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1.
Value Health ; 23(9): 1157-1162, 2020 09.
Artigo em Inglês | MEDLINE | ID: mdl-32940233

RESUMO

OBJECTIVES: Despite wide support for patient involvement in health technology assessments (HTA), determining meaningful engagement is complex. This article explores experiences and perceptions among patient groups participating in the Canadian Agency for Drugs and Technologies in Health (CADTH)'s pan-Canadian Oncology Drug Review (pCODR) process. METHODS: We created a qualitative interview study comprising 22 semi-structured telephone interviews with individuals representing 21 different patient groups registered with the pCODR process. The analysis used a qualitative descriptive approach employing techniques from grounded theory. RESULTS: Patient groups view the ability to make submissions to the pCODR process as a meaningful activity closely aligned with organizational priorities. Concurrently, they face substantial resource challenges to prepare submissions, including high opportunity costs and difficulty accessing needed literature and finding relevant patients. Although patient groups felt that CADTH is committed to transparency, they expressed considerable uncertainty around the direct impact of their submissions and desired additional avenues for engagement. CONCLUSIONS: This study suggests a strong commitment by patient groups to participate in the pCODR process despite uncertainty about how their submissions are used to inform HTA recommendations. Identifying opportunities to provide both financial and nonfinancial resources to patient groups is crucial to encouraging and supporting their meaningful participation in HTA processes.


Assuntos
Antineoplásicos/economia , Oncologia/economia , Participação do Paciente , Avaliação da Tecnologia Biomédica/organização & administração , Canadá , Análise Custo-Benefício , Tomada de Decisões , Humanos , Pesquisa Qualitativa
2.
S Afr Med J ; 110(4): 296-301, 2020 Mar 30.
Artigo em Inglês | MEDLINE | ID: mdl-32657741

RESUMO

BACKGROUND: There has been no comprehensive study determining the financial burden of breast cancer in the South African (SA) public sector. OBJECTIVES: To develop a method to determine the cost of breast cancer treatment with chemotherapy per episode of care and to quantify the associated costs relating to chemotherapy at Groote Schuur Hospital (GSH), a government hospital in SA. These costs included costs associated with the management of adverse events arising from chemotherapy. METHODS: Retrospective patient-level data were collected for 200 patients from electronic databases and patient folders between 2013 and 2015. Direct medical costs were determined from the health funder's perspective. The information collected was categorised into the following cost components: chemotherapy medicines, support medicines, administration of chemotherapy, laboratory tests, radiology scans and imaging, doctor consultations and adverse events. Time-and-motion studies were conducted on a set of new patients and the data obtained were used for the study sample of 200 patients. All the above costs were used to determine the cost of chemotherapy per episode of care. The episode of care was defined as the care provided from 2 months prior to the date of commencing chemotherapy (pre-chemotherapy phase), during chemotherapy (treatment phase) and until 6 months after the date when the last cycle of chemotherapy was administered (follow-up phase). RESULTS: A method was developed to determine the episode-of-care costs for breast cancer at GSH. The total direct medical cost for treatment of breast cancer at GSH for 200 patients was ZAR3 154 877, and the average episode-of-care cost per patient was ZAR15 774. The average cost of management of adverse events arising from the various treatment modalities was ZAR13 133 per patient. It was found that the cost of treating a patient with adverse events was 1.8 times higher than the cost of treating a patient without adverse events. Of the patients, 86.5% managed to complete their prescribed chemotherapy treatment cycles, and the average cost of treatment of these patients was 1.3 times more than the average cost for patients who could not complete their treatment, based on the number of treatment cycles received. CONCLUSION: A comprehensive method to determine the costs associated with breast cancer management per episode of care was developed, and costs were quantified at GSH according to the treatment protocol used at the hospital.


Assuntos
Antineoplásicos/economia , Carcinoma de Mama in situ/tratamento farmacológico , Neoplasias da Mama/tratamento farmacológico , Carcinoma Ductal de Mama/tratamento farmacológico , Carcinoma Lobular/tratamento farmacológico , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/economia , Custos de Cuidados de Saúde/estatística & dados numéricos , Hospitais Públicos/economia , Adulto , Idoso , Carcinoma de Mama in situ/economia , Carcinoma de Mama in situ/patologia , Neoplasias da Mama/economia , Neoplasias da Mama/patologia , Carcinoma Ductal de Mama/economia , Carcinoma Ductal de Mama/patologia , Carcinoma Lobular/economia , Carcinoma Lobular/patologia , Quimioterapia Adjuvante/economia , Técnicas de Laboratório Clínico/economia , Diagnóstico por Imagem/economia , Custos de Medicamentos/estatística & dados numéricos , Cuidado Periódico , Feminino , Humanos , Pessoa de Meia-Idade , Terapia Neoadjuvante/economia , Cuidados Paliativos/economia , Honorários por Prescrição de Medicamentos/estatística & dados numéricos , Encaminhamento e Consulta/economia , Estudos Retrospectivos , África do Sul , Estudos de Tempo e Movimento , Adulto Jovem
3.
PLoS One ; 15(7): e0236426, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32716969

