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The state of play in RWE, big data, HEOR and value-based interventions.

I recently had the honor to lead unique gathering of thought leaders in pharma and healthcare to discuss the state of real world evidence, big data, HEOR and value-based interventions.

The event I was hosting on October 14th and 15th in Bethesda was called Real World Evidence + Data Partnerships Summit which convened for the third year in a row. More than 100 executives attended representing a variety of industry stakeholders participating in over 30 panel discussions, moderated case studies, and innovative payor, policy, research, and technology insight presentations. Below is a broad-level summary of a few fascinating presentations.

Focus on the best governance, tools and internal capabilities to improve data management and quality

Our keynote session this year was led by Dr Robert Kaplan, Chief Science Officer Agency for Healthcare Research and Quality (AHRQ). Dr Kaplan spoke about Big Data and Large Sample Size emphasizing the cautions of Simpson’s paradox (correlation present in different groups is reversed when the groups are combined) and the need to personalize payor messages. He shared information from specific trials, suggesting that quite often industry might be selecting the wrong endpoints to measure impact of interventions in real world populations vs. the carefully identified patient represented in clinical trial populations. It is critical to obtain localized value from big data to determine what data adds value and real insights--not just quantity.

Sessions continued to present challenges of integrating real world evidence (RWE) from an infrastructure perspective and potential solutions for harmonizing data collection to manage fragmentation resulting from combining databases in a variety of coding systems and languages.

Our guest from Europe, Bart Vannieuwenhuyse, senior director, Janssen discussed how the European Medical Framework (EMIF) was launched to optimize efficient and currently available patient-level data. The EMIF represents 56 partners from 14 European countries and will be an ongoing five year project until 2017, including more than 53 million sets from seven EU countries.

Technological solutions from the provider side were presented by Brian Wells, AVP at Penn, ranging from integrated data warehouses of the University of Pennsylvania Health System, leveraging the data that you have to systematically gain insights, and navigate the integrated data consortiums to gain insights from collaboration with real-life subjects.

As healthcare systems evolve from volume metrics to value benchmarks, the effective organization of real-world data – both structured and unstructured – was actively discussed around issues of urgency, diversity, complexity, and practicality. Conference veteran Marc Berger, VP Real World Data & Analytics at Pfizer presented a unified definition of RWE as “healthcare data used for decision making that is not collected in conventional randomized controlled trials (RCTs)”. Amy Abernethy of Flatiron shared best practices and lessons learned from integrating clinical work flows through EHR, partnering with health care providers, technology driven chart abstraction, data verification and cleansing. Organizing Real World cancer data and addressing the challenges were applicable to a variety of technology barriers evident in large databases.

Gina Graham from GE Healthcare described agent based modeling as an effective and timely methodology to inform ever-critical health system decisions and resource allocation questions. Insights from all stakeholders revealed that storing the data is hard, integrating it is complex, and transitioning it into a value model may be the biggest challenge. Brian A. Stuelpner of Price Waterhouse Coopers stressed that successful organizations have integrated key areas into a common team with aligned incentives. As a result, the process for RWE has become more efficient, freeing resources to focus on the science and analytics. The lack of a true enterprise system or strategic business architecture results in fragmented and inefficient RWE generation.

HEOR experts offer their experience with different study designs, identification of relevant populations and how to best address validity and bias concerns

Kathleen Foley, Truven Health Analytics presented deterministic-linkage strategies that integrate claims data with EMR data to address unique challenges and gaps in real world data. The ability of outcomes research to leverage EMR data for approval, commercial and development purposes was the focus of an effective case study demonstrating how Amgen developed a data platform for business analytics, safety, clinical development, effectiveness, outcomes and epidemiologic research. Integrating nicely with earlier discussions from morning sessions leveraging biomarker data from a proprietary oncology EMR database introduced efficiency into the R&D process to benefit their entire pipeline.

Policy insights from the growing relevance of Comparative Effectiveness Research (CER) explored methodological parameters around Patient-Centered outcomes research. Jean R Slutsky, Chief Engagement and Dissemination Officer from at PCORI provided insights on study design standardization, data registries, and missing data or quality issues.

How do we extract timely and actionable information from data, knowing which patients are exposed to which interventions? CVS Caremark executive Olga Martin discussed utilization patterns and costs from a rich set of eligibility and benefit data on millions of patients allowing surveillance of unintended outcomes. These trends continue to evolve and utility of vulnerable patient indices provide timely solutions to monitor progression of treatment and outcomes.

