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Multi-stakeholder meeting 'European Perspectives on MDS Patient Management' - meeting report

Executive Summary

The first MDS-RIGHT multi-stakeholder meeting was held on 3 May 2017 in Valencia, Spain in conjunction with the biannual MDS 2017 International Symposium. It gathered more than 70 participants representing a wide range of MDS stakeholders, including medical specialists and nurses caring for MDS patients, MDS patient advocates, medical researchers and data managers, healthcare authorities, regulators, HTA experts and industry representatives.

The 2-hour meeting was organised to raise awareness and generate endorsement of accepted evidence-based recommendations for MDS patient management (MDS-RIGHT Task 6.3), to obtain insights on general MDS challenges and solutions across Europe, to stimulate stakeholder collaboration and to obtain feedback on the new MDS therapeutic algorithm interactive tool and the MDS-RIGHT/MDS-Europe website. The meeting included short presentations as well as panel and audience discussions on MDS-RIGHT, MDS patient management-related challenges and solutions and MDS patient management recommendations.

MDS is a relatively rare disease that may affect as many as 2 million (predominantly elderly) people across Europe.  Given that the average survival of MDS patients is only about 3-4 years and the MDS disease burden keeps growing, all stakeholders agreed that new and better drugs and drug combinations are urgently needed and they expressed their willingness to increase international stakeholder collaboration with a view to generating and exchanging more MDS-related evidence, thus helping to accelerate the development of new MDS treatment options.

This includes access to state-of-the-art diagnostic tools (including next-generation sequencing/NGS, but also tools for geriatric assessment), disease-specific Quality of Life (QoL) assessment tools, up-to-date guidance for taking the right treatment decisions upfront based on therapeutic recommendations that include all aspects of MDS patient management (as foreseen by the MDS-RIGHT therapeutic algorithm interactive tool), cross-border MDS patient support, large international clinical trials that fully reflect patient needs (as well as comorbidities such as cancer) and industry collaboration and support for academic clinical trials.

As even well-designed MDS trials may not be able to answer all relevant questions, it was considered vital by all stakeholders to collect and analyse more robust real-world data (RWD) on all subtypes of MDS - as well as on comorbidities, outcomes, QoL and health economics - with the help of well-funded patient registries.  In fact, according to the European Medicines Agency (EMA), RWD may become a requirement for marketing authorisiation applicants or holders and, therefore, the EUMDS Registry (on which the MDS-RIGHT study is based) and the MDS-RIGHT project are considered a "unique resource" for informing clinical, industry, regulatory and HTA decisions.

To read more information, including summaries of all presentations from the stakeholder meeting, download the full report here.

Download a combined pdf of the full presentations from the stakeholder meeting here.