RESUMEN
ABSTRACTInjectable antiretroviral treatment (ART) represents a new effective and potentially more convenient alternative to oral ART for people living with HIV (PLWH). This study assessed preferences of PLWH for long-acting injectable compared with oral ART in the Netherlands. A labelled discrete choice experiment presented 12 choice sets of long-acting injectable and oral ART. PLWH were asked to select their preferred ART, described by six attributes: location of administration, dosing frequency, risk of short-term side effects, drug-drug interaction, forgivability, and food and mealtime restrictions. Random parameters logit and latent class models were used to estimate preferences of PLWH. 98.6% of 76 respondents were experienced oral ART users that had taken ART for a median of 12 years (Q1-Q3: 7.0-20.0). 30 (39.5%) respondents chose long-acting injectable ART in all choice tasks and 22 (28.9%) always chose oral ART. The random parameter model showed that, on average, respondents significantly favoured long-acting injectable ART over oral ART, preferred administration of the long-acting injectable ART at home, and a less frequent regimen. The latent class model confirmed one class strongly preferring long-acting injectable ART and one class slightly preferring oral ART. This study highlights the value for both long-acting injectable and oral ART.
Asunto(s)
Infecciones por VIH , Humanos , Países Bajos , Infecciones por VIH/tratamiento farmacológico , Prioridad del Paciente , Antirretrovirales/uso terapéutico , Encuestas y CuestionariosRESUMEN
BACKGROUND: The choice between disease-modifying drugs (DMDs) for the treatment of multiple sclerosis (MS) becomes more often a shared decision between the patient and the neurologist and MS nurse. This study aimed to assess which DMD attributes are most important for the healthcare professionals in selecting a DMD for a patient. Subsequently, within this perspective, the neurologists' and nurses' perspectives were compared. Lastly, the healthcare professionals' perspective was compared with the patients' perspective to detect any differences that may need attention in the communication about DMDs. DESIGN: A best-worst scaling (BWS) was conducted among 27 neurologists and 33 MS nurses treating patients with MS to determine the importance of 27 DMD attributes. These attributes were identified through three focus groups with MS patients in a previous study (N=19). Relative importance scores (RISs) were estimated for each attribute. Multivariable linear regression analyses were used to compare the different perspectives. RESULTS: According to the neurologists and nurses, safety of the DMD was the most important DMD attribute in the treatment decision, closely followed by effect on disability progression, quality of life and relapse rate. Patients with MS agreed with the importance of the last three attributes, but valued safety significantly lower (b=-2.59, P<.001). CONCLUSIONS: This study suggests that, overall, neurologists and nurses regard the same DMD attributes as important as MS patients with the notable exception of safety. This study provides valuable information for the development of interventions to support shared decision making and highlights which attributes of DMDs may need additional attention.
Asunto(s)
Toma de Decisiones , Personal de Salud , Esclerosis Múltiple/tratamiento farmacológico , Prioridad del Paciente , Femenino , Grupos Focales , Humanos , Masculino , Persona de Mediana Edad , Calidad de Vida , Recurrencia , Encuestas y CuestionariosRESUMEN
BACKGROUND: Direct-to-patient research via Web-based questionnaires is increasingly being used. Missed data or delayed reporting of data may negatively affect the quality of study results. It is insufficiently known to what degree patients adhere to agreed self-assessment schedule over the long term and whether questionnaires are filled out in a timely manner. OBJECTIVE: The objective of this study was to investigate patients' adherence to a self-assessment schedule with low-frequency long questionnaires versus that with a high-frequency short questionnaire. METHODS: In this study, the 36-item MS Impact Profile (MSIP) questionnaire measured (perceived) disabilities and the 54-item MS Quality of Life-54 (MSQoL-54) questionnaire measured health-related quality of life at 6-month intervals. Additionally, the 2-item Medication and Adherence (MA) questionnaire documented medication and adherence to