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1.
J Mark Access Health Policy ; 7(1): 1648973, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-31489150

RESUMO

Background and Objectives: Utility elicitation studies for schizophrenia generate different utility values for the same health states. We reviewed utility values used in schizophrenia pharmacoeconomic evaluations and evaluated the impact of their selection on the incremental cost-effectiveness ratio (ICER). Methods: A systematic search was performed in Medline and Embase. Health state definitions, associated utility values, elicitation studies, and value selection processes were extracted. Sets of utility values for all schizophrenia health states were used in a cost-effectiveness model to evaluate the ICER. Results: Thirty-five cost-utility analyses (CUAs) referring to 11 utility elicitation studies were included. The most frequent health states were 'stable' (28 CUAs, 7 utility elicitation studies, 10 values, value range 0.650-0.919), 'relapse requiring hospitalisation' (18, 5, 7, 0.270-0.604), 'relapse not requiring hospitalisation' (18, 5, 10, 0.460-0.762), and 'relapse only' (10, 5, 6, 0.498-0.700). Seventeen sets of utility values were identified with difference in utility values between relapse and stable ranging from -0.358 to -0.050, resulting in ICERs ranging from -56.2% to +222.6% from average. Conclusion: The use of utility values for schizophrenia health states differs among CUAs and impacts on the ICER. More rigorous and transparent use of utility values and sensitivity analysis with different sets of utility values are suggested for future CUAs.

2.
J Mark Access Health Policy ; 7(1): 1618661, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-31156762

RESUMO

The importance of understanding the impact of disease and treatment on children's Health-Related Quality of Life (HRQoL) has given rise to an increasing use of child self-report and observer or proxy instruments. In this article, we review the status quo and challenges of HRQoL measurement specific to children under five. A number of HRQoL questionnaires exist for use with children and/or proxies, and both guidelines and reviews have been published on paediatric HRQoL. However, none address the challenges of measurement for children under five, for whom proxy measures should be used. In reality, there is significant heterogeneity in the cut-off age for self-report questionnaires. Recommendations are that proxies should be used for observable concepts, but not for concepts that require interpretation. Some research has been undertaken on dimensions/concepts in paediatric HRQoL questionnaires. However, no HRQoL models have been developed specifically for children, and heterogeneity in questionnaire dimensions underlines that there is no clear grasp of what HRQoL means in paediatric populations. There is a need to carry out research in order to develop theoretical models of HRQoL that are specific to children at different developmental stages, in order to evaluate and support new and existing measures for paediatric HRQoL and their use in clinical practice as well as clinical trials.

3.
Curr Med Res Opin ; 35(8): 1355-1364, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-30810054

RESUMO

Objective: Baloxavir marboxil (baloxavir) is the first cap-dependent endonuclease inhibitor being studied for the treatment of influenza in single oral dosing regimen. This network meta-analysis (NMA) evaluated the efficacy and safety of baloxavir compared to other antivirals for influenza in otherwise healthy patients. Methods: A systematic literature review was performed on 14 November 2016 in Medline, Embase, CENTRAL, and ICHUSHI to identify randomized controlled trials assessing antivirals for influenza. A NMA including 22 trials was performed to compare the efficacy and safety of baloxavir with other antivirals. Results: The time to alleviation of all symptoms was significantly shorter for baloxavir compared to zanamivir (difference in median time 19.96 h; 95% CrI [3.23, 39.07]). The time to cessation of viral shedding was significantly shorter for baloxavir than zanamivir and oseltamivir (47.00 h; 95% CrI [28.18, 73.86] and 56.03 h [33.74, 87.86], respectively). The mean decline in virus titer from baseline to 24 h was significantly greater for baloxavir than for the other drugs. Other differences in efficacy outcomes were not significant. No significant differences were found between baloxavir and the other antivirals for safety, except total drug-related adverse events where baloxavir demonstrated a decrease compared to oseltamivir and laninamivir. Conclusions: The NMA suggests that baloxavir demonstrated better or similar efficacy results compared to other antivirals with a comparable safety profile. Baloxavir led to a significant decrease in viral titer versus zanamivir, oseltamivir and peramivir and decreased viral shedding versus zanamivir and oseltamivir.

