ArticlePharmacoEconomics - open2019
The Role of Real-World Evidence in UK Reimbursement: Case Study of Lenalidomide in Myelodysplastic Syndrome Deletion 5q.
Article in PharmacoEconomics - open, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 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
2 citing papers in PubMed.
- Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective.Journal for immunotherapy of cancer · 2020Article
- The quality of research with real-world evidence.Colombia medica (Cali, Colombia) · 2019Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundUncertainty within cost-effectiveness analysis, often driven by lack of mature data from large clinical trials, plays a key role in decisions made by the National Institute for Health and Care Excellence (NICE), particularly for early access medicines and orphan drugs.
objectivesIn this case study, we used real-world evidence to address the uncertainty in the cost-effectiveness case for lenalidomide in transfusion-dependent low- and intermediate-1-risk myelodysplastic syndrome (MDS) deletion 5q [del(5q)], affecting a small but unique subpopulation with an orphan disease.
methodsAs part of a submission to NICE, we developed a cost-effectiveness model for lenalidomide, resulting in eventual recommendation.
resultsDue to data limitations within the trial evidence available, the model was based on surrogate outcomes supported by a disease-wide literature review. The validity of modelled estimates for critical long-term outcomes in terms of time on treatment (32% reaching 26 cycles when the patient access scheme applied in the model vs. 28% in the real-world data) and survival was confirmed using real-world evidence (projected median survival for best supportive care of 4.3 years vs. real-world evidence showing median survival with low- and intermediate-1-risk MDS of 5.7 and 3.5 years, respectively).
conclusionThis case study demonstrates the usefulness and relevance of the application of real-world data when trial data are limited.
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.