Evidence map›Paper›PMID 31317510›Full record

Trial reportApplied health economics and health policy2019

Phase I/II Clinical Trial-Based Early Economic Evaluation of Acalabrutinib for Relapsed Chronic Lymphocytic Leukaemia.

Rick A Vreman, Joost W Geenen, Anke M Hövels, Wim G Goettsch, Hubert G M Leufkens, Maiwenn J Al

Abstract readClinical Trial, Phase IClinical Trial, Phase II
In one paragraph

Trial report in Applied health economics and health policy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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

11 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Systematic Literature Review of Economic Evaluations of Treatment Alternatives in Chronic Lymphocytic Leukemia.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2023
    Pooled it
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  11. Cardiovascular Toxicities of Bruton's Tyrosine Kinase Inhibitors.Current treatment options in oncology · 2020
    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

6 authors.

Rick A VremanDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Universiteitsweg 99, 3584 CG, Utrecht, The Netherlands. r.a.vreman@uu.nl.ORCID 0000-0002-2076-322X
Joost W GeenenDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Universiteitsweg 99, 3584 CG, Utrecht, The Netherlands.
Anke M HövelsDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Universiteitsweg 99, 3584 CG, Utrecht, The Netherlands.
Wim G GoettschDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Universiteitsweg 99, 3584 CG, Utrecht, The Netherlands. w.g.goettsch@uu.nl.
Hubert G M LeufkensDivision of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Universiteitsweg 99, 3584 CG, Utrecht, The Netherlands.
Maiwenn J AlErasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, The Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesThe objective of this study was to construct an early economic evaluation for acalabrutinib for relapsed chronic lymphocytic leukaemia (CLL) to assist early reimbursement decision making. Scenarios were assessed to find the relative impact of critical parameters on incremental costs and quality-adjusted life-years (QALYs).

methodsA partitioned survival model was constructed comparing acalabrutinib and ibrutinib from a UK national health service perspective. This model included states for progression-free survival (PFS), post-progression survival (PPS) and death. PFS and overall survival (OS) were parametrically extrapolated from ibrutinib publications and a preliminary hazard ratio based on phase I/II data was applied for acalabrutinib. Deterministic and probabilistic sensitivity analyses were performed, and 1296 scenarios were assessed.

resultsThe base-case incremental cost-effectiveness ratio (ICER) was £61,941/QALY, with 3.44 incremental QALYs and incremental costs of £213,339. Deterministic sensitivity analysis indicated that survival estimates, utilities and treatment costs of ibrutinib and acalabrutinib and resource use during PFS have the greatest influence on the ICER. Probabilistic results under different development scenarios indicated that greater efficacy of acalabrutinib would decrease the likelihood of cost effectiveness (from 63% at no effect to 2% at maximum efficacy). Scenario analyses showed that a reduction in PFS did not lead to great QALY differences (- 8 to - 14% incremental QALYs) although it did greatly affect costs (- 47 to - 122% incremental pounds). For OS, the opposite was true (- 89 to - 93% QALYs and - 7 to - 39% pounds).

conclusionsAcalabrutinib is not likely to be cost effective compared with ibrutinib under current development scenarios. The conflicting effects of OS, PFS, drug costs and utility during PFS show that determining the cost effectiveness of acalabrutinib without insight into all parameters complicates health technology assessment decision making. Early assessment of the cost effectiveness of new products can support development choices and reimbursement processes through effective early dialogues between stakeholders.

Indexed as

Antineoplastic AgentsBenzamidesCost-Benefit AnalysisHumansLeukemia, Lymphocytic, Chronic, B-CellMiddle AgedPyrazinesQuality-Adjusted Life YearsState MedicineSurvival AnalysisUnited KingdomacalabrutinibAntineoplastic AgentsBenzamidesPyrazines

Identifiers

PMID31317510
PMCPMC6885502

What Socratic holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

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 Socratic graph.