RESUMO
There is growing interest globally in using real-world data (RWD) and real-world evidence (RWE) for health technology assessment (HTA). Optimal collection, analysis, and use of RWD/RWE to inform HTA requires a conceptual framework to standardize processes and ensure consistency. However, such framework is currently lacking in Asia, a region that is likely to benefit from RWD/RWE for at least two reasons. First, there is often limited Asian representation in clinical trials unless specifically conducted in Asian populations, and RWD may help to fill the evidence gap. Second, in a few Asian health systems, reimbursement decisions are not made at market entry; thus, allowing RWD/RWE to be collected to give more certainty about the effectiveness of technologies in the local setting and inform their appropriate use. Furthermore, an alignment of RWD/RWE policies across Asia would equip decision makers with context-relevant evidence, and improve timely patient access to new technologies. Using data collected from eleven health systems in Asia, this paper provides a review of the current landscape of RWD/RWE in Asia to inform HTA and explores a way forward to align policies within the region. This paper concludes with a proposal to establish an international collaboration among academics and HTA agencies in the region: the REAL World Data In ASia for HEalth Technology Assessment in Reimbursement (REALISE) working group, which seeks to develop a non-binding guidance document on the use of RWD/RWE to inform HTA for decision making in Asia.
Assuntos
Medicina Baseada em Evidências , Mecanismo de Reembolso , Avaliação da Tecnologia Biomédica , Ásia , Análise Custo-Benefício , Confiabilidade dos Dados , Tomada de Decisões , Inquéritos e Questionários , TelecomunicaçõesRESUMO
BACKGROUND: Breast cancer is the leading malignancy among Filipino women, with about 23.50% of cases characterized by human epidermal growth factor receptor-2 (HER2) overexpression. Trastuzumab, in addition to standard chemotherapy, is currently recommended as primary treatment for HER2-positive early-stage breast cancer (EBC) in the adjuvant settings, and has been listed in the Philippine National Formulary (PNF) since 2008, but with no current evidence yet on its value for money, to date. Hence, despite several policy enablers, its accessibility remains to be limited in the Philippines. We performed an economic evaluation to assess the cost-effectiveness and budget impact of adjuvant trastuzumab therapy for HER2-positive EBC in the Philippines, using healthcare system and societal perspectives, in aid of guiding coverage decisions. METHODS: A Markov model-based cost-utility and budget impact analyses were conducted to estimate the total costs incurred and outcomes gained in using 1 year of adjuvant trastuzumab added to standard chemotherapy versus standard chemotherapy alone, over a lifetime horizon. We discounted both costs and outcomes at 3.5% per annum. Parameters were estimated using country survival data, systematic review and meta-analysis of the relative treatment effect, local and international cost data, and published utility data. Univariate and probabilistic sensitivity analyses were used to account for parameter uncertainty. RESULTS: Trastuzumab therapy was dominated with an incremental cost-effectiveness ratio (ICER) at PHP 453,505 per QALY gained from a healthcare system perspective or PHP 458,686 per QALY gained from a societal perspective, with 10% cost-effectiveness probability at the country cost-effectiveness threshold of PHP 120,000 per QALY gained. National implementation will cost an additional amount of PHP 13,909 million in year one alone, plus about PHP 2000 to 3000 million annually for the succeeding fiscal years. CONCLUSION: At its current cost, 1 year of adjuvant trastuzumab therapy compared to standard chemotherapy alone for HER2-positive EBC does not represent value for money in the Philippines. Its current cost will have to significantly lower down by one-half to achieve cost-effectiveness.
