Abstract:
BACKGROUND:Validation of overall survival (OS) extrapolations of immune-checkpoint inhibitors (ICIs) during the National Institute for Health and Care Excellence (NICE) Single Technology Assessment (STA) process is limited due to data still maturing at the time of submission. Inaccurate extrapolation may lead to inappropriate decision-making. The availability of more mature trial data facilitates a retrospective analysis of the plausibility and validity of initial extrapolations. This study compares these extrapolations to subsequently available longer-term data. METHODS:A systematic search of completed NICE appraisals of ICIs from March 2000 to December 2017 was performed. A targeted search was also undertaken to procure published OS data from the pivotal clinical trials for each identified STA made available post-submission to NICE. Initial Kaplan-Meier curves and associated extrapolations from NICE documentation were extracted to compare the accuracy of OS projections versus the most mature data. RESULTS:The review identified 11 STAs, of which 10 provided OS data upon submission to NICE. The extrapolations undertaken considered parametric or piecewise survival models. Additional data cut-offs provided a mean of 18 months of OS beyond the end of the original data. Initial extrapolations typically under-estimated OS from the most mature data cut-off by 0.4-2.7%, depending on the choice of assessment method and use of the manufacturer- or ERG-preferred extrapolation. CONCLUSION:Long-term extrapolation of OS is required for NICE STAs based on initial immature OS data. The results of this study demonstrate that the initial OS extrapolations employed by manufacturers and ERGs generally predicted OS reasonably well when compared to more mature data (when available), although on average they appeared to underestimate OS. This review and validation shows that, while the choice of OS extrapolation is uncertain, the methods adopted are generally aligned with later-published follow-up data and appear appropriate for informing HTA decisions.
journal_name
J Med Econjournal_title
Journal of medical economicsauthors
Bullement A,Meng Y,Cooper M,Lee D,Harding TL,O'Regan C,Aguiar-Ibanez Rdoi
10.1080/13696998.2018.1547303subject
Has Abstractpub_date
2019-03-01 00:00:00pages
205-214issue
3eissn
1369-6998issn
1941-837Xjournal_volume
22pub_type
杂志文章,评审abstract:OBJECTIVE:Compare long-term costs and outcomes of lurasidone to aripiprazole among adults with schizophrenia in the US who previously failed ≥1 atypical antipsychotic (olanzapine, risperidone, quetiapine, or ziprasidone) based on an indirect comparison of outcomes data from clinical trials. METHODS:A 5-year Markov coh...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2013.807813
更新日期:2013-07-01 00:00:00
abstract:OBJECTIVE:To assess the economic impact of initial and repeat hospitalizations associated with acute coronary syndrome (ACS) over 1 year (2009). DESIGN AND METHODS:National- and state-level data on length of stay (LOS) and related charges for ACS-associated hospital admissions were assessed using two Healthcare Utiliz...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2014.885907
更新日期:2014-03-01 00:00:00
abstract:AIMS:Improvements in information technology have granted the recent development of rapid, cloud-enabled, onsite laboratory testing for rheumatoid arthritis (RA). This study aims to quantify the value to payers of such technologies. MATERIALS AND METHODS:To calculate the value of rapid, cloud-enabled, onsite laboratory...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2018.1502191
更新日期:2018-11-01 00:00:00
abstract:BACKGROUND:Percutaneous pulmonary valve implantation (PPVI) using the Melody * transcatheter pulmonary valve is a new procedure introduced in 2000 as a less invasive treatment for right ventricular outflow tract (RVOT) dysfunction. The aim of this new procedure is to restore pulmonary valve competence without the need ...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2010.545465
更新日期:2011-01-01 00:00:00
abstract:AIMS:To compare patient characteristics, rates, and costs of medically attended falls among patients with Parkinson's disease (PD) and probable PD plus neurogenic orthostatic hypotension (PD + nOH). MATERIALS AND METHODS:MarketScan Commercial and Medicare Supplemental databases (January 1, 2009-December 31, 2013) were...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2017.1284668
更新日期:2017-05-01 00:00:00
abstract:OBJECTIVE:The objective of the current study was to propose an alternative method for measuring individual operator and peer volumes to use as predictors for adverse outcomes. STUDY DESIGN:A retrospective analysis was performed to assess the volume-outcome relationship for percutaneous coronary intervention (PCI) perf...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696990701815578
更新日期:2008-01-01 00:00:00
