ReviewNature medicine2024
Enhancing pediatric access to cell and gene therapies.
Review in Nature medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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
22 citing papers in PubMed.
- Academic anti CD19 CAR-T cell therapy for relapsed/refractory B-cell lymphomas in real-world clinical practice: a prospective cohort study.Frontiers in oncology · 2026Trial
- Point-of-care fresh CAR T cells for pediatric or young adult BCP-ALL that is relapsed/refractory or in very-high-risk first relapse.Blood advances · 2025Trial
- AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and quality.Molecular therapy. Advances · 2026Article
- Recommendations for the use of clinical outcome assessments in rare disease drug development.EClinicalMedicine · 2026Review
- Bridging development and disease: the potential of LncRNAs as biomarkers and therapeutics in pediatric neurological disorders.Pediatric research · 2026Review
- The quintessential role for CAR T cell therapy in children, adolescents and young adults with cancer.Nature reviews. Clinical oncology · 2026Review
- A blueprint to accelerate rare pediatric gene therapy approvals.Nature medicine · 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
- Seeking stability for gene addition in inborn errors of metabolism.Molecular therapy. Nucleic acids · 2026Article
- Medicine Development and Access for Rare Diseases: Can We Do Better?Journal of inherited metabolic disease · 2026Review
- CAR T cells: the missing piece needed to improve outcomes for children with cancer?Journal for immunotherapy of cancer · 2026Review
- The Role of Microemulsions in Enhancing the Stability of Biopharmaceuticals.Drug design, development and therapy · 2026Review
- Advocating for child health through storytelling.Pediatric research · 2026Article
- Enabling access to genetically modified cell therapies through flexible approaches to manufacturing and cost recovery.Journal for immunotherapy of cancer · 2025Article
- Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Perspectives on the FDA platform technology designation program for the approval of gene therapies: a Swiss multi-stakeholder exploratory interview study.Orphanet journal of rare diseases · 2025Article
- Emerging polymeric nanocarriers for mRNA and protein therapeutics: design, challenges, and clinical outlook.Nanomedicine (London, England) · 2025Review
- Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable.Nature biotechnology · 2025Article
- How to democratize cell and gene therapy: A global approach.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Unlocking patient access to gene therapy: five key practices.Gene therapy · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Increasing numbers of cell and gene therapies (CGTs) are emerging to treat and cure pediatric diseases. However, small market sizes limit the potential return on investment within the traditional biopharmaceutical drug development model, leading to a market failure. In this Perspective, we discuss major factors contributing to this failure, including high manufacturing costs, regulatory challenges, and licensing practices that do not incorporate pediatric development milestones, as well as potential solutions. We propose the creation of a new entity, the Pediatric Advanced Medicines Biotech, to lead late-stage development and commercialize pediatric CGTs outside the traditional biopharmaceutical model in the United States-where organized efforts to solve this problem have been lacking. The Pediatric Advanced Medicines Biotech would partner with the academic ecosystem, manufacture products in academic good manufacturing practice facilities and work closely with regulatory bodies, to ferry CGTs across the drug development 'valley of death' and, ultimately, increase access to lifesaving treatments for children in need.
Indexed as
Identifiers
38886624What 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.