an ICON plc Company

Bethesda, MD, United States

an ICON plc Company

Bethesda, MD, United States
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Beusterien K.,Outcomes Research Strategies in Health | Grinspan J.,an ICON plc Company | Kuchuk I.,University of Ottawa | Mazzarello S.,University of Ottawa | And 5 more authors.
Oncologist | Year: 2014

Objective. Our objective was to evaluate preferences associated with grade I/II and grade III/IV chemotherapy side effects among breast cancer patients receiving chemotherapy. We also assessed trade-offs that patients are willing to make between treatment side effects and the route and schedule of treatment administration. Methods. In this cross-sectional study, patients receiving chemotherapy for breast cancer completed a one-time Web survey. Conjoint analysis was used to elicit preferences for 17 grade I/II and III/IV side effects associated with available chemotherapies and regimens. In the analysis, the risk of each side effect was increased by 5%, holding all others constant, and the respective impact on patient preferences was identified. Results. A total of 102 women participated (mean age 54 ± 11). Among the grade I/II side effects, a 5% reduction in the risk of sensory neuropathy, nausea, and motor neuropathy had the highest impact on preferences. Among grade III/IV side effects, motor neuropathy, nausea/vomiting, and myalgia made the most difference. An oral twice-daily regimen was most preferred; however, patients were willing to receive an intravenous regimen relative to oral to avoid an increased risk of 5% in the majority of side effects. Avoiding an increased chance of grade III/IV motor neuropathy was associated with willingness to tolerate one of the least preferred administration schedules. Conclusion. This study identified relative preferences among both mild/moderate to severe side effects from the patient perspective. Patients appear to be willing to make trade-offs between side effects and different regimens. These findings may help to inform medical decision-making processes. © AlphaMed Press 2014.


Azevedo V.F.,Federal University of Paraná | Azevedo V.F.,Edumed Health and Educational Research | Sandorff E.,An ICON plc Company | Siemak B.,An ICON plc Company | And 2 more authors.
Value in Health Regional Issues | Year: 2012

Objectives: Biosimilars are increasingly attractive to payers around the globe because of mounting financial pressure. Many Latin American governments are developing abbreviated regulatory pathways for biosimilars. There are limited data regarding how certain regulatory agencies in the region plan to address biosimilar access. This study explores potential opportunities and challenges for biosimilar drugs in Brazil, Mexico, Argentina, Chile, and Venezuela. Methods: We conducted targeted literature reviews, followed by key informant interviews, to understand the expected regulatory environment for biosimilars. We also asked questions about the economic, political, and historical factors that could play a role in the extent to which biosimilar-specific pathways have been developed across countries to date, and will continue to evolve in the future. Results: Brazil has led the development of biosimilar regulation in Latin America, with two distinct pathways, one for more complex molecules such as monoclonal antibodies and a less rigorous path for simpler molecules such as pegylated interferon and low molecular weight heparin. Other countries have been slower to respond, in part because of the degree of emphasis within each country for the advancement of biosimilar regulatory standards. Signs of relaxed standards akin to those seen in Brazil's "individual development" pathway were found in other countries. Conclusions: The example of the two-pathway system coupled with governmental prioritization of local manufacturing capabilities in Brazil should promote increased biosimilar utilization within the country. Assuming that the two-pathway system demonstrates success in Brazil, we hypothesize that other Latin American countries may adapt aspects of this "local" model for developing a regulatory pathway for biosimilars. © 2012 International Society for Pharmacoeconomics and Outcomes Research (ISPOR).


Devlen J.,an ICON plc company | Beusterien K.,Outcomes Research Stategies in Health LLC | Yen L.,Global Health Economics and Outcomes Research | Ahmed A.,Beth Israel Deaconess Medical Center | And 2 more authors.
Journal of Managed Care Pharmacy | Year: 2014

