James Piercy

Real-world physician and patient behaviour across countries: Disease-Specific Programmes - a means to understand

ABSTRACT Background/objectives: Treatment guidelines and strategies are often based on data from randomized controlled trials and observational clinical studies. These sources drive treatment decisions, yet the data they provide may have limited relevance to the wider population in real-world clinical practice due to the narrow selection criteria applied to patients in trials. Information used to inform clinical practice and improve patient outcomes can, therefore, be unreflective of real-world clinical situations. The purpose of this article is to assess the value of Adelphi Disease Specific Programmes (DSPs) as sources of real world data. Methods: DSPs are large, multinational, observational studies of clinical practice for a range of common chronic diseases. Treatment practice data are collected by physicians (n = 700) who are asked to provide information for the next 10 patients consulting for a specific condition. These patients (n = 7000) are also invited to fill out a self-completion form providing their own assessment of symptoms, expectations and quality of life. Analyses: This article provides examples of the statistical techniques that have been employed to analyse the data in terms of cost/burden of illness, quality of life, disease severity and progression, compliance and adherence to therapy, impact of treatment guidelines and analyses of unmet need. Conclusions: DSPs can support clinical understanding of how diseases are managed including rationale for doctor decision-making and patient attitudes to their condition. Comparisons with other data sources and limitations of the programmes are discussed (including the fact that, unlike claims databases and registries, the DSPs are cross-sectional and not longitudinal).

Social and economic burden of walking and mobility problems in multiple sclerosis

BackgroundMultiple sclerosis (MS) is a chronic progressive neurological disease and the majority of patients will experience some degree of impaired mobility. We evaluated the prevalence, severity and burden of walking and mobility problems (WMPs) in 5 European countries.MethodsThis was a cross-sectional, patient record-based study involving 340 neurologists who completed detailed patient record forms (PRF) for patients (>18 years) attending their clinic with MS. Patients were also invited to complete a questionnaire (PSC). Information collected included demographics, disease characteristics, work productivity, quality of life (QoL; EuroQol-5D and Hamburg Quality of Life Questionnaire Multiple Sclerosis [HAQUAMS]) and mobility (subjective patient-reported and objectively measured using the timed 25 foot walk test [T25FW]). Relationships between WMPs and disease and other characteristics were examined using Chi square tests. Analysis of variance was used to examine relationships between mobility measures and work productivity.ResultsRecords were available for 3572 patients of whom 2171 also completed a PSC. WMPs were regarded as the most bothersome symptom by almost half of patients who responded (43%; 291/683). There was a clear, independent and strong directional relationship between severity of WMPs (subjective and objective) and healthcare resource utilisation. Patients with longer T25FW times (indicating greater walking impairment) were significantly more likely to require additional caregiver support (p < 0.0001), visit a variety of healthcare professionals including their primary care physicians (p = 0.0044) and require more long-term non-disease modifying drugs (p = 0.0001). A similar pattern was observed when subjective reporting of the severity of WMPs was considered. Work productivity was also markedly impacted by the presence of WMPs with fewer patients working full time and a reduction in weekly working hours as T25FW times and the subjective severity of WMPs increased.ConclusionsIn Europe, WMPs in MS represent a considerable personal and social burden both financially and in terms of quality of life. Interventions to improve mobility could have significant benefits for patients and society as a whole.

Impact of urinary incontinence on healthcare resource utilization, health-related quality of life and productivity in patients with overactive bladder

To evaluate the impact of urinary incontinence (UI) on healthcare resource utilization (HRU), health‐related quality of life (HRQoL) and productivity measures in patients with overactive bladder (OAB).

