It should come as no surprise that with the explosion of novel agents for hematologic conditions, a large proportion of clinical trials are designed and sponsored by pharmaceutical companies rather than by cooperative groups of hematologists or scientists. And yet, clinicians and translational scientists are often the individuals with key insights into practical design, realistic timelines, and methods for recruiting a diverse and typical patient population into trials.

ASH is particularly invested in ensuring fair and nimble clinical trials aimed at bringing new treatments to our patients as soon as possible, as well as increasing recruitment of diverse populations. As part of that effort, the ASH Subcommittee on Clinical Trials has been working on several projects for the annual meeting that are aimed at supporting the development of scientifically rigorous and diverse clinical trials in the field of hematology. Subcommittee Chair and Vice Chair Drs. Alan Mast and Grzegorz Nowakowski, respectively, said in a recent interview with The Hematologist that the subcommittee's goal is to help re-engage hematologists in this effort.

“So, the question was: What can ASH do to engage the academic community, working with industry, to have a more distinct voice in how trials are designed? We want to make sure that clinical trials are more real-world and are enrolling the patients in need — something that's especially challenging in hematologic conditions, which are not as common as other cancers,” explained Dr. Nowakowski. “Some of those trials are more difficult to develop than typical trials in the oncology space.”

One of the first initiatives of the subcommittee was the development of the “Trials in Progress” abstract category for the ASH annual meeting (Sidebar). This initiative, which is several years old now, is designed to allow basic scientists to see the clinical studies that are being designed or introduced and to propose ideas for correlative science to accompany the efforts.

“By spotlighting these abstracts, ASH has created an opportunity for translational scientists to interact with clinical scientists,” Dr. Nowakowski continued. “This is the kind of opportunity when they can see what's being done and say, ‘Hey, this is a really interesting study, why don't we sequence your samples for a specific mutation or capture some other interesting correlatives?’” he added.

This year, a dedicated Poster Walk at the ASH annual meeting will draw attention to these trials in progress and provide attendees with an opportunity to interact with Trials in Progress presenters.

The subcommittee is also planning a Scientific Workshop on Streamlining Hematology Clinical Trials (Friday, December 10, 2:00 p.m. Eastern time). This afternoon workshop will have an international panel of experts on trial design and efficiencies. The goal is to have a forum to discuss how clinical trials are streamlined and prioritized to improve efficiency, safety, and generalizability, while preserving scientific integrity and unbiased efficacy assessments. “This is an area where basic scientists and clinicians in hematology can actively collaborate,” Dr. Mast said. There will be a focus on 1) the regulatory drug approval process, 2) translating bench discoveries to clinical care, and 3) what we’ve learned about clinical trials during the pandemic.

The subcommittee is also keenly interested in how often clinical trials accurately reflect the populations that they are trying to help. Overly restrictive inclusion criteria and a lack of diversity in clinical trial accrual can have far-reaching effects on drug development and the extended time required to bring a drug to market. Both Drs. Mast and Nowakowski will be co-chairing a Joint Scientific and Education Program Symposia on Barriers to Successful Clinical Trial Design & Accrual with Live Q&A (Saturday, December 11, 4:00 p.m. Eastern time). Of note, this session was also an idea that stemmed from the subcommittee's work.

Drs. Mast and Nowakowski are eager to have members, including fellows of course, to attend the sessions on trial design at the annual meeting. The committee is also exploring the possibility of a fellowship program that would allow trainees or others to learn the inner workings of drug development from the industry side, not just from academia, over the next year. “This fellowship is really aimed at expanding the understanding of what industry is looking for in protocols and what regulatory pressures they are under, and then have the fellow bring that knowledge and insight back to their academic institutions,” Dr. Nowakowski explained. “We see this as a very promising idea that could bring a lot of cross pollination between industry and academia,” he concluded.

Finally, the subcommittee is also dedicated to working with the ASH Clinical Research Training Institute to foster early-career faculty in the development of clinical trials. The hope is that by demonstrating to these participants the importance of the U.S. Food and Drug Administration guidance on diversity and inclusion in their trials, this can seed future trial designs and clinical research.

Highlighting the Science of Clinical Trials at the Ash Annual Meeting