Rethinking Clinical Endpoints and Outcomes in Oncology and Cardiometabolic Clinical Trials: A Patient-Centric Approach
Explore how clinical endpoints are evolving to be more patient-centric for better outcomes in oncology and cardiometabolic clinical trials.
Endpoint strategies are under pressure to evolve. There is a mismatch between what regulators accept as relevant endpoints and what patients care about. When designing an endpoint strategy we should ask, “Are we capturing how patients feel, function and live?” Our experts in oncology and cardiometabolic disease addressed this question. Because although they come from two seemingly different therapeutic areas, they both face the same underlying tension that the science is moving faster than the frameworks we use to evaluate it.
Featuring insights from insights from our experts Wael Harb, MD, Head of Research & Development in Early Phase Oncology at Syneos Health®; Anup Sabharwal, MD MBA FACC FACE FESC FAHA FASPC FNLA, Vice President, Head of Research & Development and Scientific Strategy in Cardiometabolic at Syneos Health; and Jane Williams, MD MPH MBA, Senior Vice President, Global Head Medical Management at Syneos Health, this discussion addresses how life science leaders can better their patient-centric outcomes in cardiometabolic and oncology clinical trials.
Are you prepared to rethink your clinical trials endpoint strategy?
What Are the Clinical Endpoints That Could Enable Faster Decision-Making in Drug Development?
“When a patient walks into a clinical trial, they are hoping to see their daughter graduate, go back to work sooner, or have the energy to live the life they came in hoping to keep, and yet, that’s not what we measure,” said Dr. Harb.
In oncology trials, surrogate endpoints, such as minimal-residual disease (MRD), are shortening the decision-making time in clinical trials. Cardiometabolic trials have anchored foundational endpoints such as major adverse cardiovascular events (MACE) and mortality, that when compared to surrogate biomarkers, could fall short when getting patients earlier access to medications.
When rethinking endpoints, experts in the drug industry should be measuring the trajectory of a patient’s life, not just the trajectory of their disease. That way, clinical endpoints could focus on the outcomes that will expedite decision-making on patient treatments and keep patient priorities at the center of clinical development to alleviate their symptoms when it matters most to them.
Moving Toward a Patient-Centric Approach on Clinical Trial Outcomes
Patients judge treatment effectiveness by asking themselves: “Do I actually feel better? Am I able to live longer?” Patients prioritize endpoints that are experiential and holistic, such as:
- Reducing symptoms
- Having fewer hospital visits
- Regaining independence
- Having a sense of normalcy
- Decreasing uncertainty
“What matters to the patient most is the real-time impact. For them, it’s about having a meaningful quality of life. They want to feel they don’t have the disease anymore,” said Dr. Sabharwal.
Although oncology and cardiometabolic diseases are different, the pressure to evolve their endpoint strategy is fundamentally the same. If traditional clinical trial endpoints are episodic, but disease is continuous, what is worth measuring? There is a need for a macro shift to move toward measurements that better reflect meaningful patient outcomes. These could be variables such as days alive and out of hospital (DAOH) and quality-of-life adjusted outcomes.
In oncology, drug developers need better surrogate biomarkers. “We are evolving on this with circulating tumor DNA (ctDNA) and MRD. The FDA’s January 2026 MRD guidance in multiple myeloma became a turning point in how we think about drug development. We can now decide on transformative therapy at 12 months instead of seven years. For a patient, that’s not a regulatory detail; that’s life,” Dr. Harb emphasized.
On the other hand, if we look at cardiometabolic disease endpoints, MACE and mortality are no longer sufficient. “There is a lot of focus on hard endpoints that have delivered rigor, credibility and meaningful impact. But they also come with time, scale and cost that can slow innovation,” explains Dr. Sabharwal. In this therapeutic area, we are seeing signals of change, such as:
- The GLP-1 story reshaping expectations for demonstrated cardiovascular benefit
- Growing interest in cardiorenal composite endpoints
- Using a more thoughtful approach of weighted and patient-centered outcomes
- The emerging role of real world data, Real World Evidence (RWE) and longitudinal patient trajectories
Our experts think that if we want to move toward a thoughtful use of patient-centered outcomes, the next generation of endpoints should allow us to think about how fast, how deep and how long a drug development or treatment can take. “Cardiometabolic clinical trials are costly, lengthy, and they are usually fragmented. Instead of having separate trials for cardiovascular, cardiorenal and peripheral arteries, why can’t we do them all at once?” asked Dr. Sabharwal.
Designing the Future of Endpoint Strategies for Better Clinical Trials
Looking ahead, our experts believe the next evolution of clinical endpoints won’t be about replacing them, but layering them, such as using:
- Hard outcomes for rigor
- Smarter endpoint surrogates for speed
- RWE for context
- Patient-centered measures to anchor everything in lived experience
- And bringing patients in the room when the endpoint strategy is being designed
As clinical research becomes increasingly patient-centric, the endpoint strategy design evolves alongside scientific innovation and changing stakeholder expectations. Emerging measures are expanding how sponsors evaluate treatment impact, incorporating a holistic view of the patient’s experience, such as considering their pain levels, emotional well-being and independence to live their lives to the fullest. Organizations that take this approach will be better positioned in the drug development industry to evolve clinical endpoints for better patient outcomes.
Discover how Syneos Health helps support more efficient biomarker development in your drug development process
Contributors
Anup Sabharwal, MD MBA FACC FACE FESC FAHA FASPC FNLA
Vice President, Head of Research and Development and Scientific Strategy-Cardiometabolic
Syneos Health
Resources
Williams J, Sabharwal A, Harb W. Are We Measuring the Right Outcomes? Rethinking Clinical Endpoints for the Next Generation of Therapies. Presented at Fierce Biotech Week 2026; May 12, 2026; Boston, MA
Related Content
Looking for exclusive insight?
