Real-world evidence (RWE) captures health data from outside controlled clinical trials, such as wearable devices, patient forums, electronic health records, and symptom tracking apps. It reflects the daily realities of individuals managing chronic conditions, navigating medications, and seeking answers when the system fails to provide clarity.

As Yael Elish, one of the founders of the mega crowdsourcing transportation platform WAZE and now CEO and Founder of the patient RWE platform StuffThatWorks, has said, “Patients will never have a seat at the table until their data is considered part of the navigational guide for new medicines, devices, and indications of use.”

She’s right, but perhaps we must push ourselves to a new level of acknowledging why we are pursuing new approaches to care. This is not just about offering patients a seat at the allegorical table. It’s about acknowledging that the table of discovery and development exists for their benefit. The science, systems, and research all exist to serve their needs. Their voices and their lived experiences must inform the path forward.

A Silent Majority Speaks: The Data We Ignore

A recent survey conducted by StuffThatWorks, which gathered insights from more than 15,000 patients across 145 medical conditions, revealed a staggering disconnect. 92% of patients expressed a willingness to participate in clinical trials. Yet, 84% said they had never been asked, never invited to the table of health innovation.

This isn’t a communications oversight—it’s a justice issue. The system is failing those it claims to serve. Patients are ready to contribute. Some are out of options and want concrete action steps. They are waiting to be seen, heard, and engaged. Their personal care and experiential data they offer doesn’t just enrich our understanding—it recalibrates it.

“The Pharma Industry’s efforts to include patient voices have been a step in the right direction; however, they remain on a very small scale today,” notes Caroline Redeker, chief strategy officer at Advanced Clinical, a leading clinical research organization. “Interacting with a minimal number of patients without scale does not accurately represent the full patient population with the condition.  Using available static data (claims, EMR) detached from the patient covers the ‘what’ of patients – how many, where, with claims or treatments of a condition, and mostly in the US.”  

Redeker adds: “The more important factors in trial design include the ‘why,’ including most bothersome symptoms, effectiveness of treatments, regional differences, comorbidity considerations, and other valuable information to accurately design the right trial.  The ‘why’ information can only come directly from the patients.  The future will bring patients and their organized data to the table and include patient insights from all over the globe.”

Real-World Realities: Lessons from Ehlers-Danlos Syndrome

Consider Ehlers-Danlos Syndrome (EDS), a connective tissue disorder with 13 recognized subtypes, ranging from the more common hypermobile form to the rare, life-threatening vascular subtype. Each type carries a distinct constellation of symptoms and risks. Some people face chronic dislocations and mobility challenges, while others live with the daily fear of spontaneous organ rupture.

A single disease name masks and encompasses a spectrum of experiences. Standard clinical trial models often can’t keep up with that complexity. But thousands of patients with EDS are documenting their treatment responses, flares, and management strategies across RWE platforms such as StuffThatWorks—building a picture of this disease that’s as varied, inclusive and individualized as the people living with it.

“The StuffThatWorks survey expands the usual definition of real-world data to include this novel direct patient-inputted database,” says George Sands, MD, FAAN, FAHA, FACC, a former senior director at Pfizer and noted advocate for collaborating with patient communities  “Patients and patient advocacy groups want to partner with sponsors, whether pharma or academia, and to be fully represented in research endeavors. This is very different from sponsors looking to recruit and retain research participants more fully. This is about collaborating.” 

Dr. Sands adds, “Additionally, it is important to have all different patient populations, including people of color, so their data is included in the trials and can be used for clinical care. Otherwise, clinicians have to extrapolate based on those included in the trials; it doesn’t always work exactly.” 

That kind of data, born of day-to-day life, isn’t just complementary to clinical trials. It completes a fuller picture essential for developing effective treatments. Without it, we risk designing therapies that help in theory but fail in practice.

From Inside-Out to Outside-In

Much of medicine’s traditional approach to research is inside-out, starting with what companies aim to prove and working outward through the trial process. That’s not inherently wrong—it is how rigorous science is structured—but that is not enough.

We must also embrace outside-in realities as part of the innovation journey, where patients’ lived experiences inform what we explore, how we measure success, and who we prioritize.

