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OBJECTIVES: We present an empirical comparison of relative-efficacy estimate(s) from matching-adjusted indirect comparisons (MAICs) with estimates from corresponding standard anchored indirect treatment comparisons. METHODS: A total of 80 comparisons were identified from 17 publications through a systematic rapid review. A standardized metric that used reported relative treatment efficacy estimates and their associated uncertainty was used to compare the methods across different treatment indications and outcome measures. RESULTS: On aggregate, MAICs presented for connected networks tended to report a more favorable relative-efficacy estimate for the treatment for which individual-level patient data were available relative to the reported indirect treatment comparison estimate. CONCLUSIONS: Although we recognize the importance of MAIC and other population adjustment methods in certain situations, we recommend that results from these analyses are interpreted with caution. Researchers and analysts should carefully consider if MAICs are appropriate where presented and whether MAICs would have added value where omitted.
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Avaliação de Resultados em Cuidados de Saúde , Humanos , Avaliação de Resultados em Cuidados de Saúde/métodos , Resultado do TratamentoRESUMO
BACKGROUND: Cutaneous melanoma is amongst the most aggressive of all skin cancers. Neoadjuvant treatment is a form of induction therapy, given to shrink a cancerous tumour prior to the main treatment (usually surgery). The purpose is to improve survival and surgical outcomes. This review systematically appraises the literature investigating the use of neoadjuvant treatment for stage III and IV cutaneous melanoma. OBJECTIVES: To assess the effects of neoadjuvant treatment in adults with stage III or stage IV melanoma according to the seventh edition American Joint Committee on Cancer (AJCC) staging system. SEARCH METHODS: We searched the following databases up to 10 August 2021 inclusive: Cochrane Skin Specialised Register, CENTRAL, MEDLINE, Embase, LILACS and four trials registers, together with reference checking and contact with study authors to identify additional studies. We also handsearched proceedings from specific conferences from 2016 to 2020 inclusive. SELECTION CRITERIA: Randomised controlled trials (RCTs) of people with stage III and IV melanoma, comparing neoadjuvant treatment strategies (using targeted treatments, immunotherapies, radiotherapy, topical treatments or chemotherapy) with any of these agents or current standard of care (SOC), were eligible for inclusion. DATA COLLECTION AND ANALYSIS: We used standard Cochrane methods. Primary outcomes were overall survival (OS) and adverse effects (AEs). Secondary outcomes included time to recurrence (TTR), quality of life (QOL), and overall response rate (ORR). We used GRADE to evaluate the certainty of the evidence. MAIN RESULTS: We included eight RCTs involving 402 participants. Studies enrolled adults, mostly with stage III melanoma, investigated immunotherapies, chemotherapy, or targeted treatments, and compared these with surgical excision with or without adjuvant treatment. Duration of follow-up and therapeutic regimens varied, which, combined with heterogeneity in the population and definitions of the endpoints, precluded meta-analysis of all identified studies. We performed a meta-analysis including three studies. We are very uncertain if neoadjuvant treatment increases OS when compared to no neoadjuvant treatment (hazard ratio (HR) 0.43, 95% confidence interval (CI) 0.15 to 1.21; 2 studies, 171 participants; very low-certainty evidence). Neoadjuvant treatment may increase the rate of AEs, but the evidence is very uncertain (26% versus 16%, risk ratio (RR) 1.58, 95% CI 0.97 to 2.55; 2 studies, 162 participants; very low-certainty evidence). We are very uncertain if neoadjuvant treatment increases TTR (HR 0.51, 95% CI 0.22 to 1.17; 2 studies, 171 participants; very low-certainty evidence). Studies did not report ORR as a comparative outcome or measure QOL data. We are very uncertain whether neoadjuvant targeted treatment with dabrafenib and trametinib increases OS (HR 0.28, 95% CI 0.03 to 2.25; 1 study, 21 participants; very low-certainty evidence) or TTR (HR 0.02, 95% CI 0.00 to 0.22; 1 study, 21 participants; very low-certainty evidence) when compared to surgery. The study did not report comparative rates of AEs and overall response, and did not measure QOL. We are very uncertain if neoadjuvant immunotherapy with talimogene laherparepvec increases OS when compared to no neoadjuvant treatment (HR 0.49, 95% CI 0.15 to 1.64; 1 study, 150 participants, very low-certainty evidence). It may have a higher rate of AEs, but the evidence is very uncertain (16.5% versus 5.8%, RR 2.84, 95% CI 0.96 to 8.37; 1 study, 142 participants; very low-certainty evidence). We are very uncertain if it increases TTR (HR 0.75, 95% CI 0.31 