This Real-World Example Illustrates The Power Of Patient Centricity In Clinical Trials

By Laura Williams, MD, MPH, chief patient officer, Ardelyx

The most meaningful advances in drug development stem from a deep, sustained commitment to understanding the lived experiences of patients. From early discovery through clinical trials and beyond, patient insights must be central in every decision we as drug developers make, setting a higher standard for therapeutic innovation. Integrating the patient voice into all aspects of our business, from discovery to development and commercialization of our first-in-class medicines, must be foundational to address unmet medical needs.

Here, I reflect on how patients with chronic kidney disease (CKD) successfully guided Ardelyx’s clinical trial program and issue a broader call to our industry: When we put patients at the center, we don’t just improve clinical outcomes — we redefine what success looks like.

Understanding Patient Centricity

Patient centricity refers to the intentional inclusion of patient needs, preferences, and lived experiences at every stage of the drug development life cycle. This includes designing trials that are both scientifically robust as well as practical, tolerable, and accessible for participants. It’s a commitment to understanding the journey patients face and crafting solutions that fit into — not disrupt — their lives.

In practice, understanding and integrating patient needs in clinical trials translates to better enrollment, higher retention, improved adherence, and more meaningful data generation. Inclusion of the diverse population of patients who have the disease you aim to improve is another major prerequisite of clinical trial design.  As a result, pharmaceutical companies can better interpret study results (safety, efficacy, tolerability, and quality of life improvements) from the therapies under investigation. Subsequently, clinical packages offer strong evidence to support regulatory approval and, ultimately, have a positive impact on patients.

Over the past decade, patient centricity has become an expectation among patients, advocacy communities, and regulatory bodies who recognize that a patient-centric approach accelerates the research and development necessary to address unmet patient needs.

Challenges In Hyperphosphatemia Management

CKD affects an estimated 37 million people in the United States and many more globally. For patients with advanced CKD who require dialysis, the burden of disease is significant — not just physically but emotionally and financially. Among the many complications these patients face, hyperphosphatemia — elevated phosphate levels in the blood — is one of the most common and is associated with the highest risk for morbidity and mortality, including vascular calcification, cardiovascular disease, and bone disorders, and in some cases, it can delay or prevent placement on the transplant waitlist. It is also important to note that Blacks and Hispanics have a fourfold and twofold higher rate of kidney failure, respectively, than whites.

For patients with CKD on maintenance dialysis, managing serum phosphate levels remains a long-standing battle, despite diet, dialysis, and phosphate-lowering therapies (PLTs). Until recently, the only type of PLT available was phosphate binders (PBs), which are administered as multiple pills per meal.

An estimated 70% of CKD patients on dialysis treated with PBs are unable to consistently maintain normal phosphorus levels over six months. This creates a treatment paradox: Patients are prescribed therapies that are difficult to adhere to, leaving them at continued risk for serious complications.

Patient Centricity In Ardelyx’s CKD Clinical Trials

When Ardelyx began developing a therapy for patients with CKD on maintenance dialysis with elevated phosphorus, we wanted to ensure participants in our Phase 3 clinical trials reflected the real-world population of those impacted by kidney failure. We selected trial sites in geographic areas where the incidence of CKD was known to be high and prioritized the diverse demographic makeup of the CKD population on dialysis. To achieve this feat, we not only enrolled patients from academic sites but also from non-academic urban and rural areas in a direct effort to include those most impacted by this disease. As a result, our clinical development programs showed significant demographic diversity and reflected the real-world patient population of those impacted by this disease, as indicated by the United States Renal Data System (USRDS). Among clinical trial participants:

• 44.6% identified as Black
• 48.0% identified as white
• 26.5% identified as Hispanic
• 62.0% were male and 38.0% female

Having our trial participants reflect the real-world CKD population on dialysis in the United States was critical to understanding how patients respond to treatment and whether safety and tolerability differed across patient populations. These same patients provided scientific data points through the clinical trial process to support FDA evaluation and shared their stories publicly (“data with a soul”) during the open public forum section of a major FDA-sponsored advisory committee meeting, which proved invaluable to the approval of the therapy. Too often, underrepresented populations are excluded from pivotal trials — despite bearing a disproportionate burden of the disease. This is likely due to some of the known social determinants of health, including, but not limited to, education, access to care, potential distrust of the research community, etc.

At Ardelyx, inclusion is more than just enrollment numbers. We engage with communities by providing disease education and fundraising efforts with patient advocacy groups and providing education and awareness programs at local churches and with labor groups, and more. We build trust and ensure that patient perspectives shape our development programs from the ground up by holding quarterly meetings with our dedicated patient advisory councils and having policy discussions with patient advocacy groups and policymakers. 

The patient-centric clinical program design — from the therapeutic administration methods to the diverse and representative participant population — is intentionally crafted to help CKD patients on dialysis and generate meaningful, generalizable data.

Not Just A Buzzword

Ardelyx’s clinical development programs exemplify how a patient-centric approach can drive meaningful innovation in drug development. By listening to patients, involving them in decision-making, and designing therapies that truly meet their needs, we can do more than bring new drugs to market — we can change lives.

As we move forward, I encourage all stakeholders — researchers, clinicians, and industry leaders — to embed patient centricity into the core of their work. It is not a buzzword, checkbox, or bumper sticker. It is a foundational framework that, when embraced fully, leads to better science, stronger clinical trials, more empowered patients, and, most importantly, better patient outcomes. Always remember: The patients are waiting.

About The Author:

Laura Williams, MD, MPH, has served as Ardelyx’s chief patient officer (CPO) since 2025, having joined the company in November 2020 as senior vice president, global therapeutic strategies and patient advocacy. Laura served as chief medical officer beginning in 2021 through her transition to CPO earlier this year.

Laura is a life sciences enterprise leader with 30-plus years of experience as a pharmaceutical drug developer, healthcare policy advisor, patient advocate, and portfolio strategist. She is an accomplished, results-oriented physician-scientist and board member committed to discovering, developing, and commercializing innovative therapies that address unmet medical needs.

Laura earned her Bachelor of Science degree from Mississippi State University, a Doctor of Medicine degree from the University of Iowa, and a Master of Public Health degree in epidemiology from the University of Washington. She completed her internal medicine residency at the University of Michigan, where she also served as chief resident and junior faculty.