ArticlePharmacoEconomics - open2023
"Don't Think Twice, It's All Right": Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England.
Article in PharmacoEconomics - open, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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
16 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Approaches used to model patient and carer health-related quality of life in economic models of rare disease treatments in NICE appraisals.Health and quality of life outcomes · 2026Review
- Use of RWD in the assessment of economic evaluations of innovative health products: lessons learned from the French National Authority for health (HAS).International journal of technology assessment in health care · 2026Article
- Implementing performance-based risk-sharing agreements in non-small cell lung cancer immunotherapy: a real-world data case study.Health economics review · 2025Article
- Planning Post-Launch Evidence Generation: Lessons From France, England and Spain.Clinical pharmacology and therapeutics · 2025Review
- Real-world evidence: state-of-the-art and future perspectives.Journal of comparative effectiveness research · 2025Review
- The Use of Real-World Data for Estimating Relative Treatment Effects in NICE Health Technology Assessment Submissions: A Review.PharmacoEconomics · 2025Review
- Evidence Following Conditional NICE Technology Appraisal Recommendations: A Critical Analysis of Methods, Quality and Risk of Bias.PharmacoEconomics · 2024Article
- Calculating the Expected Net Benefit of Sampling for Survival Data: A Tutorial and Case Study.Medical decision making : an international journal of the Society for Medical Decision Making · 2024Article
- An Analysis of Uncertainties and Data Collection Agreements in the Cancer Drugs Fund.PharmacoEconomics - open · 2024Article
- Real-world evidence for coverage determination of treatments for rare diseases.Orphanet journal of rare diseases · 2024Article
- Real-world evidence in the reassessment of oncology therapies: payer perceptions from five countries.Future oncology (London, England) · 2024Article
- Expanding access to high-cost medicines under the Universal Health Coverage scheme in Thailand: review of current practices and recommendations.Journal of pharmaceutical policy and practice · 2023Article
- Real-world utilization and healthcare costs for multiple myeloma: A retrospective analysis of patients in Singapore.EJHaem · 2023Article
- The Innovative Medicines Fund: a universal model for faster and fairer access to new promising medicines or a Trojan horse for low-value creep?Journal of the Royal Society of Medicine · 2023Article
- Article
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
Abstract
objectivesThe Cancer Drugs Fund (CDF) in England uses managed access agreements to facilitate additional data collection to address uncertainties identified in the appraisals of new drugs. This study reviews the uncertainties highlighted in the original appraisals where recommendations "to use within the CDF" were made and how additional data were used to address these uncertainties in the CDF review appraisals where final decisions on routine commissioning were made.
methodsThe first 24 drugs exiting the 2016 CDF were included in this review. The information about uncertainty and the use of newly collected data were extracted from the original appraisals and the CDF review appraisals. The additional data used in the CDF review appraisals, distinguishing between clinical trial data and real-world data (RWD), were reviewed to assess the extent to which the additional data were able to reduce the original uncertainties.
resultsThe recommendation that the drug be routinely commissioned was made in 87.5% of re-appraisals. Uncertainty stemming from immaturity of the survival data in clinical trials was frequently found in appraisals. Later follow-up of clinical trials was used to address this uncertainty, whereas limited use was made of RWD. The Systemic Anti-Cancer Therapy (SACT) dataset is the most frequently used source of RWD. SACT data were mostly used in review appraisals to support the clinical outcomes based on later follow-up of trial participants and to inform modelling of subsequent treatments or treatment duration.
conclusionsWhile additionally collected RWD attracted attention when the 2016 CDF was introduced, RWD have not been widely used in CDF review appraisals and (to date) have done little to reduce uncertainty. Experience with these appraisals has highlighted the importance of longer follow-up of clinical trials and the relatively limited role of RWD, in general, and of SACT data in particular.
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.