The Clinical Trials Renaissance

In a synthesis of advanced scientific methodologies, regulatory oversight, ethical standards, and patient care never before imagined, clinical trials revolutionized prior approaches to medical treatments that had remained rooted in anecdotal evidence or traditional practices with no systematic testing. By means of formalized experiments involving multiple phases that tested the efficacy and safety of medical interventions, countless lives have been saved, and an extraordinarily high bar for evidence-based medicine was established.

In recent years, however, this traditional model appears to be reaching its limits. As the growing volume and diversity of data being collected stretches trial resources and timelines, the rigid, one-size-fits-all trial structure is ceding ground to designs that are more adaptive, flexible, and responsive to incoming data. Advances in genetics, personalized medicine, and digital technology have transformed the ways clinical trials are conducted and analyzed, expanding their scope and complexity while allowing for broader participation, more targeted therapies, and the potential for higher success rates and faster approvals.

Today the clinical trials industry stands at a critical inflection point, positioned between its traditional paradigms and the transformative potential of emerging technologies and scientific advancements. While new insights into the complexities of disease and patient heterogeneity have made clear that more adaptive trial designs are needed to match the right treatments to the right patients, the industry continues to grapple with how to modernize and optimize the drug development process while maintaining the rigor and validity that have characterized its historical success.

Over the last decade, the average clinical trial timeline has increased by 20 percent. Hobbled by inefficiencies at clinical research sites, up to 80 percent of today’s trials experience delays; the average time for a new drug to progress from initial discovery to market launch is twelve years. As the number of registered clinical trials continues to increase, failure rates remain high, with only one in ten compoundsbrought to trial reaching the market. Meanwhile, cost overruns are common, with the total cost to implement, conduct, and monitor a large, multicenter trial—ending in success or failure—regularly >exceeding $1 billion. The global cost of clinical research and trials is currently at over $50 billion annually and expected to climb to more than $85 billion by 2030.

A fundamental obstacle to success has been patient recruitment, with many trials struggling to enroll enough participants and keep them engaged throughout the study, even as the demand for participants is increasing. This is particularly true for rare disease trials, where patient populations are limited and geographically dispersed. Today, clinical trials participants travel 67 miles on average to study sites, with the number of miles rising to 135 for patients in rare disease trials.

An even more pressing challenge, however, is the lack of diversity in clinical trials participants. Recent data from the FDA shows that Black individuals make up only 8 percent of clinical trials participants, despite representing 13 percent of the US population. Similarly, Hispanic individuals represent only 11 percent of participants, despite being 19 percent of the population. More than just the representation of diverse groups, the reporting of racial, ethnic, and gender data in clinical trials remains insufficient, with under 5 percent of clinical trials data reporting racial and ethnic data.

This lack of diversity not only perpetuates health disparities and leads to significant gaps in our understanding of how treatments work across different groups, but also comes with staggering social costs. Estimates by the USC Schaeffer Center suggest that reduced life expectancy, fewer healthy years, and shortened work lives for underrepresented populations will lead to the loss of hundreds of billions of dollars over the next twenty-five years.

To address these challenges, the industry is exploring new approaches to clinical trial design. Decentralized clinical trials (DCTs)—and hybrid trials, which combine remote and in-person elements—are gaining traction as ways to improve patient access and convenience. By leveraging telemedicine, wearables, mobile apps, and remote monitoring tools, DCTs and hybrid models are opening new possibilities for patient-centric trials, while reducing the burden on participants and diversifying the pool of potential enrollees. The global wearable drug delivery market is expected to exceed $240 billion in 2024.

Toward similar goals, clinical trials are also being introduced at new research sites, like pharmacies, community clinics, and doctors’ offices. These frontier sites can also increase participant diversity while reducing logistical burdens for participants, particularly for underrepresented and economically disadvantaged patients. As site selection and management constitute critical factors in trial success, sponsors are leveraging AI and data analytics to optimize location choice around sites’ potential for patient enrollment and speed of recruitment.

But along with these advances have come new challenges. Maintaining data quality and consistency across varied trial sites, especially the new sites, is a significant hurdle. These frontier sites often lack the infrastructure and individuals with administrative expertise—such as site coordinators, principal investigators, and nurses—to support complex trials, thus requiring additional investments in training and technology.

To remedy this, key stakeholders are investing in technology platforms that unify data and insights across locations while also enabling common collaboration and communication tools. Not only do these platforms come with an array of potential cost benefits, they also represent significant improvements over today’s processes, which typically involve teams operating separately and then stitching together their siloed assessments after the fact.

Today, integrating these technologies into the existing clinical trials framework is the way forward. Decisive for success will be finding solutions for the array of data quality and standardization challenges, but equally central are the critically important issues around regulatory acceptance and patient privacy concerns. The ethical principles to which clinical trials must adhere, such as informed consent, risk-benefit assessment, and equitable access to investigational treatments, also confront new obstacles in the context of novel trial designs and emerging technologies.

For the clinical trials industry to realize the potential of these new technologies, the upcoming generation will need to combine a grasp of the latest technologies and data tools with a deep understanding of the entire clinical trials landscape. This is also true for professionals in regulatory compliance, medical writing, site management, and data analysis working in the pharmaceutical industry, at a clinical research organization, or at an academic institution. One way for people to upskill their training is to find professional development opportunities in the clinical trials field, which is not always easy to do.

University of Chicago Professional Education (UCPE) offers an online certificate program in Clinical Trials Management and Regulatory Compliancethat is designed and delivered by experts in clinical research. They are able to provide students with the skills and knowledge they need to advance their careers nd help solve some of the ongoing obstacles within this area. UCPE offers an entire educational experience within their certificate programs including advising, networking, and ongoing events and opportunities even after certificate completion. For those looking to delve into the field, UChicago offers an online global clinical research course as well.

Companies can also benefit from this program to build strong skill sets among teams and make learning opportunities available to their employees. UCPE partnered with >ZS Associates, a global professional services firm that has focused in recent years on building out its specialty in clinical trials development. Through the University’s Educational Partnership program, ZS collaborated with a skilled instructional design team and industry experts to build a customized course—rooted in UCPE’s clinical trials management certificate program—that would upskill the hundreds of ZS workers worldwide needing a deep and comprehensive dive into the history and contemporary practice of clinical trials.

Individuals looking for more program news and invitations to upcoming events can connect with an advisor today, while companies interested in partnering with the University of Chicago Professional Education can find additional information on our Corporate Partnership page.