to 1.79; 1 study, 150 participants; very low-certainty evidence). The study did not report comparative ORRs or measure QOL. OS was not reported for neoadjuvant immunotherapy (combined ipilimumab and nivolumab) when compared to the combination of ipilimumab and nivolumab as adjuvant treatment. There may be little or no difference in the rate of AEs between these treatments (9%, RR 1.0, 95% CI 0.75 to 1.34; 1 study, 20 participants; low-certainty evidence). The study did not report comparative ORRs or measure TTR and QOL. Neoadjuvant immunotherapy (combined ipilimumab and nivolumab) likely results in little to no difference in OS when compared to neoadjuvant nivolumab monotherapy (P = 0.18; 1 study, 23 participants; moderate-certainty evidence). It may increase the rate of AEs, but the certainty of this evidence is very low (72.8% versus 8.3%, RR 8.73, 95% CI 1.29 to 59; 1 study, 23 participants); this trial was halted early due to observation of disease progression preventing surgical resection in the monotherapy arm and the high rate of treatment-related AEs in the combination arm. Neoadjuvant combination treatment may lead to higher ORR, but the evidence is very uncertain (72.8% versus 25%, RR 2.91, 95% CI 1.02 to 8.27; 1 study, 23 participants; very low-certainty evidence). It likely results in little to no difference in TTR (P = 0.19; 1 study, 23 participants; low-certainty evidence). The study did not measure QOL. OS was not reported for neoadjuvant immunotherapy (combined ipilimumab and nivolumab) when compared to neoadjuvant sequential immunotherapy (ipilimumab then nivolumab). Only Grade 3 to 4 immune-related AEs were reported; fewer were reported with combination treatment, and the sequential treatment arm closed early due to a high incidence of severe AEs. The neoadjuvant combination likely results in a higher ORR compared to sequential neoadjuvant treatment (60.1% versus 42.3%, RR 1.42, 95% CI 0.87 to 2.32; 1 study, 86 participants; low-certainty evidence). The study did not measure TTR and QOL. No data were reported on OS, AEs, TTR, or QOL for the comparison of neoadjuvant interferon (HDI) plus chemotherapy versus neoadjuvant chemotherapy. Neoadjuvant HDI plus chemotherapy may have little to no effect on ORR, but the evidence is very uncertain (33% versus 22%, RR 1.75, 95% CI 0.62 to 4.95; 1 study, 36 participants; very low-certainty evidence). AUTHORS' CONCLUSIONS: We are uncertain if neoadjuvant treatment increases OS or TTR compared with no neoadjuvant treatment, and it may be associated with a slightly higher rate of AEs. There is insufficient evidence to support the use of neoadjuvant treatment in clinical practice. Priorities for research include the development of a core outcome set for neoadjuvant trials that are adequately powered, with validation of pathological and radiological responses as intermediate endpoints, to investigate the relative benefits of neoadjuvant treatment compared with adjuvant treatment with immunotherapies or targeted therapies.
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Antineoplásicos , Melanoma , Neoplasias Cutâneas , Adulto , Humanos , Antineoplásicos/efeitos adversos , Ipilimumab , Melanoma/tratamento farmacológico , Melanoma/patologia , Nivolumabe , Neoplasias Cutâneas/tratamento farmacológico , Neoplasias Cutâneas/patologia , Ensaios Clínicos Controlados Aleatórios como Assunto , Estadiamento de Neoplasias , Melanoma Maligno CutâneoRESUMO
OBJECTIVES: This study evaluates the cost-effectiveness of tisagenlecleucel (a CAR T-cell therapy), versus blinatumomab, for the treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (R/R ALL) in the Irish healthcare setting. The value of conducting further research, to investigate the value of uncertainty associated with the decision problem, is assessed by means of expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses. METHODS: A three-state partitioned survival model was developed. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 60 months; general population mortality with a standardized mortality ratio was then applied. Estimated EVPI and EVPPI were scaled up to population according to the incidence of the decision. RESULTS: At list prices, the incremental cost-effectiveness ratio was EUR 73,086 per quality-adjusted life year (QALY) (incremental costs EUR 156,928; incremental QALYs 2.15). The probability of cost-effectiveness, at the willingness-to-pay threshold of EUR 45,000 per QALY, was 16 percent. At this threshold, population EVPI was EUR 314,455; population EVPPI was below EUR 100,000 for each parameter category. CONCLUSIONS: Tisagenlecleucel is not cost effective, versus blinatumomab, for the treatment of pediatric and young adult patients with R/R ALL in Ireland (at list prices). Further research to decrease decision (parameter) uncertainty, at the defined willingness-to-pay threshold, may not be of value. However, there is a high degree of uncertainty underpinning the analysis, which may not be captured by EVPI analysis.
