Pharmacoeconomics oncology

OBJECTIVES: Evidentiary requirements for relative effectiveness assessment vary among European health technology assessment (HTA) bodies, affecting the time to HTA decision-making and potentially delaying time to patient access. Improved alignment may reduce this time; therefore, we aim to analyze the differences in evidentiary requirements for oncology drug assessments among European HTA bodies and provide recommendations toward an increased alignment. METHODS: Interviews were conducted with stakeholders in drug assessments of Italy, the Netherlands, Poland, Portugal, England and Wales, and Sweden about evidentiary requirements for several subdomains to identify differences and obtain recommendations for addressing differences...

INTRODUCTION: As a result of an increasing focus on patient-centered care within oncology and more pressure on the sustainability of health-care systems, the discussion on what exactly constitutes value re-appears. Policymakers seek to improve patient values; however, funding all values is not sustainable. AREAS COVERED: We collect available evidence from scientific literature and reflect on the concept of value, the possible incorporation of a wide spectrum of values in reimbursement decisions, and alternative strategies to increase value in oncological care...

Personalized medicine has allowed for knowledge at an individual level for several diseases and this has led to improvements in prevention and treatment of various types of neoplasms. Despite the greater availability of tests, the costs of genomic testing and targeted therapies are still high for most patients, especially in low- and middle-income countries. Although value frameworks and health technology assessment are fundamental to allow decision-making by policymakers, there are several concerns in terms of personalized medicine pharmacoeconomics...

Children's Oncology Group (COG) pharmacists and pharmacy technicians from more than 200 COG-member institutions comprise the COG Pharmacy Discipline. Discipline members serve an essential role in the design and execution of COG clinical trials. Core activities include study drug management, study drug access, clinical trial operations, protocol harmonization, and direct patient care. Discipline members are also actively involved in continuing education, membership engagement, and research across other COG committees/domains...

Patient-reported outcome (PRO) data are critical in understanding treatments from the patient perspective in cancer clinical trials. The potential benefits and methodological approaches to the collection of PRO data following treatment discontinuation (e.g., due to progressive disease or unacceptable drug toxicity) are less clear. In 2020, the FDA's Oncology Center of Excellence (OCE) and the Critical Path Institute (C-Path) co-sponsored a 2-hour virtual roundtable session to discuss this specific issue. In this article, we summarize key points from this discussion with 16 stakeholders representing academia, clinical practice, patients, international regulatory agencies, health technology assessment bodies/payers, industry, and PRO instrument development...

OBJECTIVE: To estimate the incremental health benefits of pharmaceutical innovations approved between 2011-2021 and the share that would surpass the National Institute for Health and Care Excellence (NICE) 'size of benefit' decision weight thresholds. METHODS: We identified all U.S. approved drugs between 2011-2021. Health benefits, in terms of quality-adjusted life-years (QALYs) for each treatment, were extracted from published cost-effectiveness analyses (CEA)...

An increasing interest in the identification of optimal dosage for oncology therapies has prompted key opinion leaders and regulators to encourage the integration of patient-reported outcome (PRO) assessments in phase 1 oncology clinical trials. While the potential benefits of assessing PROs in early-phase studies have been acknowledged, the difficulties that arise from such a radical shift have largely been overlooked in the public discussion. In this commentary, the authors provide insight into the challenges that industry sponsors face in integrating PRO assessments into phase 1 oncology trials, with the ultimate goal of facilitating conversations that may help to resolve some of these issues...

UNLABELLED: Trismus refers to restriction in the ability to open the mouth. Comprehensive evaluation of trismus and its treatment outcomes needs a multidimensional, self-administered, trismus specific tool. In the present scenario, Gothenburg trismus questionnaire is the only reliable instrument to quantify trismus. Translation of this questionnaire helps in providing standardized documentation of trismus related problems and to obtain a patient's perspective on treatment outcomes within various populations...

INTRODUCTION: The objective of this systematic review is to summarize the use of machine learning (ML) in predicting overall survival (OS) in patients with bladder cancer. METHODS: Search terms for bladder cancer, ML algorithms, and mortality were used to identify studies in PubMed and Web of Science as of February 2022. Notable inclusion/exclusion criteria contained the inclusion of studies that utilized patient-level datasets and exclusion of primarily gene expression-related dataset studies...

OBJECTIVES: Therapeutic drug monitoring aims to quantify the concentration of a drug in a biological matrix. In oncology, the therapeutic arsenal is vast and therapeutic drug monitoring optimizes treatment and reduces costs. This review will analyze the financial impact of therapeutic monitoring of anticancer drugs in healthcare institutions. METHODS: Keywords were selected using Decs (MeSH). Through the Pubmed, Scopus, and Virtual Health Library (VHL) databases, 74 articles were found, of which 4 meet the inclusion criteria...

