Evidence map›Paper›PMID 34268704›Full record

ArticlePharmacoEconomics - open2021

Cost-Effectiveness Analysis of rFVIIIFc Versus Contemporary rFVIII Treatments for Patients with Severe Hemophilia A Without Inhibitors in the United States.

Ash Bullement, Emma S Knowles, Pronabesh DasMahapatra, Talaha Ali, Ron Preblick

Open access · goldAbstract read
In one paragraph

Article in PharmacoEconomics - open, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
1.0field-weighted citation impact, top 24% of its field
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 1 synthesis or guideline pooled it, 9 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 2 institutions in 2 countries.

Ash BullementDelta Hat, Nottingham, UK.ORCID http://orcid.org/0000-0001-7091-0972
Emma S KnowlesDelta Hat, Nottingham, UK.ORCID http://orcid.org/0000-0002-6503-2850
Pronabesh DasMahapatraSanofi Genzyme, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-8242-0580
Talaha AliSanofi Genzyme, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-3079-7853
Ron PreblickSanofi Genzyme, Cambridge, MA, USA. ronald.preblick@sanofi.com.ORCID http://orcid.org/0000-0002-7923-041X
Sanofi (United States) · USSanofi (France) · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundA range of treatments for patients with severe hemophilia A (HA) have been developed over the last decade, allowing for reduced frequency of administration and improved outcomes (joint health and breakthrough bleeding rates). While clinically effective, the cost effectiveness of these treatments has not been established.

objectiveThis study presents a cost-effectiveness analysis of contemporary rFVIII treatments for severe HA patients without inhibitors.

methodsA published semi-Markov model was used to compare three different prophylaxis regimens: (1) extended half-life (EHL) recombinant Factor VIII (rFVIII) Fc-fusion protein (rFVIIIFc, Eloctate

resultsrFVIIIFc was shown to provide the most QALYs (27.922) compared with both PEG-rFVIII (27.454) and SHL rFVIII (27.071), at lower costs. Discounted lifetime costs were estimated at US$18.235m (rFVIIIFc), US$20.198m (PEG-rFVIII), and US$18.285m (SHL rFVIII), and were predominantly affected by model settings related to acquisition costs, patient weight, and dosing.

conclusionsrFVIIIFc may offer a cost-effective option for severe HA patients. Uncertainties owing to the limited evidence base is the main limitation of the study.

Identifiers

PMID34268704
PMCPMC8611150
OpenAlexW3182262198

What OpenQuestion holds

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Registered trials

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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.