WASHINGTON, DC—Speakers at a workshop here sponsored by the Institute of Medicine presented their vision for a transformed US clinical trials system by the year 2020. The new vision for clinical trials, a brainchild of IOM's Forum on Drug Discovery, Development, and Translation, proposes a system in which clinical research is seamlessly woven into clinical practice in a continuous learning loop to provide the best personalized patient care possible.
In 2010 the Drug Forum published a workshop summary, “Transforming Clinical Research in the United States: Challenges and Opportunities,” where speakers presented four new draft discussion papers addressing the need for:
* Innovation in clinical trials.
* Developing a robust clinical trials workforce.
* Transforming the economics of clinical trials.
* Developing a workable infrastructure for clinical trials.
The new vision proposes four workforce groups participating in clinical research in the future:
* Community practitioners, the largest group, the clinical workforce members who participate in trials as part of their clinical practices.
* Implementers, professionals such as clinical research coordinators and research site managers who implement clinical trials at the level of a hospital or research site.
* Investigators, those at the MD, PhD/MD, PhD levels who lead and design clinical trials.
* Methodologists, the smallest group, experts who do research on the methods and policies pertaining to clinical trials (including clinical investigators, biostatisticians, epidemiologists, and health services researchers).
According to the draft paper on developing a clinical trials infrastructure for the future, involving medical practitioners in the community would involve disease-specific networks that would be designed to evaluate a series of interventions, including investigational therapies or preventive agents.
Using standardized data collection and standardized protocols would reduce costs; the draft paper on the economics of clinical trials of the future envisions improved productivity as an effect of the use of health information technology.
In the best of all possible worlds, there would be earlier drug approvals based on evolving evidence; health care decisions would be based on that evolving evidence; patients would have faster access to safe and effective products targeted to their needs; and payers would pay for only what works for whom, when, said Bruce R. Luce, PhD, MBA, Senior Vice President for Science Policy at the United BioSource Corporation and Senior Scholar in the Department of Health Policy at Jefferson Medical College.
“The nation needs a health care system that learns—that is bidirectional,” said keynote speaker Richard Platt, MD, MSc, Professor and Chair of the Department of Population Medicine at Harvard Medical School, Executive Director of the Harvard Pilgrim Health Care Institute, and Co-Chair of the Clinical Effectiveness Research Innovation Collaboration of the IOM Roundtable on Value and Science-Driven Health Care. Today, “we typically practice blind,” he added. “Research needs to be so woven into practice, that the delivery of care also is generating new knowledge.”
Clinical data represent the most critical resource for progress, he noted, and “we are haltingly but certainly going to have clinical data in electronic form,” which will speed the union of clinical research and clinical practice. Dr. Platt praised the Department of Veterans Affairs for its new approach that embeds research into clinical care of its patients.
Another speaker, Richard K. Murray, MD, Head of the Global Center for Scientific Affairs at Merck & Co., said, “Today, we have a research enterprise that is largely disconnected from patient care. Bringing research into clinical practice will be the greatest area of growth and change.”
Dr. Murray, a coauthor of the IOM draft discussion paper on the need for innovation, said that at its core the drug forum's vision involves the incorporation of clinical research into the practice of health care delivery.
And, the draft paper states, while there will continue to be a need for traditional trials looking at the benefits and risks of interventions in large numbers of patients, the new model will be one of integration of research and practice – “Doctors, nurses, and other providers should regard it as their business to generate evidence as well as to consume it in practice.”
“The United Kingdom and Canada are moving in this direction,” noted another of the coauthors, Robert M. Califf, MD, Director of the Duke Translational Medicine Institute, Professor of Medicine, and Vice Chancellor for Clinical and Translational Research at Duke University Medical Center.
“We really see clinical trials linked to the EHR as a source of data. The critical missing link is the practitioner who has the time and the inclination to participate in clinical research.”
Asked by OT if clinical research and medical practice—which have traditionally been two separate systems—can really be integrated into one functional continuum, Dr. Califf said, “It's going to happen. It's a question of whether it happens in five years or 10.”
He pointed out that one factor driving the move toward an integrated clinical research/clinical practice continuum is the dramatic escalation in the number of facts per clinical decision with new sources of biological data. “The burning platform is overwhelming complexity,” he said, citing new knowledge in structural genetics (single nucleotide polymorphisms and haplotypes); functional genetics (gene expression profiles); and proteomics and other effector molecules.
He said the consolidation of medical care into large integrated health systems is another factor driving the union of clinical research with clinical practice. “Private practice as we used to know it is going away.”
“I see a big opportunity in moving to a clinical research enterprise with real-world relevance,” said Ihor W. Rak, MD, Vice President for Clinical Neuroscience at AstraZeneca, agreeing with Dr. Califf. “We are very close to a tipping point.”
Currently, clinical trials and research are embedded more in the practice of oncology than in some other specialties, said Peter Paul Yu, MD, Director of Cancer Research at the Palo Alto Medical Foundation, a multispecialty medical group serving the San Francisco Bay area. “The challenge of personalized medicine is on the mind of all oncologists and all oncology researchers. We believe this [personalized cancer care] is a model for other diseases.”
‘Wave of the Future’
Asked by Dr. Califf if he considers himself a clinician or a researcher, Dr. Yu said, “I consider myself the wave of the future; I think the line between academic research and clinical practice is blurring.”
For the good of the patient, “I think we should tear down those walls [between practice and research] as much as possible, as soon as possible,” added Dr. Yu, who is a member of the board of directors of the American Society of Clinical Oncology and Chair of the ASCO Health Information Technology (HIT) Work Group.
He predicted that in the future some hospitals will cite their research as a marketing tool that reflects their efforts at quality improvement. What is lacking right now, said Dr. Yu, is standardization of HIT data, but that, he said, is a solvable problem. Asked by OT if HIT is moving in the direction of interoperability, he said, “Oh, yes.”
As the US clinical trials enterprise changes, it may naturally evolve in ways that produce models that are quite different from those of today, said Briggs W. Morrison, MD, Senior Vice President and Head of Worldwide Medical Excellence at Pfizer Inc. For example, said Dr. Morrison—whose medical training is in medical oncology and molecular oncology—some trials of the future may have no principal investigator.
“It may be entirely patient-driven; patients will learn about a trial and enroll on the Internet. There may be no PI,” he said. Dr. Morrison said his experience is that many investigators who take part in doing clinical trials “do it as a moonlighting project,” so it is not so farfetched to think of trials assuming new structures that are different from traditional models.