Early-Phase Drug Development for Underrepresented haematological Diseases: Challenges and clinical translation
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About the Article Collection
Early-phase investigational drug clinical trials are a cornerstone of progress in addressing underrepresented hematological diseases, yet they come with a unique set of challenges. This Article Collection focuses on the complexities and future directions of early-phase clinical trials for three critical areas: malignant hematological diseases, non-malignant hematological diseases, and very rare diseases, those with as few as 10 to 100 diagnoses annually.
The collection delves into the obstacles faced in designing and conducting these trials, including limited patient populations, ethical considerations, and the need for innovative trial methodologies. It also highlights the importance of biomarker-driven approaches, adaptive trial designs, and collaborative networks to enhance the feasibility and success of these studies. By exploring these challenges and offering forward-looking insights and highlighting new investigational drugs in the pipeline for these disease, this collection aims to advance the field of early-phase clinical research, ultimately accelerating the delivery of promising therapies to patients with these often-overlooked conditions.
Authors are invited to contribute Editorials, Reviews, Critical Perspectives, and Original Research that highlight the novel approaches to treatment for rare hematological diseases. By publishing as part of this Article Collection, contributors will join a collaborative effort to drive progress in this vital area of research, ultimately improving the lives of patients worldwide.
The benefit of publishing as part of an Article Collection is that they often become a key resource, driving a research community forward. This has a synergistic effect on every article in the Collection. The articles often receive more views and higher citation numbers as the Collections receive increased promotion via social media and our marketing team.
If you are interested in contributing, or would like to learn more, then please reach out to the Commissioning Editor, Lydia Awosika, who will be happy to assist.
About the Guest Advisor
Akshay Sharma
Akshay Sharma, MBBS, MSc is a pediatric hematologist, oncologist and transplant physician at St. Jude Children’s Research Hospital in Memphis, TN. His postdoctoral work focused on understanding the genetic regulation of fetal hemoglobin and as a physician-scientist he continues to advance that work in the form of novel gene therapy clinical trials for patients with sickle cell disease. He leads several clinical studies of genetically modified autologous and allogeneic hematopoietic cell transplant for nonmalignant hematological disorders. The goal of his clinical research is to advance cellular therapeutics for children with hematological disorders. He is passionate about improving access to these novel therapies in an equitable and patient-centered manner.
Submission information
Submission deadlines:
Standard track: December 1st, 2026
Accelerated Publication: February 2nd, 2027
Accelerated Publication Plus: March 2nd, 2027
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