4.
Curr Med Res Opin ; 35(5): 785-792, 2019 05.
Artigo em Inglês | MEDLINE | ID: mdl-30681007

RESUMO

OBJECTIVES: Understanding inhaler preferences may contribute to improving adherence in COPD patients and improving long-term outcomes. This study aims to identify and quantify preferences for convenience-related inhaler attributes in French moderate-to-severe COPD patients, with discrete choice experiment (DCE) methodology. METHODS: Attributes were defined from a literature search, clinician and patient interviews: shape, dose insertion, dose preparation, dose release, dose confirmation, dose counter and reusability. An online DCE was conducted in respondents with self-reported COPD stage 2-4 recruited through a panel. The study questionnaire included twelve choice scenarios per respondent and questions on patient characteristics, treatment and disease severity. Statistical analyses used a mixed logit regression model with random effects. Utility scores were estimated for four types of inhalers: Inhaler A - soft mist inhaler; Inhaler B - reusable soft mist inhaler; Inhaler C - multi-dose dry powder inhaler; and Inhaler D - single dose dry powder inhaler. RESULTS: The study was completed by 153 patients (50 females); respondents were 50.4 years old on average; 13 different inhaler devices were reported. The most preferred inhaler is L-shaped, has dose preparation with capsule insertion and a dose counter, and is reusable. Inhaler profiles A and B had the highest utilities (mean of 1.2533 and 0.9578 respectively) compared to inhaler C (0.6315) and D (0.2200). CONCLUSIONS: This study showed statistically significant results that the strongest drivers of preference in French users of inhalation devices for COPD are shape, dose counter and reusability. Convenience-related characteristics are important to patients and should be taken into account by clinicians prescribing these devices.

5.
BMC Health Serv Res ; 19(1): 22, 2019 Jan 09.
Artigo em Inglês | MEDLINE | ID: mdl-30626376

RESUMO

BACKGROUND: Diabetic macular oedema (DMO) may lead to visual loss and blindness. Several pharmacological treatments are available on the National Health Service (NHS) to United Kingdom patients affected by this condition, including intravitreal vascular endothelial growth factor inhibitors (anti-VEGFs) and two types of intravitreal steroid implants, releasing dexamethasone or fluocinolone acetonide (FAc). This study aimed to assess the value for money (cost-effectiveness) of the FAc 0.2 µg/day implant (ILUVIEN®) in patients with chronic DMO considered insufficiently responsive to other therapies. METHODS: We developed a Markov model with a 15-year time horizon to estimate the impact of changes in best-corrected visual acuity in DMO patients on costs and quality-adjusted life years. The model considered both eyes, designated as the "study eye", defined at model entry as phakic with an ongoing cataract formation or pseudophakic, and the "fellow eye". The model compared the FAc 0.2 µg/day implant with a 700 µg dexamethasone implant (pseudophakic patients only) or with usual care, defined as a mixture of laser photocoagulation and anti-VEGFs (phakic and pseudophakic patients). Costs were estimated from the perspective of the NHS and Personal Social Services; full NHS prices were used for drugs. RESULTS: In patients who were pseudophakic at baseline, at 36 months, the FAc implant provided an additional gain of 4.01 and 3.64 Early Treatment Diabetic Retinopathy Study (ETDRS) letters compared with usual care and the dexamethasone implant, respectively. Over the 15-year time horizon, this translated into 0.185 additional quality-adjusted life years (QALYs) at an extra cost of £3066 compared with usual care, and 0.126 additional QALYs at an extra cost of £1777 compared with dexamethasone. Thus, incremental cost-effectiveness ratios (ICERs) were £16,609 and £14,070 per QALY gained vs. usual care and dexamethasone, respectively. In patients who were phakic at baseline, the FAc 0.2 µg/day implant provided an additional gain of 2.96 ETDRS letters at 36 months compared with usual care, which, over 15 years, corresponded to 0.11 additional QALYs at an extra cost of £3170, resulting in an ICER of £28,751 per QALY gained. CONCLUSION: The FAc 0.2 µg/day implant provided good value for money compared with other established treatments, especially in pseudophakic patients.


Assuntos
Retinopatia Diabética/tratamento farmacológico , Fluocinolona Acetonida/administração & dosagem , Glucocorticoides/administração & dosagem , Edema Macular/tratamento farmacológico , Análise Custo-Benefício , Retinopatia Diabética/economia , Retinopatia Diabética/fisiopatologia , Implantes de Medicamento , Fluocinolona Acetonida/economia , Glucocorticoides/economia , Humanos , Edema Macular/economia , Edema Macular/fisiopatologia , Anos de Vida Ajustados por Qualidade de Vida , Reino Unido
6.
J Mark Access Health Policy ; 6(1): 1438721, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-29686801

RESUMO

Objective: To evaluate the cost-effectiveness of solifenacin 5 mg/day versus other oral antimuscarinic agents used for overactive bladder (OAB) from a UK National Health Service (NHS) perspective. Study design: In a Markov model, hypothetical patients received solifenacin 5 mg/day or a comparator antimuscarinic, after which they could switch to an alternative antimuscarinic. The model estimated incremental cost-effectiveness ratios (ICER), expressed as cost per quality-adjusted life year (QALY) over a 5-year period. Results: Solifenacin 5 mg/day was the dominant treatment strategy (i.e., less costly and more effective) versus tolterodine extended-release (ER) 4 mg/day, fesoterodine 4 and 8 mg/day, oxybutynin ER 10 mg/day and solifenacin 10 mg/day, and was cost-effective (i.e., ICERs below the £30,000 per QALY threshold generally applied in the NHS) versus oxybutynin immediate release (IR) 10 mg/day, tolterodine IR 4 mg/day and trospium chloride 60 mg/day. The probability of solifenacin 5 mg/day being dominant/cost-effective at a willingness-to-pay threshold of £30,000 per QALY was 57-98%. Conclusions: Solifenacin 5 mg/day appears to be a cost-effective strategy for the treatment of OAB over a 5-year timeframe compared with other oral antimuscarinic agents in the UK. These findings are important for decision-makers considering the economic implications of selecting treatments for OAB.