abstract:OBJECTIVE:To assess patient characteristics, treatment patterns, and healthcare resource utilization (HRU)/costs of individuals treated for neuroendocrine tumors (NETs) in the US. METHODS:Using a US administrative claims database, this study identified commercially-insured adults newly diagnosed with carcinoid tumors ...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2014.975233
更新日期:2015-02-01 00:00:00
abstract:OBJECTIVES:This study reviewed patient characteristics, management, and medical costs of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDSs) in Spanish hospitals. METHODS:Data were extracted from the Spanish Ministry of Health records via a claims database containing patient records from 192 private and ...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2020.1840180
更新日期:2020-11-02 00:00:00
abstract:OBJECTIVE:The relationship between chronic noncancer pain (CNCP) control and pain medication (analgesic) adherence has not been widely documented. The primary aim of this study was to evaluate the relationship between pain intensity and the degree of adherence to analgesic medication prescribed in pain clinics. There w...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2011.598202
更新日期:2011-01-01 00:00:00
abstract:OBJECTIVE:To compare 1-year costs and benefits of dapagliflozin (DAPA), a sodium-glucose cotransporter-2 (SGLT-2) inhibitor, with those of other treatments for type 2 diabetes (T2D), such as glucagon-like peptide-1 receptor agonists (GLP-1RAs), sulfonylureas (SUs), thiazolidinediones (TZDs), and dipeptidyl peptidase-4 ...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2018.1434182
更新日期:2018-05-01 00:00:00
abstract::Background: IVF is now a wide spread procedure globally, and currently 65 countries report annually all or part of their IVF/ICSI cycles, from which the International Committee Monitoring progress in Assisted Reproduction Technology (ICMART) published its report. There is considerable variation in the utilization (num...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2019.1609484
更新日期:2019-09-01 00:00:00
abstract:AIMS:Fast-acting insulin analogues (FAIAs) reduce hypoglycaemia and improve administration flexibility compared with short-acting human insulin (SHI). This analysis examines whether these benefits translate into cost offsets when comparing the total treatment costs for FAIA versus SHI used as basal-bolus therapy for tr...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2011.588892
更新日期:2011-01-01 00:00:00
abstract::Aims: Due to the lack of studies evaluating compliance or persistence with Alzheimer's Disease (AD) treatment outside High-Income Countries (HICs), this study aimed to assess compliance, persistence, and factors associated with non-compliance and non-persistence by utilizing existing "real-world" information from mult...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2018.1534739
更新日期:2019-01-01 00:00:00
abstract:OBJECTIVE:A new and smaller percutaneous ventricular assist device (pVAD, Impella, Abiomed, Danvers, MA) has been developed to provide circulatory support in hemodynamically unstable patients and to prevent hemodynamic collapse during high-risk percutaneous coronary interventions (PCI). The objective of the study was t...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2012.762004
更新日期:2013-01-01 00:00:00
abstract:BACKGROUND:Patients with unresectable, metastatic colorectal cancer with wild type Kirsten ras mutational status are eligible for sequential treatments which include monoclonal antibodies as first line (1L), second line (2L), or third line (3L) regimens. OBJECTIVE:To compare the economic outcomes of different sequence...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2013.864973
更新日期:2014-02-01 00:00:00
abstract:AIM:Renal failure is a growing public health problem, and is mainly treated by hemodialysis. This study aims to estimate the societal costs of hemodialysis in Lebanon. METHODS:This was a quantitative, cross-sectional cost-of-illness study conducted alongside the Nutrition Education for Management of Osteodystrophy tri...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2016.1207653
更新日期:2016-12-01 00:00:00
abstract:OBJECTIVES:To describe treatment patterns and healthcare burden among individuals with suspected pulmonary arterial hypertension (PAH), as identified through a practice guideline-based healthcare claims algorithm. METHODS:Adults with evidence of PAH from 1 January 2004 (commercial and Medicaid) or 1 July 2006 (Medicar...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2012.690801
更新日期:2012-01-01 00:00:00
abstract:OBJECTIVES:Atazanavir (ATV) and darunavir (DRV) are protease inhibitors approved for HIV treatment in combination with ritonavir (/r). The objectives of this study were to compare persistence (time to treatment discontinuation/modification), adherence, and healthcare costs among patients with human immunodeficiency vir...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2015.1128942
更新日期:2016-01-01 00:00:00
