DELIVERING ACTIONABLE RESULTS FOR OBSERVATIONAL STUDIES THROUGH RAPID EVALUATION PROCESSES

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PRM77 PROXY DEFINITIONS IN RETROSPECTIVE BURDEN OF ILLNESS STUDIES: A SYSTEMATIC REVIEW Frame D , Pilcher L , Wallace E Frame Research, LLC, Brooklyn, NY, USA .

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Objectives: To assess frequency of proxy definitions for health conditions in retrospective studies querying existing databases.  Methods: Systematic literature review (PubMed and ISPOR Scientific Presentations Database, searched 1/1/201312/31/2015). Eligible studies focused on cost/utilization burden of any disease, published in English or presented at ISPOR annual meetings. Studies with populations explicitly limited by treatment(s) received were excluded. We used publicly available information (abstract, poster, and/or full text) to extract data on study methodology used to identify patients with the condition of interest. The primary endpoint was the proportion of studies using proxy definitions of illness, e.g. treatments received or other algorithms besides diagnosis codes, to qualify patients for inclusion. P values were computed using Fisher’s exact test.  Results: 219 studies met prospectively-defined eligibility criteria (117 journal articles and 102 ISPOR abstracts, collectively representing 35+ million patients). Twice as many studies were conducted in the US as in all other countries combined; most used insurance claims data. Key patient selection criteria were extractable in 69% of articles and 64% of ISPOR abstracts, despite a majority being publicly available only in abstract form. 17% of ISPOR abstracts used proxy definitions compared to 14% of journal articles (p= 0.64). Use of proxy definitions did not differ by country/region. Claims data analyses used proxy definitions in 19% of studies, compared to 9% for other data sources, but the difference was not statistically significant (p= 0.15). Patient counts for studies using either diagnosis codes or treatment proxy definitions were infrequently reported, but ranged from 28.3% to 99.6% qualifying by proxy definition.  Conclusions: ISPOR annual meeting abstracts were not more likely than published papers to use proxy definitions of disease conditions. Most proxy definitions involved treatments received, which can dwarf the number eligible by diagnosis code. Cost of illness estimates may be influenced by patient selection using proxy definitions. PRM78 DELIVERING ACTIONABLE RESULTS FOR OBSERVATIONAL STUDIES THROUGH RAPID EVALUATION PROCESSES Mack C 1, Parmenter L 2, Velentgas P 1, Franke K 1, Jablonski R 1, Dreyer N A 3 1Quintiles, Durham, NC, USA, 2Quintiles Real World & Late Phase, Reading, UK, 3Quintiles, Cambridge, MA, USA .

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Objectives: Increased transparency, protocol registration and more rigorous quality standards have improved confidence in the validity of observational research. However, limiting analyses to a predefined protocol without allowing for flexibility reduces the potential to uncover new insights during study conduct. Rapid Evaluation Processes (REP) supplement standard research with ad-hoc hypothesis generation, evaluation, and delivery of actionable results within short timeframes.  Methods: REP adapts research processes into a streamlined yet flexible format whereby a study-specific analytic platform is developed in advance and then customized based on “breaking news”. A focused team produces results for immediate discussion and action, generating high impact results far more quickly than typical research timelines. Case examples include a global avian influenza outbreak, which produced a critical need to evaluate effectiveness of off-label treatment for rapidly occurring cases; sports injury surveillance, where effectiveness of rule changes require in-season evaluation based on trends in week-to-week injury occurrence; and market uptake of new therapies which impact enrollment and analytic methods.  Results: REP adds tangible value in settings where current events such as safety reports, new and highly utilized treatments, rapid development of cases (e.g., diseases or injuries), reimbursement or policy changes, evolving epidemics, or media speculation drive changes in treatment or outcome recognition. Vaccine and antibiotic surveillance programs and evaluation of risk minimization programs within drug utilization studies, often mandated by regulators, are also key research objectives that can be addressed by REP.  Conclusions: REP enables timely exploratory analyses that generate hypotheses, provides understanding of rapidly changing disease settings or medication use, delivers inputs into needed changes in active study enrollment or operations, and answers time-sensitive questions about patient health. PRM79 A NOVEL METHOD USING PRESCRIPTION TREATMENT PATTERNS TO ESTIMATE HEART FAILURE PREVALENCE IN CANADA Jobin-Gervais K 1, Zaour N 1, Caron J 2, Borrelli R 3, Fischer A A 3 1Novartis Pharmaceuticals Canada Inc., Dorval, QC, Canada, 2IMS Health Brogan, Kirkland, QC, Canada, 3IMS Health Brogan, Mississauga, ON, Canada .

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Objectives: Currently, it is estimated that more than 600,000 Canadians have heart failure (HF). Existing HF prevalence estimates use hospital and, where available, primary care diagnosis. However, diagnosis databases are limited in availability and coverage. Therefore, this study intended to develop a model identifying treated Canadian HF patients using retail prescription patterns from a national database to validate, and build upon Canadian literature prevalence estimates.  Methods: A decision tree model was constructed using 675 HF diagnosed (ICD-9 428) patients and 804 randomly sampled non-HF patients within IMS Health Brogan’s (IMS) E360 Canadian electronic medical record database. All patients were aged ≥ 18, had ≥ 2 doctor visits between January 2006 - January 2015, and had an ATC-1 cardiovascular prescription. The model’s CHF predictive accuracy was validated using a test dataset. Afterward, clinical opinion was engaged to confirm the rules generated by the statistical model. We applied the model to IMS’s patient-level longitudinal prescription database to identify the prevalence of treated HF patients by age group.  Results: Age and standardized duration treated with a beta-blocker, ACE/ARB, high-ceiling diuretic, and vasodilator were strong predictors of HF, while physician referral and gender were not. The model had an 80.0% accuracy, 81.1% positive predictive value, and 79.5% negative predictive value on a

