Observational studies are critical in evaluating the long-term safety and effectiveness of new therapies on patients’ lives. However, two major shifts are significantly changing the research environment.
First, groundbreaking and often curative treatments like CAR-T cell therapies demand research that extends far beyond traditional timelines. These innovative therapies require studies spanning decades, making traditional site-based approaches impractical.
For instance, how many patients would enroll and stay engaged in a fifteen-year study? Likely none. To meet this new requirement, we need a different approach that reduces the burden on patients and research sites, allows access to broader, more diverse patient groups, and adapts to changing regulatory needs.
The second major shift is a growing call to integrate the patient voice throughout the research process, from design to execution. Stakeholders across the board – sponsors, regulators, payers, clinicians, and most importantly, patients – are demanding this increased transparency and engagement.
Including the patient perspective is not only ethically right, but it also leads to better, more holistic patient-centered evidence. This is crucial as we advance towards delivering personalized treatments to those who need them most.
These shifts underscore the urgency for a new approach to observational research that is patient-centric, flexible, and sustainable. Recent technological advances, including purpose-built large language models (LLMs), and regulatory support now make it possible to realize this new model of research.
Looking ahead, a direct-to-patient model for observational research will ease the participation burden, better incorporate the patient voice, and empower individuals to take control of their health – all while meeting the growing demands for robust evidence in developing new drugs and therapeutics.
Making studies easier and more flexible for patients and sites
As the expectations around observational studies evolve, the traditional site-based model struggles to meet the demands of modern evidence generation.
Site-based research puts a heavy burden on patients to attend scheduled visits over many years, requiring significant travel and time commitments. This leads to high patient dropout rates and undermines data quality, despite the high costs of maintaining these sites.
Additionally, clinical research sites and investigators frequently lack the resources necessary to effectively run observational studies. Many are already engaged in interventional trials where support structures are more robust, so they often decline to participate in observational research. Others already have overburdened clinical schedules, so collecting additional patient data during routine encounters only adds to their heavy workloads.
To alleviate the burden on patients, in the next generation of observational studies patients can consent to provide researchers with direct access to their past, current, and future medical records, regardless of where they receive care. Patients can participate in studies remotely, making it easier and more convenient to contribute to the long-term research of innovative new therapies.
The role of a research site also changes within this new model. Every care site can become a research site that produces high-quality, informative data. LLMs, a type of AI that uses deep learning techniques to understand human language, can extract clinical observations from medical records, including a clinicians’ unstructured narrative notes.
These technologies, combined with expert human verification, ensure data is clean and ready for analysis, enhancing the quality of evidence generation. When designed thoughtfully with researchers and patients in mind, these technologies enable real-time tracking, decrease the burden on all stakeholders, and keep patients engaged and invested in their health. As a result, patient retention rates and study success increase.
Harnessing the patient voice to enhance research
In 2020, the FDA published its initial guidance on Patient-Focused Drug Development, fulfilling both a statutory requirement under the 21st Century Cures Act and a commitment outlined in the Prescription Drug User Fee Act (PDUFA) VI. The guidance covers four key topics:
Collecting comprehensive and representative patient input
Identifying what is important to patients through various methods
Selecting, developing, or modifying fit-for-purpose clinical outcomes assessments (COAs)
Incorporating COAs into endpoints for regulatory decision-making
This guidance serves as a framework for patient-centered research, addressing key study design and implementation decisions. It marks a key milestone in the FDA’s recognition of the vital role patient engagement plays – moving beyond merely asking participants to attend study visits to treating them as equal partners in the research process.
Historically, patient experience data was often treated as an afterthought or of secondary importance in many studies. The guidance, however, points towards a future where patient experience becomes central to study design and drug approval processes.
By prioritizing insights into how patients perceive risks, manage symptoms, cope with disease progression, and handle treatment side effects, research can capture the real impact of therapies on day-to-day life – not just during scheduled site visits, but also in real-time by leveraging technology.
Advancing the next generation of observational research
We are at an important turning point in observational research. It’s clear that our existing evidence generation infrastructure is inadequate for today’s challenges. The industry needs a new model that can meet evolving study requirements and is flexible enough to adapt to whatever comes next.
To succeed, we must engage a broader, more diverse group of patients, ease the burden on both patients and research sites, and fully embrace patients as true research partners. By doing so, will can ensure that the next generation of life-saving and altering treatments reach those who need them most.
This evolution is imperative for the future of healthcare and medical innovation.
Picture: DrAfter123, Getty Images
Daniel R. Drozd, MD MSc, is a physician, epidemiologist, engineer, and Chief Medical Officer at PicnicHealth, where he works extensively with product and commercial teams and oversees scientific collaborations with the company’s industry and academic partners. Among his many contributions to improving health outcomes for patients everywhere, he helped design and develop the registry database for the largest multicentered observational HIV cohort in North America to study the long-term impact of HIV treatments.
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