7.
Eur Urol ; 74(3): 324-333, 2018 09.
Artigo em Inglês | MEDLINE | ID: mdl-29699858

RESUMO

BACKGROUND: Mirabegron is an established treatment alternative to antimuscarinic therapy for patients with overactive bladder (OAB), as shown by efficacy and tolerability data from phase III trials. OBJECTIVE: To assess efficacy and tolerability of mirabegron 50mg versus antimuscarinic monotherapies and combination therapies. DESIGN, SETTING, AND PARTICIPANTS: Systematic literature review and network meta-analysis of randomised controlled trials (2000-2017) assessing eligible treatments for OAB. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: Efficacy assessments included micturition frequency, urgency urinary incontinence, dry rate, and 50% reduction in incontinence. Tolerability assessments included dry mouth, constipation, blurred vision, and hypertension. RESULTS AND LIMITATIONS: A total of 64 studies (n=46 666) were included in the network meta-analysis. Mirabegron 50mg was significantly more efficacious than placebo for all efficacy endpoints. Comparable overall efficacy was observed for mirabegron 50mg versus most active treatments, but solifenacin 10mg monotherapy and solifenacin 5mg plus mirabegron 25 or 50mg in combination were more efficacious for some/all outcomes. Mirabegron 50mg was significantly better tolerated regarding dry mouth, constipation, and urinary retention than 21/22, 9/20, and 7/10 active comparators, respectively; similar overall tolerability was observed between mirabegron 50mg and all treatments (including placebo) for the remaining endpoints. Limitations of the study included between-trial variations in the definition of certain endpoints and heterogeneity of the available data (eg, number of studies and patients assessed) for comparator treatments across different endpoints. CONCLUSIONS: The relief of key OAB symptoms produced by mirabegron 50mg is significantly better than placebo, and similar to a range of common antimuscarinics, with the benefit of significantly fewer bothersome anticholinergic side effects such as dry mouth. Combination treatment of solifenacin 5mg plus mirabegron 25 or 50mg appears to provide an efficacy benefit compared with mirabegron 50mg, with the expected side effects of individual antimuscarinics. PATIENT SUMMARY: This study assessed the efficacy and tolerability of different drug treatments for OAB. Mirabegron 50mg was as effective as antimuscarinic therapy, with fewer common, bothersome side effects such as dry mouth, constipation, and urinary retention. Combination treatment of solifenacin 5mg plus mirabegron 25 or 50mg was more effective than mirabegron 50mg alone, but with more anticholinergic side effects.


Assuntos
Acetanilidas/uso terapêutico , Agonistas de Receptores Adrenérgicos beta 3/uso terapêutico , Antagonistas Muscarínicos/uso terapêutico , Tiazóis/uso terapêutico , Bexiga Urinária Hiperativa/tratamento farmacológico , Bexiga Urinária/efeitos dos fármacos , Urodinâmica/efeitos dos fármacos , Agentes Urológicos/uso terapêutico , Acetanilidas/efeitos adversos , Agonistas de Receptores Adrenérgicos beta 3/efeitos adversos , Quimioterapia Combinada , Humanos , Antagonistas Muscarínicos/efeitos adversos , Recuperação de Função Fisiológica , Tiazóis/efeitos adversos , Resultado do Tratamento , Bexiga Urinária/fisiopatologia , Bexiga Urinária Hiperativa/diagnóstico , Bexiga Urinária Hiperativa/fisiopatologia , Agentes Urológicos/efeitos adversos
8.
Eye (Lond) ; 32(6): 1028-1035, 2018 06.
Artigo em Inglês | MEDLINE | ID: mdl-29386666

