RESUMEN
BACKGROUND: Criticisms have emerged that cancer medicines offer modest benefits at increasingly high prices. Reimbursement decisions made by health technology assessment (HTA) agencies have become a complex endeavour for cancer medicines. Most high-income countries (HICs) use HTA criteria to identify high-value medicines for reimbursement under public drug coverage plans. We compared HTA criteria specific for cancer medicines in economically similar HICs, to understand how these criteria contribute to reimbursement decisions. METHODS: We did an international, cross-sectional analysis in collaboration with author investigators across eight HICs, from the Group of Seven (known as G7; Canada, England, France, Germany, Italy, and Japan) and Oceania (Australia and New Zealand). Publicly available data from HTA agency reports and official documentation were extracted and analysed between Aug 15, 2021, and July 31, 2022. We collected data pertaining to the decision-making criteria used by the national HTA agency; HTA reimbursement status for 34 medicine-indication pairs corresponding to 15 unique US top-selling cancer medicines; and HTA reimbursement status for 18 cancer medicine-indication pairs (13 unique medicines) with minimal clinical benefit (score of 1 on the European Society of Medical Oncology Magnitude of Clinical Benefit Scale). Descriptive statistics were used to compare HTA decision criteria and drug reimbursement recommendations (or for Germany and Japan, final reimbursement status) across the eight countries. FINDINGS: Therapeutic impact related to clinical outcomes of the new medicine was a uniform criterion across the eight countries, whereas quality of evidence (under the remit of therapeutic impact assessment) and equity were infrequently cited criteria. Only the German HTA agency mandated that surrogate endpoints be validated in therapeutic impact assessment. All countries except Germany included formal cost-effectiveness analyses within HTA reports. England and Japan were the only countries that specified a cost-effectiveness threshold. Of the 34 medicine-indication pairs corresponding to US top-selling cancer medicines, Germany reimbursed the maximum (34 [100%]), followed by Italy (32 [94%] recommended for reimbursement), Japan (28 [82%] reimbursed), Australia, Canada, England, and France (27 [79%] recommended for reimbursement), and New Zealand (12 [35%] recommended for reimbursement). Of the 18 cancer medicine-indication pairs with marginal clinical benefit, Germany reimbursed 15 (83%) and Japan reimbursed 12 (67%). France recommended nine (50%) for reimbursement, followed by Italy (seven [39%]), Canada (five [28%]), and Australia and England (three [17%] each). New Zealand did not recommend any medicine-indications with marginal clinical benefit for reimbursement. Considering the overall cumulative proportion across the eight countries, 58 (21%) of 272 indications for the US top-selling medicines and 90 (63%) of 144 marginally beneficial medicine-indications were not recommended for reimbursement or reimbursed. INTERPRETATION: Our findings indicate discordance in public reimbursement decisions across economically similar countries, despite overlapping HTA decision criteria. This suggests a need for improved transparency around the nuances of the criteria to ensure improved access to high-value cancer medicines, and deprioritisation of low-value cancer medicines. Health systems have opportunities to improve their HTA decision-making processes by learning from the systems in other countries. FUNDING: None.
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Neoplasias , Evaluación de la Tecnología Biomédica , Humanos , Estudios Transversales , Francia , Neoplasias/tratamiento farmacológico , OceaníaRESUMEN
OBJECTIVES: Precision oncology is generating vast amounts of multiomic data to improve human health and accelerate research. Existing clinical study designs and attendant data are unable to provide comparative evidence for economic evaluations. This lack of evidence can cause inconsistent and inappropriate reimbursement. Our study defines a core data set to facilitate economic evaluations of precision oncology. METHODS: We conducted a literature review of economic evaluations of next-generation sequencing technologies, a common application of precision oncology, published between 2005 and 2018 and indexed in PubMed (MEDLINE). Based on this review, we developed a preliminary core data set for informal expert feedback. We then used a modified-Delphi approach with individuals involved in implementation and evaluation of precision medicine, including 2 survey rounds followed by a final voting conference to refine the data set. RESULTS: Two authors determined that variation in published data elements was reached after abstraction of 20 economic evaluations. Expert consultation refined the data set to 83 unique data elements, and a multidisciplinary sample of 46 experts participated in the modified-Delphi process. A total of 68 elements (81%) were selected as required, spanning demographics and clinical characteristics, genomic data, cancer treatment, health and quality of life outcomes, and resource use. CONCLUSIONS: Cost-effectiveness analyses will fail to reflect the real-world impacts of precision oncology without data to accurately characterize patient care trajectories and outcomes. Data collection in accordance with the proposed core data set will promote standardization and enable the generation of decision-grade evidence to inform reimbursement.