RESUMO

BACKGROUND: For stage IV lung cancer patients receiving add-on Viscum album L. (VA) treatment an improved overall survival was detected. Information regarding cost-effectiveness (CE) for comparisons between chemotherapy (CTx) and CTx plus additive VA in stage IV lung cancer treatment is limited. The present study assessed the costs and cost-effectiveness of CTx plus VA (V) compared to CTx alone (C) for stage IV non-small cell lung cancer (NSCLC) patients treatment in a hospital in Germany. METHODS: In the observational real-world data study, data from the Network Oncology clinical registry were utilized. Enrolled stage IV lung cancer patients received the respective therapy (C or V) in a certified German Cancer Center. Cost and cost-effectiveness analyses from the hospital's perspective were investigated on the basis of overall survival (OS) and routine financial controlling data. In addition, the incremental cost-effectiveness ratio (ICER) was calculated. The primary result of the analysis was tested for robustness in a bootstrap-based sensitivity analysis. RESULTS: 118 patients (C: n = 86, V: n = 32) were included in the analysis, mean age 63.8 years, the proportion of male patients was 55.1%. Adjusted hospital's total mean costs for patients from the C and V group were €16,289, 95%CI: 13,834€-18,744€ (over an adjusted mean OS time of 13.4 months) and €17,992, 95%CI: 13,658-22,326 (over an adjusted mean OS time of 19.1 months), respectively. The costs per additional OS year gained (ICER) with the V-therapy compared to C therapy were €3,586. CONCLUSION: The findings of the present study suggest that the combined use of chemotherapy and VA was clinically effective and comparably cost-effective to chemotherapy alone in our analysed patient sample from the hospital's perspective. Further randomized and prospective cost-effectiveness studies are necessary to complement our findings.


Assuntos
Antineoplásicos/administração & dosagem , Antineoplásicos/economia , Carcinoma Pulmonar de Células não Pequenas/tratamento farmacológico , Carcinoma Pulmonar de Células não Pequenas/economia , Análise Custo-Benefício , Neoplasias Pulmonares/tratamento farmacológico , Extratos Vegetais/administração & dosagem , Viscum album/química , Antineoplásicos/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Carcinoma Pulmonar de Células não Pequenas/patologia , Economia Hospitalar , Feminino , Humanos , Tempo de Internação , Neoplasias Pulmonares/patologia , Masculino , Pessoa de Meia-Idade , Análise Multivariada , Estadiamento de Neoplasias , Extratos Vegetais/economia , Extratos Vegetais/uso terapêutico , Análise de Sobrevida
4.
Biochim Biophys Acta Rev Cancer ; 1874(1): 188382, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32522600

RESUMO

Liver cancer is highly malignant and insensitive to cytotoxic chemotherapy and is associated with very poor patient prognosis. In 2007, the small-molecule targeted drug sorafenib was approved for the treatment of advanced liver cancer. In the subsequent ten years, sorafenib has been the only first-line therapeutic targeted drug for advanced hepatocellular carcinoma (HCC). However, a number of clinical studies show that a considerable percentage of patients with liver cancer are insensitive to sorafenib. The number of patients who actually benefit significantly from sorafenib treatment is very limited, and the overall efficacy of sorafenib is far from satisfactory, which has attracted the attention of researchers. Based on previous studies and reports, this article reviews the potential mechanisms of sorafenib resistance (SR) and summarizes the biomarkers and clinicopathological indicators that might be used for predicting sorafenib response and developing personalized therapy.


