ArticleMolecular therapy. Methods & clinical development2020
Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals.
Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 1 of them a synthesis that pooled it.
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
29 citing papers in PubMed, 1 synthesis or guideline pooled it, 51 citations in OpenAlex.
- Outlook of Cell Gene Therapies Development and Approval from Quality and Regulatory Perspective.Therapeutic innovation & regulatory science · 2026Pooled it
- Expanding Patient Access to Advanced Therapies.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- A tumor-on-a-chip for in vitro study of CAR-T cell immunotherapy in solid tumors.Nature biotechnology · 2026Article
- Characteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov.Gene therapy · 2026Article
- Adoptive T-cell therapies in the clinic.Bioengineering & translational medicine · 2026Review
- Advanced therapy medicinal product (ATMP) classification - a case study on spinal cord injury.Journal of translational medicine · 2026Review
- Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review.Molecular therapy. Advances · 2026Review
- Clinical evidence deficiencies drive CHMP negative opinions and pre-opinion withdrawals in the EU centralized procedure (2021-2025).Frontiers in medicine · 2026Article
- Redefining quality in cell and gene therapies: Lessons from implementing electronic QMS in academic cGMP facility.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Mapping the Clinical Development Trajectory of Cell and Gene Therapy Products.Clinical pharmacology and therapeutics · 2025Article
- Advanced Therapy Medicinal Products: Availability, Access and Expenditure in Italy.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Article
- Synthesizing regulatory guidance for demonstrating preclinical efficacy and translating promising cell therapies to early phase clinical trials: a scoping review.BMC medicine · 2024Article
- Regulatory Aspects for Approval of Advanced Therapy Medicinal Products in the EU.Handbook of experimental pharmacology · 2024Article
- Applying a clinical lens to animal models of CAR-T cell therapies.Molecular therapy. Methods & clinical development · 2022Review
- Compliance in Non-Clinical Development of Cell-, Gene-, and Tissue-Based Medicines: Good Practice for Better Therapies.Stem cells translational medicine · 2022Article
- Pre-approval and post-approval availability of evidence and clinical benefit of conditionally approved cancer drugs in Europe: A comparison with standard approved cancer drugs.British journal of clinical pharmacology · 2022Article
- Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment.Frontiers in public health · 2022Review
- Advanced Therapy Medicinal Products' Translation in Europe: A Developers' Perspective.Frontiers in medicine · 2022Article
- GMP Compliant Production of a Cryopreserved Adipose-Derived Stromal Cell Product for Feasible and Allogeneic Clinical Use.Stem cells international · 2022Article
- 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 at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Advanced therapy medicinal products (ATMPs) comprising cell therapy, gene therapy, and tissue-engineered products, offer a multitude of novel therapeutic approaches to a wide range of severe and debilitating diseases. To date, several advanced therapies have received marketing authorization for a variety of indications. However, some products showed disappointing market performance, leading to their withdrawal. The available evidence for quality, safety, and efficacy at product launch can play a crucial rule in their market success. To evaluate the sufficiency of evidence in submissions of advanced therapies for marketing authorization and to benchmark them against more established biological products, we conducted a matched comparison of the regulatory submissions between ATMPs and other biologicals. We applied a quantitative assessment of the regulatory objections and divergence from the expected data requirements as indicators of sufficiency of evidence and regulatory flexibilty, respectively. Our results demonstrated that product manufacturing was challenging regardless of the product type. Advanced therapies displayed critical deficiencies in the submitted clinical data. The submitted non-clinical data packages benefited the most from regulatory flexibility. Additionally, ATMP developers need to comply with more commitments in the post-approval phase, which might add pressure on market performance. Mitigating such observed deficiencies in future product development, may leverage their potential for market success.
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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.