Designing Trials That Reflect Real Lives

A turning point emerged in oncology, when patients voiced a preference that challenged long-held assumptions.

And it underscored a gap between traditional endpoints and what patients considered meaningful.

This shift prompted Sanofi to evolve from a model of patient centricity – having empathy for patients – toward integrated patient engagement, where patients help drive scientific development from the earliest stages of medical research.

To support this, Sanofi unified more than a decade of work into the Patient Community Promise, co-created with over 80 patient and caregiver groups. The work began with a direct question to patient advocates worldwide: What do you wish pharmaceutical companies would be transparent about? Their answers were specific: How many programs involve patient communities? How many clinical trials? Are diversity goals being set – and met? And, critically: Are you willing to report this publicly, even when results fall short?

In 2023, Sanofi published the Patient Community Promise, which defines how patient perspectives inform development programs, clinical trials, and evidence-generation plans. Sharing it openly created accountability across countries and functions, setting a benchmark for what patient partnership means in practice.

The revolution has been tangible. Patient insights are being integrated before a trial is designed. Clinical Outcomes Scientists, experts in identifying the measurements that matter to patients, are involved from preclinical research onward. They sit on protocol development committees and work with teams to understand what impacts daily life for people living with a particular disease. “We start in the pre-clinical space,” Vicky explains, “and we ask: what do patients living with this disease say matters most to their quality of life?” That insight shapes which signals Sanofi teams look for in Phase 1 studies, much earlier than industry standard.

This integrated approach is supported by PALADIN (Patient Advocacy Leaders and Drug Development Industry Network), a consortium launched in partnership with patient advocacy groups and biopharma organizations. One of its outputs, a framework for co-creating Target Product Profiles (the blueprint that defines the desired characteristics of a new medical product to guide its development), is now publicly available and guides early development planning.

The impact of this approach is visible across many areas of development, where patient insight has reshaped how studies are designed and how outcomes are measured.

• In atopic dermatitis (AD), Sanofi worked with patient communities to co-develop the Target Product Profile using the PALADIN framework. This ensured that the goals set at the very beginning of development reflected what people living with the condition said mattered most in their daily lives.
• In sickle cell disease (SCD), listening sessions revealed that pain crises often go unrecorded in clinical settings because they happen unpredictably and often outside the hospital. To address this, Sanofi teams developed a pain eDiary that allows patients to log crises as they occur, producing a more accurate picture of the disease. Patients also emphasized the importance of fatigue, an insight that helped teams prioritize fatigue-related endpoints that better reflected real-world experience.
• In warm autoimmune hemolytic anemia (wAIHA), patients described how difficult and exhausting it was to travel frequently for blood draws and check-ins. Their feedback prompted teams to restructure the trial so that some biweekly appointments could take place at local laboratories, reducing the burden of travel. Sanofi also adjusted the frequency of certain procedures and worked with central laboratories to explore ways to reduce blood-sample volumes.
• And in oncology, patients questioned why new tissue biopsies were needed when archived tissue samples were already available. Their perspective led Sanofi teams to shift to using archived samples instead of asking patients to undergo new procedures. This insight quickly evolved into a new standard practice.

Across all these areas, the pattern is consistent and deeply grounded: when patients share how a disease affects their daily lives, the science becomes more aligned with the realities of those lives. “Sometimes the insights are simple but transformative. When you listen closely, patients will tell you how to make the science better.”

At Sanofi, we are moving beyond traditional endpoints to co-create trials that provide real impact for patients. Mapillar Dahn-Flemister, a mother and caregiver to three daughters living with sickle cell disease, shares how patient insights are essential to shaping clinical trial design to ensure they reflect patients’ real-world experiences.   

The evolution taking place inside Sanofi parallels a broader shift happening across regulatory agencies worldwide.

• In the United States, the FDA has strengthened its expectations for patient-focused drug development, encouraging companies to demonstrate clearly how patient insights shape trial design and endpoint selection.
• Across Europe, the EMA has taken similar steps, releasing policy guidance that reinforces the role of direct patient involvement in regulatory assessment and underlines the importance of outcomes that reflect daily life, not just clinical measurements.
• And in China, this movement has gone even further. Two years ago, the Center for Drug Evaluation (CDE) issued guidance on integrating patient voice into clinical development, along with detailed recommendations on what meaningful engagement should look like. The CDE now requires that clinical outcome assessments used in global studies be culturally validated with Chinese patient populations, embedding patient partnership directly into feasibility assessments, trial design, and endpoint selection.

Vicky notes that many aspects of this guidance “drew heavily on the Sanofi experience,” highlighting how consistent and thoughtful engagement can ripple outward into broader expectations across health systems.

Taken together, these developments show that listening to patients is no longer optional or supplemental. It’s become a foundational expectation of modern clinical research worldwide.

Embedding patient partnership also means being present in communities. Sanofi sets diversity recruitment goals for 100% of its U.S. clinical trials, including in rare diseases such as SCD and wAIHA.

To support this, the company co-founded Reflections, an Atlanta-based collaboration between pharmaceutical partners, trial sites, advocacy groups, and local leaders to help remove barriers to clinical trial participation. “We had to be with people, literally in the communities with them, listening,” Vicky says. Within its first year, participation in Atlanta-area trials increased five-fold.

Sanofi’s SCAN (Sanofi Community Alliance Network) helps maintain these relationships throughout the year by attending patient meetings, supporting advocacy initiatives, and discussing clinical research in familiar community settings. “This isn’t something we do randomly,” Vicky emphasizes. “It’s embedded in how we work.”

The question guiding Vicky’s team is straightforward but ambitious: How can clinical trials be perceived as a care option, where people are proactively seeking them out? The goal is to accelerate science while maintaining rigor and inclusivity, and to build systems that prevent misalignment before it happens.

This kind of change takes time, and Sanofi tracks its progress carefully. Over the years, the number of protocol optimizations (changes made after patient review) has steadily declined. For Vicky, this downward trend isn’t about doing less; it’s about doing things right from the start. “We’ve seen a reduction because we’ve been listening and learning,” she says, “and averting the need to make some of those modifications later.”

The question now is no longer whether patient voices belong in clinical development; that’s settled. As we look across the journey for developing new medicines, the challenge becomes how consistently these partnerships can be embedded across every disease, every region, and every stage of decision-making.

Because for many patients, the choice should never be between treatments that extend life and treatments that make life worth living. They should have both.

To help research keep rising, learn about our clinical trials and how you can volunteer at https://www.sanofistudies.com/global/en/.