Assuntos
Antineoplásicos/farmacologia , Carcinoma Hepatocelular/tratamento farmacológico , Resistencia a Medicamentos Antineoplásicos/genética , Neoplasias Hepáticas/tratamento farmacológico , Medicina de Precisão , Sorafenibe/farmacologia , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Biomarcadores Tumorais/genética , Carcinoma Hepatocelular/economia , Carcinoma Hepatocelular/genética , Carcinoma Hepatocelular/mortalidade , Análise Custo-Benefício , Progressão da Doença , Humanos , Fígado/patologia , Neoplasias Hepáticas/economia , Neoplasias Hepáticas/genética , Neoplasias Hepáticas/mortalidade , Taxa de Depuração Metabólica/genética , Estadiamento de Neoplasias , Seleção de Pacientes , Prognóstico , Ensaios Clínicos Controlados Aleatórios como Assunto , Sorafenibe/economia , Sorafenibe/uso terapêutico , Análise de Sobrevida , Resultado do Tratamento , Microambiente Tumoral/efeitos dos fármacos , Microambiente Tumoral/genética
5.
BMC Health Serv Res ; 20(1): 424, 2020 May 14.
Artigo em Inglês | MEDLINE | ID: mdl-32410676

RESUMO

BACKGROUND: More alternatives have become available for the diagnosis and treatment of cancer in low- and middle-income countries. Because of increasing demands, governments are now facing a problem of limited affordability and availability of essential cancer medicines. Yet, precise information about the access to these medicines is limited, and the methodology is not very well developed. We assessed the availability and affordability of essential cancer medicines in Mexico, and compared their prices against those in other countries of the region. METHODS: We surveyed 21 public hospitals and 19 private pharmacies in 8 states of Mexico. Data were collected on the availability and prices of 49 essential cancer medicines. Prices were compared against those in Chile, Peru, Brazil, Colombia and PAHO's Strategic Fund. RESULTS: Of the various medicines, mean availability in public and private sector outlets was 61.2 and 67.5%, respectively. In the public sector, medicines covered by the public health insurance "People's Health Insurance" were more available. Only seven (public sector) and five (private sector) out of the 49 medicines were considered affordable. Public sector procurement prices were 41% lower than in other countries of the region. CONCLUSIONS: The availability of essential cancer medicines, in the public and private sector, falls below World Health Organization's 80% target. The affordability remains suboptimal as well. A national health insurance scheme could serve as a mechanism to improve access to cancer medicines in the public sector. Comprehensive pricing policies are warranted to improve the affordability of cancer medicines in the private sector.


Assuntos
Antineoplásicos/economia , Antineoplásicos/provisão & distribução , Medicamentos Essenciais/economia , Medicamentos Essenciais/provisão & distribução , Neoplasias/tratamento farmacológico , Comércio/estatística & dados numéricos , Custos e Análise de Custo/estatística & dados numéricos , Hospitais Públicos , Humanos , México , Farmácias , Setor Privado , Setor Público , Inquéritos e Questionários
6.
Lancet Oncol ; 21(5): 664-670, 2020 05.
Artigo em Inglês | MEDLINE | ID: mdl-32359489

RESUMO

BACKGROUND: Increasing cancer drug prices are a challenge for patients and health systems in the USA and Europe. By contrast with the USA, national authorities in European countries often directly negotiate drug prices with manufacturers. The American Society of Clinical Oncology (ASCO) and the European Society for Medical Oncology (ESMO) developed frameworks to evaluate the clinical value of cancer therapies: the ASCO-Value Framework (ASCO-VF) and the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS). We aimed to assess the association between the clinical benefit of approved cancer drugs based on these frameworks and their drug prices in the USA and four European countries (England, Switzerland, Germany, and France). METHODS: For this cost-benefit analysis, we identified all new drugs with initial indications for adult cancers that were approved by the US Food and Drug Administration between Jan 1, 2009, and Dec 31, 2017, and by the European Medicines Agency up until Sept 1, 2019. For drugs indicated for solid tumours, we assessed clinical benefit using ASCO-VF and ESMO-MCBS. We compared monthly drug treatment costs between benefit levels using hierarchical linear regression models, and calculated Spearman's correlation coefficients between costs and benefit levels for individual countries. FINDINGS: Our cohort included 65 drugs: 47 (72%) drugs were approved for solid tumours and 18 (28%) were approved for haematological malignancies. The monthly drug treatment costs in the USA were a median of 2·31 times (IQR 1·79-3·17) as high as in the assessed European countries. There were no significant associations between monthly treatment costs for solid tumours and clinical benefit in all assessed countries, using the ESMO-MCBS (p=0·16 for the USA, p=0·98 for England, p=0·54 for Switzerland, p=0·52 for Germany, and p=0·40 for France), and for all assessed countries except France using ASCO-VF (p=0·56 for the USA, p=0·47 for England, p=0·26 for Switzerland, p=0·23 for Germany, and p=0·037 for France). INTERPRETATION: Cancer drugs with low or uncertain clinical benefit might be prioritised for price negotiations. Value frameworks could help identify therapies providing high clinical benefit that should be made rapidly available across countries. FUNDING: Swiss Cancer Research Foundation (Krebsforschung Schweiz).