RESUMO

AIMS: To analyze the risk of incident cataract (diagnosis or extraction) in patients with or without diabetes focusing on other comorbid conditions, antidiabetic drug use, and diabetes duration. METHODS: The study population comprised newly diagnosed diabetes patients (≥40 years) from the UK-based Clinical Practice Research Datalink (CPRD) between 2000 and 2015, and a random sample of the general population matched for age, sex, general practice, and year of diabetes diagnosis. We assessed cataract incidence rates (IRs) and performed a nested case-control analysis in the diabetic cohort to assess potential risk factors for a cataract. RESULTS: There were 56,510 diabetes patients included in the study. IRs of cataract were 20.4 (95% CI 19.8-20.9) per 1000 person-years (py) in patients with diabetes and 10.8 (95% CI 10.5-11.2) per 1000 py in the general population. IRs increased considerably around the age of 80 years and with a concomitant diagnosis of macular edema. The incidence rate ratio (IRR) was highest in patients of the age group of 45-54 years. In the nested case-control study, we identified 5800 patients with cataract. Risk of cataract increased with increasing diabetes duration (adj. OR 5.14, 95% CI 4.19-6.30 diabetes for ≥10 years vs. diabetes <2 years). CONCLUSIONS: According to our study, diabetes is associated with an approximately two-fold increased detection rate of cataract. The risk of cataract associated with diabetes is highest at younger ages. Patients with diabetic macular edema are at an increased risk for cataract as well as patients with long-standing diabetes.


Assuntos
Catarata/epidemiologia , Diabetes Mellitus Tipo 2/complicações , Adulto , Idoso , Idoso de 80 Anos ou mais , Estudos de Casos e Controles , Feminino , Humanos , Hipoglicemiantes/administração & dosagem , Incidência , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Fatores de Risco , Reino Unido/epidemiologia
9.
Neurourol Urodyn ; 37(3): 986-996, 2018 03.
Artigo em Inglês | MEDLINE | ID: mdl-29140559

RESUMO

AIMS: To compare efficacy and tolerability of solifenacin 5 mg/day versus other oral antimuscarinic agents for the treatment of overactive bladder (OAB). METHODS: Literature searches of MEDLINE, Embase, and the Cochrane Library were undertaken to identify randomized controlled trials in OAB (2000-2015) for antimuscarinic agents. A network meta-analysis (NMA) was performed to estimate efficacy and tolerability outcomes for solifenacin 5 mg/day relative to other antimuscarinics. RESULTS: The NMA included 53 eligible trials (published, n = 48; unpublished on search date, n = 5). Solifenacin 5 mg/day was significantly more effective than tolterodine 4 mg/day for reducing incontinence and urgency urinary incontinence (UUI) episodes, but significantly less effective than solifenacin 10 mg/day for micturition; no other statistically significant differences were noted for efficacy. Solifenacin 5 mg/day had a statistically significant lower risk of dry mouth compared with darifenacin 15 mg/day, fesoterodine 8 mg/day, oxybutynin extended-release 10 mg/day, oxybutynin immediate-release (IR) 9-15 mg/day, tolterodine IR 4 mg/day, propiverine 20 mg/day, and solifenacin 10 mg/day. There were no significant differences between solifenacin 5 mg/day and other antimuscarinics for risk of blurred vision, or for 11 of 17 active comparators for risk of constipation. CONCLUSIONS: This NMA suggests that the efficacy of solifenacin 5 mg/day is at least similar to other common antimuscarinics across the spectrum of OAB symptoms analyzed, and is more effective than tolterodine 4 mg/day in reducing incontinence and UUI episodes. Solifenacin 5 mg/day has a lower risk of dry mouth compared with several agents.


Assuntos
Antagonistas Muscarínicos/uso terapêutico , Succinato de Solifenacina/uso terapêutico , Bexiga Urinária Hiperativa/tratamento farmacológico , Compostos Benzidrílicos/efeitos adversos , Compostos Benzidrílicos/uso terapêutico , Benzilatos/efeitos adversos , Benzilatos/uso terapêutico , Humanos , Ácidos Mandélicos/efeitos adversos , Ácidos Mandélicos/uso terapêutico , Antagonistas Muscarínicos/efeitos adversos , Meta-Análise em Rede , Succinato de Solifenacina/efeitos adversos , Tartarato de Tolterodina/efeitos adversos , Tartarato de Tolterodina/uso terapêutico , Resultado do Tratamento
10.
J Stud Alcohol Drugs ; 78(6): 867-876, 2017 11.
Artigo em Inglês | MEDLINE | ID: mdl-29087821

RESUMO

OBJECTIVE: A microsimulation model was adapted to evaluate the cost-effectiveness of nalmefene combined with psychosocial support (NMF + PS) versus psychosocial support alone (PS). The economic impact of alcohol reduction using nalmefene treatment was not evaluated. METHOD: The model simulates patient-level alcohol consumption over a 5-year time horizon across different treatment cohorts. Study outcomes included probabilities of alcohol-attributable diseases and injuries as well as deaths from these events. The approach used nalmefene clinical trial data, a time horizon of 1 and 5 years, and a U.K. societal perspective. Extensive deterministic and probabilistic sensitivity analyses were conducted. RESULTS: Compared with the PS strategy, NMF + PS was associated at Year 5 with a gain of 0.047 quality-adjusted life years (QALYs) and an additional £503, leading to an incremental cost-effectiveness ratio (ICER) of £10,613 per QALY gained. When compared with the strategy without treatment, NMF + PS was associated with a gain of 0.228 QALYs and an additional £1,795, leading to an ICER of £1,758 per QALY gained. The NMF + PS strategy dominated both treatment strategies when considering the U.K. societal perspective. Sensitivity analyses confirmed the robustness of the results. CONCLUSIONS: A combination of NMF and PS was better than PS alone, considering a 5-year time horizon and a societal perspective.