abstract:BACKGROUND:In the last decade, the number of new agents, including monoclonal antibodies, being developed to treat metastatic colorectal cancer (mCRC) increased rapidly. While improving outcomes, these new treatments also have distinct and known safety profiles with toxicities that may require hospitalizations. However...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2011.610394
更新日期:2011-01-01 00:00:00
abstract:AIM:Prescription drug prices in the United States are considered rather extreme. Americans spend over $460 billion on drugs annually, or almost 17 percent of total national healthcare spending. How innovation incentives and insurance coverage drive pricing, diffusion, and utilization of drugs, under conditions of risk ...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2020.1772797
更新日期:2020-09-01 00:00:00
abstract:OBJECTIVE:To carry out a cost-utility analysis comparing initial treatment with solifenacin 5 mg/day vs oxybutynin immediate-release (IR) 15 mg/day for the treatment of patients with overactive bladder (OAB) from the perspective of the U.K. National Health Service (NHS). METHODS:A Markov model with six health states w...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2013.829079
更新日期:2013-10-01 00:00:00
abstract:OBJECTIVE:Duloxetine is indicated for treatment of major depressive disorders in the UK. While clinical trials have documented its clinical effectiveness, little is known regarding the relationship between duloxetine use and healthcare utilization in community practice. This study quantifies the impact of treatment wit...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2012.672941
更新日期:2012-01-01 00:00:00
abstract:OBJECTIVE:To assess the cost-effectiveness of panitumumab in combination with mFOLFOX6 (oxaliplatin, 5-fluorouracil, and leucovorin) vs bevacizumab in combination with mFOLFOX6 as first-line treatment of patients with wild-type RAS metastatic colorectal cancer (mCRC) in Spain. METHODS:A semi-Markov model was developed...
journal_title:Journal of medical economics
pub_type: 杂志文章,随机对照试验
doi:10.1080/13696998.2017.1285780
更新日期:2017-06-01 00:00:00
abstract::Background: The potential impact of disease management to optimize quality of care, health outcomes, and total healthcare costs across a range of cardiac disease states is unknown. Methods: A trial-based cost-utility analysis was conducted alongside a randomized controlled trial of 335 patients with chronic, non-valvu...
journal_title:Journal of medical economics
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1080/13696998.2019.1631831
更新日期:2019-09-01 00:00:00
abstract:OBJECTIVE:This analysis was conducted to compare the direct medical costs of treatment with darbepoetin alfa every 3 weeks (Q3W) and epoetin alfa every week (QW) in patients with chemotherapy-induced anaemia (CIA) from the payer's perspective. METHODS:An analysis was conducted from a US health plan perspective to comp...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696990801959656
更新日期:2008-01-01 00:00:00
abstract:INTRODUCTION:Triple therapy using a protease inhibitor (PI) with peginterferon and ribavirin (PR) is increasingly used in patients with chronic hepatitis C virus (HCV) infection. The most recently introduced PI, simeprevir (SMV), offers high levels of viral eradication combined with a reduced overall duration of therap...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2015.1044457
更新日期:2015-01-01 00:00:00
abstract:OBJECTIVE:To review, summarize, and analyze both similarities and differences of pharmacoeconomic (PE) guidelines, to enable researchers to access their characteristics and the current state of PE guidelines; furthermore, to learn which methodological issues still remain contested and to promote the methodological deve...
journal_title:Journal of medical economics
pub_type: 杂志文章,评审
doi:10.1080/13696998.2017.1387118
更新日期:2018-01-01 00:00:00
abstract:BACKGROUND:Residential step-up/step-down services provide transitional care and reintegration into the community for individuals experiencing episodes of subacute mental illness. This study aims to examine psychiatric inpatient admissions, length of stay, and per capita cost of care following the establishment of a ste...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.1080/13696998.2019.1588126
更新日期:2019-05-01 00:00:00
abstract:OBJECTIVE:Cost-effectiveness analyses of new treatments for cardiovascular disease frequently require input parameters whose values are known with uncertainty due to limited data. The objective of this paper is to examine the extent to which published sensitivity analyses addressing this uncertainty adhere to Health Te...
journal_title:Journal of medical economics
pub_type: 杂志文章,评审
doi:10.3111/13696990903123813
更新日期:2009-06-01 00:00:00
abstract:OBJECTIVE:To assess predictors and costs of multiple sclerosis (MS) relapse, a potential outcome measure in payer-manufacturer risk-sharing agreements for disease-modifying drugs (DMDs). METHODS:A retrospective cohort analysis of medical/pharmacy claims was used. Study patients had ≥1 DMD (interferon beta, glatiramer,...
journal_title:Journal of medical economics
pub_type: 杂志文章
doi:10.3111/13696998.2013.823868
更新日期:2013-09-01 00:00:00