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Leucemia-Linfoma Linfoblástico de Células Precursoras , Criança , Análise Custo-Benefício , Atenção à Saúde , Humanos , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Anos de Vida Ajustados por Qualidade de Vida , Receptores de Antígenos de Linfócitos T , Adulto JovemRESUMO
This study aims to estimate the theoretical excess expenditure that would be incurred by the Irish state-payer, should drugs be reimbursed at their original asking ("list") price rather than at a price at which the drug is considered cost-effective. In Ireland, all new drugs are evaluated by the National Centre for Pharmacoeconomics. For this study, drugs that were submitted by pharmaceutical companies from 2012 to 2017 and considered not cost-effective at list price were reviewed. A total of 43 such drugs met our inclusion criteria, and their pharmacoeconomic evaluations were further assessed. The price at which the drug could be considered cost-effective (cost-effective price) at the upper cost-effectiveness threshold used in Ireland ( 45 000/quality adjusted life-year) was estimated for 18 drugs with an available cost-effectiveness model. Then, for each drug, the list price and cost-effective price (both per unit) were both individually applied to 1 year of national real-world drug utilization data. This allowed the estimation of the expected expenditures under the assumptions of list price paid and cost-effective price paid. The resulting theoretical excess expenditure, the expenditure at list price minus the expenditure at the cost-effective price, was estimated to be 108.2 million. This estimate is theoretical because of the confidentiality of actual drug prices. The estimation is calculated using the list price and likely overestimates the actual excess expenditure, which would reduce to zero if cost-effective prices are agreed. Nevertheless, this estimate illustrates the importance of a process to assess the value of new drugs so that potential excess drug expenditure is identified.
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Análise Custo-Benefício/métodos , Custos de Cuidados de Saúde/estatística & dados numéricos , Resultado do Tratamento , Análise Custo-Benefício/estatística & dados numéricos , Uso de Medicamentos/normas , Uso de Medicamentos/estatística & dados numéricos , Custos de Cuidados de Saúde/normas , Humanos , Irlanda , Programas Nacionais de Saúde/economia , Programas Nacionais de Saúde/normas , Programas Nacionais de Saúde/estatística & dados numéricosRESUMO
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.
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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 TratamentoRESUMO
It is expected that the coronavirus disease 2019 (COVID-19) pandemic will leave large deficits in the budgets of many jurisdictions. Funding for other treatments, in particular new treatments, may become more constrained than previously expected. Therefore, a robust health technology assessment (HTA) system is vital. Many clinical trials carried out during the pandemic may have been temporarily halted, while others may have had to change their protocols. Even trials that continue as normal may experience external changes as other aspects of the healthcare service may not be available to the patients in the trial, or the patients themselves may contract COVID-19. Consequently, many limitations are likely to arise in the provision of robust HTAs, which could have profound consequences on the availability of new treatments. Therefore, the National Centre for Pharmacoeconomics Review Group wishes to discuss these issues and make recommendations for applicants submitting to HTA agencies, in ample time for these HTAs to be prepared and assessed. We discuss how the pandemic may affect the estimation of the treatment effect, costs, life-years, utilities, discontinuation rates, and methods of evidence synthesis and extrapolation. In particular, we note that trials conducted during the pandemic will be subject to a higher degree of uncertainty than before. It is vital that applicants clearly identify any parameters that may be affected by the pandemic. These parameters will require considerably more scenario and sensitivity analyses to account for this increase in uncertainty.
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Comitês Consultivos , Infecções por Coronavirus , Pandemias , Pneumonia Viral , Avaliação da Tecnologia Biomédica , Betacoronavirus , Orçamentos , COVID-19 , Infecções por Coronavirus/tratamento farmacológico , Farmacoeconomia , Humanos , Pneumonia Viral/tratamento farmacológico , Qualidade de Vida , SARS-CoV-2 , Resultado do Tratamento , Suspensão de TratamentoRESUMO
BACKGROUND: Recent advances in the development of biomarker-directed therapy and immunotherapy, for advanced and metastatic gastric cancers, have the potential to improve survival and quality of life. Much attention has been directed towards second- and later-line treatments, and the landscape here is evolving rapidly. However, uncertainty in relative effectiveness, high costs and uncertainty in cost effectiveness represent challenges for decision makers. OBJECTIVE: To identify economic evaluations for the second-line or later-line treatment of advanced and metastatic gastric cancer. Also, to assess key criteria (including model assumptions, inputs and outcomes), reporting completeness and methodological quality to inform future cost-effectiveness evaluations. METHODS: A systematic literature search (from database inception to 5 March 2023) of EconLit via EBSCOhost, Cochrane Library (restricted to National Health Service [NHS] Economic Evaluation Database and Health Technology Assessment [HTA] Database), Embase, MEDLINE and of grey literature was conducted. This aimed to identify systemic treatments that align with National Comprehensive Cancer Network (NCCN) and European Society for Medical Oncology (ESMO) Clinical Practice Guidelines. Data were collected on key criteria and on reporting completeness and methodological quality. A narrative synthesis focussed on cost-effectiveness and cost-of-illness studies. Outcomes of interest included total and incremental costs and outcomes (life-years and quality-adjusted life-years), ratios of incremental costs per unit outcome and other summary cost and outcome measures. Also, for cost-effectiveness studies, reporting completeness and the methodological quality were assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and the Philips Checklist, respectively. RESULTS: A total of 19 eligible economic evaluations were identified (cost-effectiveness studies [n = 15] and cost-of-illness studies [n = 4]). There was a general lack of consistency in the methodological approaches taken across studies. In the main, the cost-effectiveness studies indicated that the intervention under consideration was more effective and more costly than the comparator(s). However, most interventions were not cost effective. No studies were fully compliant with reporting-completeness and methodological-quality requirements. Given the lack of consistency in the approaches taken across cost-of-illness studies, outcomes could not be directly compared. CONCLUSIONS: To our knowledge, this is the first published systematic literature review that has qualitatively synthesised economic evaluations for advanced and metastatic gastric cancer. There were differences in the approaches taken across the cost-effectiveness studies and the cost-of-illness studies. The conclusions of most of the cost-effectiveness studies were consistent despite identified differences in approaches. In the main, the interventions under consideration were not cost effective, presenting challenges to sustainability and affordability. We highlight a requirement for cost-effectiveness evaluations and for second-line or later-line treatments of advanced and metastatic gastric cancer that consider all relevant comparators and that are compliant with reporting-completeness and methodological-quality requirements. By addressing the methodological gaps identified here, future healthcare decision-making, within the context of this rapidly changing treatment landscape, would be better informed. PROSPERO REGISTRATION NUMBER: CRD42023405951.