Assuntos
Análise Custo-Benefício , Custos de Medicamentos , Oncologia/economia , Neoplasias/economia , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Inglaterra/epidemiologia , Europa (Continente)/epidemiologia , França/epidemiologia , Alemanha/epidemiologia , Humanos , Neoplasias/epidemiologia , Estados Unidos/epidemiologia
7.
Am J Clin Oncol ; 43(7): 517-525, 2020 07.
Artigo em Inglês | MEDLINE | ID: mdl-32304434

RESUMO

OBJECTIVE: To examine the association among tyrosine kinase inhibitor (TKI) out-of-pocket costs, adherence, and health care costs and utilization in a large group of commercially insured patients with chronic myeloid leukemia (CML). MATERIALS AND METHODS: Patients with CML aged 18 to 64 years were identified using IBM MarketScan Commercial Database between April 1, 2011 and December 31, 2014. Patients were required to be continuously enrolled 3 months before and 12 months after TKI (imatinib, dasatinib, or nilotinib) initiation. TKI adherence is estimated using the proportion of days covered (PDC), defined as the percentage of the PDC by the prescription fill during the 12-month study period (adherent patients have PDC ≥80%). Health care cost differences between adherent and nonadherent patients were estimated using generalized linear models. Health care utilization was compared using negative binomial regression models. All models were controlled for potential confounding factors. RESULTS: The study sample consisted of 863 patients, where 355 (41.1%) patients were classified as adherent. Over the study period, nonadherent patients incurred US$10,974 more in medical costs (P<0.001), and US$1663 more in non-TKI pharmacy costs (P<0.01). Adherent patients incurred US$28,184 more in TKI pharmacy costs (P<0.001) that resulted in US$18,305 more in overall total health care costs (P<0.001). Adherent patients, however, were estimated to be less likely to have all-cause hospitalizations (incidence rate ratio, 0.32; P<0.001), or CML-specific hospitalizations (incidence rate ratio, 0.31; P<0.01). CONCLUSIONS: Patients with CML with better adherence experienced fewer hospitalizations, resulting in medical service cost savings. These lower medical costs, however, were more than offset by higher TKI medication costs observed during the first year of TKI therapy.


Assuntos
Custos de Cuidados de Saúde , Leucemia Mielogênica Crônica BCR-ABL Positiva/tratamento farmacológico , Aceitação pelo Paciente de Cuidados de Saúde , Inibidores de Proteínas Quinases/economia , Inibidores de Proteínas Quinases/uso terapêutico , Adolescente , Adulto , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Dasatinibe/economia , Dasatinibe/uso terapêutico , Feminino , Humanos , Mesilato de Imatinib/economia , Mesilato de Imatinib/uso terapêutico , Revisão da Utilização de Seguros , Masculino , Adesão à Medicação/estatística & dados numéricos , Pessoa de Meia-Idade , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Pirimidinas/economia , Pirimidinas/uso terapêutico , Estudos Retrospectivos , Adulto Jovem
8.
Value Health ; 23(4): 441-450, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32327161

RESUMO

OBJECTIVES: In the field of relapsed or refractory multiple myeloma (RRMM), between-trial or indirect comparisons are required to estimate relative treatment effects between competing interventions based on the available evidence. Two approaches are frequently used in RRMM: network meta-analysis (NMA) and unanchored matching-adjusted indirect comparison (MAIC). The objective of the current study was to evaluate the relevance and credibility of published NMA and unanchored MAIC studies aiming to estimate the comparative efficacy of treatment options for RRMM. METHODS: Twelve relevant studies were identified in the published literature (n = 7) and from health technology assessment agencies (n = 5). Data from trials were extracted to identify between-trial differences that may have biased results. Credibility of the performed analyses and relevance of the research questions were critically appraised using the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) checklist and feedback based on consultations with clinical experts. RESULTS: The identified studies concerned NMAs of randomized controlled trials (RCTs; n = 7), unanchored MAICs (n = 4), or both types of analyses (n = 1). According to clinical expert consultation, the majority of the identified NMAs did not consider differences in prior therapies or treatment duration across the RCTs included in the analyses, thereby compromising the relevance. CONCLUSION: Based on the results and feedback from clinicians, the majority of NMAs did not consider prior treatment history or treatment duration, which resulted in nonrelevant comparisons. Furthermore, it may have compromised the credibility of the estimates owing to differences in effect-modifiers between the different trials. Pairwise comparisons by means of unanchored MAICs require clear justification given the reliance on non-randomized comparisons.


