ReviewPharmacoEconomics - open2019
Strimvelis
Review in PharmacoEconomics - open, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 4 of them syntheses 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
15 citing papers in PubMed, 4 syntheses or guidelines pooled it, 29 citations in OpenAlex.
- Newborn screening for severe combined immunodeficiency in the NHS newborn blood spot screening programme: a systematic review.Health technology assessment (Winchester, England) · 2026Pooled it
- A systematic review on the appropriate discounting rates for the economic evaluation of gene therapies: whether a specific approach is justified to tackle the challenges?International journal of technology assessment in health care · 2024Pooled it
- Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review.PharmacoEconomics · 2021Pooled it
- A systematic review of economic evaluations of advanced therapy medicinal products.British journal of clinical pharmacology · 2021Pooled it
- ERAD-level gene switches for on-demand protein secretion and rapidly controlled gene therapies.Nature communications · 2026Article
- Culturing Potential: advances in ex vivo cell culture systems for haematopoietic cell-based regenerative therapies.Regenerative therapy · 2025Review
- Review
- Microneedle-Mediated Transdermal Delivery of Genetic Materials, Stem Cells, and Secretome: An Update and Progression.Pharmaceutics · 2023Review
- Adding recombinant AAVs to the cancer therapeutics mix.Molecular therapy oncolytics · 2022Review
- Research advances in treatment methods and drug development for rare diseases.Frontiers in pharmacology · 2022Review
- Critical Reflections on Reimbursement and Access of Advanced Therapies.Frontiers in pharmacology · 2022Article
- The Progression of Treatment for Refractory Hypercholesterolemia: Focus on the Prospect of Gene Therapy.Frontiers in genetics · 2022Review
- Improved functionality and potency of next generation BinMLV viral vectors toward safer gene therapy.Molecular therapy. Methods & clinical development · 2021Article
- The Limitless Future of RNA Therapeutics.Frontiers in bioengineering and biotechnology · 2021Review
- Health economic evaluation of gene replacement therapies: methodological issues and recommendations.Journal of market access & health policy · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
Abstract
The Centre for Reviews and Dissemination and Centre for Health Economics Technology Assessment Group at the University of York was commissioned by the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies (HST) programme to act as the independent Evidence Review Group (ERG) for an appraisal of Strimvelis
Identifiers
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.