ReviewBMJ open2022
Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals.
Review in BMJ open, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 2 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
11 citing papers in PubMed, 2 syntheses or guidelines pooled it, 18 citations in OpenAlex.
- Pooled it
- Pooled it
- The UK early access to medicines scheme: uptake, approvals, and reimbursement.International journal of technology assessment in health care · 2025Article
- Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology.International journal of technology assessment in health care · 2025Review
- How Can General Managers Best Leverage Medical Affairs Now and in the Future?Pharmaceutical medicine · 2024Article
- Journey from an Enabler to a Strategic Leader: Integration of the Medical Affairs Function in ESG Initiatives and Values.Pharmaceutical medicine · 2023Article
- Selection Bias in Real-World Data Studies Used to Support Health Technology Assessments: A Case Study in Metastatic Cancer.Current oncology (Toronto, Ont.) · 2023Article
- Access to medicines for rare diseases: A European regulatory roadmap for academia.Frontiers in pharmacology · 2023Article
- Incremental benefits of novel pharmaceuticals in the UK: a cross-sectional analysis of NICE technology appraisals from 2010 to 2020.BMJ open · 2022Article
- Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations.Frontiers in pharmacology · 2022Article
- 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
4 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesTo quantify and characterise the usage of expanded access (EA) data in National Institute for Health and Care Excellence (NICE) technology appraisals (TAs). EA offers patients who are ineligible for clinical trials or registered treatment options, access to investigational therapies. Although EA programmes are increasingly used to collect real-world data, it is unknown if and how these date are used in NICE health technology assessments.
designCross-sectional study of NICE appraisals (2010-2020). We automatically downloaded and screened all available appraisal documentation on NICE website (over 8500 documents), searching for EA-related terms. Two reviewers independently labelled the EA usage by disease area, and whether it was used to inform safety, efficacy and/or resource use. We qualitatively describe the five appraisals with the most occurrences of EA-related terms. PRIMARY OUTCOME MEASURE: Number of TAs that used EA data to inform safety, efficacy and/or resource use analyses.
resultsIn 54.2% (206/380 appraisals), at least one reference to EA was made. 21.1% (80/380) of the TAs used EA data to inform safety (n=43), efficacy (n=47) and/or resource use (n=52). The number of TAs that use EA data remained stable over time, and the extent of EA data utilisation varied by disease area (p=0.001).
conclusionNICE uses EA data in over one in five appraisals. In synthesis with evidence from well-controlled trials, data collected from EA programmes may meaningfully inform cost-effectiveness modelling.
Indexed as
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.