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Neoplasias , Análisis Costo-Beneficio , Humanos , Neoplasias/genética , Neoplasias/terapia , Medicina de Precisión , Calidad de Vida , Encuestas y CuestionariosRESUMEN
BACKGROUND: In Canada, the Canadian Agency for Drugs and Technologies in Health (CADTH) evaluates and makes recommendations for the reimbursement of cancer drugs. One component of its recommendation is based on an economic evaluation, which typically takes the form of a cost-utility analysis. A cost-utility analysis measures the effects of competing therapies with quality-adjusted life-years (QALYs). The data for this calculation typically come from generic, preference-based measures of health-related quality of life (HRQOL). The objective of this review is to determine the frequency at which HRQOL data are collected alongside cancer drug trials and used in the cost-utility analysis submitted to the CADTH pan-Canadian Oncology Drug Review (pCODR). METHODS: Submissions between 2015 and 2018 to pCODR, the group charged with evaluating cancer drug submissions at CADTH, were reviewed. All pCODR submissions, either in progress or completed, were publicly available online. The search was restricted to completed evaluations. RESULTS: Forty-three submissions met the inclusion criteria. The incremental gain in QALYs in most submissions from the new technology was small (median incremental gain, 0.86; interquartile range, 0.6-1.39). More than half of the submissions (56%) did not include original data on HRQOL, with most relying on previous studies of variable relevance and quality. Re-analyses by pCODR based on concerns over HRQOL data used in the submitted model were common (52%). CONCLUSIONS: Drug manufacturers do not consistently collect data on HRQOL alongside clinical trials and instead rely on evidence generated in previous studies to inform cost-utility analyses. These findings should induce manufacturers to collect original HRQOL data that are simultaneously relevant to patients and decision makers.
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Antineoplásicos/economía , Oncología Médica/economía , Neoplasias/tratamiento farmacológico , Neoplasias/economía , Antineoplásicos/uso terapéutico , Canadá/epidemiología , Análisis Costo-Beneficio/economía , Toma de Decisiones , Costos de los Medicamentos/estadística & datos numéricos , Humanos , Neoplasias/epidemiologíaRESUMEN
Inhaled corticosteroids (ICSs) are often prescribed in patients with chronic obstructive pulmonary disease (COPD). Their impact on the risk of lung cancer, a leading cause of mortality in COPD patients, remains uncertain.Population-based linked administrative data between the years 1997 and 2007 from the province of British Columbia, Canada, were used to evaluate the association between lung cancer risk and ICS use in COPD patients. COPD was defined on the basis of receipt of three COPD-related prescriptions in subjects ≥50â years of age. Exposure to ICS was incorporated into multivariable Cox regression models using several time-dependent methods ("ever" exposure, cumulative duration of use, cumulative dose, weighted cumulative duration of use and weighted cumulative dose).There were 39â676 patients who met the inclusion criteria. The mean±sd age of the cohort was 70.7±11.1â years and 53% were female. There were 994 (2.5%) cases of lung cancer during follow-up. In the reference case analysis (time-dependent "ever" exposure), ICS exposure was associated with a 30% reduced risk of lung cancer (HR 0.70 (95% CI 0.61-0.80)). ICS exposure was associated with a decrease in the risk of lung cancer diagnosis over all five methods of quantifying exposure.This population-based study suggests that ICS use reduces the risk of lung cancer in COPD patients.
