From PI to Payer to Patient: RWE From the Perspective of Each Key Stakeholder

The pharma industry strives to improve human health. However, the industry is often caught in a narrow view of value creation. It doesn’t have to be this way. Creating shared value across stakeholders is essential and attainable for the healthcare industry. Embracing strategies that optimize value across the ecosystem is a collective effort that can benefit all healthcare stakeholders, from principal investigators to payers to patients, and ultimately drive long-term value for the healthcare system.

Shared value can take shape in a variety of ways-through public-private partnerships, therapeutic area alliances, and provider team healthcare models.

(RWD), data relating to patient health status and/or the delivery of healthcare routinely collected from a variety of sources, is a critical component and central to fostering optimal performance within the healthcare ecosystem.

Over the past five years, RWD has become a major element of clinical and health economic research. Data from electronic medical records, insurance claims, prescriptions filled, and even social media provide insights into the safety and effectiveness of new and existing drugs. In the UK, the WEB-RADR initiative, a multi-stakeholder initiative led by the MHRA, seeks to investigate technologies for gathering ADR data through a mobile app to add to the established safety profiles of medicines, enable earlier detection of new signals, reveal new patterns or trends in reporting, and even provide a means for geo-pharmacovigilance. Increased access and use to this wealth of data helps payers determine a value-based cost for those treatments. RWE (real-world evidence) also helps investigators to design efficient and effective trials.

In December 2018, the FDA released its framework on how RWE can be used to support drug effectiveness. Derived from the analysis of RWD, RWE is the clinical evidence regarding the usage and potential benefits or risks of a medical product. It can be generated by different study designs or analyses. As displayed in the diagram below, RWD and RWE are interdependent and connected and while the agency’s guidance on use of RWE is still crystallizing, this pivotal first step symbolizes its potential to advance drug development and regulation. 

A healthcare ecosystem that optimizes shared value is “data hungry,” requiring ongoing RWD mining and RWE generation. RWD sets are not a one-size-fits-all solution and require custom refining to ensure fit-for-purpose applications. Every stakeholder requires different types of data to solve unique problems and explore opportunities to create better value and outcomes. A framework to appraise RWD “goodness-of-fit” for RWE generation should include: data access (privacy and use of data), data elements (types of data), and data quality (structure, linkage, and coverage of data). Janssen’s PHEDRA (Platform for Hematology in EMA: Data for Real World Analysis) is an example of a platform that utilizes these elements to enable various stakeholders, such as payers, to compare patient populations, treatment configurations, and outcomes over multiple lines of therapy.  

Payers want proof

When bringing new drugs to market, payers want to see proof of effectiveness in a real-world setting; however, they often have to make coverage and access decisions based solely on results from randomized controlled trials (RCTs). While these clinical data may prove that a drug works in a controlled setting with a very specific patient population, it does not demonstrate its effectiveness in real-world patients, who tend to be older, sicker, and less likely to adhere to exact treatment regimens.

Biogen and Imperial College London are currently working on project OPTIMISE which is developing and deploying tools for collecting a wide range of data from people with multiple sclerosis, in addition to routine clinical assessment. OPTIMISE data and tools will allow researchers and physicians to better monitor outcomes and evaluate new treatments. When trial sponsors have the foresight to generate RWE as a complement to their RCT data, it helps bridge that gap of uncertainly that payers face when determining a new coverage policy for a new treatment.

Evidence may be derived by using active comparators or benchmarks in real-world treatment settings. These real-world insights are especially valuable in evaluating unmet medical need in traditionally under-represented trial populations. Payers often view pharmaceutical prices as too high and not proportionate to value. By demonstrating effectiveness among the core patient demographic and total cost-benefit, RWE can create shared valued among payers and investigators.

Investigators want guidance

When investigators integrate RWD into clinical research at the very beginning of a trial, it can have a cascading effect that drives better insights and efficiency throughout the development lifecycle creating additional value in the study.

Some investigators prime their trials with retrospective studies to learn more about certain subpopulations before launching the research. This takes extra time on the front end, but it can accelerate recruiting and result in a better-designed trial that moves faster and costs less than anticipated.

A top-10 pharma company spending $2 billion on clinical development annually, of which $500 million is addressable by RWE, can gain significant value by applying RWE to gain 20 to 40 percent efficiency in spending, or $100 to $200 million in savings. The key to creating shared value across the ecosystem is to capture RWE as part of the development strategy from the outset and cascade that value of the life asset by ensuring optimal use to address unmet need.

Patients want to be heard

Patients are the source of the most valuable real-world data and an essential piece to creating shared value. They are the subjects of EMRs and medical claims, and their social media posts and safety event reports create the foundation of real-world evidence that shapes healthcare decision-making. However, they can also benefit from these data in the short and long term.

Wearable and implantable devices, for example, provide both investigators and patients with critical data about the individual’s health, which can be used to more rapidly address adverse events and improve their own health outcomes as well as those of patients like them. Patients can also use real-world data from other consumers to make more informed decisions about the physicians and facilities they visit, and the treatments they agree to follow.

The use of digital health apps in just five patient populations where they have proven reductions in acute care utilization, such as diabetes prevention, diabetes, asthma, cardiac rehabilitation and pulmonary rehabilitation, could save the U.S. healthcare system an estimated $7 billion per year.

Over the long term, these data will inform pharma, investigators, payers, and physicians as they pursue new treatments and garner insights into which therapies are appropriate for every patient. More importantly, as stakeholders come to recognize the value of real-world data, the role of the patient in this process is heightened. The healthcare system is becoming increasingly connected and interdependent, with RWD serving as the value creation mechanism flowing throughout it. Instead of passive recipients of medicine, patients are becoming active stakeholders contributing to the knowledge base that will shape the future of healthcare.

Circle of data

Healthcare companies that embrace the idea of creating shared value will strengthen their businesses by expanding markets and differentiating themselves. A recent public-private partnership that emphasizes creating value across the system is the U.K.’s second life sciences sector deal that includes 10 different companies investing in the U.K. and pledging to healthcare innovation. A part of the effort, IQVIA is partnering with Genomics England to launch the first real-world research platform with integrated clinical and genomic data. This alliance will enable faster and more efficient drug research and more robust evidence to support treatment value.

With the availability of RWE, its generation and use occur across the entire healthcare continuum, where all stakeholders collaboratively create, analyze, and share results for better individual and population health outcomes.

Another example of connecting data across stakeholders to create a “learning system” of RWE generation, to improve quality and outcomes, is the first digital pill approved by the FDA, Abilify Mycite, which tracks if patients have taken their medication through a tiny ingestible sensor. This integrated digital-pharmaceutical solution will facilitate groundbreaking information sharing about how schizophrenia patients are taking their medication so that physicians, patients, and caregivers can optimize the treatment and outcomes. As more patients adopt this novel product and data matures, investigators can design better clinical trials and observational studies and payers can more precisely appraise real-world value and outcomes, thereby continuously improving system quality for schizophrenia patients.

Creating shared value by utilizing RWE can be a difficult concept for an industry built on a foundation of privacy and intellectual property. But the culture of data-sharing must evolve if the industry is going to benefit fully from this trend. When all stakeholders embrace a new value model of data sharing across the healthcare ecosystem, we can generate more and better data, which will steadily increase its value and impact over time and most importantly, help us all in our mutual goal to improve public health.

John Doyle, SVP & GM, Real-World Enterprise Solutions, IQVIA