Fixed-Dose Combination Antidiabetic Therapy: Real-World Factors Associated with Prescribing Choices and Relationship with Patient Satisfaction and Compliance

IntroductionCompliance with antidiabetic therapy has the potential to impact on the risk for complications by an effect on glycemic control. Fixed-dose combinations (FDCs) offer a simplified dosing regimen that may improve patient compliance. We undertook a retrospective database analysis to understand the real-world association between FDCs, treatment practices, glycated hemoglobin (HbA1c) levels, and patient perspectives in type 2 diabetes.MethodsData were drawn from the Adelphi Diabetes Disease Specific Programme (DSP), a multicenter, patient recordbased market research study of primary care physicians and diabetologists/endocrinologists in Europe. The study is based on physician interviews, completion of detailed patient record forms by physicians, and a self-completion questionnaire by patients. Regression analyses were used to identify factors associated with (1) physician-reported dipeptidyl peptidase-4 inhibitor (DPP-4)/metformin FDC prescribing in dual or triple therapy regimens; (2) HbA1c of patients prescribed a DPP-4 FDC alone versus free-form DPP-4 plus metformin dual therapy regimens; and (3) differences between patients prescribed any oral antidiabetic therapy (OAD) FDC therapy (alone or in combination with one other OAD) versus those prescribed dual or triple OAD free-form combination therapy.ResultsPhysician-reported data were available for 5891 patients (mean age 61.5 years, 43% female, mean duration since diagnosis 5.7 years). Factors associated with DPP-4 FDC usage included physicians’ reason for choice being “improves patient compliance.” The relative mean % HbA1c level associated with being on a DPP-4 FDC rather than free-form independent of the physician perception of patient compliance was 0.25 lower (CI −0.40 to −0.09). When physician-perceived patient compliance was described as “fairly compliant” rather than “poorly compliant” or “not at all compliant,” the relative mean % HbA1c level was 0.42 lower (CI −0.67 to −0.18). Similarly, being perceived as “fully compliant” rather than “fairly compliant” was associated with a relative mean % HbA1c level that was 0.17 lower (CI −0.31 to −0.02). A significant predictor for the current regimen being any FDC (alone or in combination with one other OAD) regimen was patients’ satisfaction with treatment (odds ratio 1.32; 95% CI 1.10 to 1.58; P=0.003).ConclusionsThese results suggest that DPP-4 FDC prescribing is considered to be a positive prescribing choice to improve compliance and that choice is associated with improved glycemic control. From the patient’s perspective, the decision to prescribe an FDC is associated with improved satisfaction with treatment.

Economic evaluation of MF59 adjuvanted vaccine against influenza in the high-risk elderly population in France

Summary It is accepted that routine vaccination of elderly patients against influenza is a cost-effective strategy. This economic evaluation compares standard vaccination with MF59 adjuvanted vaccine - an adjuvanted trivalent influenza vaccination - in the high-risk elderly population in France. A cohort approach was developed to estimate the costs saved and deaths avoided by using adjuvanted vaccination instead of standard vaccination in the high-risk elderly. Using the established link between immunogenicity and protective efficacy, adjuvanted vaccination provides increased efficacy against influenza-related events, such as hospitalisations, primary care consultations and death. When the cost of the vaccination strategies and influenza-related events are accounted for, it was found that adjuvanted vaccination results in fewer deaths and more life years gained than standard vaccination. This is achieved with relatively little extra cost. At attack rates approaching 10%, adjuvanted vaccination is a dominant strategy. This implies that both a reduction in costs and an increase in benefits is observed. Therefore, on the evidence presented in this paper, the MF59 adjuvanted vaccine should be the vaccine of choice.