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Corticoesteroides/administración & dosificación , Neoplasias Pulmonares/epidemiología , Enfermedad Pulmonar Obstructiva Crónica/complicaciones , Enfermedad Pulmonar Obstructiva Crónica/tratamiento farmacológico , Administración por Inhalación , Anciano , Anciano de 80 o más Años , Colombia Británica/epidemiología , Estudios de Cohortes , Femenino , Humanos , Neoplasias Pulmonares/prevención & control , Masculino , Persona de Mediana Edad , Análisis Multivariante , Modelos de Riesgos Proporcionales , Medición de Riesgo , Factores de RiesgoRESUMEN
Expenditure on cancer therapies is rising rapidly in many countries, particularly for cancer drugs. In recent years, this has stimulated a global debate among the public, patients, clinicians, decision-makers, and the pharmaceutical industry on value, affordability, and sustainability propositions relating to cancer therapies. In this article, we discuss some recent developments in evidence-based approaches to priority setting and resource allocation in Canadian cancer systems. These developments include new methods for deliberative public engagement, generating and using real-world evidence, multi-criteria decision analysis, and handling uncertainty with evidence for gene therapies.
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Medicina Basada en la Evidencia , Financiación de la Atención de la Salud , Oncología Médica/economía , Canadá , Análisis Costo-Beneficio , Toma de Decisiones , Técnicas de Apoyo para la Decisión , Costos de la Atención en Salud , Política de Salud , Prioridades en Salud/economía , Humanos , Oncología Médica/organización & administración , Neoplasias/terapia , Formulación de Políticas , Asignación de Recursos/economía , Asignación de Recursos/métodosRESUMEN
Chronic obstructive pulmonary disease is a common, preventable and treatable disease. Exercise training programmes (ETPs) improve symptoms, health-related quality of life (HRQoL) and exercise capacity, but the optimal setting is unknown. In this review, we compared the effects of ETPs in different settings on HRQoL and exercise capacity. We searched (5 July 2016) the Cochrane Airways Group Specialised Register, ClinicalTrials.gov and World Health Organization trials portal. We selected studies, extracted data and assessed risk of bias with two independent reviewers. We calculated mean differences (MD) with 95% CI. We assessed the quality of evidence using Grades of Recommendation, Assessment, Development and Evaluation. Ten trials (934 participants) were included. Hospital (outpatient) and home-based ETPs (seven trials) were equally effective at improving HRQoL on the Chronic Respiratory Questionnaire (CRQ) (dyspnoea: MD -0.09, 95% CI: -0.28 to 0.10; fatigue: MD -0.00, 95% CI: -0.18 to 0.17; emotional: MD 0.10, 95% CI: -0.24 to 0.45; and mastery: MD -0.02, 95% CI: -0.28 to 0.25; moderate quality) and on the St George's Respiratory Questionnaire (SGRQ) (MD -0.82, 95% CI: -7.47 to 5.83, low quality). Hospital (outpatient) and community-based ETPs (three trials) were equally effective at improving HRQoL (CRQ dyspnoea: MD 0.29, 95% CI: -0.05 to 0.62, moderate quality; fatigue: MD -0.02, 95% CI: -1.09 to 1.05, low quality; emotional: MD 0.10, 95% CI: -0.40 to 0.59, moderate quality; and mastery: MD -0.08, 95% CI: -0.45 to 0.28, moderate quality). There was no difference in exercise capacity. There was low to moderate evidence that outpatient and home-based ETPs are equally effective. See related Editorial.