Assuntos
Consumo de Bebidas Alcoólicas/prevenção & controle , Alcoolismo/terapia , Naltrexona/análogos & derivados , Anos de Vida Ajustados por Qualidade de Vida , Consumo de Bebidas Alcoólicas/economia , Análise Custo-Benefício , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Naltrexona/administração & dosagem , Risco
11.
J Mark Access Health Policy ; 5(1): 1344088, 2017.
Artigo em Inglês | MEDLINE | ID: mdl-28804600

RESUMO

Background: Despite the guidelines for Economic and Public Health Assessment Committee (CEESP) submission having been available for nearly six years, the dossiers submitted continue to deviate from them, potentially impacting product prices. Objective: to review the reports published by CEESP, analyse deviations from the guidelines, and discuss their implications for the pricing and reimbursement process. Study design: CEESP reports published until January 2017 were reviewed, and deviations from the guidelines were extracted. The frequency of deviations was described by type of methodological concern (minor, important or major). Results: In 19 reports, we identified 243 methodological concerns, most often concerning modelling, measurement and valuation of health states and results presentation and sensitivity analyses; nearly 63% were minor, 33% were important and 4.5% were major. All reports included minor methodological concerns, and 17 (89%) included at least one important and/or major methodological concern. Global major methodological concerns completely invalidated the analysis in seven dossiers (37%). Conclusion: The CEESP submission dossiers fail to adhere to the guidelines, potentially invalidating the health economics analysis and resulting in pricing negotiations. As these negotiations tend to be unfavourable for the manufacturer, the industry should strive to improve the quality of the analyses submitted to CEESP.

12.
BMC Pulm Med ; 17(1): 99, 2017 Jul 06.
Artigo em Inglês | MEDLINE | ID: mdl-28683805

RESUMO

BACKGROUND: Dry powder inhalers (DPIs) are often used in asthma and chronic obstructive pulmonary disease (COPD) therapies. Using the discrete choice experiment (DCE) methodology, this study conducted in France was designed to assess patients' preferences for different attributes of DPIs. METHODS: Attributes of DPIs were defined based on a literature review, patient focus group discussions and interviews with healthcare professionals (qualitative phase of the study). An online survey was then conducted among French patients with asthma or COPD to elicit patient preferences and willingness to pay (WTP) for these attributes using the DCE methodology (quantitative phase). A fractional factorial design including three blocks of 12 choice sets was created. Each choice set comprised three alternatives: two fictitious inhalers and the patient's current inhaler. Marginal utilities were estimated using a ranked ordered logit model. Interactions between attributes and disease (asthma or COPD) were tested. RESULTS: Six DPI attributes were defined based on the qualitative phase: ease of use/fool-proof priming; accurate and easy-to-read dose counter; dose confirmation; hygiene of the mouthpiece; flexibility of the device handling; ability to use the inhaler with breathing difficulties. Overall, 201 patients with asthma and 93 with COPD were included in the online survey. Patients with asthma placed most value on an inhaler that requires one step for dose preparation (WTP €4.83 [95% CI: €3.77-€5.90], relative to an inhaler requiring four steps) and one that could be used during episodes of breathing difficulties (WTP €4.49 [95% CI: €2.95-€6.02]). Patients with COPD placed most value on an inhaler that could be used during episodes of breathing difficulties (WTP €7.70 [95% CI: €5.65-€9.76]) and on the accuracy of the dose counter (WTP €5.87 [95% CI: €3.98-€ 7.77]). CONCLUSION: This study suggests that asthma and COPD patients would be willing to change their inhaler if they were offered the option of a new inhaler with improved characteristics and they place a high value on an inhaler with ease of use during breathing difficulty episodes.


