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Barbosa C.D.,Mapi Consultancy | Balp M.-M.,Novartis | Kulich K.,Novartis | Germain N.,Mapi Consultancy | Rofail D.,Mapi Values
Patient Preference and Adherence | Year: 2012

Purpose: To explore the published evidence on the link between treatment satisfaction and patients' compliance, adherence, and/or persistence. Methods: Articles published from January 2005 to November 2010 assessing compliance, adherence, or persistence and treatment satisfaction were identified through literature searches in Medline, Embase, and PsycInfo. Abstracts were reviewed by two independent researchers who selected articles for inclusion. The main attributes of each study examining the link between satisfaction and adherence, compliance, or persistence were summarized. Results: The database searches yielded 1278 references. Of the 281 abstracts that met the inclusion criteria, 20 articles were retained. In the articles, adherence and compliance were often used interchangeably and various methods were used to measure these concepts. All showed a positive association between treatment satisfaction and adherence, compliance, or persistence. Sixteen studies demonstrated a statistically significant link between satisfaction and compliance or persistence. Of these, ten demonstrated a significant link between satisfaction and compliance, two showed a significant link between satisfaction and persistence, and eight demonstrated a link between either a related aspect or a component of satisfaction (eg, treatment convenience) or adherence (eg, intention to persist). An equal number of studies aimed at explaining compliance or persistence according to treatment satisfaction (n = 8) and treatment satisfaction explained by compliance or persistence (n = 8). Four studies only reported correlation coefficients, with no hypothesis about the direction of the link. The methods used to evaluate the link were varied: two studies reported the link using descriptive statistics, such as percentages, and 18 used statistical tests, such as Spearman's correlation or logistic regressions. Conclusion: This review identified few studies that evaluate the statistical association between satisfaction and adherence, compliance, or persistence. The available data suggested that greater treatment satisfaction was associated with better compliance and improved persistence, and with lower regimen complexity or treatment burden. © 2012 Dias Barbosa et al, publisher and licensee Dove Medical Press Ltd.

Dubois D.,Patient Value Solutions | Gilet H.,Mapi Values | Viala-Danten M.,Mapi Values | Tack J.,University Hospital
Neurogastroenterology and Motility | Year: 2010

Background The Patient Assessment of Constipation-Quality of Life (PAC-QOL) is a self-reported questionnaire measuring health-related quality of life (HRQL) of constipated patients and was used as secondary endpoint in three identical double-blind, randomized, placebo-controlled Phase III clinical trials. These 12-week trials in subjects with severe chronic constipation evaluated the effects of prucalopride, a selective 5-HT4 agonist given orally once daily. Methods To consolidate the main treatment effect results observed in the prucalopride trial populations, analyses were undertaken on the pooled data of the three trials to confirm the psychometric properties of the PAC-QOL and to provide guidance for the interpretation of the clinical significance of its scores. Key Results The evaluation of the psychometric properties confirmed the PAC-QOL reliability, validity and responsiveness to measure the impact of chronic constipation symptoms on HRQL in the prucalopride trials. The 1-point improvement in PAC-QOL scores used as target response level for the main treatment effect analyses was validated as a relevant definition of response for treatment group comparisons. Cumulative distribution curves, drawn for each treatment group to provide more complete information on treatment effects than single minimal important difference point estimates, demonstrated consistent superior effects of prucalopride over placebo on all PAC-QOL scores. Conclusions & Inferences The PAC-QOL questionnaire is a useful measurement tool to assess, from a patient perspective, the potential therapeutic value of chronic constipation treatments in clinical trials and, by directly reflecting the patient's own perspective on constipation and its treatment, eventually also for informing daily medical practice. © 2009 Blackwell Publishing Ltd.

