Google Gemini, Cure
Overview
Regulators and investors still want clean data, but patients want proof a therapy changes daily life. Here’s how early-stage biotech teams can choose endpoints that are measurable, meaningful, and feasible.
Your biotech’s success is not defined solely by receiving regulatory approval or showing statistically significant trial results. Increasingly, investors, regulators, and, most importantly, patients and caregivers want to know how a product or therapeutic will actually change lives. For that reason, founders need to consider patient endpoints, meaning what patients experience and value, early in study design. Otherwise, they risk slowing recruitment, weakening engagement, and creating downstream regulatory risk.
By making endpoints patient-centered from the outset, founders ensure the focus remains on how patients feel, function, and survive, said Amin Zargar, PhD, CEO of ResVita Bio, a company using synthetic biology and metabolic engineering to develop therapies for skin diseases such as Netherton Syndrome. These endpoints reflect the aspects of treatment and disease management that matter most to patients themselves. When defining them, Zargar said, founders should prioritize factors like symptom burden, quality of life, functional status, and treatment tolerability.
Doing so also provides insights that traditional biomarkers often cannot, and can help differentiate a therapy in crowded markets while strengthening clinical relevance, added Alyssa Master, PhD, a biomedical engineer and director of venture development at the University of Chicago’s Polsky Center for Entrepreneurship and Innovation. Prioritizing patient-centered endpoints has also become a regulatory expectation. By aligning with those expectations early, founders can reduce late-stage risk and improve the odds of successful market entry, she said.
Below is a guide for early-stage founders navigating how to identify, define, and justify patient-centered endpoints. It also outlines how endpoints done well can strengthen evidence, build stakeholder trust, and ultimately support innovations that meaningfully affect patient lives.
How to Identify the Outcomes Patients Prioritize in Daily Life
Selecting patient-centered endpoints begins with understanding what patients actually value in their day-to-day lives, not just what is easiest to measure in a clinical trial, said Zargar. Symptoms, functional limitations, emotional burden, and treatment expectations should shape trial design more than traditional clinical metrics alone.
For ResVita Bio, that process involved meeting with patient advocacy groups, clinicians treating the disease, and patients themselves. “It’s very helpful to think about what patients consider to be a successful treatment, and not just what you think, or even what the clinician thinks,” Zargar said.
Those priorities can vary widely by disease stage, demographics, and lived experience. But through intentional engagement with patient communities, real-world observation, and ongoing communication, patterns begin to emerge. Those patterns, Zargar said, should directly inform endpoint selection.
One way to operationalize that process is to involve a chief medical officer early, rather than relying solely on a research and development team. “Having a chief medical officer involved in these conversations, going back with clinicians, patients, and your CRO, helps ensure your endpoints are as real-world and translatable as possible,” he said. “The last thing you want is to spend years running a clinical trial and end up with endpoints that aren’t meaningful. That can kill a program, kill a company, and kill a therapy.”
How to Translate Patient Priorities Into Measurable, Validated Endpoints
Understanding what matters to patients is only the first step. The harder task is translating those priorities into endpoints that can be measured reliably and accepted by regulators and other stakeholders.
Patient experiences are often nuanced, subjective, and context-dependent, which makes them difficult to capture using traditional quantitative tools. Founders need to invest time in developing measures that are scientifically sound, sensitive to change, and feasible within the constraints of a clinical trial, said Master.
“This can be tricky depending on the therapeutic area,” she said. “I recommend working closely with physicians to understand what scoring systems are already used for the condition you’re studying, then getting as quantitative as possible. If a patient says, ‘I can’t get out of bed in the morning,’ look for a way to quantify improvement, like working toward a specific number of hours out of bed each day. You want something measurable that still maps back to quality of life.”
For caregivers, that translation may look different. It could mean a measurable reduction in the level of support required after treatment. But founders should be cautious when selecting measures. Many commonly used scoring systems are only semi-quantitative, relying on subjective assessments rather than objective data. Pain and fatigue scales often fall into this category.
“When you’re selecting endpoints, avoid relying too heavily on semi-quantitative measures,” Master said. “Instead, look for parameters you can clearly measure. For instance, before treatment a patient could walk one block, and afterward they can walk a mile. That’s much more concrete.”
How to Create Endpoints That Are Feasible and Low Burden for Patients
Patient-centered endpoints lose value if they become too burdensome or impractical for participants to complete consistently. Excessive assessments, long questionnaires, painful procedures, or frequent clinic visits can increase dropout rates, reduce data quality, and undermine trial integrity, said Zargar.
Feasibility is not just an operational concern. It directly affects patient experience and data reliability. Unrealistic expectations can lead to incomplete or biased data and ultimately weaken a study’s conclusions. The goal is to strike a balance between clinical rigor and participant burden.
“I always recommend talking to the FDA as early as possible,” said Master. “The more you communicate with them about your design and rationale, the better.”
Founders should also map the patient journey in detail. Talk directly with patients about what they will tolerate, then design trials within those boundaries rather than pushing against them. High dropout rates create missing data, which can compromise statistical significance.