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Considerable variety in how patients respond to treatments, driven by differences in their geno- and/ or phenotypes, calls for a more tailored approach. This is already happening, and will accelerate with developments in personalized medicine. However, its promise has not always translated into improvements in patient care due to the complexities involved. There are also concerns that advice for tests has been reversed, current tests can be costly, there is fragmentation of funding of care, and companies may seek high prices for new targeted drugs. There is a need to integrate current knowledge from a payer's perspective to provide future guidance. Multiple findings including general considerations; influence of pharmacogenomics on response and toxicity of drug therapies; value of biomarker tests; limitations and costs of tests; and potentially high acquisition costs of new targeted therapies help to give guidance on potential ways forward for all stakeholder groups. Overall, personalized medicine has the potential to revolutionize care. However, current challenges and concerns need to be addressed to enhance its uptake and funding to benefit patients.
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Atenção à Saúde/métodos , Assistência ao Paciente/métodos , Medicina de Precisão/métodos , Atenção à Saúde/tendências , Estudos de Viabilidade , Previsões , Humanos , Assistência ao Paciente/tendências , Farmacogenética/métodos , Farmacogenética/tendências , Medicina de Precisão/tendênciasRESUMO
BACKGROUND: Dupilumab was the first biological medicine to receive marketing authorisation, in Ireland, for the treatment of atopic dermatitis (AD). In 2019, Ireland's National Centre for Pharmacoeconomics recommended that dupilumab not be reimbursed at the submitted price; it was not cost effective. Following confidential price negotiations, the Health Service Executive (HSE) reimbursed dupilumab subject to a HSE-Managed Access Protocol (MAP). Patients with refractory, moderate-to-severe AD were deemed eligible to receive treatment under the MAP; the cohort for which dupilumab is expected to be most effective and most cost effective (versus standard of care). Approval, for treatment, is granted on an individual patient basis by the HSE-Medicines Management Programme. AIMS: Applications for approval of treatment with dupilumab were analysed to determine the percentage of patients deemed eligible. Key characteristics of this population were investigated. METHODS: Data from individual patient applications were analysed. Key characteristics of the approved population were investigated using IBM SPSS Statistics® version 27. Data were derived from the HSE-Primary Care Reimbursement Service pharmacy claims database. The number of patients who received dupilumab over the study period was determined. RESULTS: In total, 96% of submitted applications were deemed eligible. Of these, 65% were male and 87% were adults. In the main, the approved patient population had severe refractory AD; the mean Eczema Area Severity Index score was 28.72. CONCLUSION: The majority of applications submitted were approved. This work highlights how a MAP can facilitate access to treatment in patients who are deemed eligible whilst containing overall expenditure.
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Anticorpos Monoclonais Humanizados , Adulto , Humanos , Masculino , Feminino , Irlanda , Resultado do Tratamento , Índice de Gravidade de Doença , Anticorpos Monoclonais Humanizados/uso terapêutico , Método Duplo-CegoRESUMO
Understanding the functional characteristics of antibodies produced against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) will assist in the determination of disease outcomes for this virus. In this study, the ability of antibodies to inhibit viral entry into the host cell through the interaction of the receptor binding domain of the viral spike protein and the angiotensin-converting enzyme 2 receptor on the human cell surface was investigated. The SARS-CoV-2 IgG levels in 20 SARS-CoV-2 positive patients were measured using an enzyme-linked immunosorbent assay, and the samples were further analyzed using a functional binding assay. Inhibition of viral infectivity was also measured using a pseudovirus neutralization assay against a D614G SARS-CoV-2 virus strain. A significant correlation between IgG levels and neutralizing antibody 50% inhibitory concentration (IC50) titers was observed (p < 0.05). Similarly, the IC50 titers obtained in the neutralization and binding assays were significantly correlated (p < 0.001). Varying levels of IgG and IC50 titers were observed for the SARS-CoV-2 antibody-positive samples, with one sample not showing any neutralizing capability despite detectable IgG levels. Gender comparisons showed no statistical differences in any of the assays. These results suggest that increased SARS-CoV-2 IgG levels correlate with greater protection against the entry of the virus into cells; however, further investigations in larger studies are needed to confirm the correlates of protection.
