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Improving MDS patient management in Europe - and what you can do right away

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MDS-RIGHT consortium

13 June 2017

An unprecedented MDS stakeholder meeting was held on May 3rd, right before the opening of the MDS 2017 International Symposium in Valencia, Spain. Hosted by MDS-RIGHT, the 2-hour meeting on ‘European Perspectives on MDS Patient Management’ gathered more than 70 MDS specialists, nurses, patient advocates, researchers, regulatory/HTA experts and industry representatives. Leading representatives from each stakeholder group provided a series of short presentations and engaged in panel and audience discussions about what is needed to improve the situation for MDS patient in Europe:

More and better MDS data are needed because MDS is a relatively rare disease and information from clinical trials alone may not be able to answer all relevant questions. Therefore, more real-world data (RWD) on all subtypes of MDS — including comorbidities, outcomes, disease-specific Health-Related Quality of Life (HRQoL), and health economics — should be collected and analysed with the help of robust and well-funded patient registries. It was also noted that the European Medicines Agency (EMA) may make RWD mandatory for marketing authorisation applicants or holders. The EUMDS Registry, on which the MDS-RIGHT study is based and which is expanded to include all MDS subtypes and many other important research targets, is a 'unique resource' for informing clinical, industry, regulatory and HTA decisions.

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.

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The MDS-Europe community online platform is a unique and convenient tool for facilitating and enhancing international MDS stakeholder collaboration, e.g., by informing other stakeholders about planned and ongoing projects or about important MDS-related challenges that need tackling. The platform will be continuously expanded and more than 3,000 MDS stakeholders across Europe have already been informed about the opportunity to use this tool to engage in the discussion — by contributing a discussion article, or simply by commenting on one of the articles already online.

Five things you can do right away:

  1. Let us know if you feel the above listing of what is needed to improve the situation for MDS patients in Europe is complete or if any important point needs to be added. Simply click the speech bubble icon below to leave a comment.

  2. Tell us if you believe the above listing needs and to dos should be turned into a 'European MDS Stakeholder Manifesto', firmly summarising what the MDS community aims and how this could be achieved - and then to invite institutions, organizations and companies active in the MDS field to show their commitment by signing it. An 'MDS Stakeholder Manifesto' could then be used by all stakeholders for advocacy purposes at European and national levels.

  3. Send us any relevant and important topics that you feel need addressing on the MDS-Europe community online platform and do actively engage in online discussions. There will be at least one new discussion article every month.

  4. Encourage your friends and colleagues to regularly visit the MDS-Europe website — and to contribute to the MDS-Europe community online discussions — and ask them to help spread the word about it.

  5. Consider adding a hyperlink on your own institutional, organizational or company website to the MDS-Europe online platform.

Thank you in advance for your commitment and support

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