Craig Lipset, founder of Clinical Innovation Partners, and co-chair of the unifying NGO/research industry Decentralized Trials & Research Alliance, and former Head of Clinical Innovation at Pfizer, has long championed a rebalancing of power in clinical trials.

“To earn and maintain trust, research participation must become a symbiotic relationship powered by a bidirectional flow of data and learnings, says Lipset. As patients share insight into study design, bring real-world data into trials, and share experience that shapes new endpoints, sponsors must commit to ensuring that study data and results are flowing back.”

That vision is becoming increasingly possible. With technologies that enable decentralized trials, remote monitoring, and electronic patient-reported outcomes, we can now collect real-world data at scale. However, that only matters if we respect what the data tells us and are willing to let patient experiences guide the research agenda. We give lip service to the importance of patient voice in biopharma research and marketing, but do we see them as “customers” of innovation?

The Science of Listening

Real-world evidence is the patient’s language—a narrative built not in labs but in lived experience. It’s not peripheral to science—it is science when equity is the goal. When patients share their stories, whether on online platforms, through wearables, or in digital symptom trackers, they are offering far more than an anecdote. They are offering insight. They contribute to the science of what works, for whom, and under what conditions.

And when we listen, we don’t just become better researchers. We become better healers.

We stand at a pivotal moment. Innovation can either deepen the divide between patients and science or bridge it. Real-world evidence is the support structure for that bridge. It offers us the opportunity to democratize discovery and reimagine research collectively.

Reflects StuffThatWorks Elish, “The current methods of involving patients in the clinical and health spaces are doomed to failure.  Patients’ voice will be heard ONLY when turned into organized data, at scale.  Because no one can argue or ignore organized data at scale. Once that’s accomplished, the integration of patient voice will proliferate and become easily accessible to everyone in multiple forms, including a simple AI-based chat question.  We are doing that at StuffThatWorks; it’s core to our mission and vision.” 

Ultimately, the patient wants to be invited to the table—but not as a token guest. Without them, there is no table worth setting. The patient is not a peripheral player in this system—they are the system’s purpose. They are not guests in the system. They may not be the core customer of the health system, a universe that centers around its economic viability. Still, in the world of health system kinetics, where we examine the relationship and priority of the sector pieces, they are the reason we unite to invent solutions to confront disease, despair and death.  And the future of medicine depends on how well we recognize that truth.

The future of medicine hinges on one choice—do we continue treating patients as passive subjects, or as co-creators of the care that might save their lives? That’s not philosophy. That’s leadership.

Interested in this topic – here is more to explore:

Aman Gupta: Patient Inclusivity: The Missing Piece In Pharma’s Global Innovation Strategy

The Need for High Quality, Reliable Information that is Data-Driven – A Conversation with Yael Elish

Gil Bashe: Real-World Evidence Unlocks Consumer Voice to Improve Care

The post How Real-World Evidence Proves the Power of Patient Engagement appeared first on Medika Life.

Doctors are evaluated on patient throughput, not the depth of their engagement. Pharmaceutical and medical device companies create and perfect new products for patients but often neglect their preferences, stated needs, and lived experiences in recruitment and development efforts.

Real-world evidence (RWE), a promising frontier in health settings from innovation to care decisions, is poised to change this dynamic. If we are open to acting upon it, it can become the basis for centering efforts directly on the patient experience.

As a father and caregiver, I’ve walked this journey firsthand. My child’s health challenges have brought me face-to-face with the limitations of a system that often measures success in drugs prescribed rather than resolutions achieved. I’ve seen the toll that rushed consultations and fragmented care take on families searching for answers. It is a stark reminder that even as the science of medicine advances, the art of listening lags.

Recently, we visited a doctor’s office to consult about our young adult’s rare disease. We hired a patient advocate to bridge the chasm between clinical expertise and lived experience. They translate medical jargon, connect patients with appropriate specialists, and, most importantly, validate the patient’s voice.

Listening to the advocate prompted me to ask a critical question about this complex clinical case: “What is the underlying cause of the symptoms?”When our non-medically trained patient advocate suggested surgery to address the root causes of our child’s GI problems, one doctor we consulted, who came from a noted teaching hospital, confronted us.

“Who are you going to believe,” he asked. A patient or me?”  Ultimately, the patient advocate, who consulted with other patients and their physicians, was spot-on. The operation took place; the outcome was outstanding. That is the power of RWE as a catalyst for objective conversation.

