With thousands of targeted drugs and therapies under development and a large selection in the pipeline undergoing preclinical evaluation, there are far more plausible treatments than what can be tested in clinical trials. In fact, in 2017, a striking 600,000 patients were needed to fill more than 2,000 immuno-oncology trials alone. The problem: There were only around 50,000 total patients participating in research across all areas of oncology.
That equates to a meager 5 percent of cancer patients around the world involved in research, suggesting that most studies in progress will never reach enrollment goals. Thus, data will not hit the statistical prowess the trials are aiming for, trials will be delayed, millions of dollars will be left on the table, and potential blockbuster drugs will be delayed to market.
From the patient perspective, this scenario is especially disturbing. Too often, drug developers focused on trial enrollment fail to consider the care and future of the patient. In today’s system, patients are first offered opportunities to participate in trials that are not accruing — not necessarily the best trial for them. Furthermore, after a patient commits to a trial in the current system, it’s very possible they will miss out on an opportunity that would be better for them.
To compound the issue, patients who have exhausted the standard of care and are not participating in a research trial are often treated with off-label drugs, rational cocktails and increasingly expanded access to singular right-to-try options. Unfortunately, these individualized experiments are not coordinated, and their results are seldom reported, so little is learned. One of the biggest missteps in the research continuum today are these lost learning opportunities.
Those familiar with the current drug development process express skepticism that the system can ever be changed — and for good reason; many have tried and failed. However, a confluence of trends, from patient activism to the explosion of new therapies to an unprecedented open regulatory environment, make a paradigm shift from drug-developer-centric to patient-centric trials both necessary and possible.
It is time to reimagine clinical research and care as a unified, patient-centric, data and knowledge sharing network that efficiently learns from all patients on all treatments all the time. With a learning health care system, we can help physicians and their patients access relevant treatments and trials and arm them with the knowledge they need to achieve the best possible outcomes.
Shifting the paradigm will align the desires of all stakeholders — from patients and physicians to researchers, pharmaceutical companies and payers. Not only will we be able to treat each patient in accord with the best available knowledge, but we will continuously update this knowledge to benefit subsequent patients. Moreover, treatment plans can be coordinated across all patients to maximize collective learning, positively influencing the number of therapies we are able to test and their speed to market.
Reinventing clinical research in the envisioned manner requires an unprecedented level of cooperation among many stakeholders. Together we must unravel and evaluate many of our current practices, including: how we gather and analyze data, how we disseminate real-time information and recommendations to relevant stakeholders, how we close the learning loop, how new technologies and incentives influence efficiencies, and how new regulatory pathways and laws can accelerate the end goal.
I invite you to join me and four prominent panelists from the U.S. Food and Drug Administration (FDA), Dana-Farber Cancer Institute and more to expand and collaborate on these issues at the 2019 Bridging Clinical Research & Clinical Health Care Collaborative, March 4-5, 2019, in Washington, D.C. Register Now >