Objectives

Within MDS-RIGHT six main objectives have been addressed to developing more effective and safer interventions for elderly European citizens with anaemia and/or lower-risk MDS:

1. Comparison of outcomes and costs of existing health care interventions and policies using both established (e.g. survival and progression to high-risk myeloid cancers) and new Core Outcome Sets (COS; blood transfusion dependency, bone marrow failure, and health related quality of life (HRQoL), over and above the established set). Together with health technology assessment, this provides robust evidence to underpin the sustainable use of health care resources.
2. Enhanced compliance with diagnostic procedures in MDS by introducing, validating and comparing new minimally invasive diagnostic methods, which contributes to increased the number of correctly and timely diagnosed MDS patients.
3. Raised awareness of obtaining the right diagnosis in elderly by comparing HRQoL in MDS-patients against a control group. Raised awareness leads to improvement of HRQoL through better tailoring of health care interventions.
4. Better outcome prediction models, enriched with (epi)genetic and new Flow cytometry (FCM) techniques, supporting personalised medicine and robust economic analyses.
5. Improved, evidence-based diagnostic and therapeutic guidelines providing up-to-date, evidence-based information and regulatory guidance to health care providers, patients and regulatory agencies.
6. Establishment of an European MDS competence network encompassing all stakeholders in the field for dissemination and utilization of project results.

Expected impacts

We believe our results will have a significant impact on both individual patient outcomes and MDS-related health care use:

• Safer and more efficient health care: our results are expected to lead to more sustainable cost effective utilization of existing health care interventions and to better access of this elderly MDS-population to the right care.
• Enhanced diagnostic compliance: using new and less invasive diagnostic technologies and raising the awareness of the relevance of the right diagnosis contributes to an increased number of correctly diagnosed cases.
• Improved patient outcomes and improved HRQoL through better tailoring of interventions.
• Evidence: better treatment-outcome prediction models will support more rational, personalised treatment decisions and robust economic analyses.
• Practice: results have been (and will continue to be) incorporated into new online evidence-based diagnostic and interventional European guidelines. These new guidelines improve sustainability and equity of European health and health care.
• Guidance and information: results of the project have been (and will continue to be) disseminated in order to provide regulatory guidance to professionals and authorities and information to patients.

Overall: awareness of the importance of the right diagnosis and the right treatment for the right patient at the right time will lead to better health and health care use.