ArticlePharmaceutical medicine2023
The Cost of Biotech Innovation: Exploring Research and Development Costs of Cell and Gene Therapies.
Article in Pharmaceutical medicine, 2023. 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.
- Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies.British journal of haematology · 2026Review
- Precision modification of heart failure signaling by CRISPR-Cas9 base editing.Journal of molecular and cellular cardiology · 2026Review
- Discontinued therapies for sickle cell disease: status and future directions.Expert opinion on investigational drugs · 2026Review
- Assessing the Early Economic Feasibility of a Curative Gene Therapy for Multiple Sclerosis Using a Risk-Adjusted Valuation Framework.Healthcare (Basel, Switzerland) · 2026Article
- Gene Editing Applications as Future Cardiovascular Therapies.Annual review of genetics · 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
- Research and development investments for biologics independently developed by US biotechnology startups, 2017-2023.Health affairs scholar · 2025Article
- Mapping the Clinical Development Trajectory of Cell and Gene Therapy Products.Clinical pharmacology and therapeutics · 2025Article
- Article
- ACT To Sustain: Adoptive Cell Therapy To Sustain Access to Non-Commercialized Genetically Modified Cell Therapies.Transplantation and cellular therapy · 2024Review
- Integrating Diversity, Equity, and Inclusion into Preclinical, Clinical, and Public Health Mathematical Models.Bulletin of mathematical biology · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundClinical development paradigms for cell and gene therapies appear to be different to those of more conventional treatments: therefore, it is informative to explore this from the perspective of investments required to bring a new cell and/or gene therapy to the market. While there are a number of studies in the literature analyzing clinical-stage R&D costs for novel therapeutics, these are 'modality-agnostic' and thus do not elucidate costs specifically for the emerging class of cell and gene therapies.
objectivesThe objective of this study was to understand the research and development (R&D) costs associated with the clinical development of new cell and gene therapy assets
methodsAs part of our analysis of clinical-stage R&D costs for cell and gene therapies, we focused our efforts on cell and gene therapy assets recently approved by the US Food and Drug Administration (FDA) or expected to receive FDA approval by the end of 2024. A total of 25 therapies were identified for the study, 11 of which had sufficient level of detail for our clinical-stage R&D costing study. We calculated the clinical-stage R&D costs to bring a new cell and/or gene therapy to the market following a three-step approach, starting with (1) calculation of the out-of-pocket investment reported in US SEC reports; (2) we adjusted these figures for the risk of failure by applying a clinical trial phase-dependent attrition risk rate; (3) we accounted for the cost of capital of 10.5%.
resultsAfter accounting for R&D attrition rate (i.e., costs of failed programs) and applying a cost of capital at 10.5%, we estimate that the clinical-stage R&D investment required to bring a new cell and/or gene therapy to market is US$1943 M (95% CI US$1395 M, US$2490 M).
conclusionThis knowledge can inform financial planning for biopharma companies looking to enter the space and inform policy makers within the context of the commercialization and pricing of such therapies.
Indexed as
Identifiers
37286928What 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.