More and better MDS clinical trials and observational studies are needed because eligible MDS study populations are small and interventional study endpoints do not always fully reflect unmet patient needs. Therefore, more and larger clinical trials should be conducted, recruiting MDS patients from many countries. MDS investigations should be designed and conducted with greater flexibility and fully reflect the patient perspective. In addition, more industry support should be provided for academic clinical investigations carried out by co-operative study groups.
More and better MDS treatment options are urgently needed, including drug combinations, because the average survival of MDS patients is still poor (even in lower-risk MDS) and the MDS disease burden keeps growing, due to the ageing of the population in Europe. It is therefore of crucial importance that pharmaceutical companies invest heavily in the development of novel treatment options for patients with MDS and have them approved by European regulators as well as by national HTA and reimbursement bodies.
Better professional guidance for MDS patient care is needed because about 50% of MDS patients become transfusion-dependent early after diagnosis and it is vital to make the right treatment decisions upfront and to avoid both over- and undertreatment. It is therefore important to have access to state-of-the-art MDS diagnostic tools, including Next-Generation Sequencing (NGS), but also to tools for upfront geriatric assessment and to disease-specific HRQoL assessment tools. What is more, comprehensive real-time guidance for clinical decision-making has to be provided, based on state-of-the-art therapeutic recommendations that include every aspect of MDS patient management, as foreseen by the MDS-RIGHT therapeutic algorithm interactive tools (that are currently in development).
More and better international stakeholder collaboration is an essential prerequisite for achieving the above, because no MDS stakeholder is able to make substantial progress alone, i.e., without the input and support from other MDS stakeholders. Therefore, more willingness is needed from all stakeholders to increase international collaboration in terms of generating and sharing more MDS-related evidence, providing cross-border patient support (also for participating in clinical trials in other countries), involving more MDS patients in study design and accelerating the development of new treatment options by sharing and complementing clinical studies data with RWD.