test dataset. The study estimated a 2014 national ≥ 18 aged HF prevalence of 2.31%, or 657,902 patients, which aligned well with literature estimates, 590,416 and 626,199 patients. When the model’s prevalence estimates were stratified by patient age group, the majority differed by < 0.5% from literature prevalence estimates.  Conclusions: The model’s HF prevalence estimates closely match the literature both in overall and age stratified prevalence. As of 2014, approximately 2.31% of Canadians aged ≥ 18 are treated for HF. Overall, this study provides a mechanism to calculate detailed prevalence estimates in Canada using retail prescription data. PRM80 IMPROVING PRECISION BY APPLYING DISEASE PROGRESSION EQUATIONS FROM MULTIPLE SOURCES IN THE ALZHEIMER’S DISEASE ARCHIMEDES CONDITION EVENT (ACE) SIMULATOR Tafazzoli A 1, DosSantos R 1, Ishak K J 2, Krotneva S 3, Kansal A 1 .

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1Evidera, Bethesda, MD, USA, 2Evidera, Montreal, QC, Canada, 3Evidera, St-Laurent, QC, Canada

Objectives: AD is a highly prevalent condition with a tremendous total burden on society. As treatments are developed for use in early stages of AD, simulations need to predict decades of disease progression. Our objective was to evaluate how different sources for estimating disease progression impact simulated long-term outcomes.  Methods: We simulated disease progression over 15 years using the AD ACE simulator in a cohort of patients from the Alzheimer’s Disease Neuroimaging Initiative (ADNI) with Mini-Mental State Exam (MMSE) scores ≥ 25 (normal cognition or mild cognitive impairment). Predicted changes in cognitive function over time were simulated using equations based on ADNI data, which consists of individuals from normal cognition to mild AD with few observations in more severe stages, and published equations from the AHEAD model, which were developed using data from patients already diagnosed with mild to severe AD. Several thresholds for switching between the equations were tested for the impact on patient progression in terms of MMSE and need for institutional care, in the absence of mortality.  Results: When ADNI equations were used in patients from baseline until development of moderate AD (MMSE =  15) and AHEAD equations subsequently, it took 13.9 years to reach a population mean MMSE of 10. This value was stable when the threshold for switching between equations varied between MMSE scores of 5 and 20. Simulations in which disease progression is exclusively based on ADNI data or AHEAD model, however, both predict faster progression, requiring 12.9 and 6.6 years, respectively. Accordingly, fewer patients required institutional care with the combined equations (56%) than using ADNI (60%) or AHEAD equations (74%) alone.  Conclusions: Equations based on ADNI data and those from AHEAD predict faster progression than an appropriate combination of the two sources, potentially overestimating the rate at which patients develop severe AD and their need for costly institutional care. PRM81 MODELING ECODEVELOPMENTAL CONTEXT OF STDS/HIV RISK AND PROTECTIVE BEHAVIORS AMONG AFRICAN AMERICAN ADOLESCENTS Li Y 1, Mgbere O 2, Abughosh S 1, Chen H 1, Cuccaro P 3, Essien E J 1 of Houston, Houston, TX, USA, 2Houston Department of Health and Human Services, Houston, TX, USA, 3University of Texas School of Public Health, Houston, TX, USA .

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Objectives: Risk and protective processes work together as an integrated developmental process and may affect target behaviors directly or indirectly, thus require an understanding of the multiple and interrelated processes contributing to behavioral outcomes. The purpose of this study was to model the ecodevelopmental chain of relationships associated with sexually transmitted disease (STDs) and HIV risk behaviors among African American (AA) adolescents.  Methods: Data used for this study comprised 1,619 AA adolescents with an average age of 16±1.8 years obtained from the first wave of the National Longitudinal Study of Adolescent Health. Confirmatory factor analysis was conducted, followed by structural equation modeling to determine latent constructs that reflect the social-interactional components of ecodevelopmental theory using observed variables. Data analyses were conducted using Mplus software version 7.1.  Results: Findings indicate that the school domain and feeling of father’s love play significant roles in risky sexual behavior of AA adolescents. AA adolescents who reported having their father’s love and a strong bond with school personnel leaned towards better health status. Parents’ attitude towards use of birth control influenced their sexual behavior through direct pathway, mediated by gender and age of AA adolescents. The key variables found to predict risky sexual behavior of AA adolescents include: knowing more people who contracted STDs, having more friends with adequate knowledge about condom use, being older in age, male and a sexual minority. These factors indicate that both peer’s influence and individual characteristics have significant impact on the risk of contracting STDs/HIV among AA adolescents.  Conclusions: Our findings suggest that any intervention aimed at maximal protection against STD/HIV-related risk among AA adolescents should adopt both self- and contextbased concepts, and strategies that promote positive functioning in the family, school, and peer microsystems. Mediational relationships noted in the present study allude to important hypotheses for future research. PRM82 THE EVOLUTION OF ECONOMIC MODELING IN ALZHEIMER’S DISEASE: WHERE DO WE GO FROM HERE? Hernandez L Evidera, Lexington, MA, USA .

Objectives: Numerous economic evaluations have assessed the cost-effectiveness of treatments for Alzheimer’s disease (AD) in the last two decades. New challenges for the future models of AD include: the new definition of AD as a continuous spectrum from mild-cognitive impairment (MCI) through the dementia stage, the emergence of disease-modifying therapies (DMTs), and the growing interest in biomarkers for the early diagnosis of AD. Therefore, it is important to understand the existing models of AD and how they could inform new model-based economic evaluations of treatments for AD.  Methods: A systematic review of cost-effectiveness studies of treatments for AD published in the last decade, including studies