Big data and acute leukemia – Use of big data to improve outcomes #bigdataforbloodcancer
ALAN is involved in HARMONY and is representing the views of AML, ALL and APL patient organisations, patients and families.
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.
As many blood cancers are rare, and healthcare practice varies across EU, a lack of data on relevant outcomes represents a challenge for clinicians, researchers, and decision-makers alike.
Background to project
The key outstanding questions in this field can only be answered by studying large numbers of patients. Therefore, the HARMONY Alliance developed the HARMONY Big Data Platform, assembling harmonized clinical data from over 100.000 of European patients (update December 2021).
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.
The project results in a pan-European network and brings together key stakeholders in the clinical, academic, patient, health technology assessment, regulatory, economical, ethical and pharmaceutical fields to:
- Developing a data sharing platform that empowers clinicians and policy stakeholders to improve decision-making
- Establishing a network reflecting the European blood cancers landscape
- Defining clinical endpoints and standard outcomes in various blood cancers
- Alignment of key stakeholders on relevance of these outcomes (policy makers, payers, patients)
- Providing means for analysing complex data sets comprising different layers of information
- Identifying specific markers for early registration of innovative and effective therapies for blood cancers
The project is part of IMI’s BigData for Better Outcomes programme, which aims to facilitate the use of diverse data sources to deliver results that reflect health outcomes of treatments that are meaningful for patients, clinicians, regulators, researchers, healthcare decision-makers, and others.
For more information: https://www.harmony-alliance.eu/
What is BigData and why is it important in hematology?
For blood cancers, BigData means gathering into one single database clinical, genetic and molecular information on patients and diseases which is currently maintained in a number of individual databases from clinical trials and registries in different countries. HARMONY is orchestrating the consolidation of all databases into one common platform. This will harness the enormous potential of BigData and BigData analytics to deliver insights into how the care of patients with blood cancers can be improved.
HARMONY’s partners from industry are providing access to clinical trials data, showing how drugs work in controlled circumstances. Public registries collect data on how patients are treated and the outcomes of therapies in the real world. Together, these sources are greater than the sum of their parts. For example, by combining the populations from several clinical trials, it is possible to analyze populations with rare diseases with greater statistical power than individual databases can provide.
Other BigData analyses can reveal molecular data on genomes, what proteins are expressed in different cancers, how treatments affect the expression of important genes, and more.
Know more about Harmony Alliance and Big Data in Blood Cancer
For more information on the HARMONY initiative: www.harmony-alliance.eu
HARMONY aims to tackle key issues of relevance for acute leukemia patients:
- Break down the silos: get clinicians, industry, health technology assessment agencies, patients to collaborate
- Rapid sharing, consolidation and learning from data by analysing multi-source complex data sets
- Identify areas of utmost patient need to improve patient management, reduce waste in unnecessary interventions
- Improve characterization of blood cancers in order to improve treatment strategies
- Re-define treatment goals based on individualized risk assessments and outcome measures;
- Foster the design of innovative clinical studies;
- Involve the patient perspective in all of this.
It is a unique group of 9 European patient organisations working in the different areas of hematological diseases within the HARMONY Alliance, combining years of expertise in patient-led scientific research and policy advocacy, and use this experience to the benefit of the affected patient communities.
The Patient Cluster and their patient communities are consistently involved in the definition of outcomes, the design of research projects and the information of patients. In addition the patient cluster also make sure that there is a reliable bi-directional flow of information within and outside of the HARMONY Alliance. For the first time in a major research project, the patient community sits at the table as truly equal partners to researchers, industry and regulators.
The HARMONY Alliance Patient Cluster experience will also be captured and promoted in publications and on social media.