Yi Y.,Mapi Values | Lindemann M.,Bayer AG | Colligs A.,Bayer AG | Snowball C.,Mapi Values
European Journal of Pediatrics | Year: 2011

Neural tube defects (NTDs) are the second most common group of serious birth defects. Although folic acid has been shown to reduce effectively the risk of NTDs and measures have been taken to increase the awareness, knowledge, and consumption of folic acid, the full potential of folic acid to reduce the risk of NTDs has not been realized in most countries. To understand the economic burden of NTDs and the economic impact of preventing NTDs with folic acid, a systematic review was performed on relevant studies. A total of 14 cost of illness studies and 10 economic evaluations on prevention of NTDs with folic acid were identified. Consistent findings were reported across all of the cost of illness studies. The lifetime direct medical cost for patients with NTDs is significant, with the majority of cost being for inpatient care, for treatment at initial diagnosis in childhood, and for comorbidities in adult life. The lifetime indirect cost for patients with spina bifida is even greater due to increased morbidity and premature mortality. Caregiver time costs are also significant. The results from the economic evaluations demonstrate that folic acid fortification in food and preconception folic acid consumption are cost-effective ways to reduce the incidence and prevalence of NTDs. This review highlights the significant cost burden that NTDs pose to healthcare systems, various healthcare payers, and society and concludes that the benefits of prevention of NTDs with folic acid far outweigh the cost. Further intervention with folic acid is justified in countries where the full potential of folic acid to reduce the risk of NTDs has not been realized. © 2011 The Author(s).

Vissers D.C.J.,Mapi Values | Tolley K.,Tolley Health Economics Ltd. | Jakobsson J.,Karolinska Institutet | Jansen J.P.,Mapi Values
Value in Health | Year: 2011

Objective: Breakthrough cancer pain (BTCP) represents a considerable economic burden. A decision-analysis model was developed to evaluate the cost-effectiveness of intranasal fentanyl spray (INFS) compared with oral transmucosal fentanyl citrate (OTFC) and fentanyl buccal tablet (FBT) for the treatment of BTCP. Methods: The model was parameterized for Sweden to estimate the costs and benefits associated with treatments. Expected reductions in pain intensity (PI; measured on a numeric rating scale ranging from 0 to 10) per BTCP episodes were translated into resource use and quality-adjusted life years (QALYs). Relative analgesic efficacy of interventions was derived from a mixed treatment comparison of six randomized controlled trials. The relationship between PI and utility was obtained from a time-trade off study in the general population. Resource use and unit cost data were obtained from the literature and validated by Swedish clinical experts. The base case scenario assumed three BTCP episodes/day, a background PI of 2, and a time horizon of 180 days. Prices of INFS and OTFC were assumed to be equal with FBT ̃14% less. Uncertainty in the source data was incorporated by probabilistic sensitivity analyses and different scenario analyses. Results: With INFS, 55% of BTCP (95% uncertainty interval [UI]: 46-68%) was avoided, which is greater than expected with OTFC (29%; UI 22-38%) or FBT (31%; UI 25-39%). INFS was dominating OTFC (resulting in 0.046 QALY gain and saving 174 Euros with a time horizon of 180 days) and cost-effective versus FBT (incremental cost-effectiveness ratio 12203 Euros/ QALY). Despite uncertainty in the source data, there is a >99% probability that INFS is the most cost-effective intervention. Conclusion: Given inherent limitations of modelling studies, the greater efficacy of INFS translates to cost and QALY advantages over competing interventions in the treatment for BTCP in Sweden. Copyright © 2011, International Society for Pharmacoeconomics and Outcomes Research (ISPOR).