Background: The causes for nonadherence to mesalamine in patients with inflammatory bowel disease (IBD) have been characterized using mostly indirect methods. Patient-reported barriers are lacking in this population. OBJECTIVE: To identify patient-reported barriers to mesalamine adherence through direct interviews. Methods: Focus groups and one-on-one interviews were undertaken in adult patients with IBD. Transcripts from the focus groups and interviews were analyzed to identify themes and links between these themes, assisted by qualitative data software MaxQDA. Results: Of 27 patients participating, 21 (78%) had ulcerative colitis, and 6 (22%) had Crohn's disease. Their self-reported adherence ranged from complete adherence (n = 3) to intermittent nonadherence (n = 24). Patients frequently indicated that they were resistant to taking medications for their condition. The barriers to adherence that emerged from interviews could be categorized under a number of themes: competing priorities, social stigma, refill inconvenience, costs, efficacy values, side effects, and pill characteristics. Efficacy values reported to influence adherence included doubts about efficacy, consequences of missed doses, and doubts about need for maintenance medication. Pill characteristics reported as barriers included pill size and pill frequency. Despite use of electronic prescribing, obtaining refills was reported as an obstacle to adherence in this cohort. Decanting of pills to multiple containers to increase accessibility was also reported. Conculsions: Patients with both ulcerative colitis and Crohn's disease report a number of common barriers to mesalamine adherence. Factors in medication- taking behavior and beliefs were reported in this study that may have implications for strategies to improve adherence by health care providers. ©2014, Academy of Managed Care Pharmacy.


Khan N.,IMS Health | Shah D.,an ICON plc company | Tongbram V.,an ICON plc company | Verdian L.,Eisai Inc | Hawkins N.,an ICON plc company
Current Medical Research and Opinion | Year: 2013

Objectives: This paper compares the efficacy and tolerability of perampanel (PER) relative to other recently approved anti-epileptic drug (AEDs)-lacosamide (LCS), retigabine (RTG), and eslicarbazepine (ESL) for the adjunctive treatment of partial onset seizures with or without secondary generalization and specifically in the secondary generalization subgroup. Materials and methods: A systematic literature review of all RCTs of PER and selected AEDs in EMBASE, Medline, and the Cochrane Central from 1998 to January 2011 with an update in PubMed in March 2013 was performed. A network meta-analysis was conducted for 50% responder rate for overall seizures; withdrawal due to adverse events; seizure freedom; and 50% responder rate for secondary generalized seizures. Results: Twelve RCTs (three PER, three LCS, three RTG and three ESL) were included. PER performed significantly better than placebo for 'responder rate' (OR 2.151, 95% CrI 1.348-3.472) and 'seizure freedom' (OR 2.507, 95% CrI 1.067-7.429). When compared to other agents, PER was found to be equally effective. For 'withdrawal due to adverse events', PER had the lowest odds ratio vs. placebo compared with other AEDs. In the analysis for the subgroup of patients with secondary generalization, only four RCTs (three PER and one LCS) met the inclusion criteria for one outcome (responder rate) for LCS. In this subgroup, PER was statistically significantly better than placebo (OR 2.448, 95% CrI 1.088-5.828). Conclusion: PER was statistically significantly superior to placebo in responder rate, seizure freedom, and responder rate in the secondary generalization population. Though PER had statistically significant greater withdrawal compared to placebo, it had the lowest ORs vs. placebo, suggesting a superior safety profile among the comparators included in this analysis. In patients with partial onset seizure with secondary generalization, PER had a statistically significant effect on responder rate compared to placebo. © 2013 Informa UK Ltd.


Pocoski J.,Bayer AG | Benjamin K.,An ICON plc. Company | Michaels L.A.,Bayer AG | Flood E.,An ICON plc. Company | Sasane R.,Bayer AG
European Journal of Haematology | Year: 2014

Aim: This review summarises the importance, recent progress and issues in measuring patient-reported outcomes (PROs) in haemophilia research. Methods: A critical review of recent advances and trends in measuring haemophilia-related PROs was conducted, using current regulatory guidelines and methodological recommendations to evaluate these instruments. Results: Although regulators, payers and policymakers increasingly consider the patient's perspective to be important in treatment decision-making, to date, few haemophilia intervention studies have meaningfully applied PRO endpoints. Condition-specific PRO instruments have been developed, but most are not fully validated; sensitivity to subgroup differences and changes over time is unclear. Generic PROs and instruments developed for other conditions have been used to measure health-related quality of life (HRQL) in haemophilia patients, but little evidence of their validity for this purpose exists. Haemophilia presents a number of challenges to developing valid, reliable and responsive PRO instruments, including the rarity of the disorder; necessitating research in multiple counties to attain sufficient sample size; the chronic nature of the condition; acute exacerbations of illness; age and geographical region variations with respect to treatment; differences in treatment regimens, range of disease severity and phenotypes; and changes in patients' perceived health status over time. Given that haemophilia begins at birth, the illness has an impact on the lives of caregivers, although the extent of the impact is largely unknown. Conclusions: Patient perspectives are crucial to understanding the best and most cost-effective haemophilia treatment approaches. More research is needed on the ability of current disease-specific and generic PRO instruments to capture responsiveness to treatments over time and subgroup differences in outcomes. Inclusion of PROs in clinical trials is necessary to answer these questions. © 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.


