Putting Patients First In Clinical Trial Designs — A Multifaceted Approach
By Gwen Nichols, M.D., chief medical officer, The Leukemia & Lymphoma Society
Patient enrollment is critical to the success of a clinical trial and yet clinical trial designs are not always conducive to the participation of cancer patients. According to a study by Dr. Joseph Unger and colleagues at Fred Hutchinson Cancer Research Center, more than half (55.6 percent) of all cancer patients do not participate in trials because no appropriate trial is available for the patient’s cancer type and stage at the center where they are being treated. Further, when a trial was available, an additional 21.5 percent were ineligible due to exclusion criteria.
While a trial may exist elsewhere that is a fit for the patient, having to travel to participate in a trial can be an overwhelming burden for the patient and their family due to physical and financial obstacles, limited means of transportation, and other factors. Further, regardless of access or eligibility, many patients and their healthcare providers are simply not aware of clinical trials for which they may be eligible. Moreover, providers may not be equipped and trained for the complex biomarker and genetic testing that may be required to ensure the patient is eligible for the study. Lack of understanding about what a clinical trial is and fear of taking an “experimental” treatment, or of receiving a placebo, also impact patient enrollment.
As a clinician, researcher, and chief medical officer of The Leukemia & Lymphoma Society (LLS), I am responsible for finding ways to remove these barriers for blood cancer patients and their caregivers. This charge is one I take seriously and requires an approach that is multifaceted, including addressing national health policy, increasing access to experimental medicines, and building awareness of clinical trials. In the following sections, I will elaborate on several elements of our approach that pharma companies should consider implementing in their own clinical trials.
Make Clinical Trial Design Patient-Centered
LLS teamed up with researchers to design a patient-centered survey to assess patient preferences for the benefits and risks of acute myeloid leukemia (AML) treatments. Patients express clear preferences for certain outcomes (e.g., complete remission) and aversions to others (e.g., time spent in a hospital). The information collected from this study can allow us to make small changes in trials, like minimizing time in hospital or number of trips back and forth for testing, that may help reduce barriers to participation.
Increase The Speed — Deliver More Treatment Options To Patients More Quickly
In 2016 we launched our Beat AML Master Clinical Trial, a multisite collaborative trial to test multiple targeted therapies simultaneously. Beat AML showcases the ability to rapidly match patients with the therapy best suited to their disease subtype using next generation genomic technology. Our primary purpose in running Beat AML is not just to move drugs forward, although we hope that the data from the study will support such an outcome. We designed Beat AML to bring more treatment options to the patient. Based on the success of Beat AML, LLS is launching other precision medicine trials (e.g., LLS PedAL for pediatric acute leukemia).
Bring Trials To The Communities Where Patients Live
LLS is working on expanding access to high-quality clinical trials to patients served by community-based sites of care, particularly rural, minority, and/or economically disadvantaged blood cancer patients, through our IMPACT (impactful medicine providing access to clinical trials) initiative. We will be awarding grants to major cancer treatment centers that will serve as clinical trial hubs from which they will establish a network of partnerships with doctors in community settings. This will enable patients to participate in major cancer center trials at their local community centers, while minimizing travel to the hub to receive treatment. The grants will be announced and effective July 1, 2020.
Help Patients Overcome Access Hurdles
In LLS’s Clinical Trial Support Center, our nurse navigators are specially trained in blood cancers, clinical trials, and patient education and support, and they work one-on-one with patients and caregivers to create a tailored list of appropriate clinical trials that the patient can share with their physician and care team. They also work with our patient support team to help patients overcome barriers to enrolling in a trial outside their treatment center, including finding travel and lodging support. The nurses provide personal assistance throughout the entire clinical trial process, from the search to the enrollment process, and while on a trial.
In January we are launching a collaboration with The American Society of Hematology (ASH) to give its 2,500 North American member clinicians dedicated access to our nurse navigators through a new process designed to improve the flow of information between the patient, physician, and nurse navigator.
We also make available financial assistance, including help with travel costs, to help patients get to clinical trials.
Conclusion
The opportunities to improve patients’ access to clinical trials abound. We must keep the patient at the center of every effort as they hold the key to expanding our understanding of the underlying drivers of cancer and advancing new treatment. Additionally, clinical trials often give patients access to their best option for the newest available treatment in a closely supervised and supportive setting. We will not rest until patients have more barrier-free opportunities to participate in clinical studies. We are open to collaborating, so if you have ideas, I encourage you to get in touch.
About The Author:
Gwen Nichols, M.D., chief medical officer of The Leukemia & Lymphoma Society, oversees the society’s scientific research portfolio, patient services, and policy and advocacy initiatives. Before joining LLS, Dr. Nichols was the oncology site head of the Roche Translational Clinical Research Center and director of the Hematologic Malignancies Program at Columbia University. Trained in internal medicine at the University of Chicago, she completed post-doctoral research and a hematology-oncology fellowship at Memorial Sloan-Kettering Cancer Center while the attending physician. She received her medical degree with honors from the State University of New York at Buffalo.