disease-modifying medication every month. An experienced MS nurse assessed the Expanded Disability Status Scale (EDSS) score via phone. For both the self-assessment schedules, we calculated the percentage of patients who had completed all the questionnaires in the first 2 years (completion adherence), the percentage of patients who completed all the questionnaires within set time frames (interval adherence), the relationship between adherence and the EDSS score, and the timing of EDSS assessment. RESULTS: Of the 331 patients who enrolled themselves, 301 patients completed at least one questionnaire. At month six (M6), M12, M18, and M24, the MSIP was completed by 83.4% (251/301), 71.8% (216/301), 68.1% (205/301), and 58.5% (176/301) of the patients, respectively; the MSQoL-54 by 82.1% (247/301), 71.8% (216/301), 66.8% (201/301), and 57.1% (172/301), respectively; and the MA questionnaire by 80.1% (241/301), 70.4% (212/301), 62.1% (187/301), and 53.5% (161/301), respectively. For the MSIP, 56.8% (171/301) of the patients were 2-year completion adherent; 55.5% (167/301) and 53.5% (161/301) of the patients were completion adherent for the MSQoL-54 and MA questionnaires, respectively. Whereas 85.5% (142/166) of the patients were interval adherent for the MSIP and MSQoL-54, 25.5% (41/161) were interval adherent for the MA questionnaire, with 73.9% (119/161) exceeding the maximum MA monthly interassessment interval. Completion adherence for the monthly short MA questionnaire was higher in patients with moderately high disability (EDSS 5.0-5.5) than for those with no or minimal disability (EDSS 0-2.5) (OR 5.47, 95% CI 1.08-27.69; P=.040). Completion adherence was also higher in patients with EDSS assessment within 6 months after baseline than in those with later assessment (OR 1.810, 95% CI 0.999-3.280; P=.050). CONCLUSIONS: The 2-year completion adherence to Web-based self-assessments did not differ between the low-frequency long questionnaires and a high-frequency short questionnaire, but the interval adherence was substantially higher for the low-frequency long questionnaires. Personal contact with a member of the research team regarding a clinically relevant professional-reported outcome early in the study might positively affect the long-term completion adherence in direct-to-patient studies.
Asunto(s)
Encuestas Epidemiológicas , Internet , Esclerosis Múltiple/diagnóstico , Esclerosis Múltiple/psicología , Cooperación del Paciente/estadística & datos numéricos , Autoevaluación (Psicología) , Adolescente , Adulto , Anciano , Personas con Discapacidad/psicología , Personas con Discapacidad/estadística & datos numéricos , Femenino , Humanos , Masculino , Persona de Mediana Edad , Esclerosis Múltiple/fisiopatología , Calidad de Vida , Adulto JovenRESUMEN
OBJECTIVE: This study assessed the Health-Related Quality of Life (HRQoL) and utilities of Multiple Sclerosis (MS) patients in Lebanon using generic and MS-specific QoL instruments, categorized by disease severity, and explored factors associated with HRQoL. METHODS: This was a cross-sectional, retrospective HRQoL study collecting data through face-to-face interviews using the EQ-5D-5 L and the Multiple Sclerosis International Quality of Life (MusiQoL) questionnaires. We enrolled Lebanese patients aged ≥18 years, diagnosed with MS for >6 months. Patients were categorized by disease severity using the expanded disability status scale (EDSS) scores: 0-3 (mild MS), 4-6.5 (moderate MS), and 7-9 (severe MS). Bivariate and linear regression analyses were performed to study factors associated with HRQoL. RESULTS: A total of 210 patients (mean age: 43.3 years; 65.7 % females) were included. The mean EQ-5D-5 L utility score was 0.74. This score decreased significantly with disease severity (p < 0.001 for the trend): 0.93, 0.60, and 0.32 for mild, moderate, and severe MS, respectively. The mean MusiQoL global index score was 71.33 and was significantly lower for severe MS (58.68), than for moderate (65.23) and mild (77.80), (p < 0.001 for the trend). Higher educational level, lower EDSS scores, and longer disease duration were associated significantly with a higher EQ-5D-5 L utility (R2 = 0.67), while employment, lower EDSS scores, and decrease in cognitive difficulties were associated with better MusiQoL utility (R2 = 0.46). CONCLUSIONS: This study reveals a significant and gradual deterioration in HRQoL as MS progresses, resulting in low utility scores for patients with severe MS.
