specific characteristics of personalised cell therapies.
The full position paper is available in the Members' Area of the ECA ATMP Group website.
If you are not yet a member, simply register; membership is free of charge.
You can also learn more about the paper and join the discussion at the ATMP Conference at the PharmaCongress on 24/25 March in Wiesbaden.
Source: https://www.gmp-compliance.org/gmp-news/eca-atmp-group-publishes-position-paper-on-specifications-considering-patient-centricity
Primary Keywords (Short-Tail) ATMP, Cell Therapy, Gene Therapy, Regulatory Compliance, ICH Guidelines, Patient-Centric.
Targeted Phrases (Long-Tail) Patient-centric specifications for ATMPs, Autologous cell therapy regulatory guidelines, Overcoming out-of-specification in cell therapies, ECA ATMP Group position paper summary, Pharmaceutical validation for advanced therapies.
Hashtags #ATMP #CellTherapy #RegulatoryCompliance #PatientCentricity #PharmaTech #PersimmonEngineering #Validation