2021 is poised to be a year of epochal change and transformation in the clinical trial ecosystem. Consider the numerous significant events that have occurred in less than six months.
In January, the Clinical Trials Transformation Initiative (CTTI) unveiled its Transforming Trials 2030 vision stating that clinical trials should be patient-centered, easily accessible, designed with a quality approach, and fully integrated into routine healthcare delivery processes.
Signaling a significant departure from the status quo, the vision also recommends that future trials need to maximally leverage already available “real-world” clinical and nonclinical data, including data collected through digital technologies, to minimize the collection of additional trial-specific data.
Ultimately, CTTI stated that trials should contribute knowledge about how to prevent, diagnose, and treat disease that can be acted upon to improve population health. Using the same outdated research methods and processes to design and conduct clinical trials, however, will no longer be sufficient.
Antiquated State of Clinical Trials
Further illustrating this need to improve clinical trial infrastructure and conduct, Janet Woodcock, MD, acting commissioner of the U.S. Food and Drug Administration (FDA), during a CTTI webinar in January, highlighted results from her agency’s analysis of trials evaluating repurposed therapies to treat COVID-19 infections.
The results were not encouraging. Dr. Woodcock highlighted that only 5% of all trials were adequately designed to generate evidence that could change the standard of care for COVID-19 and most trials suffered from poor enrollment, particularly among U.S. sites.
In response, Dr. Woodcock called for the development of a reusable technology infrastructure and the creation of strong networks of community trial sites to facilitate the efficient conduct and execution of trials across the U.S.
In the months following these two events, collaborative projects were launched by CTTI and the Duke Margolis Center for Health Policy to evaluate best practices and future approaches for embedding clinical trials in routine healthcare delivery settings, which should deliver key learnings needed to determine how to improve and transform trials.
Leveraging Real-World Data to Transform Clinical Trials
Given this tumultuous and exciting time in the clinical trial space, I am honored to be actively contributing and sharing my experience with both CTTI and the Duke Margolis Center for Health Policy projects on embedding clinical trials, on behalf of Verana Health.
After spending approximately 15 years designing and coordinating cardiovascular clinical trials with multiple industry partners while on the faculty at the Duke Clinical Research Institute, I was fortunate to be invited to co-lead the ADAPTABLE trial starting in 2015 and continuing for the next 5 years. This pivotal trial was the first interventional study conducted within the PCORnet distributed research network of health systems across the U.S. connected via a common electronic health record (EHR) data platform.
The final ADAPTABLE results were presented on May 15 and showcased how a large clinical trial, fully enabled by real-world data (RWD), could be conducted with high-quality metrics, all of which align precisely with the CTTI Transforming Trials 2030 vision as well as Dr. Woodcock’s recommendations. Below is an overview of the ADAPTABLE study design:
Developing a World-Class, Scalable Technology Platform
Inspired by my experiences with leading the ADAPTABLE trial, I joined Verana Health in January 2020 because I was motivated to harness the full potential of diverse RWD sources to disrupt and transform clinical research, particularly prospective randomized clinical trials. Verana Health has the necessary resources, expertise, innovative spirit, and technical capabilities to accelerate change and drive innovation in the clinical trials ecosystem and achieve the goals of the CTTI vision.
At Verana Health, we are developing a world-class, scalable technology platform across multiple therapeutic areas to collect, normalize, curate, and analyze near real-time EHR data from any EHR platform used by medical specialty practices across the U.S. Our direct connections to these practices, through our partnerships with medical professional societies, enables us to leverage our platform to spearhead data-driven approaches for trial protocol development and optimization, trial site selection, and efficient recruitment of trial participants. Our product offerings are driving these transformational approaches and will facilitate innovative partnerships with life science companies and government agencies that sponsor clinical trials.
In addition to aligning with CTTI’s vision, our aspirations as a technology company for impacting clinical trials align directly with a framework for driving innovation articulated by Amy Abernethy, MD, Ph.D., (former FDA Principal Deputy Commissioner) during the National Academies of Sciences, Engineering and Medicine workshop on Envisioning a Transformed Clinical Trials Enterprise for 2030, a four-part series which concluded in May. The framework, states:
- Software technology and data innovators must be involved as equal partners
- Keep patients at the center of all innovation
- Focus upon data quality and integrity to generate credible study results
- Incorporate iterative innovation to solve difficult tasks like ensuring trial participant privacy
- Use a “product mindset” to continuously test and validate new approaches
- Pressure-test innovation and disruptive approaches with all stakeholders, including regulatory authorities to build confidence and familiarity
Developing Effective Solutions
As we build data and technology solutions to meet the clinical trial challenges using this framework, we must focus on solving clear problems that exist rather than just developing slick tools without understanding the applications and implications of these tools for trial stakeholders. Furthermore, as we develop the reusable infrastructure referenced by Dr. Woodcock, it must be able to incorporate disparate data and technology solutions into integrated, easy-to-use tools and platforms that result in more efficient processes and outcomes.
Although much has happened already in 2021, we expect even more disruption in the clinical trial space in the remaining months. As these events unfold, we have a sense of urgency to achieve the CTTI’s vision well before 2030 and we are eager to collaborate with all partners and stakeholders to transform clinical trials so they are more efficient and timely, but most importantly, also lead to meaningful results that improve clinical care delivery and patient outcomes and quality of life.