Asunto(s)
Esclerosis Múltiple , Calidad de Vida , Índice de Severidad de la Enfermedad , Humanos , Femenino , Masculino , Adulto , Líbano , Estudios Transversales , Esclerosis Múltiple/psicología , Esclerosis Múltiple/fisiopatología , Persona de Mediana Edad , Estudios Retrospectivos , Encuestas y CuestionariosRESUMEN
In 2019, we embarked on a study on the economic burden of multiple sclerosis (MS) in Lebanon, in collaboration with a premier Lebanese MS center. This coincided with a triple disaster in Lebanon, comprising the drastic economic and financial crisis, the COVID-19 pandemic, and the consequences of the explosion of Beirut's port. Specifically, the economic and financial turmoil made the valuation of costs challenging. Researchers could face similar challenges, particularly in low- and middle-income countries (LMICs) where economic crises and recessions are recurrent phenomena. This paper aims to discuss steps taken to overcome the fluctuation of the prices of resources to get a valid valuation of societal costs during times of a financial and economic crisis. In the absence of local costing data and guidelines for conducting cost-of-illness (COI) studies, this paper provides empirical recommendations on the valuation of costs that are particularly relevant in LMICs. We recommend (1) clear reporting and justification of the country-specific context, year of costing, assumptions, data sources, and valuation methods, as well as the indicators used to adjust cost for inflation during different periods of fluctuation of prices; (2) collecting prices of each resource from multiple and various sources; (3) conducting a sensitivity analysis; and (4) reporting costs in local currency and Purchasing Power Parity dollars (PPP$). Precision and transparency in reporting prices of resources and their sources are markers of the reliability of the COI studies.
Asunto(s)
COVID-19 , Pandemias , Humanos , Líbano , Reproducibilidad de los Resultados , Economía MédicaRESUMEN
INTRODUCTION: This study assessed the societal costs of multiple sclerosis (MS) in Lebanon, categorized by disease severity. METHODS: This was a cross-sectional, prevalence-based, bottom-up study using a face-to-face questionnaire. Patients were stratified by disease severity using the expanded disability status scale (EDSS); EDSS scores of 0-3, 4-6.5, and 7-9 indicating respectively mild, moderate, and severe MS. All direct medical, nonmedical, and indirect costs related to reduced productivity were accounted for regardless of who bore them. Costs, collected from various sources, were presented in international US dollars (US$) using the purchasing power parity (PPP) conversion rate. RESULTS: We included 210 Lebanese patients (mean age: 43.3 years; 65.7% females). The total annual costs per patient were PPP US$ 33,117 for 2021, 12.4 times higher than the nominal GDP per capita. Direct costs represented 52% (US$ 17,185), direct nonmedical costs 8% (US$ 2,722), and indirect costs 40% (US$ 13, 211) of the mean annual costs. The total annual costs per patient increased with disease severity and were PPP US$ 29,979, PPP US$ 36,125, PPP US$ 39,136 for mild, moderate, and severe MS, respectively. CONCLUSION: This study reveals the huge economic burden of MS on the Lebanese healthcare system and society.
Asunto(s)
Esclerosis Múltiple , Femenino , Humanos , Adulto , Masculino , Esclerosis Múltiple/terapia , Estudios Transversales , Costo de Enfermedad , Líbano , Encuestas y Cuestionarios , Costos de la Atención en Salud , Índice de Severidad de la Enfermedad , Calidad de VidaRESUMEN
This protocol describes the estimation of the societal costs and quality-of-life (QOL) burden of multiple sclerosis (MS) in Lebanon. This cross-sectional, prevalence-based burden-of-illness study was carried out in a premier MS center in Lebanon. We enrolled Lebanese patients aged 18 years and older who had been diagnosed with MS more than 6 months. The study uses a bottom-up approach to estimate the cost-of-illness (COI) and QOL using a retrospective face-to-face interview questionnaire. This resource utilization questionnaire was adapted to the Lebanese context by clinical and health economics experts. The methodologies used to estimate the consumption of healthcare resources, informal care, and productivity losses are well-defined and aligned with the Lebanese healthcare system. Costs are presented overall and by MS severity levels. QOL is measured using the EuroQOL (EQ-5D-5 L) and Multiple Sclerosis International Quality of Life (MusiQoL) instrument. This protocol pioneers in informing the design of future COI and QOL studies in low - and middle-income countries (LMICs), as the methods used could be applied in similar LMICs. Furthermore, we provide recommendations and discuss the challenges of conducting a high-quality burden-of-illness study in LMICs and the steps taken to meet them, using the case of Lebanon.
