Latest Trends In Design Of Clinical Trials For Real-World Health Economic Evidence Generation

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to ensure clear understanding by those entering data and developed a standardized electronic case report form, which ensured complete data entry and minimal errors. A data validation plan was implemented to ensure quality control and was used to validate all data entered into the study. To ensure consistency across MRI scans taken at different locations in clinical practice, a single MRI reading facility was used to provide the quantitative MRI parameters.  Conclusions: By rethinking the use of retrospective clinical data and warehoused MRI images we enhanced the use of RCR in MS and further developed our understanding of MS disease activity in a real-world setting. PRM191 Latest Trends In Design Of Clinical Trials For Real-World Health Economic Evidence Generation Aggarwal S1, Kumar S2, Topaloglu H1 1NOVEL Health Strategies, Chevy Chase, MD, USA, 2Institute for Global Policy Research, Washington, DC, USA

Objectives: Due to high healthcare costs, all stakeholders are increasingly demanding economic evidence for new medical products. While modeling provides useful estimates for economic impact, such data can also be potentially collected in a trial setting. The objective of this study was to review the trends in design of clinical trials focussed on health economics.  Methods: Systematic review was conducted to identify new and on-going clinical and observational studies for health economics. The inclusion criteria were term ‘economic’ and study completion date of 2016 or after. The data field extracted were study title, intervention, sponsor, age subgroups, planned enrollment, study type, study design, completion date and outcome measures.  Results: Overall, 59 clinical and observational studies with total planned enrollment of 128,254 patients were identified. The median enrollment for the studies was 230 patients. Majority of the studies were for medical devices (45%) and remaining were for drugs (18%), behavioral interventional (15%) and procedures (11%). For primary outcomes, 16 of the 59 studies measured a cost based outcome, 5 were hospitalization and 5 were QALY/ICER or cost effectiveness based outcomes. For secondary outcomes, 23 of the 59 studies measured cost and quality of life based outcomes, 9 were hospitalization and 2 were QALY/ICER or cost effectiveness based outcomes. Both primary and secondary outcomes show use of several non-traditional measures. Regarding sponsorship, 56 of 59 were sponsored by academia and only 3 were sponsored by the industry.  Conclusions: Health economic clinical and observational studies offer opportunity to generate high quality economic evidence, however, currently most of these studies are being sponsored by the academia. PRM192 The Validity And Reliability Of Cohort Identification Algorithms For Real World-Studies Zhou Y1, Murray JF2 of Michigan AND Eli Lilly and Company, Indianapolis, IN, USA, 2Eli Lilly and Company, Carmel, IN, USA

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Objectives: Assess the use of validated cohort extraction algorithms (e.g., algorithms with known characteristics of sensitivity, specificity, etc.) from observational study literature. We selected diseases the from 2012 CDC National Vital Statistics Report on Deaths amenable to retrospective cohort identifications. We targeted diagnosis, procedure and/or pharmacy-based algorithms used to identify patient cohorts; this abstract contains an initial focus on diabetes, pancreatic cancer, mild cognitive impairment (MCI) and migraine but the full analysis will have 16 targeted diseases.  Methods: We selected search terms to find articles that reported the actual criteria and algorithm used to identify a study cohort retrospectively. We searched in Medline on OVID platform using queries that combined diseases, study types and databases. The results were limited to human studies in United States in English published between 1990 and the present. One investigator assessed the retrieved studies against pre-determined search criteria for inclusion and exclusion. Two investigators independently assessed and classified the included studies into pre-defined categories of studies with unreported, non-validated algorithms and validated algorithms. We also extracted and collated the reported validation methods and operating characteristics.  Results: Literature search identified 441 studies for diabetes, 63 studies for pancreatic cancer, nine studies for MCI and 23 studies for migraine. Of these identified studies, 30.1%, 16.7%, 0% and 20% studies used validated algorithms for diabetes, pancreatic cancer, MCI and migraine respectively.  Conclusions: Algorithms extracted from the identified studies vary significantly in their nature (i.e., single codes or combinations of multiple codes), validation status and known operating characteristics. Use of non-validated algorithms can cause undesirable heterogeneity as well as the inability to validate and replicate findings. This can be a serious confounder and source of bias in cohort identification. The use of validated algorithms should be advocated and used over the development of new or ad hoc algorithms. PRM193 Combining Multiple Data Sources In A Single Study: Strengths Of Hybrid Study Methodology Kritikou P1, Stein D2, De Cock E3, Hanson KA2 1UBC: An Express Scripts Company, London, UK, 2UBC: An Express Scripts Company, Dorval, QC, Canada, 3United BioSource Corporation, Barcelona, Spain

Objectives: When existing secondary data cannot address multiple stakeholder needs, primary data collection (retrospective and/or prospective) should be considered in combination to or as a standalone study design. Our objective was to explore the strengths of hybrid studies that combine more than one data source based on practical research experience.  Methods: Six case studies representing different hybrid designs are described: two combined chart review with one-time survey (physician and patient), two prospectively collected time data on drug administration processes (one with chart review and one with Health Care Professional [HCP] survey) and two combined patient surveys with administrative data (one with