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COVID-19 , Humanos , Enzima de Conversão de Angiotensina 2 , Anticorpos Neutralizantes , Anticorpos Antivirais , Imunoglobulina G , SARS-CoV-2 , Glicoproteína da Espícula de Coronavírus/químicaRESUMO
BACKGROUND: Polypharmacy and associated potentially inappropriate prescribing (PIP) place a considerable burden on patients and represent a challenge for general practitioners (GPs). Integration of pharmacists within general practice (herein 'pharmacist integration') may improve medications management and patient outcomes. This systematic review assessed the effectiveness and costs of pharmacist integration. METHODS: A systematic search of ten databases from inception to January 2021 was conducted. Studies that evaluated the effectiveness or cost of pharmacist integration were included. Eligible interventions were those that targeted medications optimization compared to usual GP care without pharmacist integration (herein 'usual care'). Primary outcomes were PIP (as measured by PIP screening tools) and number of prescribed medications. Secondary outcomes included health-related quality of life, health service utilization, clinical outcomes, and costs. Randomised controlled trials (RCTs), non-RCTs, interrupted-time-series, controlled before-after trials and health-economic studies were included. Screening and risk of bias using Cochrane EPOC criteria were conducted by two reviewers independently. A narrative synthesis and meta-analysis of outcomes where possible, were conducted; the certainty of evidence was assessed using the Grading of Recommendations, Assessment, Development and Evaluation approach. RESULTS: In total, 23 studies (28 full text articles) met the inclusion criteria. In ten of 11 studies, pharmacist integration probably reduced PIP in comparison to usual care (moderate certainty evidence). A meta-analysis of number of medications in seven studies reported a mean difference of -0.80 [-1.17, -0.43], which indicated pharmacist integration probably reduced number of medicines (moderate certainty evidence). It was uncertain whether pharmacist integration improved health-related quality of life because the certainty of evidence was very low. Twelve health-economic studies were included; three investigated cost effectiveness. The outcome measured differed across studies limiting comparisons and making it difficult to make conclusions on cost effectiveness. CONCLUSIONS: Pharmacist integration probably reduced PIP and number of medications however, there was no clear effect on other patient outcomes; and while interventions in a small number of studies appeared to be cost-effective, further robust, well-designed cluster RCTs with economic evaluations are required to determine cost-effectiveness of pharmacist integration. TRIAL REGISTRATION: CRD42019139679.
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Medicina Geral , Farmacêuticos , Humanos , Avaliação de Resultados em Cuidados de Saúde , Polimedicação , Atenção Primária à SaúdeRESUMO
BACKGROUND AND OBJECTIVE: In Ireland, similar to other jurisdictions, health technology assessment (HTA) is used to inform the health payer's drug reimbursement decisions. These HTAs are conducted by the National Centre for Pharmacoeconomics (NCPE). In 2009, the NCPE introduced the Rapid Review process to identify drugs that do not require further assessment in the form of the previously established full HTA process. METHODS: A retrospective analysis of all Rapid Reviews submitted to the NCPE from 2010 to 2019, inclusive, was conducted. Rapid Review recommendation was recorded (i.e. full HTA required or not required). For those submitted from 2012 to 2019, additional data relating to the drug, economic and clinical evidence-related factors were collected. Multivariable logistic regression methods were used to model the relationship between these factors and the likelihood of requiring a full HTA. An exploratory analysis estimated the additional NCPE appraisal time that would have been required to evaluate all drugs, had the Rapid Review process not been established. RESULTS: Of the 446 Rapid Reviews submitted, approximately half (49.6%) were deemed to require a full HTA. Drugs for cancer indications, drugs designated first-in-class status, and high-cost drugs were positively and significantly associated with the likelihood of requiring a full HTA. No significant association was found for drugs for orphan indications when factors relating to cost and clinical evidence were included in the model. Without the Rapid Review process, an estimated additional 15,631 NCPE appraisal days would have been required to evaluate all drugs submitted over the 10-year period. CONCLUSIONS: This is the first study to use data uniquely available to the NCPE to evaluate factors associated with the requirement for a full HTA following a Rapid Review. The process has reduced the NCPE appraisal time required to evaluate all submissions over the study period. The NCPE's Rapid Review process allows for appropriate resource prioritisation within a national HTA agency.