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Atención Ambulatoria/métodos , Terapia por Ejercicio/métodos , Servicios de Atención de Salud a Domicilio , Pacientes Ambulatorios , Evaluación de Programas y Proyectos de Salud , Enfermedad Pulmonar Obstructiva Crónica/rehabilitación , Humanos , Enfermedad Pulmonar Obstructiva Crónica/fisiopatología , Calidad de VidaRESUMEN
Purpose: Patients with coronary heart disease (CHD) experience reduced quality of life which may be associated with mortality in the longer term. This study explores whether patient-rated physical and mental health status was associated with mortality at 6-year follow-up among patients with CHD attending primary care in Ireland and Northern Ireland. Methods: This study is a secondary data analysis of patients with CHD recruited to a cluster randomized controlled trial from 2004 to 2010. Data collected included patient-rated physical component summary (PCS) and mental component summary (MCS) scores of health status (from the 12-Item Short-Form Health Survey (SF-12)), demographics and clinical parameters at baseline, and all-cause mortality at 6-year follow-up. Multivariate regression was conducted using generalized estimating equations (GEE) with a log-link function. Results are presented as odds ratios (ORs) and 95% confidence intervals (CIs). Results: The study consisted of 762 individuals with mean age 67.6 years [standard deviation (SD): 9.8], and was 29% female. Mean baseline SF-12 mental (MCS) and physical (PCS) component scores were 50.0 (SD: 10.8) and 39.6 (SD: 11.2), respectively. At 6-year follow-up, the adjusted OR for the baseline MCS for mortality was 0.97 (95% CI: 0.95-0.99) and for the PCS 0.97 (95% CI: 0.95-0.99). For every five-point increase in MCS and PCS scores, there was a 14% reduction in the likelihood of all-cause mortality. Conclusions: Overall, the magnitude of effect for both mental health status and physical health status was similar; higher scores were significantly associated with a lower risk of mortality at 6-year follow-up.
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Enfermedad Coronaria/mortalidad , Estado de Salud , Calidad de Vida , Anciano , Causas de Muerte , Enfermedad Coronaria/fisiopatología , Enfermedad Coronaria/psicología , Femenino , Estudios de Seguimiento , Humanos , Irlanda/epidemiología , Masculino , Salud Mental , Persona de Mediana Edad , Análisis Multivariante , Irlanda del Norte/epidemiología , Análisis de Regresión , Autoinforme , Factores de TiempoRESUMEN
This Viewpoint discusses the flawed assumptions and potential negative impacts of a proposed federal bill that would ban government health care programs from using the quality-adjusted life-year (QALY) and "similar measures" when determining insurance coverage or negotiating prices.
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Gobierno Federal , Años de Vida Ajustados por Calidad de Vida , Estados Unidos , Legislación como AsuntoRESUMEN
Inhaled corticosteroids (ICS) are commonly prescribed to COPD patients, particularly those with more advanced stages of the disease. These patients are also at increased risk of lung cancer. A systematic review was undertaken to identify studies that examined the association between lung cancer risk and ICS therapy in COPD patients. The search strategy was created in MEDLINE and extended to EMBASE as well as other relevant databases. Both randomized controlled trials (RCTs) and observational studies were considered for inclusion. Studies were required to have incident lung cancer or deaths from lung cancer as an outcome in order to be included in the review. Six studies met the inclusion criteria. Two observational studies directly addressed the specific research. Four RCTs presented sufficient data to calculate the relative risk of lung cancer in COPD patients. None of the identified RCTs showed a statistically significant association of ICS use with lung cancer risk. Observational studies showed a protective effect from ICS use, particularly at high doses. Given the observational evidence and the low numbers of lung cancer events in the RCTs, these results may be prone to type II error. The observational studies dealt with very specific patient populations and exposure definitions, which might not have adequately captured the complex relationship between ICS exposure and lung cancer risk. Results from RCTs suggest no effect of ICS on the risk of lung cancer. However, results from observational studies suggest the potential that ICS may confer a protective effect, particularly at high doses.
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Glucocorticoides/farmacología , Neoplasias Pulmonares/epidemiología , Enfermedad Pulmonar Obstructiva Crónica , Administración por Inhalación , Humanos , Gravedad del Paciente , Enfermedad Pulmonar Obstructiva Crónica/diagnóstico , Enfermedad Pulmonar Obstructiva Crónica/tratamiento farmacológico , Enfermedad Pulmonar Obstructiva Crónica/epidemiología , Medición de RiesgoRESUMEN
OBJECTIVES: This study examines the cost-effectiveness of the OPTI-SCRIPT intervention on potentially inappropriate prescribing in primary care. METHODS: Economic evaluation, using incremental cost-effectiveness and cost utility analyses, conducted alongside a cluster randomized controlled trial of twenty-one general practices and 196 patients, to compare a multifaceted intervention with usual practice in primary care in Ireland. Potentially inappropriate prescriptions (PIPs) were determined by a pharmacist. Incremental costs, PIPs, and quality-adjusted life-years (QALYs) at 12-month follow-up were estimated using multilevel regression. Uncertainty was explored using cost-effectiveness acceptability curves. RESULTS: The intervention was associated with a nonsignificant mean cost increase of 407 (95 percent CIs, -357-1170), a significant mean reduction in PIPs of 0.379 (95 percent CI, 0.092-0.666), and a nonsignificant mean increase in QALYs of 0.013 (95 percent CIs, -0.016-0.042). The incremental cost per PIP avoided was 1,269 (95 percent CI, -1400-6302) and the incremental cost per QALY gained was 30,535 (95 percent CI, -334,846-289,498). The probability of the intervention being cost-effective was 0.602 at a threshold value of 45,000 per QALY gained and was at least 0.845 at threshold values of 2,500 per PIP avoided and higher. CONCLUSIONS: While the OPTI-SCRIPT intervention was effective in reducing potentially inappropriate prescribing in primary care in Ireland, our findings highlight the uncertainty with respect to its cost-effectiveness. Further studies are required to explore the health and economic implications of interventions targeting potentially inappropriate prescribing.