Objective: Compare the efficacy of 2 NRTIs combined with raltegravir (RAL), efavirenz (EFV), or protease inhibitors (PI) in the management of antiretroviral-naïve HIV adult patients.Methods: By means of a systematic literature view, 7 randomized controlled trials were identified: 2 RAL vs EFV trials; 1 ritonavir-boosted lopinavir (LPV/RTV) vs EFV trial; 1 ritonavir-boosted atazanavir (ATV/RTV) vs LPV/RTV trial; 1 ritonavir-boosted darunavir (DRV/RTV) vs LPV/RTV trial; 1 ritonavir-boosted fosamprenavir (FPV/RTV) vs LPV/RTV trial; and 1 FPV/RTV vs ATV/RTV trial. Endpoints concerned virological suppression and immunologic efficacy. Trials were analyzed with Bayesian mixed treatment comparison meta-analysis.Results: For up to 24 weeks of treatment, a PI-based regimen resulted in a lower proportion of patients with virological response than an EFV-based regimen, whereas RAL seems more efficacious than EFV up to at least 12 weeks. After 48 weeks, the odds ratio (OR) of virological suppression with RAL relative to EFV was 1.34 (95% credible interval [CrI], 0.87-2.07). ORs for PIs relative to EFV varied from 0.68 (0.41-1.07) with LPV/RTV to 0.99 (0.52-1.84) with DRV/RTV. RAL demonstrated a greater improvement in CD4+ T cell counts than EFV at 48 weeks. The PI regimens showed all similar improvements relative to EFV.Conclusion: Based on available RCTs, the fastest virological suppression is expected with RAL followed by EFV and PIs. Over time, RAL appears to be at least as good as PI and EFV regimens. CD4+ cell recovery seems the greatest with LPV/RTV, DRV/RTV, and RAL. Given the limited number of RCTs, additional studies are recommended. © 2011 Thomas Land Publishers, Inc.

Ghosh S.,Mapi Values | Chandran A.,Pfizer | Jansen J.P.,Mapi Values
AIDS Research and Human Retroviruses | Year: 2012

We sought to identify and summarize the incidence and prevalence of neuropathy among HIV patients and subgroups. A systematic search of the literature was performed using MEDLINE and EMBASE. The relevant literature was identified based on predefined criteria. Prevalence data were collected from cross-sectional and cohort studies. Incidence data were collected from cohort and case-control studies. Thirty-seven studies were included of which there were 23 cohort studies, 13 cross-sectional studies, and one case-control study. The prevalence of neuropathy among HIV patients derived from 25 studies varied from 1.2% to 69.4%. Regarding the development of neuropathy among HIV-positive patients, standardized by study duration, the rates per 100 person-years ranged from 0.7 to 39.7. Among older patients there is a greater risk of neuropathy. The same seems to be the case for patients with more severe disease. Currently available studies providing information on the incidence and prevalence of neuropathy among HIV patients suggest a significant burden, but there is a great variation in results across studies. There is no definitive explanation for the variation. However, it underscores the fact that complexity of the disease, along with absence of standardized diagnostic criteria, has considerably influenced the methodologies and outcomes of the studies. © Copyright 2012, Mary Ann Liebert, Inc.

Fleurence R.L.,United Biosource Corporation | Naci H.,United Biosource Corporation | Jansen J.P.,Mapi Values
Health Affairs | Year: 2010

Although not the gold standard of clinical research, observational studies can play a central role as the nation's health care system embraces comparative effectiveness research. Investigators generally prefer randomized trials to observational studies because the former are less subject to bias. Randomized studies, however, often don't represent real-world patient populations, while observational studies can offer quicker results and the opportunity to investigate large numbers of interventions and outcomes among diverse populations-sometimes at lower costs. But some decisions based on observational studies have turned out to be wrong. We recommend that researchers adopt a "body of evidence" approach that includes both randomized and observational evidence. © 2010 Project HOPE- The People-to-People Health Foundation, Inc.

Gater A.,Mapi Values | Thomson T.A.,Mapi Values | Strandberg-Larsen M.,Novo Nordisk AS
Thrombosis and Haemostasis | Year: 2011

Worldwide, haemophilia is the most common hereditary bleeding disorder. The incidence of haemophilia B, however, is considerably less than haemophilia A and consequently appears to have received less attention in the research literature. This article aims to summarise the available evidence documenting the patient and economic burden associated with haemophilia B and current methods of disease management. Both the immediate and long-term clinical consequences of haemophilia B can have significant implications for patients in terms of functional limitations and diminished health-related quality of life (HRQOL). Evidence demonstrates that primary prophylaxis is the optimal strategy for replacing missing clotting factor IX (FIX) and managing haemophilia B. Use of recombinant FIX (rFIX) over plasma-derived FIX (pd-FIX) is also generally preferred for safety reasons. Prophylaxis using currently available rFIX products, however, requires a demanding regimen of intravenous infusions 2-3 times a week which may have significant implications for adherence and ultimately the long-term efficacy of such regimens. Only limited assessments of the cost-effectiveness of prophylactic versus on-demand FIX treatment regimens have been conducted to date. Prophylaxis, however, is generally more costly as greater quantities of FIX are consumed. Any reduction in FIX replacement dosing frequency is expected to improve patient adherence and contribute to improved clinical outcomes, further supporting the costeffectiveness of such interventions. Although a rare disease, as economic constraints for healthcare increase, generating further information regarding the key clinical, patient and economic outcomes associated with haemophilia B will be essential for supporting improvements in care for people with haemophilia B. © Schattauer 2011.