Assessing physician and patient acceptance of infliximab biosimilars in rheumatoid arthritis, ankylosing spondyloarthritis and psoriatic arthritis across Germany

Objectives We examined rheumatologists’ motivation for prescribing biosimilars, assessed their treatment preferences in relation to prescribing behavior and explored patient attitudes to biosimilars. Methods Data were taken from the Adelphi Real World Biosimilars Programme, a real-world, cross-sectional study undertaken with German rheumatologists and patients with rheumatoid arthritis, ankylosing spondyloarthritis or psoriatic arthritis in 2015–2016. Rheumatologists provided data on their prescribing behavior and attitudes toward biosimilars and invited the next eight eligible consecutive consulting patients to complete a questionnaire. Rheumatologists were split into “investigative”, “conservative” and “other” groups. Results Overall, 50 rheumatologists and 261 patients participated. Biosimilars accounted for <10% of all biologic therapy prescriptions, and >95% of rheumatologists would prescribe a biooriginator rather than biosimilar as the first- or second-line therapy if unrestricted. Patients showed some reluctance to accept biosimilars, and a small proportion of patients were unhappy when switched from a biooriginator to a biosimilar. Satisfaction with treatment was highest in patients who started treatment with a biooriginator prior to biosimilar availability. Patient concerns when starting treatment with a biooriginator or a biosimilar included not knowing enough about the drug (25%–41%), potential side effects (26%–32%) and potential long-term problems (19%–30%). Conclusion Study results demonstrate that there is some reluctance from patients to accept biosimilars and the need to educate patients who are unsure to allow them to be involved in decision making, highlighting the importance of patient and physician communication. There remains a need for further research into nonclinical switching and the long-term impact of prescribing biosimilars.

Assessing the impact of nocturia on health-related quality-of-life and utility : results of an observational survey in adults

Abstract Background and aim: The impact of nocturia (getting up at night to void) on health-related quality-of-life (HRQoL) is often under-estimated. This study investigated the relative burden in terms of HRQoL and utilities of nocturia in a real-world setting. Methods: Patient data were collected from two surveys: a nocturia-specific, cross-sectional survey of physicians and their patients (DSP), and a general UK population health survey (HSFE). Utilities (EQ-5D-5L), productivity (Work Productivity and Activity Index), and the impact of nocturia symptoms (Nocturia Impact Diary and Overactive Bladder Questionnaires) were assessed against the number of voids. A robust linear regression model with propensity score weights was used to control for confounding factors in estimating utilities. Results: Physician-recorded data were available from 8,738 patients across the US, Germany, Spain, France, and the UK; of these, 5,335 (61%) included patient-reported outcomes. In total, 6,302 controls were drawn from the two surveys and compared to 1,104 nocturia patients. Deterioration of HRQoL was associated with increasing number of night-time voids (p < 0.0001). In particular, significant differences were observed between 0–1 and ≥2 voids (p < 0.001). The regression model demonstrated that nocturia (≥2 per night) is associated with a modest but significant deterioration in utility of 0.0134 (p < 0.05). Limitations: The cause of nocturia is multifactorial and the mostly elderly patients may have several concomitant diseases. The authors tried to adjust for the most common ones, but there may be diseases or unknown relationships not included. Conclusions: Nocturia negatively affected HRQoL and patient utility. A clear effect is seen already at two voids per night. Every effort should, therefore, be made to reduce nocturia below the bother threshold of two voids per night.

Trends in medication use in patients with type 2 diabetes mellitus: a long-term view of real-world treatment between 2000 and 2015

Background Despite the availability of a variety of treatments, many patients with type 2 diabetes mellitus (T2DM) are not achieving glucose control. We analyzed successive waves of the Adelphi Real World Diabetes Disease Specific Programmes (DSPs) to assess treatment patterns reported by primary care physicians (PCPs) and specialists and the effect of treatment on levels of glucose control. Methods Data were collected between 2000 and 2015 in the US and EU5 (France, Germany, Italy, Spain, and the UK). Physicians completed patient record forms for the next 10 patients consulting with T2DM. Key aspects captured were change over time in therapy usage, time to insulin introduction, and glycated hemoglobin (HbA1c) levels. Results Over 12 DSP waves, 3,555 specialists and 5,109 PCPs completed questionnaires for 70,657 patients. Treatment patterns changed considerably over time as new agents were introduced. The number of agents prescribed per patient increased over time, as did HbA1c levels at which physicians stated they would introduce insulin. The greatest improvements in HbA1c levels occurred during 2000–2008, with little improvement since 2008. Conclusion In this real-world setting, the proportion of patients with T2DM achieving good glucose control has not increased greatly since 2008. A better understanding of how to individualize treatment pathways may be required to improve control in these patients.