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Farmacoeconomia , Avaliação da Tecnologia Biomédica , Custos de Medicamentos , Humanos , Organizações , Estudos Retrospectivos , Avaliação da Tecnologia Biomédica/métodosRESUMO
When treating malnutrition, oral nutritional supplements (ONSs) are advised when optimising the diet is insufficient; however, ONS usage and user characteristics have not been previously analysed. A retrospective secondary analysis was performed on dispensed pharmacy claim data for 14,282 anonymised adult patients in primary care in Ireland in 2018. Patient sex, age, residential status, ONS volume (units) and ONS cost (EUR) were analysed. The categories of 'Moderate' (<75th centile), 'High' (75th-89th centile) and 'Very High' ONS users (≥90th centile) were created. The analyses among groups utilised t-tests, Mann-Whitney U tests and chi-squared tests. This cohort was 58.2% female, median age was 76 years, with 18.7% in residential care. The most frequently dispensed ONS type was very-high-energy sip feeds (45% of cohort). Younger males were dispensed more ONSs than females (<65 years: median units, 136 vs. 90; p < 0.01). Patients living independently were dispensed half the volume of those in residential care (112 vs. 240 units; p < 0.01). 'Moderate' ONS users were dispensed a yearly median of 84 ONS units (median cost, EUR 153), 'High' users were dispensed 420 units (EUR 806) and 'Very High' users 892 yearly units (EUR 2402; p < 0.01). Further analyses should focus on elucidating the reasons for high ONS usage in residential care patients and younger males.
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Suplementos Nutricionais/estatística & dados numéricos , Disparidades em Assistência à Saúde/estatística & dados numéricos , Padrões de Prática Médica/estatística & dados numéricos , Prescrições/estatística & dados numéricos , Atenção Primária à Saúde/estatística & dados numéricos , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Irlanda , Masculino , Desnutrição/terapia , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Instituições Residenciais/estatística & dados numéricos , Estudos RetrospectivosRESUMO
INTRODUCTION: Managing patients with multiple conditions (multimorbidity) is a major challenge for healthcare systems internationally, particularly in older patients. Multimorbidity and subsequent polypharmacy increase treatment burden and the risk of potentially inappropriate prescribing, and both are complex to manage in primary care. Limited evidence suggests integration of pharmacists into general practice teams could improve medication management for patients with multimorbidity and polypharmacy. Building on findings from a non-randomised, uncontrolled General Practice Pharmacist (GPP) feasibility study conducted in Irish primary care, the aim of this study is to conduct a pilot cluster randomised controlled trial (cRCT) of the GPP study, to assess feasibility, intervention impact, costs and appropriateness of continuing to a definitive cRCT. METHODS AND ANALYSIS: This pilot cRCT will involve 8 general practitioner (GP) practices and 120 patients. Practices will identify and recruit patients aged ≥65 years, who are taking ≥10 regular medications. Practices will be allocated to intervention or control after baseline data collection. Intervention practices will have a pharmacist integrated within their service, working with GPs, patients and practice staff to optimise prescribing and other medication-related activities. Control practices will provide standard GP care. The primary feasibility outcomes will include recruitment rate, uptake of medication reviews and study retention. For the primary clinical outcome, the number of potentially inappropriate prescribing incidences per patient will be collected. Secondary outcomes will include medication-related outcomes, patient-reported outcome measures, and data pertaining to the role and impact of the pharmacist on prescribing. In addition, economic and process evaluations will be conducted. ETHICS AND DISSEMINATION: This trial has been approved by the Irish College of General Practitioners Research Ethics Committee and will be performed in accordance with the Declaration of Helsinki. The results will be reported in peer-reviewed journals and be presented at national and international conferences. TRIAL REGISTRATION NUMBER: ISRCTN Registry (https://doi.org/10.1186/ISRCTN18752158).
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Medicina Geral , Farmacêuticos , Idoso , Humanos , Prescrição Inadequada , Multimorbidade , Polimedicação , Ensaios Clínicos Controlados Aleatórios como AssuntoRESUMO
BACKGROUND & AIMS: Understanding how older adults perceive their nutritional needs and malnutrition risk is important to inform strategies to improve prevention and management of the condition. This scoping review aimed to identify, characterize and summarize the findings from studies analysing community-dwelling older adults' opinions and perceptions towards their nutritional needs and malnutrition risk. METHODS: An electronic literature search was carried out using three databases, Pubmed, Embase, and CINAHL up to January 2020. Articles were reviewed following PRISMA guidelines. RESULTS: A total of 16,190 records were identified and reviewed with 15 studies being included, all of which were conducted in high income countries. Common conceptual categories that were identified included; older community-dwelling adults consider that a healthy diet for them is the same as that recommended for the general population, consisting of fruits, vegetables, reduced fat and reduced sugar. Weight loss was seen as a positive outcome and a normal component of the ageing process. Lack of appetite was identified by participants in the majority of studies as a barrier to food intake. CONCLUSIONS: This review shows how older community-dwelling adults, with a high risk of malnutrition, follow dietary public health recommendations for the general population and have a greater awareness of the risks of overweight. The implementation of nutritional guidelines that consider the nutritional needs of all older adults and education of non-dietetic community healthcare professionals on providing appropriate nutritional advice to this population are warranted.
