Home> News> #bigdataforbloodcancer blog: The importance of patient involvement and Patient Reported Outcomes in data-driven blood cancer research

#bigdataforbloodcancer blog: The importance of patient involvement and Patient Reported Outcomes in data-driven blood cancer research

September 29, 2022 17:15 - x 00, 0 - 00:00

#bigdataforbloodcancer, Patients

Despite the many advances in medicine, healthcare’s Achilles’ heel remains a one-size-fits-all model that often overlooks the patient’s perspective. Data collected directly from patients in real-world settings and analyzed by AI has the potential to become the loudest voice in research.

In this blog the HARMONY Patient Cluster leads share their perspectives. This blog is published on the 2nd Big Data for Blood Cancer Awareness Day, at the end of Blood Cancer Awareness Month.



What patients face in their daily lives does not always reach scientists working in laboratories. These include not only the outcomes of the treatments captured precisely where the therapies are applied but also individual experiences, side effects of medications and their impact on quality of life, compliance, and unmet needs. Besides all of this, the same pill or therapy can be applied to different patients who do not fit the study cohorts: young and older individuals, with exacerbated symptoms or a mild course of disease, with comorbidities or living in different environments.

What has been missing is a feedback loop between patients and scientists, close cooperation beyond the classical randomized clinical trials. It has been changing recently with the rise of technologies like Big Data, artificial intelligence algorithms, and wearable technologies that are able to collect a wide range of data. Patient Reported Outcomes (PRO) allows for more granular insights into objective, measurable patient experiences along the course of the treatment. The new bridge between individuals and science built on data gives the patients a stronger voice and enhances their advocacy.


According to the Lancet Haematology paper Improving outcomes through patient-generated evidence - the next step in patient advocacy, there are meaningful datasets that patients can generate: data on treatment adherence, inequalities in access to diagnostics or therapies, current treatment patterns, quality of life, the burden of disease, the impact of illness on society, disease-related outcomes, and patient preferences on the risks and benefits. Download the paper in PDF here >

Jan Geissler, LeukaNET, HARMONY Partner and author of the Lancet Haematology paper: “We are talking about reliable data that captures patients’ daily lived experiences and should unquestionably become a part of modern evidence generation”.


The HARMONY Alliance amplifies patient voice in research

HARMONY recognized this from the beginning of the project, setting new standards for patient involvement in big data research and bringing patient involvement in biomedical research to the next level. It started with including the alliances of the patient organizations of the nine hematologic malignancies covered by the HARMONY Alliance. Thanks to the Patient Cluster [link], blood cancer patients are involved in the development, submission, conduct and analysis of research proposals. 

“By representing the patient perspective, the Patient Cluster can enrich research and development (R&D) through prioritizing core outcomes, matching the patients’ true unmet needs, communicating research projects and results to the patient communities in an understandable way,” according to Tamás Bereczky, LeukaNET, HARMONY Partner.

The evidence shows this can lead to better outcomes and healthier cooperation across the entire research and development continuum. “HARMONY gives patients the opportunity to drive research toward their own interests and answer their own questions,” remarks Eglys Gonzalez, LeukaNET, HARMONY Partner and working on strengthening the scientific profile of the patient community in the HARMONY Alliance. Gonzalez adds: “Within the HARMONY Alliance the role of the patients is related to their involvement in the processes of research proposals, representing the patient perspective along every step of the project, among others.”.


Data generated by individuals return to them as improved therapies

Patients in hematology can collect and deploy scientific evidence that is then integrated with the evidence generated by academia or the pharmaceutical industry. This was made possible by a fusion of three factors. First, data generated by patients can be captured automatically using breakthrough technologies or entered in a digitalized, standardized form. Second, the Harmony Alliance Big Data Platform unlocked new opportunities to harmonize data from different sources and analyze vast datasets. Third, HARMONY Alliance has successfully included patient organizations that can bring their expertise and activate the patient networks.  According to Bereczky, the patient perspective – backend by the data they deliver – sometimes conflicts with the hypotheses and assumptions of healthcare professionals and academics working in biomedical research. However, it is a valuable asset that bridges the gap between patients and research.

“The inclusion of patient-generated evidence in biomedical research will always lead to outcomes that are more patient-centric and relevant,” notes Bereczky.

The approach to seizing the opportunity of Patient Reported Outcomes (PRO) is a win-win. Researchers can finally closely examine the patient’s view and – from the opposite perspective – the patients gain the chance to accompany, figuratively, the researchers when they develop new therapies.

“Supplementing the missing data with PGO data can significantly contribute to complete research, moving medicine toward being more patient-centered,” emphasizes Gonzalez.


To capture the right data, a trusted ecosystem must be in place

It takes a lot of coordination and careful planning to develop a multi-stakeholder collaboration like the HARMONY Alliance. Technical infrastructure enabling seamless cooperation is one thing; another challenge is connecting people with various backgrounds and empowering them to work together. 

“Being part of the HARMONY Alliance has taught us about the importance of patient-reported outcomes (PROs), especially quality of life (QoL) for patients with hematologic malignancies. While other variables and outcomes – like overall survival (OS) and disease-free survival (DFS) – are much more prevalent and accepted, the decisions and outlooks of patients are fundamentally influenced by factors like QoL. They determine adherence to treatment, retention in clinical trials, and the acceptance of new treatment options,” mentions Bereczky.

The HARMONY Alliance Patient Cluster has already contributed to groundbreaking work in the field of PROs. What also matters is the communication structures created across stakeholder groups, the implementation of procedures for patient involvement and the establishment of pathways to better include patients in research and development. The HARMONY Alliance has already achieved significant results in putting patients, researchers, and clinicians at the same table. And it is data that has the power to bring the various parties together. The first steps have been taken to achieve patient-centered research. Much is yet to be done, however. With patients willing to generate and donate data, the future of inclusive and responsible research looks brighter. 



#BigDataforbloodcancer | Big Data accelerate better and faster treatment for Patients with Hematologic Malignancies

The HARMONY Alliance is a European Public–Private Partnership for Big Data in Hematology that is capturing and mining Big Data on various Hematologic Malignancies. The HARMONY Alliance, funded by Innovative Medicines Initiative, unites more than 100 organizations such as European medical associations, hospitals, research institutes, patient organizations, pharmaceutical companies, IT companies, and health technology assessment/regulatory agencies. The inclusion of all these stakeholders reflects the HARMONY ambition to develop tools that not only can be adopted by clinicians and patients but also be of interest for regulators, payers, and health technology assessment bodies.

HARMONY uses Big Data analytics to accelerate the development of more effective treatments for blood cancer patients. Data are stored in the HARMONY Big Data Platform, which has already identified over 100,000 anonymized patient records, making it one of the largest databases of its kind. Leading research teams are currently using this wealth of information to answer critical questions about hematologic malignancies that cannot be addressed with other methods. In addition, HARMONY is running projects to develop core outcome sets, as well as Health Technology Assessment projects and multi-stakeholder activities. The HARMONY Alliance is currently running two projects: HARMONY (November 2016-June 2023) and HARMONY PLUS (October 2020-October 2023). Both have work packages dedicated to HTA and multi-stakeholder engagement.


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