18.03.2026

ECA ATMP Group publishes position paper on specifications considering patient-centricity
[中譯] ECA ATMP 小組發布關於考量「以病患為核心」之規格制定立場文件

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.
[中譯] 由 ECA ATMP 小組發布的立場文件「關於 ATMP 規格考量以病患為核心之省思」，探討了一項核心法規與科學挑戰：如何為細胞與基因治療（特別是自體細胞治療）制定適當的產品規格。

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.
[中譯] 在傳統藥物開發與製造模式中，規格（即品質屬性、檢驗方法與認可標準）是根據受控的製造製程以及具再現性的起始物料來定義的。

This concept is enshrined in regulatory guidelines such as the ICH Guidelines and works well for conventional medicinal products or biotechnologically produced proteins.
[中譯] 此概念已深植於如 ICH 指南等法規準則中，且對於傳統藥品或生物技術生產的蛋白質藥物運作良好。

However, this approach has its limitations when it comes to ATMPs.
[中譯] 然而，當應用於先進醫療產品 (ATMPs) 時，這種做法便產生了局限性。

The main reason for this is the high variability of the starting material.
[中譯] 其主要原因在於起始物料的高度變異性。

In many ATMPs, especially autologous cell therapies, the starting cells come directly from the patient and are naturally very different in their properties.
[中譯] 在許多 ATMP（特別是自體細胞治療）中，起始細胞直接取自病患，其特性在本質上便有極大差異。

Factors such as age, disease status, previous therapies, inflammatory processes or tumour environment can significantly influence the quality and function of the cells.
[中譯] 諸如年齡、疾病狀態、過往治療史、發炎反應或腫瘤環境等因素，皆會顯著影響細胞的品質與功能。

This makes the manufacture of ATMPs with these starting materials much more difficult to control than in classic industrial production processes.
[中譯] 這使得以這些起始物料製造 ATMP 時，在製程管控上比傳統工業生產流程困難許多。

Another problem is that the mechanisms of action of many ATMPs are complex and often not yet fully understood.
[中譯] 另一個問題在於，許多 ATMP 的作用機轉極為複雜，且通常尚未被完全理解。

In particular, the development of suitable potency or bioactivity assays presents a major challenge.
[中譯] 特別是開發合適的效價 (Potency) 或生物活性分析方法，構成了重大挑戰。

In some cases, the therapeutic effects are patient-specific, for example due to individual antigens.
[中譯] 在某些案例中，治療效果具有病患特異性，例如因個體抗原而異。

This makes it difficult to establish standardised reference materials or clear in vitro tests.
[中譯] 這導致難以建立標準化的對照標準品或明確的體外測試方法。

At the same time, small batch sizes and short shelf lives make it difficult to comprehensively characterise the products.
[中譯] 同時，小批量產與短架效期的特性，也增加了全面進行產品特性鑑別的難度。

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.
[中譯] 在此背景下，該文件提出了質疑：現行高度聚焦於「基於製程的規格」與「批次數據統計評估」的法規手段，是否充分適用於 ATMP。

In particular, there is a risk that overly narrow specification limits will lead to frequent out-of-specification results.
[中譯] 特別是存在一種風險：過於窄小的規格限值將導致頻繁的檢驗超標 (OOS) 結果。

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.
[中譯] 就自體治療而言，這將對病患產生直接影響：若產品未獲放行，可能需要再次採集組織樣品或進行活檢，這對病患意味著額外的風險與壓力。

As a possible solution, the position paper discusses the concept of patient-centric specifications.
[中譯] 作為可能的解決方案，立場文件討論了「以病患為核心之規格」的概念。

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.
[中譯] 此概念更側重於哪些品質屬性與病患的安全及療效有實質關聯，而非僅反映製程的可製造性。

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.
[中譯] 其目標在於設計能兼顧 ATMP 固有變異性的規格，並實現安全且有效的治療。

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.
[中譯] 該文件呼籲對 ATMP 品質要求的法規與科學評估進行反思：從純粹基於製程的標準，轉向更導向病患的品質理解，以更準確地反映個性化細胞治療的特定性質。

The full position paper is available in the Members' Area of the ECA ATMP Group website.
[中譯] 完整立場文件可於 ECA ATMP 小組網站的會員專區取得。

If you are not yet a member, simply register; membership is free of charge.
[中譯] 若您尚未成為會員，只需註冊即可；會員資格是免費的。

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.
[中譯] 您亦可在 3 月 24/25 於威斯巴登舉行的 PharmaCongress ATMP 會議上了解更多關於此文件的內容並參與討論。

Source: https://www.gmp-compliance.org/gmp-news/eca-atmp-group-publishes-position-paper-on-specifications-considering-patient-centricity
[中譯] 來源：https://www.gmp-compliance.org/gmp-news/eca-atmp-group-publishes-position-paper-on-specifications-considering-patient-centricity