Assuntos
Antineoplásicos/administração & dosagem , Mieloma Múltiplo/tratamento farmacológico , Avaliação de Resultados em Cuidados de Saúde , Antineoplásicos/economia , Farmacoeconomia , Humanos , Mieloma Múltiplo/economia , Metanálise em Rede , Ensaios Clínicos Controlados Aleatórios como Assunto , Avaliação da Tecnologia Biomédica
9.
Value Health ; 23(4): 471-480, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32327164

RESUMO

OBJECTIVES: Our goal was to estimate the relative importance assigned to health technology assessment (HTA) criteria by stakeholders involved in the HTA process. HTA is an increasingly common framework used in the appraisal of drugs for public reimbursement. It identifies clinical, economic, social, and organizational criteria to be considered. The criteria can vary across jurisdictions and are typically appraised by multidisciplinary expert committees. Guidance on the relative weighing of criteria is often absent. METHODS: We elicited stakeholders' preferences using a single-scenario discrete choice experiment and a best-worst scaling model with conviction scores to assess the weights assigned to selected criteria by HTA stakeholders. We recruited 111 HTA stakeholders across multiple jurisdictions, including members of expert committees, clinical and economic experts, patients, and public payer representatives. Each judged twelve hypothetical cancer drug profiles for suitability for public funding and identified which characteristics were best and worst. In addition to standard discrete choice experiment and best-worst scaling models, we estimated a hybrid model to obtain a ranking of criteria by importance they played in the appraisal. RESULTS: A strong clinical benefit proved the most important criterion, followed by cost considerations, presence of adverse events, and availability of other treatments. The importance of clinical benefit was moderated by unmet need, adverse events, and number of patients. CONCLUSION: Policymakers might want to consider providing an explicit weighing scheme, or moving to a 2-stage selection process with an assessment of the quality of clinical evidence as a gatekeeping step for a full HTA review.


Assuntos
Antineoplásicos/administração & dosagem , Neoplasias/tratamento farmacológico , Mecanismo de Reembolso , Avaliação da Tecnologia Biomédica/métodos , Antineoplásicos/efeitos adversos , Antineoplásicos/economia , Comportamento de Escolha , Humanos , Neoplasias/economia , Projetos Piloto
11.
Adv Ther ; 37(5): 2116-2126, 2020 05.
Artigo em Inglês | MEDLINE | ID: mdl-32193809

RESUMO

INTRODUCTION: The purpose of this study was to estimate the cost-effectiveness of atezolizumab plus chemotherapy in patients with metastatic non-squamous non-small cell lung cancer (NSCLC) from the United States (US) payers' perspective in the first-line treatment. METHODS: A mathematical Markov model was developed to estimate cost and effectiveness of atezolizumab combination therapy versus carboplatin plus nab-paclitaxel alone in the first-line therapy of metastatic non-squamous NSCLC from the data of IMpower130. Costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) were analyzed, and model robustness was assessed by sensitivity analysis. Additional subgroup analyses were performed as well. RESULTS: Compared to chemotherapy, treatment with atezolizumab plus chemotherapy yields an increase of 0.16 QALYs with an increase in cost of $109,809.13, resulting in an ICER of $670,309.66 per QALY. The most influential factor in this model was the cost of atezolizumab. Probabilistic sensitivity analysis showed that there was 0% probability that atezolizumab plus chemotherapy was cost-effective at willingness-to-pay (WTP) values of $150,000 per QALY. The results of subgroup analyses showed that the ICER remained greater than $150,000/QALY across the all patient subgroups. CONCLUSION: First-line treatment with atezolizumab in combination with carboplatin plus nab-paclitaxel is not a cost-effective option in patients with metastatic non-squamous NSCLC.


Assuntos
Anticorpos Monoclonais Humanizados/economia , Antineoplásicos/economia , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Carcinoma Pulmonar de Células não Pequenas/tratamento farmacológico , Análise Custo-Benefício/estatística & dados numéricos , Neoplasias Pulmonares/tratamento farmacológico , Metástase Neoplásica/tratamento farmacológico , Idoso , Idoso de 80 Anos ou mais , Anticorpos Monoclonais Humanizados/uso terapêutico , Antineoplásicos/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Carcinoma Pulmonar de Células não Pequenas/epidemiologia , Estudos de Coortes , Feminino , Humanos , Neoplasias Pulmonares/epidemiologia , Masculino , Cadeias de Markov , Anos de Vida Ajustados por Qualidade de Vida , Estados Unidos/epidemiologia
12.
Value Health ; 23(3): 319-327, 2020 03.
Artigo em Inglês | MEDLINE | ID: mdl-32197727

