ALAN is involved in HARMONY and is representing the views of AML and ALL patient organisations: use of BigData to improve outcomes
Blood cancers, or hematologic cancers (e.g. leukemia, lymphoma and myeloma), affect the production and function of blood cells and account for about one third of cancer cases in children and about one third of cancer deaths. 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 HARMONY project aims to use ‘BigData’ to deliver information that will help to improve the care of patients with these diseases. Specifically, the project gathers together, integrates and analyses anonymous patient data from a number of high quality sources. This helps the team to define clinical endpoints and outcomes for these diseases that are recognised by all key stakeholders. Meanwhile the project’s data sharing platform facilitates and improves decision making for policy makers and clinicians alike to help them to give the right treatment to the right patient at the right time.
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: www.bd4bo.org
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.
Project objectives and scope
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
For more information on the HARMONY initiative: www.harmony-alliance.eu
Pilot study involving AML
Despite recent scientific advances, AML cure rates remain poor compared to other hematologic malignancies. In addition to accelerating clinical research, defining a core outcome set (COS) may help to improve the clinical management of AML.
A COS is a minimum set of outcomes that should be collected and reported in future clinical trials and functions as an international standard to evaluate treatments in clinical trials and other research settings (e.g., registry and observational studies) to assess real world data. Implementing a COS will improve the comparability of clinical trials and other researches, improve the consistency of reporting, and reduce selective reporting bias. In addition, the COS can be incorporated in clinical guidelines and be used to improve patient management.
HARMONY uses the Delphi method for this survey. This method will help to ensure that the COS represents the priorities of clinicians, industry, health authorities, and patients alike.
The survey is open for AML patients (18+). Click here: delphimanager.liv.ac.uk/HARMONY