Assuntos
Antineoplásicos Imunológicos/economia , Neoplasias da Mama/tratamento farmacológico , Quimioterapia Adjuvante/economia , Trastuzumab/economia , Anticorpos Monoclonais Humanizados/economia , Antineoplásicos/uso terapêutico , Antineoplásicos Imunológicos/uso terapêutico , Neoplasias da Mama/economia , Terapia Combinada , Análise Custo-Benefício , Custos de Medicamentos , Feminino , Humanos , Cadeias de Markov , Pessoa de Meia-Idade , Filipinas , Anos de Vida Ajustados por Qualidade de Vida , Receptor ErbB-2 , Trastuzumab/uso terapêuticoRESUMO
BACKGROUND: Cervical cancer is the second leading cause of cancer cases and deaths among Filipino women because of inadequate access to screening and treatment services. This study aims to evaluate the health and economic benefits of HPV vaccination and its combination with different screening strategies to find the most optimal preventive strategy in the Philippines. METHODS: A cost-utility analysis was conducted using an existing semi-Markov model to evaluate different screening (i.e., Pap smear, visual inspection with acetic acid) and vaccination strategies against HPV infection implemented alone or as part of a combination strategy at different coverage scenarios. The model was run using country-specific epidemiologic, cost and clinical parameters from a health system perspective. Sensitivity analysis was performed for vaccine efficacy, duration of protection and costs of vaccination, screening and treatment. RESULTS: Across all coverage scenarios, VIA has been shown to be a dominant and cost-saving screening strategy with incremental cost-effectiveness ratio (ICER) ranging from dominant to Php 61,059 (1443 USD) per QALY gained. VIA can reduce cervical cancer cases and deaths by 25%. Pap smear screening was found to be not cost-effective due to its high cost in the Philippines. Adding HPV vaccination at a cost of 54 USD per vaccinated girl on top of VIA screening was found to be potentially cost-effective using a threshold of 1 GDP per capita (i.e., Php 120,000 or 2835 USD/ QALY) with the most favorable assumption of providing lifelong immunity against high-risk oncogenic HPV types 16/18. The highest incremental QALY gain was achieved with 80% coverage of the combined strategy of VIA at 35 to 45 years old done every five years following vaccination at 11 years of age with an ICER of Php 33,126 (783 USD). This strategy may result in a two-thirds reduction in cervical cancer burden. HPV vaccination is not cost-effective when vaccine protection lasts for less than 20 years. CONCLUSION: High VIA coverage targeting women aged 35-45 years old at five-year intervals is the most efficient and cost-saving strategy in reducing cervical cancer burden in the Philippines. Adding a vaccination program at high coverage among 11-year-old girls is potentially cost-effective in the Philippines assuming a life-long duration of vaccine efficacy.
Assuntos
Infecções por Papillomavirus/economia , Infecções por Papillomavirus/prevenção & controle , Vacinas contra Papillomavirus/economia , Neoplasias do Colo do Útero/economia , Neoplasias do Colo do Útero/prevenção & controle , Vacinação/economia , Adolescente , Adulto , Análise Custo-Benefício , Detecção Precoce de Câncer/economia , Feminino , Humanos , Programas de Assistência Gerenciada/economia , Pessoa de Meia-Idade , Teste de Papanicolaou/economia , Vacinas contra Papillomavirus/administração & dosagem , Filipinas/etnologia , Vacinação/estatística & dados numéricosRESUMO
INTRODUCTION: As the availability of new economic evaluations (EE) on adjuvant trastuzumab therapy for early-stage breast cancer (EBC) with HER2-positive since last search and other EEs missed warrant a more extensive review, this study aimed to systematically review EEs of adjuvant trastuzumab compared with chemotherapy alone for HER2-positive EBC. AREA COVERED: The search was performed in February 2019 using MEDLINE and Scopus. Reviewers independently selected studies based on eligibility criteria, extracted data, assessed quality of reporting, and appraised quality of data sources. EXPERT OPINION: 22 studies were included which were from high-income (HICs) and upper-middle income countries (UMICs). Incremental cost-effectiveness ratios (ICERs) from HICs were within their cost-effectiveness thresholds and ranged from 6,018 to 78,929 USD per quality-adjusted life year (QALY) gained. ICERs from UMICs mostly exceeded their thresholds ranging from 3,526 to 174,901 USD per QALY gained. Evidence shows cost-effectiveness of trastuzumab for HER2-positive EBC in HICs. There were no methodological variations. The extent and adequacy of reporting were high. The quality of data sources was moderate to high. The quality of future EEs can be improved by enhancing the reporting quality, by using context-based data and real-world efficacy data, which would impact cost-effectiveness.
Assuntos
Antineoplásicos Imunológicos/administração & dosagem , Neoplasias da Mama/tratamento farmacológico , Trastuzumab/administração & dosagem , Antineoplásicos Imunológicos/economia , Neoplasias da Mama/economia , Quimioterapia Adjuvante/economia , Análise Custo-Benefício , Feminino , Humanos , Estadiamento de Neoplasias , Anos de Vida Ajustados por Qualidade de Vida , Receptor ErbB-2/metabolismo , Projetos de Pesquisa , Trastuzumab/economiaRESUMO
Objective: Breast cancer remains to be the globally leading female cancer. About 15% to 20% of breast cancers have human epidermal growth factor receptor 2 (HER2)-positive tumors - a more aggressive breast cancer subtype with shortened survival. In the light of new and updated trial data on trastuzumab therapy for HER2-positive early-stage breast cancer (EBC), we conducted a systematic review and meta-analysis to update the pooling of its relative treatment effects. Methods: Systematic search was performed through Pubmed and Scopus to identify studies comparing survival outcomes and risks of heart toxicity effects of adjuvant trastuzumab with chemotherapy versus chemotherapy alone for HER2-positive EBC patients. Results: Based on the eight included studies in the review, combining trastuzumab with chemotherapy continues to show lowered death and relapse risks by one-third. The decision to initiate trastuzumab, however, needs to be prudently deliberated as two to three times more cardiotoxicity risk was shown to be associated with its use. Conclusion: Administering adjuvant trastuzumab in a weekly cycle concurrently with anthracycline-taxane chemotherapy regimen appears to be a preferable option to optimize its favorable effect in improving DFS and to prevent significantly higher risk for cardiotoxic effects.