Assuntos
Asma/tratamento farmacológico , Inaladores de Pó Seco , Preferência do Paciente , Doença Pulmonar Obstrutiva Crônica/tratamento farmacológico , Adulto , Idoso , Idoso de 80 Anos ou mais , Comportamento de Escolha , Inaladores de Pó Seco/economia , Desenho de Equipamento , Feminino , Grupos Focais , França , Humanos , Masculino , Pessoa de Meia-Idade , Preferência do Paciente/economia , Literatura de Revisão como Assunto , Inquéritos e Questionários , Adulto Jovem
13.
Transpl Infect Dis ; 19(5)2017 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-28599091

RESUMO

BACKGROUND: We investigated the impact of early- (E-CMV) and late onset (L-CMV) cytomegalovirus disease on the probability of graft rejection, graft failure, mortality, and healthcare resource use, following solid organ transplantation (SOT) in France. METHODS: A retrospective analysis of data from the French 'Programme de Médicalisation des Systèmes d'Information' database (2007-2011) was conducted to identify SOT recipients who developed CMV disease in an inpatient setting. Recipients were stratified by time to CMV disease onset: E-CMV (≤3 months), L-CMV-3M (>3-24 months), and L-CMV-6M (>6-24 months). Data were analyzed by comparing recipients with CMV disease or without (controls) in a 1:2 ratio, matched according to age, gender, target organ, and previous/simultaneous occurrence of graft rejection. Graft failure, graft rejection, all-cause in-hospital mortality, and resource utilization (including hospitalization costs) were assessed over 12 months following CMV disease diagnosis. RESULTS: Among 20 473 SOT recipients, 2430 (11.86%) were reported to have CMV disease within 24 months after transplantation. CMV disease was significantly associated with an increased risk of graft rejection and mortality, as indicated by logistic regression analysis. Odd ratios (ORs) for the risk of graft rejection were E-CMV=1.43, L-CMV-3M=1.50, and L-CMV-6M=1.61 (all P<.05), while ORs for mortality were E-CMV=2.85, L-CMV-3M=4.22, and L-CMV-6M=4.77 (all P<.0001). Only L-CMV was significantly correlated with a higher risk of graft failure: E-CMV=1.18 (P=.1906), L-CMV-3M=1.77 (P=.0013), and L-CMV-6M=3.12 (P<.0001). Hospitalization costs increased by €7078 (range €6270-€22 111), €6523 (range €5328-€10 295), and €6311 (range €5295-€9184) in recipients with E-CMV, L-CMV-3M, and L-CMV-6-M, respectively. CONCLUSION: This study, based on French national data, demonstrates the considerable burden of CMV disease in SOT recipients and highlights the importance of developing new strategies to prevent and manage CMV disease and improve clinical outcomes for SOT patients.


Assuntos
Infecções por Citomegalovirus/epidemiologia , Infecções por Citomegalovirus/etiologia , Transplante de Órgãos/efeitos adversos , Transplantados , Estudos de Casos e Controles , Estudos de Coortes , França , Rejeição de Enxerto , Humanos , Incidência , Pacientes Internados , Razão de Chances , Transplante de Órgãos/mortalidade , Fatores de Risco , Análise de Sobrevida
14.
Orphanet J Rare Dis ; 12(1): 75, 2017 04 21.
Artigo em Inglês | MEDLINE | ID: mdl-28427466

RESUMO

BACKGROUND: The introduction of the orphan drug legislation led to the increase in the number of available orphan drugs, but the access to them is often limited due to the high price. Social preferences regarding funding orphan drugs as well as the criteria taken into consideration while setting the price remain unclear. The study aimed at identifying the determinant of orphan drug prices in France using a regression analysis. METHODS: All drugs with a valid orphan designation at the moment of launch for which the price was available in France were included in the analysis. The selection of covariates was based on a literature review and included drug characteristics (Anatomical Therapeutic Chemical (ATC) class, treatment line, age of target population), diseases characteristics (severity, prevalence, availability of alternative therapeutic options), health technology assessment (HTA) details (actual benefit (AB) and improvement in actual benefit (IAB) scores, delay between the HTA and commercialisation), and study characteristics (type of study, comparator, type of endpoint). The main data sources were European public assessment reports, HTA reports, summaries of opinion on orphan designation of the European Medicines Agency, and the French insurance database of drugs and tariffs. A generalized regression model was developed to test the association between the annual treatment cost and selected covariates. RESULTS: A total of 68 drugs were included. The mean annual treatment cost was €96,518. In the univariate analysis, the ATC class (p = 0.01), availability of alternative treatment options (p = 0.02) and the prevalence (p = 0.02) showed a significant correlation with the annual cost. The multivariate analysis demonstrated significant association between the annual cost and availability of alternative treatment options, ATC class, IAB score, type of comparator in the pivotal clinical trial, as well as commercialisation date and delay between the HTA and commercialisation. CONCLUSION: The orphan drug pricing is a multivariate phenomenon. The complex association between drug prices and the studied attributes and shows that payers integrate multiple variables in decision making when setting orphan drug prices. The interpretation of the study results is limited by the small sample size and the complex data structure.