Bladder cancer remains one of the costliest malignancies to manage. We provide a narrative review of literature assessing the economic burden and cost-effectiveness of bladder cancer treatment and surveillance. This is an update to a previous review and focuses on data published within the past 10 years. We queried PubMed and MEDLINE for all bladder cancer cost-related literature between 2013 and 2023. After initial screening, 117 abstracts were identified, 50 of which were selected for inclusion in our review...

OBJECTIVES: Novel therapies improve clinical outcomes in chronic lymphocytic leukemia (CLL), although adverse event (AE) profiles differ. This study evaluated time and personnel costs of AE management among healthcare professionals (HCPs) treating patients with CLL with novel therapies. METHODS: A non-interventional prospective survey was conducted over 2 months. Eligible HCPs reported the time per day spent performing AE management activities for CLL patients treated with acalabrutinib, ibrutinib, or venetoclax...

BACKGROUND: New pharmaceuticals are increasingly being developed for use across multiple indications. Countries across Europe and North America have adopted a range of different approaches to capture differences in the value of individual indications. OBJECTIVE: The three aims of this study were (i) to review the price-setting practice over the past 5 years for multi-indication products across England, France, Italy, Spain, Belgium, Switzerland, Turkey, Canada and the USA; (ii) to assess the impact of current practices on launch strategy; and (iii) to identify issues in the implementation of indication-based pricing...

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OBJECTIVES: Precision medicine is increasingly important in cancer treatment. Tumor-agnostic therapies are used regardless of tumor entity, as they target specific biomarkers in tumors. In Germany, the benefit assessment of oncological pharmaceuticals has traditionally been entity-specific. Thus, the assessment of tumor-agnostic therapies leaves stakeholders with various challenges. Our aim was to systematically identify challenges and possible solutions for the benefit assessment of therapies in tumor-agnostic indications using a two-step sequential qualitative approach...

INTRODUCTION: Willingness to pay (WTP) studies examine the maximum amount of money an individual is willing to pay for a specified health intervention, and can be used to inform coverage and reimbursement decisions. Our objectives were to assess how people value cancer-related interventions, identify differences in the methodologies used, and review the trends in studies' publication. AREAS COVERED: We extracted PubMed and EconLit articles published in 1997-2020 that reported WTP for cancer-related interventions, characterized the methodological differences and summarized each intervention's mean and median WTP values...

BACKGROUND: The administration of chemotherapy positively correlates with diverse adverse drug reactions, including the significant impact of hematological hazards such as anemia, leukopenia-neutropenia, thrombocytopenia, and pancytopenia. This pilot pharmacoeconomic study aimed to estimate the total direct costs of treating hematological toxicity induced by chemotherapy and its main determinants. METHODS: The study was conducted as a retrospective cost of illness study using the "from bottom to the top" approach from the perspective of the Republic Health Insurance Fund...

OBJECTIVES: Network meta-analysis (NMA) of time-to-event outcomes based on constant hazard ratios (HRs) can result in biased findings when the proportional hazards (PH) assumption does not hold in a subset of trials. We aimed to summarize the published non-PH NMA methods for time-to-event outcomes, demonstrate their application, and compare results. METHODS: The following non-PH NMA methods were compared through an illustrative case study in oncology of 4 randomized controlled trials (RCTs) in terms of progression-free survival (PFS) and overall survival (OS): 1) 1-step or 2) 2-step multivariate NMAs based on traditional survival distributions or fractional polynomials (FP), 3) NMAs with restricted cubic splines (RCS) for baseline hazard, and 4) restricted mean survival NMA...

AIM: In January 2017, a new funding model for diagnostic genetic testing in cancer was introduced in Poland. OBJECTIVES: The aim of this study was to assess the impact of changing the funding model for genetic diagnosis in oncology on improving access to personalized medicine in Poland between 2017 and 2019. METHODS: The analysis included data on settlements with the National Health Fund for genetic tests in cancer under a contract of the hospital treatment type and under the contract in the type of separately contracted services between 2017 and 2019...

Objective: This study aimed to (1) translate the PG-SGA into Korean according to a translation guideline, (2) validate the translated version against the Mini-Nutritional Assessment, and (3) determine the prevalence of malnutrition in patients with gastric cancer. Methods: The translation of the PG-SGA was based on the Guidelines for Translation by the International Society for Pharmacoeconomics and Outcomes Research Task Force for Translation and Cultural Adaptation Group...