RESUMO

OBJECTIVE: To assess whether using surrogate versus patient-relevant endpoints in pivotal trials of cancer drugs was associated with health technology assessment recommendations in England (National Institute for Health and Care Excellence [NICE]) and Canada (Canadian Agency for Drugs and Technologies in Health [CADTH]). METHODS: Cancer drug approvals from 2012 to 2016 were categorized by demonstrating benefit on overall survival (OS), progression-free survival, disease response, or having no comparator. Approvals were analyzed by benefit category and health technology assessment recommendation. The association between benefit (surrogate vs OS) and recommending a drug was examined using descriptive statistics and linear probability models controlling for unmet need, orphan designation, and cost-effectiveness. RESULTS: Of 42 cancer indications that NICE recommended, 15 (36%) demonstrated OS benefit. Cancer indications with OS benefit were less likely to receive a recommendation from NICE than those without (P = .04). In linear probability models, availability of OS benefit was no longer associated with a recommendation from NICE (P = .32). Cost-effective cancer drugs had a 55.6% (95% CI: 38.9%-72.3%) higher probability of receiving a recommendation from NICE than those that were not. In Canada, 15 of 37 (41%) cancer indications that were recommended showed OS benefit. There was no detectable association between surrogate measures and CADTH recommendations based on descriptive statistics (P = .62) or in linear probability models (P = .73). CONCLUSION: When cost-effectiveness was considered, pivotal trial endpoints were not associated with NICE recommendations. Pivotal trial endpoints, unmet need, orphan status, and cost-effectiveness did not explain CADTH recommendations.


Assuntos
Antineoplásicos/uso terapêutico , Ensaios Clínicos como Assunto , Aprovação de Drogas , Determinação de Ponto Final , Neoplasias/tratamento farmacológico , Projetos de Pesquisa , Avaliação da Tecnologia Biomédica , Antineoplásicos/efeitos adversos , Antineoplásicos/economia , Canadá , Análise Custo-Benefício , Técnicas de Apoio para a Decisão , Aprovação de Drogas/economia , Custos de Medicamentos , Humanos , Modelos Econômicos , Neoplasias/economia , Neoplasias/mortalidade , Formulação de Políticas , Intervalo Livre de Progressão , Estudos Retrospectivos , Avaliação da Tecnologia Biomédica/economia , Fatores de Tempo , Reino Unido
13.
Eur J Cancer ; 129: 23-31, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32120272

RESUMO

OBJECTIVE: Health-related quality of life (HRQoL) is one of the most important patient-relevant study end-points for the direct measurement of the benefit of cancer drugs. Therefore, our aim is to detect cancer indications with no published information on HRQoL at the time of European Medicines Agency (EMA) approval and monitor any reported HRQoL evidence updates after at least three years of follow-up. METHODS: We included all cancer indications that were approved by the EMA between January 2009 and October 2015. Our main sources of information were the EMA website, clinicaltrials.gov and a systematic literature search in PubMed. Information on HRQoL outcomes was extracted alongside evidence on median overall survival. RESULTS: In total, we identified 110 indications, of which more than half (n = 58, 53%) were lacking available information on HRQoL assessments at the time of EMA approval. After a monitoring period of at least three years, 24 updates were identified, resulting in 34 (31%) therapies where information on HRQoL was still not available. For the 76 therapies with reported information on HRQoL, cancer-specific instruments were mostly used (n = 49/76). Regarding cumulative evidence on median overall survival and HRQoL, 33 (n = 33/110, 30%) as well as 15 (n = 15/110, 14%) cancer drugs were lacking information on both study end-points at the time of approval and after monitoring, respectively. CONCLUSION: Our results demonstrate that there is an urgent need of routine re-evaluation of reimbursed cancer drugs with initially missing information on major outcomes. Standardisation of the typology and quality of HRQoL assessments need to be improved to allow better comparability of results.