Asunto(s)
Esclerosis Múltiple , Calidad de Vida , Cuidadores , Costo de Enfermedad , Estudios Transversales , Costos de la Atención en Salud , Humanos , Esclerosis Múltiple/terapia , Estudios RetrospectivosRESUMEN
BACKGROUND: Although the economic burden of multiple sclerosis (MS) in high-income countries (HICs) has been extensively studied, information on the costs of MS in low- and middle-income countries (LMICs) remains scarce. Moreover, no review synthesizing and assessing the costs of MS in LMICs has yet been undertaken. OBJECTIVE: Our objective was to systematically identify and review the cost of illness (COI) of MS in LMICs to critically appraise the methodologies used, compare cost estimates across countries and by level of disease severity, and examine cost drivers. METHODS: We conducted a systematic literature search for original studies in English, French, and Dutch containing prevalence or incidence-based cost data of MS in LMICs. The search was conducted in MEDLINE (Ovid), PubMed, Embase (Ovid), Cochrane Library, National Health Service Economic Evaluation Database (NHS EED), Econlit, and CINAHL (EBSCO) on July 2020 without restrictions on publication date. Recommended and validated methods were used for data extraction and analysis to make the results of the COI studies comparable. Costs were adjusted to $US, year 2019 values, using the World Bank purchasing power parity and inflated using the consumer price index. RESULTS: A total of 14 studies were identified, all of which were conducted in upper-middle-income economies. Eight studies used a bottom-up approach for costing, and six used a top-down approach. Four studies used a societal perspective. The total annual cost per patient ranged between $US463 and 58,616. Costs varied across studies and countries, mainly because of differences regarding the inclusion of costs of disease-modifying therapies (DMTs), the range of cost items included, the methodological choices such as approaches used to estimate healthcare resource consumption, and the inclusion of informal care and productivity losses. Characteristics and methodologies of the included studies varied considerably, especially regarding the perspective adopted, cost data specification, and reporting of costs per severity levels. The total costs increased with greater disease severity. The cost ratios between different levels of MS severity within studies were relatively stable; costs were around 1-1.5 times higher for moderate versus mild MS and about two times higher for severe versus mild MS. MS drug costs were the main cost driver for less severe MS, whereas the proportion of direct non-medical costs and indirect costs increased with greater disease severity. CONCLUSION: MS places a huge economic burden on healthcare systems and societies in LMICs. Methodological differences and substantial variations in terms of absolute costs were found between studies, which made comparison of studies challenging. However, the cost ratios across different levels of MS severity were similar, making comparisons between studies by disease severity feasible. Cost drivers were mainly DMTs and relapse treatments, and this was consistent across studies. Yet, the distribution of cost components varied with disease severity.
Asunto(s)
Costo de Enfermedad , Esclerosis Múltiple , Países en Desarrollo , Costos de los Medicamentos , Costos de la Atención en Salud , Humanos , Medicina EstatalRESUMEN
BACKGROUND: Considering the heavy economic burden of osteoporotic fractures, the limits of healthcare resources, and the recent availability of new anti-osteoporosis drugs, there is continuing interest in economic evaluation studies of osteoporosis management strategies. OBJECTIVES: This study aims to (1) systematically review recent economic evaluations of drugs for osteoporosis and (2) to apply an osteoporosis-specific guideline to critically appraise them. METHODS: A literature search was undertaken using PubMed, EMBASE, National Health Service Economic Evaluation database, and the Cost-Effectiveness Analysis Registry to identify original articles containing economic evaluations of anti-osteoporosis drugs, published between 1 July, 2013 and 31 December, 2019. A recent European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases-International Osteoporosis Foundation (ESCEO-IOF) guideline for the conduct and reporting of economic evaluations in osteoporosis was used to assess the quality of included articles. RESULTS: The database search retrieved 3860 records, of which 27 studies fulfilled the inclusion criteria. These studies were conducted in 15 countries; 12 active drugs were assessed, including various traditional pharmacological treatments such as bisphosphonates, raloxifene, strontium ranelate, denosumab, and teriparatide, and new agents such as abaloparatide, romosozumab, and gastro-resistant risedronate. Eight out of 12 studies that compared traditional oral bisphosphonates to other active interventions (denosumab, zoledronic acid, gastro-resistant risedronate, and teriparatide) suggested that the other active agents were generally cost-effective or dominant. Additionally, the cost-effectiveness of sequential therapy has recently been assessed and indications are that it can lead to extra health benefits (larger gains in quality-adjusted life-year). The key drivers of cost effectiveness included baseline fracture risk, drug effect on the risk of fractures, drug cost, and medication adherence/persistence. The current average score for quality assessment was 17 out of 25 (range 2-15); room for improvement was observed for most studies, which could potentially be explained by the fact that most studies were published prior to the osteoporosis-specific guideline. Greater adherence to guideline recommendations was expected for future studies. The quality of reporting was also suboptimal, especially with regard to treatment side effects, treatment effect after discontinuation, and medication adherence. CONCLUSIONS: This updated review provides an overview of recently published cost-effectiveness analyses. In comparison with a previous review, recent economic evaluations of anti-osteoporosis drugs were conducted in more countries and included more active drugs and sequential therapy as interventions/comparators. The updated economic evidence could help decision makers prioritize health interventions and the unmet/unreported quality issues indicated by the osteoporosis-specific guideline could be useful in improving the transparency, quality, and comparability of future economic evaluations in osteoporosis.