Assuntos
Custos de Cuidados de Saúde , Produção de Droga sem Interesse Comercial/economia , Doenças Raras/tratamento farmacológico , Doenças Raras/economia , Aprovação de Drogas , Custos de Medicamentos , França , Humanos , Análise de Regressão
16.
Clin Ther ; 38(10): 2204-2226, 2016 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-27692977

RESUMO

PURPOSE: In Europe, 4 inhaled antibiotics (tobramycin, colistimethate sodium, aztreonam, and levofloxacin) are currently approved for the treatment of chronic Pseudomonas aeruginosa lung infection in patients with cystic fibrosis (CF). Levofloxacin inhalation solution (LIS) is the most recently approved inhaled antibiotic for adult patients with CF. A systematic literature review and Bayesian network meta-analysis (NMA) was conducted to compare the relative short-term (4 weeks) and long-term (24 weeks) outcomes of these inhaled antibiotics versus LIS. METHODS: A systematic literature search was conducted on February 16, 2016, using EMBASE and Medline via OvidSP. All randomized controlled trials comparing any of the aforementioned inhaled antibiotics with 4 or 24 weeks of follow-up were evaluated. NMA was performed for the following outcomes: relative and absolute percent changes from baseline in forced expiratory volume in 1 second (FEV1%) predicted, change in P aeruginosa sputum density, respiratory symptoms score from the CF questionnaire-revised, hospitalization, additional antibiotics use, and study withdrawal rates. RESULTS: Of the 685 articles identified, 7 unique studies were included in the 4 weeks' NMA and 9 unique studies were included in the 24 weeks' NMA. Aztreonam was predicted to result in the greatest numerically increase in FEV1% predicted at 4 weeks, whereas LIS were predicted to be numerically greater than colistimethate sodium, tobramycin inhaled solution (TIS), and tobramycin inhaled powder (TIP). However, all of the 95% credibility intervals (CrIs) of these comparisons included zero. At 24 weeks, none of the treatments was significantly more effective than LIS. The estimates for the mean change from baseline to 24 weeks in relative FEV1% versus LIS was -0.55 (95% CrI, -3.91 to 2.80) for TIS, -2.36 (95% CrI, -7.32 to 2.63) for aztreonam, -2.95 (95% CrI, -10.44 to 4.51) for TIP, and -9.66 (95% CrI, -15.01 to -4.33) for placebo. Compared with LIS, the odds ratio for hospitalization at 24 weeks was 1.92 (95% CrI, 1.01-3.30) for TIS, 2.25 (95% CrI, 1.01-4.34) for TIP, and 3.16 (95% CrI, 1.53-5.78) for placebo, all statistically worse than LIS. P aeruginosa sputum density scores, additional use of antipseudomonal antibiotics, and study withdrawal rates were comparable among all inhaled antibiotics at all times. IMPLICATIONS: Based on this NMA, the analyses for many of the outcomes did not provide significant evidence to indicate that the other approved inhaled antibiotics were more effective than LIS for the treatment of chronic P aeruginosa lung infection in patients with CF. Study withdrawal rates seemed to be comparable among these inhaled antibiotics.


Assuntos
Antibacterianos/uso terapêutico , Fibrose Cística/tratamento farmacológico , Infecções por Pseudomonas/tratamento farmacológico , Administração por Inalação , Adulto , Teorema de Bayes , Doença Crônica , Europa (Continente) , Volume Expiratório Forçado/efeitos dos fármacos , Humanos , Pseudomonas aeruginosa/efeitos dos fármacos , Ensaios Clínicos Controlados Aleatórios como Assunto , Infecções Respiratórias/tratamento farmacológico
18.
Clin Drug Investig ; 36(11): 945-956, 2016 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-27470429

RESUMO

BACKGROUND AND OBJECTIVE: When modelling the pathophysiology of a disease, it is important to select a modelling approach that can adequately replicate its course. The objective of this paper was to compare the outcomes obtained by the Markov and discrete-time microsimulation modelling approaches using nalmefene clinical trial data. METHODS: Markov and microsimulation modelling approaches assessing alcohol dependence treatment with psychosocial support with or without nalmefene were compared in terms of the modelled evolution of patients' alcohol consumption and the resulting occurrence of alcohol-attributable harmful events over 1 year. RESULTS: Comparison of the proportion of the modelled population at different levels of alcohol consumption over time revealed systematic differences arising from the different modelling techniques: a lower number of patients reaching abstinence, a higher number of patients at higher drinking levels, and, overall, a smoother evolution of alcohol consumption in the microsimulation. Reasons are discussed in the paper. While the models produced similar occurrences of alcohol-attributable harmful events as a whole, distinct results for the individual events were observed, explained by the specific pathophysiology of occurrence of these events and how their implementation was adapted to fit the limitations of the compared modelling approaches; however, these differences were only statistically significant for one of the eight events. CONCLUSIONS: For a general public health or health economic assessment of alcohol use disorders, it is possible to achieve similar results with the compared approaches. To assess a patients' disease course, taking into consideration alcohol-attributable harmful events, the microsimulation approach may provide more precise results. However, further external validation of the models is needed and this additional precision may be outweighed by the greater computational burden of a microsimulation approach.