Assuntos
Antineoplásicos/uso terapêutico , Aprovação de Drogas/legislação & jurisprudência , União Europeia/organização & administração , Neoplasias/tratamento farmacológico , Qualidade de Vida , Antineoplásicos/economia , Ensaios Clínicos como Assunto , Aprovação de Drogas/organização & administração , Custos de Medicamentos/legislação & jurisprudência , Europa (Continente)/epidemiologia , Medicina Baseada em Evidências/economia , Medicina Baseada em Evidências/legislação & jurisprudência , Seguimentos , Humanos , Oncologia/economia , Oncologia/legislação & jurisprudência , Neoplasias/complicações , Neoplasias/economia , Neoplasias/mortalidade , Mecanismo de Reembolso/legislação & jurisprudência , Análise de Sobrevida , Resultado do Tratamento
14.
Clin Trials ; 17(2): 119-125, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32114790

RESUMO

BACKGROUND: Pivotal clinical trials provide critical evidence to regulators regarding a product's suitability for marketing approval. The objectives of this study are (1) to characterize select features of trials for oncology products approved by the U.S. Food and Drug Administration between 2015 and 2017; and (2) to quantify the costs of these trials and how such costs varied based on trial characteristics. METHODS: We identified novel oncology therapeutic drugs, and their respective pivotal trials, approved between 2015 and 2017 using annual summary reports from the Food and Drug Administration. Cost estimates for each pivotal trial were calculated using IQVIA's CostPro, a clinical trial cost estimating tool based on executed contracts between pharmaceutical manufacturers and contract research organizations. Measures of drug and trial characteristics included trial design, end point, patient enrollment, and regulatory pathway. We also performed sensitivity analyses that varied assumptions regarding how efficiently each trial was conducted. RESULTS: A total of 39 pivotal clinical trials provided the basis for Food and Drug Administration approval of 30 new oncology drugs from 2015 to 2017. Among these trials, primary end points were objective response rate in 20 (51.3%), progression-free survival in 13 (33.3%), and overall survival in 6 (15.4%). Twenty trials (51.3%) were single-arm studies. The median estimated cost of oncology pivotal trials was $31.7 million (interquartile range = $17.0-$60.4 million). Trials with objective response rate as primary end point had a median estimate of $17.7 million (interquartile range = $11.9-$27.1 million), compared with trials examining progression-free survival ($42.3 million, interquartile range = $34.6-$101.2 million) or overall survival ($79.4 million, interquartile range = $56.9-$97.7 million) (p < 0.001). Estimated costs for single-arm trials ($17.7 million, interquartile range = $11.9-$23.7 million) were less than for trials with a placebo-controlled ($56.7 million, interquartile range = $40.9-$103.9 million) or active control arm ($67.6 million, IQR = $35.5-$93.5 million) (p < 0.001). CONCLUSIONS: Relative to the estimated costs of drug development, the costs of these oncology pivotal trials were modest, with trials that produced more valuable scientific information costing more than their counterparts.


Assuntos
Antineoplásicos/uso terapêutico , Ensaios Clínicos como Assunto/economia , Neoplasias/tratamento farmacológico , United States Food and Drug Administration , Antineoplásicos/economia , Custos e Análise de Custo , Aprovação de Drogas/economia , Humanos , Estados Unidos
15.
J Manag Care Spec Pharm ; 26(2): 186-196, 2020 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-32011965

RESUMO

BACKGROUND: Oral oncolytic therapies have improved survival in hematologic cancers, such as chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and multiple myeloma (MM), which are now being managed like chronic conditions. However, compared with other cancers, there is a lack of studies assessing adherence, health care resource utilization, and costs in patients with these cancers. OBJECTIVE: To assess factors associated with adherence to oral oncolytic therapies, health care utilization, and costs in patients with CLL/SLL or MM. METHODS: A retrospective database study was conducted using the IBM MarketScan Commercial Claims and Medicare Supplement databases. Adults (aged ≥ 18 years) diagnosed with and prescribed an oral oncolytic for CLL/SLL (ibrutinib or idelalisib) or MM (thalidomide, lenalidomide, or pomalidomide) between 2013 and 2016 and with continuous eligibility 6 months before and 12 months after oral oncolytic initiation were identified. Adherence to oral oncolytics was measured using the proportion of days covered (PDC) metric. Multiple linear regression and multivariable logistic regression were used to identify adherence predictors. Count models assessed the relationship between adherence and resource utilization, and generalized linear models assessed the relationship between adherence and health care costs. RESULTS: A total of 701 and 2,385 patients were identified with CLL/SLL or MM, respectively. Mean PDC (SD) for CLL/SLL and MM patients was 75.3 (22.5) and 57.6 (26.5), respectively. For CLL/SLL patients, those aged ≥ 65 years (beta [B] = -4.00) had lower medication use. Among MM patients, multiple predictors of higher medication use emerged: aged ≥ 65 years (B = 3.44), higher than average outpatient resource utilization (B = 3.53), insurance plan other than preferred provider organization (PPO; B = -2.58), previous cancer therapy (B = -2.81), higher number of concurrent unique therapeutic classes (B = -0.35), and higher comorbidity burden (B = -2.55). Patients with CLL/SLL and enrolled in plans other than a PPO were more likely to be adherent (OR = 1.41, 95% CI = 1.01-1.98), whereas patients who were aged ≥ 65 years, were residents of the southern United States, and had visited the emergency department in the baseline period were less likely to be adherent. For MM patients, those aged ≥ 65 years (OR = 1.68, 95% CI = 1.38-2.04) and with higher than average outpatient services utilization (OR = 1.24, 95% CI = 1.01-1.52) were more likely to be adherent, whereas those enrolled in plans other than a PPO, previously treated with cancer therapy, and with higher comorbidity burden were less likely to be adherent. In both cohorts, adherent patients had significantly lower odds of health care utilization and incurred lower medical costs, but higher prescription costs, following oncolytic initiation; however, total costs were not significantly lower in those adherent. CONCLUSIONS: Factors were identified that influenced adherence at the patient, treatment, and health system levels. These factors can be used to identify patients requiring interventions for improving medication-taking behavior and associated health care burden. DISCLOSURES: This study received no outside funding. Dashputre was recently employed by Novartis; K. Gatwood has received speaker fees from Jazz Pharmaceuticals; and J. Gatwood has received research funding from Merck & Co. and GlaxoSmithKline, unrelated to this study..


