PI Talks Recruitment, Retention, & Trial Design For Multiple Myeloma Studies
A conversation with James Berenson, MD, founder, Institute for Myeloma & Bone Cancer Research
James Berenson, MD specializes in treating multiple myeloma (MM), staying abreast of any advancements through peer-reviewed papers, reviews, conferences, and even patients who relay new findings. But as often as he’s on the receiving end of drug development news, he’s also part of creating it. Berenson has been conducting clinical trials for nearly 40 years.
In this Q&A, Berenson shares learnings from his nearly four-decade foray into clinical research, from how he picks his trials, what matters most to patients, and how sponsor companies should approach trial design and patient recruitment for the MM patient population.
Clinical Leader: When seeking to serve as a principal investigator (PI) on an MM clinical trial, what criteria do you use to determine if it is a good fit for you, your site, and your patients?
Berenson: I am interested in innovative trials that drive the field forward and not as much in the types of trials that are confirmatory. I also want to make sure the sponsor and CRO have expertise in conducting clinical trials related to the disease (MM or a related disease) that we will be enrolling patients. As a result, I rarely participate in randomized Phase 3 studies. I want to be involved in the design and interpretation of the data and not just “a cog in the wheel.” Finally, I want to make sure we have the personnel to carry out the study, the patient population to conduct the trial, and that we do not have too many competitive trials involving the same patient population.
Why do you choose to serve as a PI on MM clinical trials?
Being a PI allows me to be an active participant in the drug development process. I want hands-on experience with the drugs so that my experience and recommendations are based on “real-world” data. The benefit to me is to be part of drug discovery, and the best part is to be able to use what we find in our research laboratory and put it to test in our clinic. This is very exciting and rewarding to me and our whole team of both laboratory scientists and clinical team.
When it comes to designing trials for patients with multiple myeloma, what do sponsor companies need to understand about patients?
Sponsors need to understand that the goal of new therapy is not only to make the patients’ lives longer but better. That means side effects and quality of life are important to consider in the design of clinical trials. Also, it means considering practical aspects of the trial, such as how often and for how long each visit is.
How best can sponsors ensure their trials meet the wants and needs of this patient population?
Sponsors should get patients and prospective participants involved early in the development of the trial. Patients are the ones who should matter most to the healthcare professionals, clinical staff, CRO, and drug sponsor. Also, sponsors should rely on prior testing of the drug to determine ways to improve the patient experience as patients deal with the impacts of both the study treatment and their underlying disease. Drug companies should not rely on trial data alone, as some sites do not fully evaluate patients’ experiences with their drugs thoroughly. This means, when selecting sites, sponsors should stay away from places in the world that do not fully engage patients. Sites that do not fully engage patients are susceptible to insufficient reporting of patient side effects. In the end, if this does not happen, the drug company will be sorry.
What is unique about the MM patient population that might affect how sponsors design their trials, whether it relates to site location, transportation needs, visit frequency, etc.?
MM patients are elderly with an average age of 70 years old and have significant comorbidities. They are generally well-informed, whether educated by site staff, personal research, and/or patient support and myeloma educational meetings. Ideally, the location of the site should be close to the patient’s home and/or work. It should allow for transportation that is easily obtained and convenient. The sponsors should be willing to underwrite travel costs which may include not only transportation for overnight stays as well if they are required as part of the trial. Ideally, the drug is oral. If not, the trial should only require monthly visits, although more frequent visits may be necessary for the first several months).
Along those lines, what are some specific ways in which sponsor companies, CROs, and/or sites can best recruit this patient population? Are there any nuances to consider?
Advertise properly. For this patient population, digital advertising, with myeloma support groups, and at hematology meetings is preferred. Inform healthcare professionals (HCPs), patient support groups, and nonprofit organizations that disseminate information about the trial. As much as possible, inform them of how this may help bring new drugs to the clinic. Provide as much information as possible on the clinical benefit and, hopefully, lack of significant side effects from the treatment.
In addition to developing a therapy that cures MM, what is the most persistent need in the MM space right now?
The most important need for patients, in general, is more treatment options that are effective, safe, and well-tolerated. Multiple myeloma remains incurable, so having other treatment options remains an overarching need. Ultimately, one would like to find a cure for this incurable form of bone marrow-based cancer. But it also may be possible that some therapies although not curative will be able to be given intermittently based on better ways to assess changes in clinical status and allow patients to have long drug holidays which would reduce side effects and drug costs. In the meantime, it will be important to personalize treatment based on the patient’s myeloma markers, blood counts, kidney function, and bone-related problems as well as other comorbidities and lifestyle.
About The Expert:
James R. Berenson, MD has been a specialist in the treatment of patients with multiple myeloma, MGUS, amyloidosis, Waldenstrom’s macroglobulinemia, and metastatic bone disease for more than 30 years. He has been involved in many breakthroughs that brought new treatments to patients with these diseases resulting in both an improvement in the length and quality of their lives. Dr. Berenson has served as a member of the National Institutes of Health – Center for Scientific Review, Clinical Oncology Study Section. He has served as a member of the Scientific Boards of the Multiple Myeloma Research Foundation, the International Myeloma Foundation, and the International Waldenstrom’s Macroglobulinemia Foundation. He also has served on both the Foundation and the Scientific Boards of the Leukemia, Lymphoma, and Myeloma Society.