Flood E.,An ICON plc. Company | Pocoski J.,Bayer AG | Michaels L.A.,Bayer AG | Bell J.A.,An ICON plc. Company | And 2 more authors.
European Journal of Haematology | Year: 2014

Introduction/Aims: Haemophilia and its treatment have a significant impact on patients' lives. The study objectives were to understand the impacts of haemophilia and its treatment from the patient perspective and to inform the development of comprehensive health-related quality-of-life (HRQL) conceptual models to illustrate these impacts. Methods: The study included two phases. Phase I involved a review of literature published from 1995 to 2010, qualitative analysis of six patient (N = 31) and three healthcare provider (N = 15) focus group transcripts, and interviews with two experts to inform draft conceptual models of mild/moderate and severe haemophilia. Phase II involved interviews with 20 haemophilia patients and qualitative analysis of transcripts to confirm the concepts and structure of the conceptual models. Results: The literature search resulted in 66 publications assessing HRQL, four of which were qualitative studies on the impact of haemophilia from the patient perspective. Results from Phase I indicated that acute bleeding events result in pain, swelling, bruising and restricted joint movement; repeated joint bleeds result in chronic symptoms, such as pain and arthropathy. Acute bleeds cause interruptions in daily activities and interfere with work/school. Patients have fears about having bleeds, which can affect their participation in activities, such as sports or crowded events. Patients also expressed feelings of depression, frustration, isolation and embarrassment. Results of Phase II corroborated findings from Phase I. Conclusions: The conceptual models illustrate the substantial impact of haemophilia and its treatments on patients' lives and can help inform clinical study design and the selection of endpoints to assess treatment benefit. © 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.


PubMed | an ICON plc Company
Type: | Journal: Health and quality of life outcomes | Year: 2013

Diabetic Macular Edema (DME) is a common cause of impaired vision and blindness amongst diabetics. If not detected and treated early, the resulting vision loss can lead to considerable health costs and decreased health-related quality of life (HRQoL). The aim of this study was to provide evidence of the psychometric properties of the National Eye Institute - Visual Functioning Questionnaire (VFQ-25) for use in a cohort of DME patients who participated in a clinical efficacy and safety trial of pegaptinib sodium (Macugen).A phase 2/3 randomised, double masked trial evaluated pegaptanib injection versus sham injection in patients with DME. The analysis was conducted using baseline HRQoL data of the VFQ-25 and the EQ-5D, on a modified intent-to-treat sample of 235 patients. These measures were administered by a trained interviewer by telephone in all but one of the study countries, where face-to-face interviews were conducted in the clinic. The measures were completed in the week prior to baseline, and after 54 weeks of treatment. Distance visual acuity, measured according to the Early Treatment Diabetic Retinopathy Study (ETDRS), was assessed at all time points. Psychometric properties of the VFQ-25 assessed included domain structure, reliability, concurrent and construct validity, responsiveness.The VFQ-25 was found to consist of 11 domains slightly different than those proposed. Nevertheless, none of the eight established multi-item scales met the criterion for further splitting and the VFQ-25 was scored as in the developers instructions. Internal consistency reliability was demonstrated for six out of the eight original multi-item scales, with Cronbachs alpha ranging from 0.58 (Distance Activities) to 0.85 (Vision Specific: Dependency). The VFQ-25 domains generally showed a low to moderate correlation with EQ-5D visual analogue scale (range 0.16-0.43) and with the visual acuity score (range 0.10-0.41). Construct validity was upheld with higher VFQ-25 scores for patients who saw more letters according to the ETDRS. Almost all scales were shown to be responsive with Guyatts statistic ranging from 0.10 to 0.56 at 54 weeks.The VFQ-25 has evidence to support its validity and reliability for measuring HRQoL in DME. However, some operating characteristics of the instrument need further consideration and discussion in the case of DME patients. Further research is therefore warranted in this indication.


