When Dorothy’s character from The Wizard of Oz inadvertently surrounds herself with a motley cast of perfectly imperfect outsiders, ranging from a skittish lion to a foolish scarecrow to an unfeeling tin man, the instinct might be to think, “She’s doomed. She’d be better off finding her way back to Kansas on her own (#protagonistgirlpower).” Could this crew ever find the cohesion and competencies necessary to surmount the challenges to come? You bet. As sure as Dorothy’s slippers were ruby red… Though one of the major underlying themes of the story remains self-determination and the notion that we each possess the very qualities we may feel we lack, another central, perhaps more subtle, message emphasizes the power of teamwork. As Dorothy treks the yellow brick road (aka, the symbol of hope) to find the (in the end, not so) Great and Powerful Wizard, she befriends the aforementioned trio who not only enable her to reach her end-goal but become fiercely loyal companions on a most unconventional road trip. The road, and their journey, are bumpy — paved with cackling wicked witches, armies of flying monkeys, and a deadly poppy field … and that’s not counting the lions and tigers and bears (oh my!). But, you see, in the end, the Scarecrow had a great head on his shoulders, the Tinman was as kind as they come, and the Lion was intrepid, even noble. Could Dorothy have made it on her own? Maybe. But it wouldn’t have been nearly as convivial, nor as productive. Forging collaborative networks in hematologic research is analogous to the venture of Dorothy et al: It, too, requires contribution of unique skillsets, galvanized partnerships, solidarity in diversity, and a concerted, committed effort to not only follow, but in fact, pave a path toward the future of hematology.
In 2018, when ASH established the ASH Research Collaborative (ASH RC), a non-profit organization whose objective is to “foster collaborative partnerships that accelerate progress in hematology, with the goal of improving the lives of people affected by blood diseases,” it was ostensibly paving the inaugural blocks of its own yellow brick road. Instead of Dorothy and her diversiform allies, there is an even more robust squad: a consortium of Clinical Trials Networks serving to expedite drug development, promote clinical trials, and harmonize practices. And sure, there’s no Emerald City, but there’s is an equally impressive Data Hub technology platform facilitating data exchange on malignant and nonmalignant hematologic conditions on a global scale. Two sessions that brilliantly highlighted these no-less-than-magical resources and their substantial outputs are presented below.
The first stop on our “somewhere-over-the-rainbow” odyssey was the Special-Interest Session on “The Role of Real World Data Collection in Hematologic Malignancies” that occurred on Sunday. With a remarkably diverse group of panelists representing scientific organizations, regulatory agencies, and patient perspectives, this session illuminated the position of the ASH RC and HARMONY Alliance at the very real heart of real-world data (RWD) generation. Dr. John Gribben led off with the dominant theme of needing data that “truly reflect the diversity of our populations,” and underlined the strength of RWD sets in being “more representative.” ASH President Dr. Martin Tallman chaired the session and also presented on “The Role of Real-World Data in Hematologic Malignancy Research (ASH Research Collaborative)." Dr. Guillermo Sanz covered “Real-World Data Expectations, Challenges, and Potential Solutions to Accelerate Research (HARMONY),” voicing the “increasing interest in the role of RWD to accelerate research and speed-up drug development for more effective treatments of blood cancer patients.” He added, “The HARMONY Alliance, a European Public-Private Partnership for Big Data in Hematology, is collecting RWD from multiple sources and has built a secure Big Data platform, containing over 80,000 patient data sets.” Dr. Sanz described this Special-Interest session as “inspiring,” covering “challenges and potential solutions to accelerate research, regulatory perspective on data sharing and RWD … (as well as) patient ... and clinical viewpoints on RWD for research.” Dr. William A. Wood (ASH RC) reiterated the importance of RWD in hematology in his coverage of “Real-World Data Expectations, Challenges, and Potential Solutions to Accelerate Research.” These data can “inform the ways in which we generate hypotheses for further scientific discovery… contribute to clinical practice improvement at the local or multisite collaborative level… and help us evaluate the safety and effectiveness of therapies and other care delivery strategies,” Dr. Wood stated. This makes real-world evidence generation “increasingly important to many members of the hematology research and practice