Assuntos
Antineoplásicos/administração & dosagem , Leucemia Linfocítica Crônica de Células B/tratamento farmacológico , Adesão à Medicação/estatística & dados numéricos , Mieloma Múltiplo/tratamento farmacológico , Administração Oral , Adolescente , Adulto , Idoso , Antineoplásicos/economia , Bases de Dados Factuais , Feminino , Custos de Cuidados de Saúde/estatística & dados numéricos , Humanos , Leucemia Linfocítica Crônica de Células B/economia , Masculino , Pessoa de Meia-Idade , Mieloma Múltiplo/economia , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Estudos Retrospectivos , Adulto Jovem
18.
Value Health ; 23(1): 52-60, 2020 01.
Artigo em Inglês | MEDLINE | ID: mdl-31952674

RESUMO

BACKGROUND: Many high cost treatments for advanced melanoma have become available in recent years. National health technology assessment agencies have raised concerns regarding uncertainty in their clinical and cost-effectiveness. OBJECTIVE: The aim of this systematic review is to identify economic evaluations of treatments for advanced melanoma and review model assumptions, outcomes, and quality as preparation for a health technology assessment. METHODS: A search of Embase, MEDLINE, EconLit, and the Cochrane Database was conducted. Only studies using decision-analytic models were included. Two authors independently completed full-text review and data extraction. RESULTS: Fifteen studies were identified. There were major differences in the structural assumptions underpinning the models. There was general agreement in study conclusions, although the predicted costs and quality-adjusted life years for each treatment varied. BRAF monotherapy (vemurafenib, dabrafenib) or BRAF/MEK combination therapy (BRAF monotherapy with cobimetinib or trametinib) has not been shown to be cost-effective in any jurisdiction. PD-1 inhibitors (pembrolizumab, nivolumab) are consistently found to be cost-effective compared with ipilimumab, although their cost-effectiveness compared with chemotherapy is not established. Combination therapy with nivolumab and ipilimumab is unlikely to be cost-effective in any setting. One study including all agents found that none of the new treatments were cost-effective relative to chemotherapy. Publication of the study in a health economics journal is associated with better reporting of and higher-quality assessment than those published in clinical journals. CONCLUSION: Despite differences in model structures and assumptions, the conclusions of most included studies were consistent. Health technology assessment has a key role in maximizing value from high-cost innovative treatments. Consideration should be given to divestment from BRAF/MEK inhibitors and ipilimumab in favor of reimbursement of PD-1 inhibitors.


Assuntos
Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Custos de Medicamentos , Alocação de Recursos para a Atenção à Saúde/economia , Política de Saúde/economia , Melanoma/tratamento farmacológico , Melanoma/economia , Neoplasias Cutâneas/tratamento farmacológico , Neoplasias Cutâneas/economia , Avaliação da Tecnologia Biomédica/economia , Antineoplásicos/efeitos adversos , Análise Custo-Benefício , Humanos , Melanoma/patologia , Modelos Econômicos , Terapia de Alvo Molecular/economia , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Neoplasias Cutâneas/patologia , Fatores de Tempo , Resultado do Tratamento
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