PubMed | an ICON plc. Company
Type: | Journal: European journal of haematology | Year: 2014

Haemophilia and its treatment have a significant impact on patients lives. The study objectives were to understand the impacts of haemophilia and its treatment from the patient perspective and to inform the development of comprehensive health-related quality-of-life (HRQL) conceptual models to illustrate these impacts.The study included two phases. Phase I involved a review of literature published from 1995 to 2010, qualitative analysis of six patient (N = 31) and three healthcare provider (N = 15) focus group transcripts, and interviews with two experts to inform draft conceptual models of mild/moderate and severe haemophilia. Phase II involved interviews with 20 haemophilia patients and qualitative analysis of transcripts to confirm the concepts and structure of the conceptual models.The literature search resulted in 66 publications assessing HRQL, four of which were qualitative studies on the impact of haemophilia from the patient perspective. Results from Phase I indicated that acute bleeding events result in pain, swelling, bruising and restricted joint movement; repeated joint bleeds result in chronic symptoms, such as pain and arthropathy. Acute bleeds cause interruptions in daily activities and interfere with work/school. Patients have fears about having bleeds, which can affect their participation in activities, such as sports or crowded events. Patients also expressed feelings of depression, frustration, isolation and embarrassment. Results of Phase II corroborated findings from Phase I.The conceptual models illustrate the substantial impact of haemophilia and its treatments on patients lives and can help inform clinical study design and the selection of endpoints to assess treatment benefit.


Despite collection of patient reported outcome (PRO) data in clinical trials of antiepileptic drugs (AEDs), PRO results are not being routinely reported on European Medicines Agency (EMA) and Food and Drug Administration (FDA) product labels. This review aimed to evaluate epilepsy-specific PRO instruments against FDA regulatory standards for supporting label claims. Structured literature searches were conducted in Embase and Medline databases to identify epilepsy-specific PRO instruments. Only instruments that could potentially be impacted by pharmacological treatment, were completed by adults and had evidence of some validation work were selected for review. A total of 26 PROs were reviewed based on criteria developed from the FDA regulatory standards. The ability to meet these criteria was classified as either full, partial or no evidence, whereby partial reflected some evidence but not enough to comprehensively address the FDA regulatory standards. Most instruments provided partial evidence of content validity. Input from clinicians and literature was common although few involved patients in both item generation and cognitive debriefing. Construct validity was predominantly compromised by no evidence of a-priori hypotheses of expected relationships. Evidence for test-retest reliability and internal consistency was available for most PROs although few included complete results regarding all subscales and some failed to reach recommended thresholds. The ability to detect change and interpretation of change were not investigated in most instruments and no PROs had published evidence of a conceptual framework. The study concludes that none of the 26 have the full evidence required by the FDA to support a label claim, and all require further research to support their use as an endpoint. The Subjective Handicap of Epilepsy (SHE) and the Neurological Disorders Depression Inventory for Epilepsy (NDDI-E) have the fewest gaps that would need to be addressed through additional research prior to any FDA regulatory submission, although the NDDI-E was designed as a screening tool and is therefore unlikely to be suitable as an instrument for capturing change in a clinical trial and the SHE lacks the conceptual focus on signs and symptoms favoured by the FDA.


PubMed | An ICON plc Company
Type: Journal Article | Journal: Journal of medical economics | Year: 2013

Relapsing-remitting multiple sclerosis (MS) is usually managed with disease modifying drugs (DMDs), most commonly administered via self-injection. The aim of this study was to estimate the influence that different treatment-related attributes have for MS patients on their choice of MS DMD device. By establishing the relative importance of these characteristics for patients it should be possible to better understand the acceptability of a given device and to optimize the development of future devices.A discrete choice experiment (DCE) survey was developed on the basis of a review of published literature. Attributes identified for inclusion in the survey were: ease of use; comfort of use; presence of additional functions, needle visibility; practicality and efficacy. Choice sets were presented as pairs of hypothetical treatments based upon a fractional factorial design. One-hundred device-using MS patients completed the survey online. Analysis was conducted using a mixed-logit approach.Analysis of the DCE data revealed that all attributes significantly predicted treatment choice. Efficacy exhibited the largest effect on treatment selection and this provided context for understanding the magnitude of impact for the other attributes. Reducing the discomfort associated with device use and eliminating the necessity for assembly or drug reconstitution were highly valued. The addition of reminder and time-stamping functions, improved needlestick injury prevention, and reduction in device size were secondary concerns but still deemed desirable.Efficacy is of primary importance to MS patients, but characteristics of drug delivery devices can play an important role in treatment decision-making. Not all device characteristics could be included, and results are based upon 100 participants only. Findings suggest there is significant potential value in developing self-injection devices that are not only efficacious but also convenient and comfortable to use. Reducing barriers to adherence could potentially translate into improved treatment outcomes for patients with MS.

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