Asunto(s)
Osteoporosis , Fracturas Osteoporóticas , Preparaciones Farmacéuticas , Análisis Costo-Beneficio , Humanos , Osteoporosis/tratamiento farmacológico , Fracturas Osteoporóticas/prevención & control , Medicina EstatalRESUMEN
BACKGROUND: Up to 31% of patients with relapsing-remitting multiple sclerosis (RRMS) discontinue treatment with disease-modifying drug (DMD) within the first year, and of the patients who do continue, about 40% are nonadherent. Shared decision making may decrease nonadherence and discontinuation rates, but evidence in the context of RRMS is limited. Shared decision making may, however, come at additional costs. This study aimed to explore the potential cost-effectiveness of shared decision making for RRMS in comparison with usual care, from a (limited) societal perspective over a lifetime. METHODS: An exploratory economic evaluation was conducted by adapting a previously developed state transition model that evaluates the cost-effectiveness of a range of DMDs for RRMS in comparison with the best supportive care. Three potential effects of shared decision making were explored: 1) a change in the initial DMD chosen, 2) a decrease in the patient's discontinuation in using the DMD, and 3) an increase in adherence to the DMD. One-way and probabilistic sensitivity analyses of a scenario that combined the 3 effects were conducted. RESULTS: Each effect separately and the 3 effects combined resulted in higher quality-adjusted life years (QALYs) and costs due to the increased utilization of DMD. A decrease in discontinuation of DMDs influenced the incremental cost-effectiveness ratio (ICER) most. The combined scenario resulted in an ICER of 17,875 per QALY gained. The ICER was sensitive to changes in several parameters. CONCLUSION: This study suggests that shared decision making for DMDs could potentially be cost-effective, especially if shared decision making would help to decrease treatment discontinuation. Our results, however, may depend on the assumed effects on treatment choice, persistence, and adherence, which are actually largely unknown.
Asunto(s)
Análisis Costo-Beneficio/normas , Toma de Decisiones Conjunta , Cumplimiento de la Medicación/psicología , Esclerosis Múltiple Recurrente-Remitente/tratamiento farmacológico , Esclerosis Múltiple Recurrente-Remitente/economía , Análisis Costo-Beneficio/tendencias , Humanos , Cadenas de Markov , Cumplimiento de la Medicación/estadística & datos numéricos , Esclerosis Múltiple Recurrente-Remitente/psicología , Países Bajos , Años de Vida Ajustados por Calidad de VidaRESUMEN
Aims: Different methods have been used to analyze "object case" best-worst scaling (BWS). This study aims to compare the most common statistical analysis methods for object case BWS (i.e. the count analysis, multinomial logit, mixed logit, latent class analysis, and hierarchical Bayes estimation) and to analyze their potential advantages and limitations based on an applied example. Methods: Data were analyzed using the five analysis methods. Ranking results were compared among the methods, and methods that take respondent heterogeneity into account were presented specifically. A BWS object case survey with 22 factors was used as a case study, tested among 136 policy-makers and HTA experts from the Netherlands, Germany, France, and the UK to assess the most important barriers to HTA usage. Results: Overall, the five statistical methods yielded similar rankings, particularly in the extreme ends. Latent class analysis identified five clusters and the mixed logit model revealed significant preference heterogeneity for all, with the exception of three factors. Limitations: The variety of software used to analyze BWS data may affect the results. Moreover, this study focuses solely on the comparison of different analysis methods for the BWS object case. Conclusions: The most common statistical methods provide similar rankings of the factors. Therefore, for main preference elicitation, count analysis may be considered as a valid and simple first-choice approach. However, the latent class and mixed logit models reveal additional information: identifying latent segments and/or recognizing respondent heterogeneity.