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Prescripción Inadecuada/economía , Prescripción Inadecuada/prevención & control , Pautas de la Práctica en Medicina/organización & administración , Atención Primaria de Salud/organización & administración , Anciano , Anciano de 80 o más Años , Algoritmos , Análisis Costo-Beneficio , Femenino , Humanos , Irlanda , Masculino , Administración del Tratamiento Farmacológico/organización & administración , Pautas de la Práctica en Medicina/economía , Atención Primaria de Salud/economía , Años de Vida Ajustados por Calidad de VidaRESUMEN
BACKGROUND: Changes to physiological parameters precede deterioration of ill patients. Early warning and track and trigger systems (TTS) use routine physiological measurements with pre-specified thresholds to identify deteriorating patients and trigger appropriate and timely escalation of care. Patients presenting to the emergency department (ED) are undiagnosed, undifferentiated and of varying acuity, yet the effectiveness and cost-effectiveness of using early warning systems and TTS in this setting is unclear. We aimed to systematically review the evidence on the use, development/validation, clinical effectiveness and cost-effectiveness of physiologically based early warning systems and TTS for the detection of deterioration in adult patients presenting to EDs. METHODS: We searched for any study design in scientific databases and grey literature resources up to March 2016. Two reviewers independently screened results and conducted quality assessment. One reviewer extracted data with independent verification of 50% by a second reviewer. Only information available in English was included. Due to the heterogeneity of reporting across studies, results were synthesised narratively and in evidence tables. RESULTS: We identified 6397 citations of which 47 studies and 1 clinical trial registration were included. Although early warning systems are increasingly used in EDs, compliance varies. One non-randomised controlled trial found that using an early warning system in the ED may lead to a change in patient management but may not reduce adverse events; however, this is uncertain, considering the very low quality of evidence. Twenty-eight different early warning systems were developed/validated in 36 studies. There is relatively good evidence on the predictive ability of certain early warning systems on mortality and ICU/hospital admission. No health economic data were identified. CONCLUSIONS: Early warning systems seem to predict adverse outcomes in adult patients of varying acuity presenting to the ED but there is a lack of high quality comparative studies to examine the effect of using early warning systems on patient outcomes. Such studies should include health economics assessments.