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Avaliação Geriátrica/métodos , Conhecimentos, Atitudes e Prática em Saúde , Vida Independente , Desnutrição/prevenção & controle , Necessidades Nutricionais , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Masculino , Política Nutricional , RiscoRESUMO
BACKGROUND: Oral nutritional supplements (ONS) are recommended for patients who are malnourished or at risk of malnutrition. Appropriate ONS prescribing requires regular monitoring to assess its continued requirement. Previous research identified long-term ONS prescriptions (>6 months) without review, with 70% of these influenced by social factors. AIM: To investigate the characteristics of long-term ONS users in Ireland and the determinants of larger volumes of ONS dispensing. DESIGN & SETTING: Secondary analysis of anonymous dispensed pharmacy claims data of patients dispensed standard ONS for 12 consecutive months in 2018 (n = 912). METHOD: Factors showing significant (P<0.05) univariate associations with above the median consumption of ONS units were entered into a multivariable model. RESULTS: Median age was 76 (range 18 to 101) years, with 66.9% of the sample being ≥65 years. Almost 70% of the samples were on polypharmacy (45.6%; ≥5 medications) or excessive polypharmacy (21.5%; ≥10 medications). Younger age and being on polypharmacy for drugs having an effect on the central nervous system (CNS) were significantly associated with being dispensed more ONS units in univariate and multivariate analysis. Those patients in the age range 18 to 44 were 2.5 fold more likely to be prescribed more ONS units (odds ratio [OR] 2.5; 95% confidence interval [CI] 1.5 to 4.3; P<0.001). Patients using CNS drugs or on CNS polypharmacy were more likely to be prescribed more ONS units (ORs 1.2 and 2.4; 95% CI 0.9 to 1.4 and 1.3 to 4.4 respectively; P = 0.029). CONCLUSION: Older age and polypharmacy characterise long-term ONS users in this study. Younger age and CNS medication polypharmacy are predictors of more ONS units prescribed over a year.
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BACKGROUND & AIM: Protein-energy malnutrition is under-recognised in the community despite being common in older adults due to physiological and social changes which are often compounded by chronic disease. This qualitative study aimed to explore the opinions of healthcare professionals (HCPs) working in the primary care and community settings about the management of malnutrition and the prescription of oral nutritional supplements (ONS), often included in the treatment of malnutrition. METHODS: Twelve healthcare professional (HCP) focus groups with 75 participants were conducted: community dietitians (n = 17), registered dietitians working in industry (n = 5), community and residential care nurses (n = 22), physiotherapists (n = 12), pharmacists (n = 9), occupational therapists (n = 6) and speech and language therapists (n = 4). Focus group discussions were audio-recorded and transcribed verbatim. The data were coded and analysed using thematic analysis and key themes with illustrative quotes extracted are presented. RESULTS: Similar views on malnutrition management existed across professions. 'Gaps in Primary Care Management' was the first key theme wherein HCPs identified limitations in malnutrition management in the community. Barriers included limited or no dietetic services available in primary care and poor communication between general practitioners and wider primary care team members which resulted in inappropriate or delayed treatment. The second key theme, 'Challenges with ONS use in the Community', encapsulated several issues HCPs experienced with ONS usage including inappropriate prescribing and lack of monitoring of treatment goals. Conflicts of interest regarding dietitians working in industry assessing and treating older adults in residential care settings was highlighted by participants. CONCLUSIONS: This study highlights that more emphasis is needed to identify patients when they are at risk of malnutrition to avoid advanced or severe malnutrition presentations currently seen. Community dietitians for older people are required to address many of the issues raised including the need for awareness, education and training, resources, and malnutrition care pathway structures.