Real-World Evidence: A Catalyst for Change

The Food and Drug Administration (FDA) defines RWE as “the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of real-world data (RWD).” These data include electronic health records, insurance claims, and patient-generated information from mobile health apps and wearable devices. Unlike randomized controlled trials, which too often exclude diverse populations, RWE provides a more comprehensive view of how treatments perform in real-world settings.

Physicians face immense pressure to see more patients in less time. Emergency physicians, for example, are expected to see 2.4 to 3.3 patients per hour, while family doctors might handle 3.3 patients or more per hour in high-volume settings. These metrics may boost productivity, yet at the same time, often come at the expense of meaningful patient interactions. The result? Missed opportunities to understand the full scope of a patient’s experience and even the true nature of the problem.

Empowering Patients and Data

One possible example of how RWE can prioritize the patient’s voice is StuffThatWorks, a platform founded by Yael Elish, one of the founders and executives of the consumer navigational company WAZE. Like WAZE, StuffThatWorks uses AI-powered crowdsourcing to connect individuals with similar diagnoses, enabling them to share experiences and generate actionable insights into treatments and outcomes. It’s a patient-led approach that democratizes data collection, making it richer and more representative. For sponsors of medical trials, the platform offers the chance to harness a massive customer focus group, gaining access to the voices of millions of people struggling with health concerns ranging from treatment to anxiety around illness to the cost burden of sickness.

StuffThatWorks – collaborating with companies and patient organizations, recently began collaborating with The Marfan Foundation, highlighting the potential to engage non-profits in leveraging RWE to benefit global disease communities. Similarly,  The StuffThatWorks partnership with Advanced Clinical, a major research services and clinical resourcing organization, allows project teams to engage with patients directly through surveys and focus groups, enhancing patient participation in clinical research. These initiatives demonstrate the transformative power of intentionally centering patient experiences in healthcare innovation.

More and more, leading academic hospitals such as Weil Cornell Medical Center’s Genetics and Personalized Cancer Prevention Department in New York City are recognizing that care extends beyond cutting-edge medicine and outstanding physicians—it calls for experts who have been on frontlines—as patients and counselors.

“I feel fortunate to bring my real-world experience as a patient to my work as a provider,” says Emily Epstein, LMSW, Genetic Social Worker/Clinical Behavioral Health Care Specialist at Weill Cornell. “This deep understanding of the patient journey inspired me to create a role where I can offer the very support I once advocated for, rooted in true empathy and knowledge. Real-world patient experience is essential to transforming healthcare, infusing it with humanity, compassion, and meaningful change—ensuring we honor and serve both the science of medicine and humanity at its heart.”

Chief Patient Officers

Several pharmaceutical and clinical research companies have established Chief Patient Officer (CPO) roles, recognizing the importance of patient engagement. Parexel, for example, appointed Stacy Hurt as a CPO to lead patient advocacy efforts. Hurt says, “Parexel’s priority focus is on providing patient-centered solutions that enhance the clinical trial experience and make participation more accessible to everyone.”  A health professional who is both a cancer survivor and a caregiver for her son, Hurt brings a patient’s perspective to the work.  Companies like Merck, Pfizer, Sanofi, and Astellas have also created similar roles, signaling a broader industry commitment toward integrating patient insights into drug development.

The National Health Council emphasizes that as RWE becomes a cornerstone of health decision-making, the experience and needs of patients must be prioritized, not addressed as an afterthought. Integrating RWE into the health system can enhance treatment efficacy, safety, and patient satisfaction. However, this requires more than technological innovation; it demands a cultural shift within the healthcare ecosystem.

Now What?

Reflecting on my child’s medical journey and the rise of patient advocacy communities that began to take form during the HIV epidemic and that patient community’s vocal demand to be included in conversations about drug development and news flow, it’s clear that the journey toward patient-centered care is still at an awkward early stage.

Platforms like StuffThatWorks offer a glimpse into what’s possible when prioritizing patient voices. By fostering communities where individuals can share their experiences, these tools generate valuable data and empower patients to take an active role in their care.

Let us commit to creating a health system where every patient’s story is heard, valued, and acted upon. By doing so, we honor the science of medicine and humanity at its heart.

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