Assuntos
Antineoplásicos Imunológicos/administração & dosagem , Neoplasias da Mama/tratamento farmacológico , Trastuzumab/administração & dosagem , Antineoplásicos Imunológicos/efeitos adversos , Neoplasias da Mama/patologia , Cardiotoxicidade/etiologia , Quimioterapia Adjuvante/métodos , Intervalo Livre de Doença , Feminino , Humanos , Estadiamento de Neoplasias , Receptor ErbB-2/metabolismo , Trastuzumab/efeitos adversosRESUMO
Direct-acting antivirals (DAAs) are modern treatments for chronic hepatitis C infection, but majority of available evidence on its treatment effect covers genotypes 1 to 4. Therefore, the efficacy and safety of DAAs for genotypes 5 and 6 need to be analysed. Studies were identified from Medline, Scopus, and CENTRAL and a Chinese database CNKI, from inception until Dec 4, 2018. Clinical trials were included if they enrolled patients with genotypes 5 and/or 6 infection, any type of second-generation DAAs was studied, and sustained virological response was assessed at the 12th week after treatment (SVR12) as outcome measure. Meta-analysis using metaprop statistical program was applied for pooling proportions if data were sufficient (i.e., at least 2 studies). Thirteen studies were included in the analysis. Four studies assessed the efficacy of four DAA regimens in genotype 5 patients, which were mainly sofosbuvir (SOF) plus pegylated-interferon/ribavirin (PR) or other DAAs, with SVR12 ranging from 94.4% to 100%. Twelve studies assessed the efficacy of seven DAA regimens among genotype 6 patients, but only two DAA regimens (i.e., SOF + PR and SOF/ledipasvir) had sufficient data for pooling. The pooled SVR12 rates (95% CI) were 99.6% (92.2 to 100) for SOF + PR and 99.2% (96.5 to 100) for SOF/ledipasvir. No treatment-related serious adverse event was reported, while the nonserious adverse events were comparable to other genotypes. In conclusion, DAAs are effective and may be safe for the treatment of chronic hepatitis C genotypes 5 and 6. However, our evidence is based on noncomparative studies; hence, further larger-scale randomized controlled trials in these genotypes are still required.
Assuntos
Antivirais/uso terapêutico , Hepacivirus/efeitos dos fármacos , Hepatite C Crônica/tratamento farmacológico , Benzimidazóis/uso terapêutico , Bases de Dados Factuais , Quimioterapia Combinada , Fluorenos/uso terapêutico , Genótipo , Hepacivirus/classificação , Hepacivirus/genética , Humanos , Interferons , Ribavirina/uso terapêutico , Sofosbuvir/uso terapêutico , Uridina Monofosfato/análogos & derivados , Uridina Monofosfato/uso terapêuticoRESUMO
The public health and economic burden of rabies has led to major intersectoral initiatives worldwide to reduce its burden. Over the last decade, the impact of rabies prevention and control programmes in real-world settings has become increasingly evident, especially in countries where most rabies exposures and deaths occur, but they have yet to successfully eradicate rabies due to limited access to health care services. We aimed to systematically review published transmission dynamic modelling studies of rabies in both humans and dogs with a focus on studies which estimated the epidemiological and economic impact of different preventive measures. The findings are intended to inform the World Health Organization's (WHO) Strategic Advisory Group of Experts on Immunization (SAGE) to improve programmatic feasibility and clinical practice in rabies. Medline and Scopus were systematically searched for peer-reviewed articles which were published up to 14th June 2017. In addition, studies identified from a meeting of the WHO Expert Consultation on Rabies on 26-28th April 2017 in Bangkok, Thailand were added, resulting in 19 articles which were included in the review. Results from the disease modelling indicated that the basic reproduction number was low (less than 2 in all but one study). All studies found that rabies control through canine vaccination was likely to be effective in terms of reducing the incidence of rabies in dogs and/or humans, with most studies suggesting 70% annual coverage was adequate. Vaccine coverage, dog density and birth rate were identified as crucial factors influencing the effectiveness of the interventions. In conclusion, the findings from this review suggest that rabies control through canine vaccination is likely to be effective in reducing the incidence of rabies. Vaccine coverage, dog density and canine birth rate were identified as critical factors influencing the effectiveness of vaccination interventions.