Conducting effective clinical trials in hematologic malignancies requires an understanding of a rapidly evolving treatment paradigm that is increasingly nuanced, complex, and patient-directed.
Just as the underlying differences in biology and prevalence between blood cancers and solid tumors necessitates differences in treating patients, they also require specialized clinical and operational expertise within the proper conduct of clinical trials.
Biotech companies developing potential hematologic oncology therapies must adapt endpoints, study designs, and data management, as well as consider patients' experiences to address the challenges related to exploring these investigational agents for blood-based cancers. This paper examines the requirements for effectively and successfully conducting hematologic oncology clinical trials.
The basics of blood cancers and blood cancer therapy
Hematologic malignancies affect blood, bone marrow, lymph nodes, and the lymphatic system. In the United States, someone is diagnosed with a blood cancer every three minutes, and every nine minutes these malignancies result in a death.
Though awareness of the three most common forms of blood-based cancers — leukemia, lymphoma, and myeloma — is high, these diseases comprise only an estimated 10 percent of all new cancers and 9.4 percent of all cancer deaths in 2019 in the United States. In comparison, the three most common US solid tumor cancers, breast, lung and prostate cancer, together will account for an estimated 38 percent of new cancer patients and 36 percent of cancer deaths in 2019. Globally, leukemia, multiple myeloma, non-Hodgkin’s lymphoma (NHL) and Hodgkin’s lymphoma collectively accounted for only 8.2 percent of all cancer patients in 2018, excluding non-melanoma skin cancer
Despite a smaller incidence within the total oncology patient population, the global market for approved hematologic cancer drugs tallied an estimated $31.3 billion in 2018, with a projected target of $66 to $87 billion by 2025, a compound annual growth rate of well over 9 percent. This 2018 hematologic oncology therapy market share accounted for about 26 percent of the total 2018 global spending on medicines of $1.2 trillion. Of drugs with cancer indications approved between 2014 and 2019, 31 percent were for hematologic cancers, with 10 drugs alone receiving 12 indication approvals for lymphoma.