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Deterioro Clínico , Servicio de Urgencia en Hospital , Monitoreo Fisiológico/métodos , Humanos , Índice de Severidad de la Enfermedad , TriajeAsunto(s)
Neoplasias Pulmonares , Enfermedad Pulmonar Obstructiva Crónica , Corticoesteroides , Sesgo , Humanos , IncidenciaRESUMEN
Importance: New gene therapies can offer substantial benefits to patients, particularly those with rare diseases who have few therapeutic options. In May 2023, the US Food and Drug Administration (FDA) approved the first topical gene therapy, beremagene geperpavec (B-VEC), for treating both autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa (DEB). However, FDA approval was based on limited data in patients with autosomal dominant disease, even though they comprise approximately 50% of all DEB cases. Objective: To estimate projected spending in the US on B-VEC therapy for treating autosomal recessive and autosomal dominant DEB. Design, Setting, and Participants: This economic evaluation used data from the National Epidermolysis Bullosa Registry to estimate the current population of US patients with autosomal dominant and autosomal recessive DEB, with the aim of estimating US spending on B-VEC therapy from an all-payers perspective during 1- and 3-year periods after FDA approval. A base-case cost of $300â¯000 per patient per year was assumed based on a report from the manufacturer (Krystal Biotech). Exposure: Treatment with B-VEC. Main Outcomes and Measures: Estimated overall spending on B-VEC in the first year and over a 3-year period after FDA approval. Several prespecified sensitivity analyses with different assumptions about the eligible patient population and the cost of therapy were performed, and lifetime total costs of treatment per patient were estimated. Results: The estimated number of US patients with DEB who were eligible for treatment with B-VEC in the first year after FDA approval was 894. The estimated total expenditure for B-VEC therapy was $268 million (range, $179 million-$357 million). Over a 3-year period, estimated spending was $805 million (range, $537 million-$1.1 billion). Estimated lifetime total costs per patient were $15 million (range, $10 million-$20 million) per patient with autosomal recessive DEB and $17 million (range, $11 million-$22 million) for patients with autosomal dominant DEB. Conclusions and Relevance: Results of this economic evaluation suggest that the FDA's broad indication for the use of B-VEC in treating both autosomal recessive and autosomal dominant DEB will have significant implications for payers.
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Epidermólisis Ampollosa Distrófica , Epidermólisis Ampollosa , Humanos , Epidermólisis Ampollosa Distrófica/tratamiento farmacológico , Epidermólisis Ampollosa Distrófica/genética , Epidermólisis Ampollosa/genética , Análisis Costo-BeneficioRESUMEN
Importance: The US lacks a systematic approach for aligning drug prices with clinical benefit, and traditional cost-effectiveness analysis (CEA) faces political obstacles. The efficiency frontier (EF) method offers policymakers an alternative approach. Objective: To assess how the EF approach could align prices and clinical benefits of biologic medications for plaque psoriasis and estimate price reductions in the US vs 4 peer countries: Australia, Canada, France, and Germany. Design and Setting: This health economic evaluation used the EF approach to compare the prices and clinical benefits of 11 biologics and 2 biosimilars for plaque psoriasis in the US, Australia, Canada, France, and Germany. Data were collected from February to March 2023 and analyzed from March to June 2023. Main Outcome Measures: EFs were constructed based on each biologic's efficacy, measured using the Psoriasis Area and Severity Index (PASI) 90 response rate, and annual treatment cost as of January 2023; US costs were net of estimated manufacturer rebates. Prices based on the EF were compared with traditional CEA-based prices calculated by the Institute for Clinical and Economic Review at a threshold of $150â¯000 per quality-adjusted life-year gained. Results: Among 13 biologics, PASI 90 response rates ranged from 17.9% (etanercept) to 71.6% (risankizumab); US net annual treatment costs ranged from $1664 (infliximab-dyyb) to $79â¯277 (risankizumab). The median (IQR) net annual treatment cost was higher in the US ($34â¯965 [$20â¯493-$48â¯942]) than prerebate costs in Australia ($9179 [$6691-$12â¯688]), Canada ($15â¯556 [$13â¯017-$16â¯112]), France ($9478 [$6637-$11â¯678]), and Germany ($13â¯829 [$13â¯231-$15â¯837]). The US EF included infliximab-dyyb (PASI 90: 57.4%; annual cost: $1664), ixekizumab (PASI 90: 70.8%; annual cost: $33â¯004), and risankizumab (PASI 90: 71.6%; annual cost: $79â¯277). US prices for psoriasis biologics would need to be reduced by a median (IQR) of 71% (31%-95%) to align with those estimated using the EF; the same approach would yield smaller price reductions in Canada (41% [6%-57%]), Australia (36% [0%-65%]), France (19% [0%-67%]), and Germany (11% [8%-26%]). Except for risankizumab, the EF-based prices were lower than the prices based on traditional CEA. Conclusions and Relevance: This economic evaluation showed that for plaque psoriasis biologics, using an EF approach to negotiate prices could lead to substantial price reductions and better align prices with clinical benefits. US policymakers might consider using EFs to achieve prices commensurate with comparative clinical benefits, particularly for drug classes with multiple therapeutic alternatives for which differences can be adequately summarized by a single outcome measurement.
