ReviewFrontiers in pharmacology2021
Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union.
Review in Frontiers in pharmacology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
11 citing papers in PubMed.
- Expanding Patient Access to Advanced Therapies.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- Advanced therapy medicinal products (ATMPs) - An analysis of the global regulatory status.British journal of clinical pharmacology · 2026Article
- Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review.Molecular therapy. Advances · 2026Review
- Addressing clinical uncertainties in ATMP reimbursement: a review of methodological guidelines and European practice.Frontiers in pharmacology · 2026Review
- Current challenges and future directions of ATMPs in regenerative medicine.Regenerative therapy · 2025Review
- The clinical assessment of studies on orphan drugs in relation to the EMA's authorization marketing decisions in Europe.Frontiers in pharmacology · 2025Article
- Emerging Ethical Challenges in Manufacturing of Cell-Based Medicines.Advances in experimental medicine and biology · 2025Review
- ATMP clinical trials in the UK.Regenerative medicine · 2024Article
- Regulatory Aspects for Approval of Advanced Therapy Medicinal Products in the EU.Handbook of experimental pharmacology · 2024Article
- The challenges of access to innovative medicines with limited evidence in the European Union.Frontiers in pharmacology · 2023Review
- Current landscape of clinical development and approval of advanced therapies.Molecular therapy. Methods & clinical development · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Several advanced therapy medicinal products (ATMPs) have been approved in the European Union (EU). The aim of this study is to analyse the methodological features of the clinical trials (CT) that supported the marketing authorization (MA) of the approved ATMPs in the EU. A systematic review of the characteristics of pivotal CT of ATMPs approved in the EU until January 31st, 2021 was carried out. A total of 17 ATMPs were approved and 23 CT were conducted to support the MA (median, 1, range, 1-3). Of those studies, 8 (34.78%) were non-controlled and 7 (30.43%) used historical controls. Only 7 (30.4%) were placebo or active-controlled studies. Among all CT, 21 (91.3%) were open-label and 13 (56.52%) had a single-arm design. To evaluate the primary endpoint, 18 (78.26%) studies used an intermediate and single variable. The median (IQR) number of patients enrolled in the studies was 75 (22-118). To date, ATMPs' approval in the EU is mainly supported by uncontrolled, single-arm pivotal CT. Although there is a trend toward an adaptive or a life cycle approach, a switch to more robust clinical trial designs is expected to better define the benefit and the therapeutic added value of ATMPs.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.