Assuntos
Consumo de Bebidas Alcoólicas/tratamento farmacológico , Transtornos Relacionados ao Uso de Álcool/tratamento farmacológico , Alcoolismo/tratamento farmacológico , Naltrexona/análogos & derivados , Adulto , Feminino , Humanos , Naltrexona/administração & dosagem
19.
Appl Health Econ Health Policy ; 14(4): 493-505, 2016 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-27283839

RESUMO

BACKGROUND: Alcohol dependence causes considerable harm to patients. Treatment with nalmefene, aiming to reduce consumption rather than maintain complete abstinence, has been licensed based on trials demonstrating a reduction in total alcohol consumption and heavy drinking days. Relating these trial outcomes to harmful events avoided is important to demonstrate the clinical relevance of nalmefene treatment. METHODS: A predictive microsimulation model was developed to compare nalmefene plus brief psychosocial intervention (BRENDA) versus placebo plus BRENDA for the treatment of patients with alcohol dependence and a high or very high drinking risk level based on three pooled clinical trials. The model simulated patterns and level of alcohol consumption, day-by-day, for 12 months, to estimate the occurrence of alcohol-attributable diseases, injuries and deaths; assessing the clinical relevance of reducing alcohol consumption with treatment. RESULTS: The microsimulation model predicted that, in a cohort of 100,000 patients, 971 (95 % confidence interval [CI] 904-1038) alcohol-attributable diseases and injuries and 133 (95 % CI 117-150) deaths would be avoided with nalmefene versus placebo. This level of benefit has been considered clinically relevant by the European Medicines Agency. CONCLUSIONS: This microsimulation model supports the clinical relevance of the reduction in alcohol consumption, and has estimated the extent of the public health benefit of treatment with nalmefene in patients with alcohol dependence and a high or very high drinking risk level.


Assuntos
Consumo de Bebidas Alcoólicas/tratamento farmacológico , Alcoolismo/tratamento farmacológico , Naltrexona/análogos & derivados , Saúde Pública/métodos , Apoio Social , Consumo de Bebidas Alcoólicas/efeitos adversos , Consumo de Bebidas Alcoólicas/psicologia , Alcoolismo/complicações , Alcoolismo/psicologia , Ensaios Clínicos como Assunto/estatística & dados numéricos , Simulação por Computador , Europa (Continente) , Redução do Dano/efeitos dos fármacos , Humanos , Estudos Multicêntricos como Assunto/estatística & dados numéricos , Naltrexona/uso terapêutico , Antagonistas de Entorpecentes/uso terapêutico , Saúde Pública/estatística & dados numéricos
20.
Patient Prefer Adherence ; 10: 27-36, 2016.
Artigo em Inglês | MEDLINE | ID: mdl-26834463

RESUMO

BACKGROUND: Renal transplantation (RT) is considered the treatment of choice for end-stage renal disease compared to dialysis, offering better health-related quality of life (HRQoL) and higher survival rates. However, immunosuppressants are essential for the long-term survival of kidney grafts and patients' non-adherence to their medication leads to poor outcomes. Immunosuppressants can also significantly alter patients' HRQoL because of their side effects and the complex chronic medication regimen they represent. PURPOSE: To elicit key concepts related to adherence to immunosuppressant therapy (IT) and reasons for non-adherence in terms of patient reported outcomes, side effects, and the impact of the medication on HRQoL in RT population, including patient preference of once daily over twice-daily immunosuppressive regimen. Results were used to develop an IT-specific conceptual framework and provide suggestions for improving patients' adherence to IT. MATERIALS AND METHODS: Interviews were conducted with three clinical experts to determine key concepts related to RT and immunosuppressants. Thirty-seven participants in four focus groups were asked to cite important concepts related to adherence and impact of IT on HRQoL and to rate them. Qualitative analysis was conducted to code participants' responses. RESULTS: Non-adherence among participants where admitted was unintentional. The reason for this included forgetfulness, interference with lifestyle, being asleep at the time the medication should be taken, change in routine, and impact of side effects. Overall, participants reported that the evening dose was more problematic to remember and that the exclusion of this dose could make them more adherent. Participants also reported that IT impacted on their HRQoL in a number of ways including: placing restrictions on their lifestyle, causing anxiety, or impairing their ability to work. CONCLUSION: This study provides qualitative evidence about the barriers to IT adherence and the components of HRQoL that are important from the perspective of RT patients. The developed conceptual framework of IT-HRQoL in RT transplants, including social, psychological, and work life domains, can be used to inform the development of a new IT-specific measure of HRQoL in RT patients for use in head-to-head clinical trials or observational studies. Despite limitations associated with the number and the age range of patients recruited, this study suggests that a change in the regimen from twice-daily to once daily among other measures could improve their adherence to IT and their HRQoL by placing less restrictions on their lifestyles.

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