ArticlePatient preference and adherence2015
Understanding patient preferences and willingness to pay for hemophilia therapies.
Article in Patient preference and adherence, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 citing papers in PubMed, 32 citations in OpenAlex.
- Disease burden and unmet medical need in severe hemophilia in Greece: insights from clinicians and patients.Frontiers in public health · 2026Article
- Prediction of COVID-19 patients' participation in financing informal care using machine learning methods: willingness to pay and willingness to accept approaches.BMC health services research · 2024Article
- Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study.JMIR public health and surveillance · 2023Article
- Assessing patient and caregiver preferences for treatment of haemophilia A: A discrete choice experiment.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Patients' and parents' satisfaction with, and preference for, haemophilia A treatments: a cross-sectional, multicentre, observational study.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Observational
- Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment.Orphanet journal of rare diseases · 2021Article
- Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Patient Preferences to Assess Value IN Gene Therapies: Protocol Development for the PAVING Study in Hemophilia.Frontiers in medicine · 2021Article
- Preferences and Health-Related Quality-of-Life Related to Disease and Treatment Features for Patients with Hemophilia A in a Canadian General Population Sample.Patient preference and adherence · 2021Article
- Postauthorization safety surveillance study of antihaemophilic factor (recombinant) reconstituted in 2 mL sterile water for injection in children with haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2020Article
- Exploring the Impact of Infusion Frequency in Hemophilia A: Exit Interviews with Patients Participating in BAY 94-9027 Extension Studies (PROTECT VIII).The patient · 2019Article
- The role of patient and healthcare professionals in the era of new hemophilia treatments in developed and developing countries.Therapeutic advances in hematology · 2018Review
- Attitudes of Individuals with Gaucher Disease toward Substrate Reduction Therapies.Journal of genetic counseling · 2018Article
- Patient preferences in the treatment of hemophilia A: impact of storage conditions on product choice.Patient preference and adherence · 2018Article
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
No grant is acknowledged in the PubMed record.
Abstract
backgroundDespite clearly improved clinical outcomes for prophylaxis compared to on-demand therapy, on average only 56% of patients diagnosed with severe hemophilia receive prophylactic factor replacement therapy in the US. Prophylaxis rates generally drop as patients transition from childhood to adulthood, partly due to patients becoming less adherent when they reach adulthood. Assessment of patient preferences is important because these are likely to translate into increased treatment satisfaction and adherence. In this study, we assessed preferences and willingness to pay (WTP) for on-demand, prophylaxis, and longer acting prophylaxis therapies in a sample of US hemophilia patients.
methodsAdult US hemophilia patients and caregivers (N=79) completed a discrete-choice survey that presented a series of trade-off questions, each including a pair of hypothetical treatment profiles. Using a mixed logit model for analysis, we compared the relative importance of five treatment characteristics: 1) out-of-pocket treatment costs (paid by patients), 2) factor dose adjustment, 3) treatment side effects, 4) availability of premixed factor, and 5) treatment effectiveness and dosing frequency. Based on these attribute estimates, we calculated patients' WTP.
resultsOut-of-pocket treatment costs (P<0.001), side effects (P<0.001), and treatment effectiveness and dosing frequency (P<0.001) were found to be statistically significant in the model. Patients were willing to pay US $410 (95% confidence interval: $164-$656) out of pocket per month for thrice-weekly prophylaxis therapy compared to on-demand therapy and $360 (95% confidence interval: $145-$575) for a switch from thrice-weekly to once-weekly prophylaxis therapy.
conclusionImprovements in treatment effectiveness and dosing frequency, treatment side effects, and out-of-pocket costs per month were the greatest determinants of hemophilia treatment choice and WTP. The positive preferences and WTP for longer acting prophylactic therapies suggest that the uptake is likely to increase adherence, improving treatment outcomes. These preferences should also inform the Food and Drug Administration's assessment of new longer acting hemophilia therapies.
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.