Novel blood cancer treatments are being tested in clinical trials across the world. Each trial uses its own outcomes to evaluate whether the treatment is effective. For instance, patients’ response to treatment may be described with complete remission, complete remission & Minimal Residue Disease negative, partial remission, or stable disease. This makes it difficult to compare and combine the study results. In addition, many studies do not assess long-term side effects of the drugs. As a result, it is difficult to draw firm conclusions about which treatments are optimal. A Core Outcome Set (COS) may offer a solution to these problems.
A COS constitutes the minimum set of outcomes that should be collected and reported in future clinical trials in order to capture all relevant information, to better compare clinical trials and to advance novel treatment approaches faster. In the past, COS were often defined by individual stakeholder groups including patients, clinicians, health authorities and the pharmaceutical industry. In HARMONY, we now for the first time will come up with COS defined by all stakeholder groups in a collaborative effort, thereby ensuring that the new COS for hematologic malignancies will be of high value to everybody.
First, the core outcome sets of the individual diseases will be determined. Patients, clinicians, EFPIA members and regulators are invited to participate. Later, a core outcome set applicable to multiple blood cancers will be defined. This may assist researchers in selecting outcomes for future trials, thereby promoting harmonization of blood cancer studies and improving clinical management of the diseases.
Watch and listen to HARMONY Partners explaining the various aspects why we are conducting Delphi Surveys and developing Core Outcome Sets:
Lars Bullinger, Charité Berlin, explains HARMONY and Outcomes Sets for blood cancer patients.
Tamás Bereczky, Leukanet, explains how can HARMONY help doctors to make the right decisions when treating blood cancer patients?
Katharina Lang, Charité Berlin, explains how HARMONY is developing Core Outcome Sets for patients.
Martje Barbus, AbbVie explains which Delphi surveys HARMONY is investigating.
The HARMONY Alliance uses big data technologies to improve the treatment of blood cancers i.e. Hematologic Malignancies. These are cancers that affect the blood and lymphatic system. In recent decades, diagnosis and treatment of these diseases has substantially improved, but many remain incurable. Taken together, hematologic malignancies rank third after lung cancer and colorectal cancer in terms of age-adjusted mortality in Europe.
HARMONY focus: Acute Lymphoblastic Leukemia (ALL) | Acute Myeloid Leukemia (AML) | Chronic Lymphocytic Leukemia (CLL) | Multiple Myeloma (MM) | Myelodysplastic Syndromes (MDS) | Non-Hodgkin Lymphoma (NHL) | Pediatric Hematologic Malignancies
HARMONY PLUS focus: Myeloproliferative (MPN) disorders and other Hematological Malignancies; Chronic Myeloid Leukemia (CML), Polycythaemia Vera (PV), Essential Thrombocythemia (ET), Myelofibrosis; Hodgkin’s Lymphoma, Waldenström Macroglobulinaemia and other rare blood cancers.
The key outstanding questions in this field can only be answered by studying large numbers of patients. Therefore, the HARMONY Alliance develop the HARMONY Big Data Platform, assembling harmonized clinical data from approx. 60.000 of European patients. This will enable the HARMONY researchers to characterize the molecular landscape of the various blood cancers, understand their pathophysiology, and identify novel drug targets. In addition, it will allow to reliably predict disease course and drug response for subgroups of patients. Ultimately, this should result in tools to rapidly select the most promising treatment strategy for a particular patient (i.e. personalized medicine). In parallel, the HARMONY Alliance is developing core outcome sets for blood cancers. These may assist researchers in selecting outcomes for future trials, thereby promoting harmonization of HM studies and improving clinical management of the diseases.
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