“If a lot of patients drop out before the study ends, you may lose the ability to demonstrate efficacy,” Master said. “That can ripple into the commercial side and ultimately affect investment. Endpoint selection can quietly balloon into a major source of risk.”
When possible, she suggested starting with a pilot study. Pilots allow teams to test feasibility, assess compliance, and identify data collection challenges before committing significant resources. “You can correct course before you’re running a full-scale trial and spending real money,” she said.
What Regulators Expect From Patient-Centered Endpoints
Regulators want to understand how patient-centered endpoints are defined, collected, and justified. While agencies are increasingly open to outcomes that reflect how patients feel and function, they still expect clear evidence of scientific validity and clinical relevance.
Poorly designed endpoints, or those unsupported by evidence, can raise concerns and slow development rather than accelerate it. For startups, understanding regulatory expectations early is critical.
“I’ll start with the caveat that I’m not a regulator,” Master said. “But from a high level, the FDA is trying to make it easier to get new drugs and devices to market. Timelines have shortened, and the emphasis is on getting effective therapies to patients faster.”
That flexibility still comes with expectations. Founders need to ensure their patient-centered endpoints are thoughtfully developed and supported by data that demonstrate both safety and efficacy.
Six Examples of Strong Patient-Centered Endpoints
Strong patient-centered endpoints help demonstrate clear links between patient experience, clinical relevance, and measurable change. While endpoints vary by disease and modality, many relate directly to quality of life for patients and, in some cases, caregivers, said Master.
Both Master and Zargar offered examples commonly viewed as meaningful across therapeutic areas. These examples require refinement and pairing with validated measurement tools to produce robust data.
Symptom severity and frequency: Patients experience fewer flare-ups, shorter symptom duration, or reduced severity, including improvements in itching, pain, or hair loss.
Functional status: Patients are better able to perform daily activities such as dressing, carrying objects, or walking longer distances.
Independence or autonomy: Patients rely less on caregivers or are able to return to work or school.
Time-based outcomes: Patients experience longer periods of symptom control without treatment escalation.
Disease-specific milestones: Therapies prevent disease-related events like hospitalizations or cognitive or motor decline.
Treatment tolerability: Dosing, administration, side effects, and time burden do not significantly disrupt daily life.
How Endpoint Choices Affect Retention, Trial Burden, and Statistical Power
Endpoint selection shapes patient experience and retention. Endpoints that are overly complex, invasive, or misaligned with patient priorities increase trial burden, leading to higher dropout rates and missing data.
“You have to be careful not to unduly burden patients,” said Zargar. He pointed to punch biopsies in dermatology trials as an example. While biopsies can provide rich scientific data, they are painful and invasive. “Patients understandably don’t want a piece of their skin removed unless it’s absolutely necessary.”
At ResVita Bio, the team opted for newer, FDA-compliant outcome methods that allowed them to gather meaningful data noninvasively. “We were careful not to burden patients with punch biopsies or tape stripping, especially in a phase one study where the primary endpoint is safety,” Zargar said.
Zargar recommends that new biotechs look beyond classical approaches when possible and evaluate newer tools that can capture biological activity without unnecessary discomfort. Improved retention reduces operational costs and preserves statistical power, making it easier to detect treatment effects.
How to Communicate Your Endpoint Strategy to Investors, Partners, and Reviewers
If patient endpoints are not clearly understood and trusted by investors, partners, and reviewers, even well-designed programs can struggle. Founders should develop a clear rationale linking patient relevance to scientific and commercial outcomes.
Effective storytelling around endpoints helps demonstrate strategic discipline, reduce perceived development risk, and build confidence, said Master. She often recommends bringing in clinical trial–focused consultants to help shape messaging, particularly for technically driven founding teams.
Audience matters, she added. The way endpoints are discussed with scientific diligence teams should differ from how they are framed for investors focused on financial models.
Finally, while investors expect returns, they also invest in vision. “It’s easy for technical founders to get lost in the details,” Master said. “Remember that the real goal is making patients’ lives better. Investors are human too. They want to know they’re helping people.”
Common Mistakes Founders Make When Choosing Patient-Centered Endpoints
Even with good intentions, founders can run into issues when selecting patient-centered endpoints. Common mistakes often stem from assumptions about patient needs or prioritizing convenience over long-term value.
These missteps can weaken evidence, raise regulatory concerns, or undermine patient trust. Correcting endpoint errors late in development is costly and sometimes impossible, Master said. Common mistakes include:
Picking endpoints that sound scientific but don’t reflect lived experience
Choosing measures that are overly complex or increase dropout risk
Failing to research prior clinical work in the space
Relying on founder or clinician assumptions rather than patient input
Overlooking differences across disease stages or patient subgroups
Using endpoints that lack reliability or sensitivity to change
Underestimating data collection and analysis challenges
“This is where early FDA interaction is invaluable,” Master said. She also recommends reviewing publicly available trial data on ClinicalTrials.gov to understand how similar studies were designed and where they ran into trouble.
Pilots, she added, allow teams to identify and fix problems early. “You can make mistakes cheaply and quickly, then invest heavily once the details are worked out.”