James Sorace, from the Office of Science and Data policy, HHS, presented thought-provoking strategies to address the complexity of biomedical systems. The complexity of disease combinations in the Medicare populations for example require key strategic objectives addressed during the early phases of development of observational studies. These data sources such as those captured by the FDA provide insight of the real world impact of an intervention. Bespoke registries, patient centric data, and the potential of data-driven personalized care implementing mobile phone-based data capture were my highlights from the afternoon as the “drive” for personalized data from representative populations continued. The final case study of day presented by Amanda Bruno, HEOR Lead at Bristol-Myers Squibb, was of particular interest for leveraging HEOR field teams to evolve the medical perspective to develop RWE that better reflects the particular gaps and needs in evidence required from specific customers.

Opportunities for engaging with payors on RWE and discover how their access decisions are evolving.

On Day 2, the morning keynote introduced the sessions with a focus on communicating the evidence effectively. Solutions presented include evolving the commercial model, R&D model and medical model. Historically medical affairs engagement occurred in phase III. This is not sustainable and medical affairs is evolving to integrate the “3 Ps” patients, healthcare providers and policy makers not just net product value! Evidence is not a dichotomy but should be a continuum. RWE can reduce the evidence gap between the “bench and the bedside”. Tehseen Salimi, VP Global medical affairs from AstraZeneca envisions medical affairs as the face of the molecule to the world and collaboration with efficacy and efficiency role of R&D to extend the continuum from early phase I all the way to the mature brand.

Jane Reese-Coulbourne, executive director of the Reagan-Udall Foundation for the FDA, highlighted innovative strategies in research design describing PredicTox, a new approach to understand how drugs lead to adverse effects within the field of regulatory science. The FDA is exploring “systems biology” as a substitute to the use of animal models. Systems biology is an emerging scientific discipline that blends biology with computational modeling to fully understand all the potential effects of a drug, and better understand these complex networks.

Marcus Wilson, President of Healthcore identified unsustainable cost, variation in quality and lack of coordinated care as a key driver in suboptimal health outcomes. Using an evaluation of evidence base examples in cardiovascular disease, he highlighted the need to examine utilization not evidence. If we publish data that reports 45% care inconsistencies, the real opportunity is to study the variance of clinical behavior. Each data source contributes unique information as a measure of index of exposure to healthcare system.

Stakeholder panel insights on using RWE to kick start innovation consistently recommend value integration early in R&D timeline and leading with the patient perspective. We currently use a variety of surrogates to represent the end-user in care pathways but what is needed is a shared definition of patient engagement. Defining value as quality over costs is, eventually, too simplistic and lacks emotional resonance. Does technology offer a measurable difference in ways that are affordable by the patient and sustainable to provide at that cost? The care delivery model is changing. Patients with chronic disease may be using the medical system as a tactic not a strategy. Is there an unmet need? Does an asset in development bring value? All stakeholders need to think about relative value in the care pathway to capture the right data. The current healthcare system is inept at aligning medical aspiration with outcome.

Describing innovative patient-centered tools for evolving the healthcare landscape from volume to value, Matthew Rousculp, Sr. Director at GlaxoSmithKline, provided an update about a small data pilot developed in collaboration with Community Care of North Carolina (CCNC) to identify patients at risk of negative outcomes and provide medication management guidance at the point of care. Dana Evans from Genentech also highlighted the evolution from fee for service to define novel metrics that are effectively measuring patient outcomes. Shared risk strategies are being incorporated in a variety of care models that are continuing to evolve.

Effective data partnerships to realize opportunities in the growing evidence ecosystems. Adopt the best strategies, operational models and KPIs to engage stakeholders internally and externally.

Stakeholders and their evidence needs are heterogeneous. Access strategies need to be customized accordingly stated Shailja Dixit, executive director & global head of HEOR at Forest/Activis. Establishing market access as a process, understanding stakeholder evidentiary requirement, engaging the appropriate stakeholders and integrating value driven development both from a global and regional perspective describes the 4 pillars of access strategy. Value proposition, value generation, and value communication processes must be linked for all stakeholders. Perceived challenges and issues with specialty pharmacy have evolved the perception of real-world data research as a high-priority integrating earlier engagement in the value chain throughout the drug development and commercialization process.

The Clash of the Titans presentation led by Charles Shasky, Virginia Commonwealth University Health Systems provided an insightful consideration of changing the American healthcare system to unite the disparate forces—payers, government, healthcare systems, and industry to evolve into a more patient-centric proposition. Data from a report by Milbank Memorial Fund attributes payment reforms to support primary care holistic strategies such as multiple payer participation, state government oversight, primary medical home standards, team-based primary care, harmonized performance measures, and collaborative learning strategies.

Final presentations of the conference all concluded with an overview of real world data opportunities, a need to harmonize EMR data, better timeframes for RWD—namely earlier integration in R&D process and perhaps most importantly how to accommodate a particular drug or asset favorable to patient outcomes remembering to evaluate all emergent models jointly to ensure credibility of vision.



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