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Biosimilares Farmacéuticos , Psoriasis , Humanos , Infliximab/uso terapéutico , Biosimilares Farmacéuticos/uso terapéutico , Etanercept/uso terapéutico , Factores Biológicos/uso terapéutico , Psoriasis/tratamiento farmacológico , Psoriasis/economía , Terapia BiológicaRESUMEN
Costly targeted cancer treatments challenge publicly-funded healthcare systems seeking to align expected benefit with value for money. In 2021, The Canadian Agency for Drugs and Technologies in Health (CADTH) published a provisional funding algorithm for risk-based treatment of chronic lymphocytic leukemia (CLL). We estimate the cost-effectiveness of this algorithm against current standard of care. We constructed a probabilistic Markov model comparing next generation sequencing (NGS) assay-guided front-line treatment of acalabrutinib versus venetoclax with obinutuzumab to a comparator wherein patients initiate acalabrutinib. The primary outcome was the incremental cost-effectiveness ratio (ICER) per quality-adjusted life-year (QALY) gained. Analyses were conducted from the British Columbia healthcare system perspective, with outcomes discounted at 1.5%. Assay informed treatment for patients with CLL resulted in an incremental cost effectiveness ratio of $18,040 (95% CI $16,491-$19,501) per quality adjusted life-year (QALY) gained. The probability of the NGS guided treatment algorithm being cost effective was 80% at a willingness to pay threshold of $50,000 and a corresponding ICER of $18,040. Assay-guided treatment sequencing adds additional costs to healthcare but may be a cost-effective intervention for adult patients with CLL. Integration of real-world evidence would improve the validity and reliability of model estimated for decision-makers.
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Análisis Costo-Beneficio , Secuenciación de Nucleótidos de Alto Rendimiento , Leucemia Linfocítica Crónica de Células B , Años de Vida Ajustados por Calidad de Vida , Leucemia Linfocítica Crónica de Células B/tratamiento farmacológico , Leucemia Linfocítica Crónica de Células B/economía , Leucemia Linfocítica Crónica de Células B/genética , Humanos , Secuenciación de Nucleótidos de Alto Rendimiento/economía , Secuenciación de Nucleótidos de Alto Rendimiento/métodos , Compuestos Bicíclicos Heterocíclicos con Puentes/uso terapéutico , Compuestos Bicíclicos Heterocíclicos con Puentes/economía , Sulfonamidas/economía , Sulfonamidas/uso terapéutico , Benzamidas/uso terapéutico , Benzamidas/economía , Anticuerpos Monoclonales Humanizados/economía , Anticuerpos Monoclonales Humanizados/uso terapéutico , Cadenas de Markov , Pirazinas/economía , Pirazinas/uso terapéutico , Algoritmos , Análisis de Costo-EfectividadRESUMEN
Drug repurposing faces various challenges that can impede its success. We developed a framework outlining key challenges in drug repurposing to explore when and how health technology assessment (HTA) methods can address them. We identified 20 drug-repurposing challenges across the categories of data access, research and development, collaboration, business case, regulatory and legal challenges. Early incorporation of HTA methods, including literature review, empirical research, stakeholder consultation, health economic evaluation and uncertainty assessment, can help to address these challenges. HTA methods canassess the value proposition of repurposed drugs, inform further research and ultimately help to bring cost-effective repurposed drugs to patients.
