ArticlePharmacoEconomics - open2023
A Framework for a Health Economic Evaluation Model for Patients with Sickle Cell Disease to Estimate the Value of New Treatments in the United States of America.
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 4 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
4 citing papers in PubMed, 4 citations in OpenAlex.
- Model-Based Economic Analyses of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2026Review
- Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States.PharmacoEconomics · 2024Article
- End Organ Affection in Sickle Cell Disease.Cells · 2024Review
- Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.Annals of internal medicine · 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
3 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundSickle cell disease (SCD) is an inherited blood disorder associated with lifelong morbidity and increased risk of mortality that affects approximately 100,000 individuals in the United States (US), primarily of African-American descent. Due to these complications, individuals with SCD typically incur high healthcare costs. With a number of costly but potentially curative SCD therapies on the horizon, understanding the progression of SCD and economic burden to insurers and patients is vital.
objectiveThe aim is to develop a framework to understand the progression and costs of SCD that could be used to estimate how new treatments can impact the progression and costs of the disease.
methodsWe detail how we will create a simulation model that represents the natural history of a population and allows for the characterization of the impact of novel therapies on the disease, associated costs, and outcomes in comparison to current management.
conclusionIn this report, we describe a conceptual approach to modeling SCD to determine the relative clinical and economic impact of new gene therapies compared to conventional therapies with a goal of providing a flexible approach that could inform the clinical management of SCD for patients, payers, and policy makers.
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.