ecosystem, including patients, families, clinicians, researchers, industry, the FDA [U.S. Food and Drug Administration], and many others,” he added. He further expanded on how the ASH RC Data Hub is aptly “designed to accelerate research and improve clinical practice.” While this work initially focused on sickle cell disease (SCD) and multiple myeloma, “the work of the ASH RC Data Hub… should be of broad relevance to all attendees of the ASH annual meeting,” Dr. Wood assured us. He also touched upon the effort of the EU HARMONY group in malignant hematology and how the ASH RC and the former were “natural complements to one another.” He concluded by reaffirming the central motivation of this work saying, “We intend to build relationships across sites to bring researchers and clinicians together under a shared mission as part of a real-world Collaborative Hematology Network… to bring fulfillment and satisfaction to the work involved in hematologic research and practice, for the benefit of all.” Finally, one of the most unique angles of this session was its inclusion of Jan Geissler’s perspective as a chronic myeloid leukemia patient and representative of the HARMONY Alliance patient cluster. He articulated how RWD, in contrast to that generated from randomized trials, “covers broader and more diverse populations” and more fully “captures patients’ experiences, needs and priorities.” He concluded by reminding us that patient organizations are eager to contribute to and facilitate this type of research, motivating us to continue building dialogue with our patients.
The “Special Education Session on Collaborative Hematology Networks” that took place on Monday, told its own enchanting story of the critical importance of global connectedness in hematology research. Session chair, Dr. Lisa Baumann Kreuziger provided an overview saying, “Three networks, The Sickle Cell Disease Clinical Trials Network (SCD-CTN), The H. Jean Khoury Cure CML Consortium, (and) Venous Thromboembolism Network United States (VENUS), are highlighted to show the breadth of hematology research.” How critical are these collaborations? “Clinical research networks are essential to decrease the time from study question to completion,” said Dr. Baumann Kreuziger (pictured seated left) while presenting details on VENUS, whose mission is “to develop and conduct clinical trials and research about thrombosis.” Dr. Ehab Atallah (pictured speaking) discussed the H. Jean Khoury Cure CML Consortium (HJKC3), whose establishment, current activities, and future directions, powerfully focus on Dr. Khoury’s vision “to improve the care of patients with CML through collaboration.” Dr. Charles S. Abrams (pictured seated right) profiled the ASH RC Data Hub and SCD-CTN. He articulately summarized, “The ASH RC’s SCD-CTN is part of ASH’s multiprong initiative to improve the lives of patients with Sickle Cell.” Needless to say, there are a number of challenges facing those tackling trials in SCD, which Dr. Abrams short-listed: “a shortage of clinical trial sites with experienced investigators, lack of access to a centralized data repository, poor coordination among sites, and the lack of the patient’s voice in trial design that historically has led to poor patient enrollment.” The guiding light of this network is the hope that it will “accelerate research by forging new relationships with patients to increase their understanding of clinical trials and trust in researchers, and by eliminating inefficiencies through the use of a centralized data repository (the ASH RC Data Hub).” What is the network’s scope? Tremendous. Dr. Abrams anticipates that “within the next month or so, we will have onboarded 14 clinical research hubs involving 68 geographically diverse clinical research sites that care for approximately 30,000 children and adults living with SCD.”
The principle of thriving through teamwork clearly transcends fiction and has become a brave reality thanks to the unfaltering efforts of these groups. Perhaps the key, then, is to look beyond individual shortcomings, beyond what we might perceive as unsurmountable limitations (our own modern-day flying monkeys), and as these collaborative networks have exemplified, fully embrace the power in diversity to find inspiration and innovation as a form of wizardry to reach our collective goals. There is inherent value to the journey, and the company we keep, as it is our connections and relationships that effectively bring out the best in each of us, ensuring our shared success. So rather than click our heels and recite our “no place like home,” we might even decide to link arms, put on our finest (maybe even ruby red) kicks, and forge onward a little while longer on this proverbial road, welcoming new friends along the way (… and their little dogs, too).
Dr. Szuber indicated no relevant conflicts of interest.