Patient health utility, work productivity, and lifestyle impairment in chronic hepatitis C patients in France

BACKGROUND France has a high prevalence of patients with chronic hepatitis C virus (HCV). Clinical consequences of HCV are well-recognized, while health-related quality of life (HRQoL) and productivity impacts remain less understood. This study evaluates how HCV disease severity and HCV treatment outcomes impact HRQoL and productivity among patients in France. METHODS From October 2012 to January 2013, physicians treating HCV patients in France completed Patient Record Forms, which included information on patient demographics, disease stage, and treatment status. Subsequently, these HCV patients completed the EQ-5D-3L health-state instrument and the HCV-specific Work Productivity and Activity Impairment (WPAI:HepC) Questionnaire. Results are reported in descriptive and stratified analyses by disease stage and treatment status. Linear regression analyses were performed to determine independent associations between disease severity and treatment status with EQ-5D and WPAI:HepC. RESULTS There were 297 matched physician and patient response forms completed. Mean EQ-5D Index score was 0.764, and mean EQ-VAS score was 65.85. Regression analyses showed that older age and worse disease severity were significantly associated with lower EQ-5D Index and EQ-VAS scores. Stratification of EQ-5D Index and EQ-VAS scores showed significantly better scores for HCV treatment responders compared to non-responders. Stratification of WPAI:HepC questions by disease stage revealed greater productivity impact on HCV patients with more severe disease. CONCLUSIONS In a cross-sectional sample of HCV patients in France, worsening HRQoL and productivity/activity impairment was significantly associated with disease progression and increasing age. This information provides insight into the benefits of treating HCV patients and preventing disease progression.

Incremental burden of disease in patients diagnosed with pulmonary arterial hypertension receiving monotherapy and combination vasodilator therapy.

IntroductionPulmonary arterial hypertension (PAH) is a rare, severely debilitating disease with high mortality. There are limited data available on treatment patterns and burden of disease from conditions of actual care.MethodsThis analysis assesses the burden of disease for patients with PAH treated with monotherapy and combination therapies excluding and including intravenous (IV) prostacyclin analogues (PGI2). Data were drawn from the Adelphi PAH Disease Specific Programme, a cross sectional survey of consulting patients undertaken in the US, Germany, Italy and the UK in 2010. Outcomes included demographics, clinical characteristics, health-care resource utilization, and quality of life measured by the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR).ResultsData were analyzed from 446 patients receiving ≥1 of 3 PAH-specific treatment classes. Physicians comprised mainly pulmonologists and cardiologists. The symptoms, functioning and quality of life scales of the CAMPHOR instrument were completed by 218, 229, and 214 patients, respectively. Although 46.2% of patients were classified as World Health Organization (WHO) functional class III or IV, only 24.4% of the population received combination therapy. Combination therapy including IV PGI2 was used in 4.7% of all patients. Patients on monotherapy had the lowest pulmonary vascular resistance values, the highest recorded 6-min walk distance and the lowest recorded levels of dyspnea. Patients on combination therapy including IV PGI2 scored worse on all three variables and had more hospitalizations than patients on less aggressive combination therapy. With increasing therapeutic regimens, the CAMPHOR scores were higher, indicating worse states of health.ConclusionsCombination treatment and particularly the use of prostacyclins remain underused in an unselected population of PAH patients surveyed under conditions of actual care. The disease burden is substantial and increases with greater severity of disease and more aggressive treatments. This necessitates improvement in optimizing current therapy, as well as novel and innovative combination options.