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Desnutrição , Nutricionistas , Idoso , Atenção à Saúde , Humanos , Desnutrição/terapia , Percepção , Pesquisa QualitativaRESUMO
BACKGROUND: Language and communication have an impact on how a clinical condition is treated and experienced, from both the health care professional (HCP) and patient perspective. Malnutrition is prevalent among community-dwelling older adults, yet perceptions of patient understanding of the term malnutrition to date remain underexplored. OBJECTIVE: This qualitative study explored the use and perceptions of the term malnutrition among HCPs and older adults at risk of malnutrition. DESIGN: Semi-structured interviews and focus groups were conducted with HCPs and older adults with a prescription for oral nutritional supplements (ONS) in the community, to explore perspectives. PARTICIPANTS AND SETTING: HCPs with experience of working with older adults were recruited in primary care centers, general practitioner practices, community health organizations, and community pharmacies in County Dublin, Ireland, between 2018 and 2019. Older adults, aged ≥60 years, with a current or previous prescription for ONS were recruited from daycare centers. One-to-one interviews were conducted with general practitioners (n = 16) and patients (n = 13), and focus groups were conducted with other HCPs, including dietitians (n = 22), nurses (n = 22), pharmacists (n = 9), physiotherapists (n = 12), occupational therapists (n = 6), and speech and language therapists (n = 4). DATA ANALYSIS: Data from interviews and focus groups were transcribed verbatim and analyzed using thematic analysis. RESULTS: There was mutual agreement between HCPs and patients on the main theme, "malnutrition is a term to be avoided." There were three subthemes with varying input from the different HCP groups and patients: "Malnutrition is a term a patient doesn't want to hear"-malnutrition has negative connotations that imply neglect and stigma; "malnutrition is a clinical term which patients don't understand"-with perceptions that it is better to substitute the term with simpler motivating messages; and "lack of confidence identifying malnutrition"-expressed by non-dietetics HCPs who believed they had insufficient expertise on malnutrition to communicate effectively with patients. CONCLUSIONS: HCPs and patients perceived negative connotations with the term malnutrition, and HCPs used alternatives in practice. Additional consultation with HCPs and patients is recommended to explore appropriate language for conveying health risks associated with malnutrition. Future research should also address how current communication challenges can be addressed as part of strategic management programs or interventions to prevent and treat malnutrition.
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Pessoal de Saúde/psicologia , Vida Independente/psicologia , Desnutrição/psicologia , Terminologia como Assunto , Adulto , Idoso , Comunicação , Feminino , Grupos Focais , Humanos , Irlanda , Masculino , Pessoa de Meia-Idade , Atenção Primária à Saúde , Relações Profissional-Paciente , Pesquisa QualitativaRESUMO
BACKGROUND: Differing methodological requirements and decision-making criteria are recognised as barriers to transferability of cost-effectiveness analysis (CEA) across jurisdictions. OBJECTIVE: We assessed the generic and specific transferability of published CEAs of systemic treatments for advanced melanoma to the Irish setting. METHODS: CEAs of treatments for melanoma were identified by systematic review. Transferability to the Irish setting was assessed using the EUnetHTA transferability tool for Economic Evaluation. We present a narrative discussion comparing the differences in key parameter inputs and the likely impact of these differences on the model outcomes and the reimbursement recommendation. Transferability is considered within the context of the Irish cost-effectiveness threshold, using the net monetary benefit (NMB) framework. RESULTS: No published CEAs (n = 15) aligned with the Irish reference case for CEA. Changes to key parameters were unlikely to change the conclusions of the CEA when the cost-effectiveness threshold was considered. Ten studies (19 pairwise comparisons) were compared with findings by the National Centre for Pharmacoeconomics (NCPE) using NMB. Without accounting for differences in the cost-effectiveness threshold, there was alignment between the study conclusions and NCPE recommendations in 73.7% cases. When the Irish cost-effectiveness threshold was applied in the estimation of NMB, there was agreement in 89.5% of cases. CONCLUSIONS: Alignment in methodological requirements for CEA is important to facilitate joint health technology assessment (HTA) by regional collaborations in Europe. When parameter inputs are not exactly aligned, conclusions may still be comparable across jurisdictions. For international joint procurement initiatives, determining and implementing joint decision rules may be more important than trying to align rules regarding methodological and parameter inputs.
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Técnicas de Apoio para a Decisão , Farmacoeconomia , Melanoma/economia , Melanoma/terapia , Modelos Econométricos , Avaliação da Tecnologia Biomédica/economia , Análise Custo-Benefício , Humanos , Irlanda , Anos de Vida Ajustados por Qualidade de Vida , Análise de SobrevidaRESUMO
BACKGROUND: The National Centre for Pharmacoeconomics (NCPE) is a National HTA Agency in Ireland responsible for assessment of comparative clinical effectiveness, cost-effectiveness and potential budget impact of drugs on behalf of the Health Service Executive. This research aims to assess if the budget impact models submitted to the NCPE have accurate predicted utilisation, assess if the models are consistent in the parameters included, and determine if probabilistic sensitivity analyses would aid the characterization of uncertainty. METHODS: A retrospective analysis of budget impact models that had been submitted (January 2010-December 2017 inclusive) to the NCPE was performed. The input parameters in the budget impact model were recorded. For each drug, annual realised utilisation was compared with what had been predicted by the respective budget impact model. A probabilistic sensitivity analysis was also performed on each model. RESULTS: A total of 12 models were included; each model pertained to one drug for one indication. Of the 12 models, six underpredicted and six overpredicted the annual realised utilisation. There were a range of different parameters included in each of the budget impact models. A probabilistic sensitivity analysis did not improve the characterization of uncertainty. CONCLUSION: This research has demonstrated that budget impact models submitted to a national HTA agency have limited accuracy in predicting realised utilisation, and there is inconsistency among the parameters included. An electronic budget impact template for applicants has been developed, as a more systematic approach, for their submissions to the NCPE.