Harnessing AI, Patient Voices and Regulatory Pragmatism to Shape the Future of Clinical Research

As the biotech industry navigates technological innovation, new regulatory thinking and rising patient expectations, clinical research and development are undergoing a profound transformation.

The convergence of AI-driven data analytics, regulatory updates and patient-centered design is reshaping how clinical trials are conceived, executed and evaluated, with the promise of bringing more targeted, efficient and meaningful therapies to patients faster.

In a conversation for Xtalks Clinical Edge™, Mike Exton, PhD, CEO of Lexicon Pharmaceuticals, shared perspectives on these intersecting factors and how they are actively being leveraged to shape the future of clinical development.

The company is developing genetic medicines for chronic conditions, including diabetes and heart conditions like hypertrophic cardiomyopathy (HCM).

The new era of drug development will be defined by closer integration of scientific innovation, patient focus and collaboration with regulators. Despite past industry fluctuations, resilience will depend on centering patient outcomes while advancing cutting-edge science.

Xtalks Clinical Edge: Issue 6 — Harnessing AI, Patient Voices and Regulatory Pragmatism to Shape the Future of Clinical Research

Xtalks Clinical Edge™ is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials. This magazine immerses you in a world where industry leaders, patient advocates and top researchers converge to provide the most insightful perspectives on clinical trials.

Harnessing Clinical Data and AI to Refine Trial Design

Modern clinical research generates mountains of data, but historically, much of this information has been underutilized. Advances in AI and machine learning (ML) are now unlocking new ways to harness this data to optimize trial design, identify responder subgroups and enhance both efficiency and outcomes.

“I think the ability to really harness all of that together to design trials to include patients who are going to get the best benefit is critical,” explains Dr. Exton.

The goal is to leverage data to better inform and refine the drug development process and ensure medicines benefit the right patients.

Companies like Lexicon are using AI and ML in clinical research to analyze heterogeneous datasets and detect predictors of patient response that go beyond standard demographic factors like age or gender. By identifying these subtle drivers of treatment variability, statistical “spread” in trial responses can be reduced, enabling more targeted, robust and efficient trial designs, especially for pivotal Phase III studies where precision is critical.

“We’re seeing incredibly interesting results that will help us shape those Phase III trials that are critically important, both from a regulatory and a patient perspective,” Dr. Exton says. “I’m really keen to continue to expand our application and thinking around it.”

“Digitalization is helping us run things more efficiently, which reduces a pretty significant cost in the end.”
— Mike Exton, PhD, CEO, Lexicon Pharmaceuticals

Regulatory Flexibility and Patient-Relevant Endpoints

Alongside advances in data analytics, regulatory agencies are increasingly demonstrating flexibility in their approaches to clinical endpoints.

Traditionally, regulatory pathways have focused on narrow, clinically defined endpoints that may not fully capture what matters most to patients. This is now changing.

“Sponsors live and die, if you like, by what the regulators will approve,” Dr. Exton notes. “But what we are seeing is increasing engagement and flexibility and recognition from the regulators that they have to expand beyond what has been a somewhat narrow set of endpoints that are approvable. And so that’s a virtuous cycle. If you have patients, sponsors and regulators who can talk together and communicate what is important for each stakeholder, then I think we get to a much better place.”

This shift is especially visible in therapeutic areas such as chronic pain, diabetes and cardiovascular disease, where endpoints that reflect the patient voice, including patients’ daily experiences and functional outcomes, are gaining traction.

Regulators in the US, in particular, have shown growing willingness to work with industry and patient groups to ensure that trial designs and endpoints reflect real-world relevance with an emphasis on what is important to patients and patient-reported outcomes, helping to accelerate innovation while maintaining rigor.

With the FDA becoming more patient-centric, this shift could help streamline drug approval processes, explains Dr. Exton.

In Lexicon’s trial for HCM, the primary endpoint focuses on patient-reported outcomes such as fatigue, energy and daily function. “By focusing on outcomes that matter to patients, we’re not only improving the relevance of our data but also potentially accelerating approvals,” he said.

Similarly, in diabetes, the FDA is expanding its perspective beyond HbA1c levels to consider “time in range,” a metric enabled by continuous glucose monitoring.

“We’re seeing encouraging proactivity from the FDA,” he says. “With the new commissioner’s approach, there’s a real opportunity for stronger collaboration between industry and regulators.”

“If you have patients, sponsors and regulators who can talk together and communicate what is important for each stakeholder, then I think we get to a much better place.”
— Mike Exton, PhD, CEO, Lexicon Pharmaceuticals

Embedding the Patient Voice and Real-World Data

Integrating the patient voice is not merely a compliance exercise; it’s a cultural mindset that permeates the organization from the top down, explains Dr. Exton. “It’s a matter of the mindset of everyone within the company.”

He shares how over the past year, Lexicon’s advocacy team has facilitated numerous interviews with patients living with diabetic neuropathic pain, providing invaluable insights into patient needs, expectations and lived experiences.

“It’s important to hear that not only to understand what it is that we’re to address, but to hear what advice they have for companies and sponsors to either improve how we’re doing our clinical trials, how we express ourselves in the community [and] how we engage with government,” he says.

Lexicon maintains weekly touchpoints with patient advocacy groups across therapeutic areas, and these conversations directly influence trial design, community engagement and even public policy initiatives. Patient input isn’t collected once and filed away — it’s incorporated continuously, including through patient advisory boards that shape clinical programs in real time.

This patient-centered approach is also being extended to policy advocacy. In October, Lexicon hosted a roundtable on chronic pain in Washington, DC, bringing together patient advocacy groups, industry and other stakeholders to define shared priorities for the future of chronic pain treatment in patients with diabetes. The discussions will inform the development of white papers and policy recommendations to be presented to Congress and senators, aiming to drive legislative change alongside regulatory and operational improvements.

Using patient-focused insights to drive not only regulatory and operational change, but also to help shape legislation, is truly impactful and transformative.

“When you get the sweet spot of all those things coming together, then the patient is the ultimate beneficiary,” Dr. Exton says.

Digitalization and the Balancing Act of Speed, Cost and Quality

As therapies become increasingly complex and targeted, the clinical research ecosystem faces a delicate balancing act between speed, cost and quality. Digital technologies are emerging as critical enablers in achieving this balance.

“Digitalization is helping us run things more efficiently, which reduces a pretty significant cost in the end,” Dr. Exton explains. “As we get better modeling and AI, that will oftentimes help predict the future. A smaller dataset can be used to extrapolate and predict what it would look like as a bigger dataset. That has the opportunity to significantly reduce time and cost.”

Although some of these capabilities are still nascent, regulatory agencies are increasingly open to collaborating with industry to explore new technologies, signaling a future where predictive modeling and adaptive trial designs could dramatically streamline development timelines without compromising data integrity.

“As we explore this new technology together, I think there’s great opportunity and we need to do it because the patients are waiting.”

Looking ahead, opportunities for biotech are expanding through technology, real-world evidence and patient-centered trial designs. Tools like continuous glucose monitors, digital health platforms and evolving endpoints can make clinical trials more relevant and efficient. By integrating these approaches, companies can accelerate the development of therapies that meaningfully improve patients’ lives while maintaining scientific rigor.

The biotech industry is entering a new paradigm of data-driven, patient-informed and regulator-aligned clinical research. The strategic integration of AI, patient perspectives and regulatory pragmatism can drive improvements in trial efficiency and ensure therapeutic innovation is aligned with patient needs.

This convergence represents more than just operational optimization, as it’s a structural shift in how the industry thinks about evidence generation, stakeholder collaboration and the ultimate goal of clinical development: improving patient outcomes.