Asunto(s)
Interpretación Estadística de Datos , Evaluación de la Tecnología Biomédica/métodos , Teorema de Bayes , Toma de Decisiones , Europa (Continente) , Política de Salud , Humanos , Análisis de Clases Latentes , Modelos LogísticosRESUMEN
OBJECTIVES: Understanding the preferences of patients with multiple sclerosis (MS) for disease-modifying drugs and involving these patients in clinical decision making can improve the concordance between medical decisions and patient values and may, subsequently, improve adherence to disease-modifying drugs. This study aims first to identify which characteristics-or attributes-of disease-modifying drugs influence patients´ decisions about these treatments and second to quantify the attributes' relative importance among patients. METHODS: First, three focus groups of relapsing-remitting MS patients were formed to compile a preliminary list of attributes using a nominal group technique. Based on this qualitative research, a survey with several choice tasks (best-worst scaling) was developed to prioritize attributes, asking a larger patient group to choose the most and least important attributes. The attributes' mean relative importance scores (RIS) were calculated. RESULTS: Nineteen patients reported 34 attributes during the focus groups and 185 patients evaluated the importance of the attributes in the survey. The effect on disease progression received the highest RIS (RIS = 9.64, 95% confidence interval: [9.48-9.81]), followed by quality of life (RIS = 9.21 [9.00-9.42]), relapse rate (RIS = 7.76 [7.39-8.13]), severity of side effects (RIS = 7.63 [7.33-7.94]) and relapse severity (RIS = 7.39 [7.06-7.73]). Subgroup analyses showed heterogeneity in preference of patients. For example, side effect-related attributes were statistically more important for patients who had no experience in using disease-modifying drugs compared to experienced patients (p < .001). CONCLUSIONS: This study shows that, on average, patients valued effectiveness and unwanted effects as most important. Clinicians should be aware of the average preferences but also that attributes of disease-modifying drugs are valued differently by different patients. Person-centred clinical decision making would be needed and requires eliciting individual preferences.
Asunto(s)
Toma de Decisiones , Esclerosis Múltiple/tratamiento farmacológico , Adulto , Conducta de Elección , Femenino , Grupos Focales , Humanos , Masculino , Persona de Mediana EdadRESUMEN
Choroiditis serpiginosa is an infrequent disorder, which is chronic, progressive, bilateral, and recurrent; in primary form it affects the pigmented epithelium of the retina and the choriocapillary layer within the eye. It typically begins juxtapapillary and progresses to extend centrifugally to the optic disc, usually affecting the macula and thus decreasing visual acuity. It's etiology is unknown and it debutes in acute form in adult patients, without preference for sex. It's treatment includes the use of systemic corticosteroids, non steroidal anti-inflammatory drug (NSAID), and immunosuppressant agents. In this paper we document the clinical case of a diabetic, 61 year old man, having the disease in an inactive phase with serious visual sequelae.
Coroiditis serpiginosa es un desorden poco frecuente, crónico, progresivo, bilateral y recurrente que efecta en forma primaria al epitelio pigmentario de la retina y la coriocapilar. Típicamente comienza yuxtapapilar y su progresión se extiende en forma centrífuga al disco óptico, afectando usualmente la mácula, disminuyendo así la agudeza visual. Su etiología es desconocida y debuta en forma aguda en pacientes de edad madura sin predilección por sexo. El tratamiento incluye el uso de corticoesteroides sistémicos, antiinflamatorios no esteroidales (AINES) y agentes inmunosupresores. Presentamos el caso clínico de un hombre diabético de 61 años con enfermedad en fase inactiva con secuelas visuales serias.