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Reposicionamiento de Medicamentos , Evaluación de la Tecnología Biomédica , Reposicionamiento de Medicamentos/métodos , Evaluación de la Tecnología Biomédica/métodos , Humanos , Análisis Costo-BeneficioRESUMEN
BACKGROUND: Early laparoscopic cholecystectomy for acute cholecystitis is safe and effective. However, the potential cost savings of this management strategy have not been well studied in a North American context. This study aimed to estimate the cost effectiveness of early laparoscopic cholecystectomy versus delayed laparoscopic cholecystectomy in Canada. METHODS: A decision analytic model estimating and comparing costs from a Canadian providing institution after either early or delayed laparoscopic cholecystectomy was used. The health care resources consumed were calculated using local hospital data, and outcomes were measured in quality-adjusted life years (QALYs) gained during 1 year. Uncertainty was investigated with one-way sensitivity analyses, varying the probabilities of the events and utilities. RESULTS: Early laparoscopic cholecystectomy was estimated to cost approximately $2,000 (Canadian dollars) less than delayed laparoscopic cholecystectomy per patient, with an incremental gain of approximately 0.03 QALYs. Sensitivity analysis showed that only extreme values of bile duct injury or bile leak altered the direction of incremental gain. CONCLUSIONS: Adoption of a policy in favor of early laparoscopic cholecystectomy will result in better patient quality of life and substantial savings to the Canadian health care system.
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Colecistectomía Laparoscópica/economía , Colecistitis Aguda/economía , Colecistitis Aguda/cirugía , Canadá , Análisis Costo-Beneficio , Árboles de Decisión , Estado de Salud , Humanos , Modelos Económicos , Calidad de Vida , Años de Vida Ajustados por Calidad de Vida , Tiempo de Tratamiento , Resultado del TratamientoRESUMEN
The European Commission's proposal to address antimicrobial resistance using transferable exclusivity vouchers (TEVs) is fundamentally flawed. European policymakers and regulators should consider alternatives, such as better funding for basic and clinical research, use of advance market commitments funded by a pay-or-play tax, or enacting an EU Fund for Antibiotic Development.
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Antiinfecciosos , Motivación , Humanos , Unión Europea , Antibacterianos/farmacologíaRESUMEN
Chimeric antigen receptor (CAR) T-cells are a cellular immunotherapy with remarkable efficacy in treating multiple hematologic malignancies but they are associated with extremely high prices that are, for many countries, prohibitively expensive. As their use increases both for hematologic malignancies and other indications, and large numbers of new cellular therapies are developed, novel approaches will be needed both to reduce the cost of therapy, and to pay for them. We review the many factors that lead to the high cost of CAR T-cells and offer proposals for reform.
Asunto(s)
Neoplasias Hematológicas , Receptores Quiméricos de Antígenos , Humanos , Receptores Quiméricos de Antígenos/genética , Neoplasias Hematológicas/terapia , Inmunoterapia , Linfocitos TRESUMEN
INTRODUCTION: The legalization of recreational cannabis use can enable cancer survivors to manage aspects of their care with cannabinoids without medical authorization or stigmatization. However, the absence of medical guidance-from the scientific literature or the healthcare system-makes it difficult for survivors to reach informed decisions about their care. OBJECTIVE: This article outlines the qualitative research undertaken to design a discrete choice experiment (DCE) aimed at understanding Canadian cancer survivors' preferences for managing their cancer symptoms with cannabis in this complex socio-medical context. METHODS: In this study, we drew on previously published qualitative research (a literature review and interviews with cancer survivors) and the theory of planned behavior, holding weekly team meetings to review the qualitative data and identify initial attributes associated with medicinal cannabis consumption to inform the DCE design. The initial attributes were further assessed to determine whether they were sensitive to the Canadian context, modifiable to produce levels and trade-offs, and amenable to policy intervention, in order to form the DCE choice sets. The choice sets were tested via think-aloud exercises with members of the general population and included debriefing interviews. Think-aloud participants were recruited from patient groups and previous studies. RESULTS: Based on our review of the interview study, we identified the following attributes associated with selecting medicinal cannabis: effectiveness; chance of side effects; support from family, friends, and/or physicians; cost; and availability. Ability to perform everyday activities was added and monthly out-of-pocket cost was refined to render the DCE realistic to cancer survivors in the Canadian context. Revisions to the DCE instructions, terminology, and cost levels were made based on results from the think-aloud exercises (n = 10). CONCLUSIONS: This qualitative study outlines the preference evidence collected regarding Canadian cancer survivors' decisions to manage their symptoms with cannabis to inform a DCE quantitative survey. It contributes to transparent reporting of qualitative work in DCE development and to understanding cancer survivors' preferences regarding medicinal cannabis consumption under legalization.