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pharmacy and medical claims and one with pharmacy claims and device utilization reports). We have delineated hybrid methodology strengths from a scientific and operational perspective.  Results: Each case study used one protocol which allowed leveraging a single study infrastructure. Five studies employed existing data from patient medical charts or administrative claims to facilitate patient identification and enrollment; two studies allowed for direct-to-patient recruitment. The case studies addressed a wide breadth of stakeholder needs: one assessed patient-level treatment patterns from medical charts and linked physician prescribing behavior via survey; one collected chart data on treatment patterns and outcomes in combination with patient-reported quality of life; one collected time data for drug administration processes combined with HCP satisfaction survey; and another collected data pre- and post-index event (patients acted as their own controls) to assess the impact of an intervention. The final two studies allowed the combination of medication refill data with device utilization and/or patient survey data.  Conclusions: The key strength of hybrid studies is the ability to address several stakeholder needs utilizing a single framework. Combing more than one data source for the same research initiative provides more comprehensive data insights and allows stronger value messages to be disseminated.

RESEARCH ON METHODS – Conceptual Papers PRM194 Why Aren’t All Pro/Coa Clinical Trials Using Electronic Data Collection To Optimize Data Integrity And Patient Experience? Holzbaur E, Ross J, Rothrock T Almac Clinical Technologies, Souderton, PA, USA

In this conceptual paper, we will explore reasons for continued use of paper data collection for Patient Reported Outcomes (PRO) and Clinical Outcome Assessments (COA). Reasons include instruments designed specifically for paper, the inability to determine true risks of paper-use, unknown true costs of electronic vs. paper administration, instrument author barriers (only allowing paper administration, costly requirements for migration), unclear translation expectations, and a general fear of the unknown. As electronic PRO/COA (ePRO/eCOA) offers several benefits over paper, including enhanced patient experience, improved compliance, and higher data quality, we will compare the advantages of electronically captured PRO/COA data to that captured via paper in order to show that ePRO/eCOA benefits far outweigh those of paper. The support and advocacy of ePRO/eCOA in clinical trials by regulatory authorities will be reviewed, along with describing current trends of the general population shifting towards the use of electronic devices in their daily routines. In focusing on patient centered clinical trials, ePRO/eCOA makes a significant contribution in reducing patient burden and optimizing their overall experience with making their required assessment entries. Additionally, this conceptual paper will focus on how PROs can be integrated into the eClinical solution to reduce the burden to patients, sites, and clinical teams and discuss this in relation to each phase of a clinical trial including set-up, management (also referred to as maintenance), and close-out. The risks and mitigation strategies in transitioning to ePRO/eCOA collection modes will be evaluated to provide guidance for the successful transition from paper to an integrated electronic PRO/COA solution. In this age of technological advancements, including an integration plan in clinical trials, can help to alleviate many of the obstacles in transitioning from paper to electronic PRO/COA while also mitigating potential risks and minimizing the burden to patients and clinical trial personnel. PRM195 Purposes Of Qualitative Methods In Health Outcomes Research: Two Practical Examples Guillemot J, Gauthier A Amaris, London, UK

Background and Aim: Qualitative methods are often disregarded for evidence generation in health outcomes and market access research as they rarely aim to achieve goals of reproducibility and generalizability of results. This has led to these methods being underused. We aim to illustrate two purposes of using qualitative methods: individual interviews to support a quantitative study and a Delphi panel to generate a consensus among experts.  Concepts: In the context of a conjoint analysis semi-direct interviews can be used to assess the comprehensibility and relevance of the quantitative questionnaire as well as to estimate the value and preciseness of assumptions. Semi-direct interviewing follows rigorous methods, including defined themes assembled in a discussion guide and behavioural rules for the interviewer to promote axiological neutrality. Analytical methods using predefined codes for the interpretation enabled a systematic understanding of datasets. Interviews shed light on occasional complexity and lack of comprehensibility of some questions. The qualitative analysis provided insights to the respondents’ experience. In the context of a study aiming to develop a clinical guideline in a recent disease area, a Delphi panel can be used to generate an expert consensus on the definition, diagnosis and management of the condition. The Delphi technique aims to rigorously organise convergence of opinion from a small number of experts on real-world issues. It is an iterative process whereby a questionnaire is submitted in several rounds. The results provided insightful treatment preferences and recommendations, which were readily transferable to clinical practice.  Conclusions: As health outcomes studies increasingly require to expand their level of detail and sensitivity by, for example, entering subjective fields of personal preferences or professional guidelines in niche disease areas, qualitative methods hold a decisive role. PRM196 Indirect Treatment Comparisons With Guided Matching-Based Adjustment: A Hybrid Of The Stc And Maic Techniques Ishak KJ1, Rael M2, Proskorovsky I1 1Evidera, Montreal, QC, Canada, 2Evidera, San Francisco, CA, USA