Jansen J.P.,Mapi Values
BMC Medical Research Methodology | Year: 2011

Background: Pairwise meta-analysis, indirect treatment comparisons and network meta-analysis for aggregate level survival data are often based on the reported hazard ratio, which relies on the proportional hazards assumption. This assumption is implausible when hazard functions intersect, and can have a huge impact on decisions based on comparisons of expected survival, such as cost-effectiveness analysis. Methods. As an alternative to network meta-analysis of survival data in which the treatment effect is represented by the constant hazard ratio, a multi-dimensional treatment effect approach is presented. With fractional polynomials the hazard functions of interventions compared in a randomized controlled trial are modeled, and the difference between the parameters of these fractional polynomials within a trial are synthesized (and indirectly compared) across studies. Results: The proposed models are illustrated with an analysis of survival data in non-small-cell lung cancer. Fixed and random effects first and second order fractional polynomials were evaluated. Conclusion: (Network) meta-analysis of survival data with models where the treatment effect is represented with several parameters using fractional polynomials can be more closely fitted to the available data than meta-analysis based on the constant hazard ratio. © 2011 Jansen; licensee BioMed Central Ltd.

Jansen J.P.,Mapi Values | Bergman G.J.D.,Mapi Values | Huels J.,Novartis | Olson M.,Novartis
Seminars in Arthritis and Rheumatism | Year: 2011

Objective: To evaluate the efficacy of available bisphosphonate therapies regarding the prevention of vertebral, hip, and nonvertebral-nonhip fractures in postmenopausal women with osteoporosis. Methods: Eight randomized placebo controlled trials investigating the effects of zoledronic acid (1 study), alendronate (3), ibandronate (1), risedronate (2), and etidronate (1) in terms of fractures with a follow-up of 3 years (or 2 years if used for registration purposes) were identified with a systematic literature search. The endpoints of interest were morphometric vertebral fractures, hip fractures, and nonvertebral-nonhip fractures. Results of all trials were analyzed simultaneously with a Bayesian network meta-analysis by which the relative treatment effect of 1 intervention to another can be obtained in the absence of head-to-head evidence. Given the estimated treatment effects and their uncertainty, the Bayesian approach allowed for calculations of the probability of which bisphosphonate is best in terms of overall fracture reductions by weighting the impact of each by type of fracture on costs, quality of life, and incidence. Results: There is a 79% probability that zoledronic acid shows the greatest reduction in vertebral fractures of all bisphophonates compared. Zoledronic acid showed a relative risk (RR) of 0.30 (95% Credible Interval 0.23-0.37) relative to placebo, an RR of 0.55 (0.41-0.76) relative to alendronate, an RR of 0.50 (0.36-0.70) relative to risedronate, and an RR of 0.58 (0.37-0.92) relative to ibandronate. Regarding hip fractures, there is a 47% probability that zoledronic acid shows the greatest risk reduction, followed by alendronate (36%) and risedronate (11%). RRs of zoledronic acid relative to placebo, alendronate, and risedronate were 0.58 (0.41-0.82), 0.95 (0.54-1.68), and 0.73 (0.37-1.44), respectively. Risedronate showed the greatest reduction in nonvertebral-nonhip fractures, followed by zoledronic acid. The RR of zoledronic acid relative to risedronate was 1.28 (0.87-1.90). Overall, there was a 94% probability that zoledronic acid showed the greatest reduction in any fracture. Weighting the impact of the different type of fractures by incidence, cost, or quality of life showed similar results. Conclusion: Of the available bisphosphonates for osteoporosis, zoledronic acid has the highest probability of offering the best overall fracture protection. © 2011 Elsevier Inc.

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