Edition of 02 December 2021

On 24 November 2021; Members of the European Parliament (MEPs) discussed Europe’s Action Plan on rare diseases during a plenary session with Ms Stella Kyriakides, Commissioner for Health and Food Safety. MEPs expressed their support for a European Action Plan for Rare Diseases and called on the Commission to adopt it by 2023. During the debate, MEPs stressed the importance of harmonising EU policies on rare diseases, ensuring equal access to innovative treatment and care across Member States, securing access to affordable and effective drugs for patients, promoting the inclusion of rare disease patients into society as well as promoting awareness and education on rare diseases. MEPs also highlighted the importance of data collection and harmonisation across Member States.
The MEPs called on the Commission to implement the recommendations of the Rare 2030 foresight study as well as the recommendations of the Court of Auditors report of 2019 and to increase funds for the European Reference Networks.

The Commissioner for Health and Food Safety affirmed that the conclusions of the evaluation of the cross-border healthcare Directive in 2022, the evaluation of the Third Health Programme and the outcomes of the Rare 2030 pilot project will be discussed in a steering group by the end of 2022 and will, if appropriate, result in the revision of the EU policy framework for rare diseases in 2023. The Commissioner also stated that the commission is committed to continue to make improvements for the life of rare disease patients and families.

EURORDIS has issued a statement following the plenary session recalling that a European Action Plan for Rare Diseases would set out overarching objectives and priorities that would be beneficial to the rare disease community, allow a larger connection between research and innovation, increase the efficiency of national health systems and promote cross-border cooperation and social inclusion. The statement also calls for all stakeholders to engage directly with the President of the European Commission Ursula von der Leyen by sharing their reason for change, as part of their international campaign calling for the adoption of an EU Action Plan for Rare Diseases by the Commission.

The EMA and the Heads of Medicines Agencies (HMA) are launching a pilot project to support the repurposing of medicines. The aim of this project is to support not-for-profit organisations and academia to gather or generate sufficient evidence on the use of an established medicine in a new indication to have this new use formally authorised by a regulatory authority. As part of the pilot, EMA and the national medicines agencies will provide regulatory support, primarily scientific advice, to help these stakeholders generate a data package robust enough to support a future application by a pharmaceutical company. Sponsors wishing to seek EMA scientific advice should complete the File drug repurposing submission form by 28 February 2022.

The European Medicines Agency (EMA) has published a guideline on registry-based studies. This guideline provides method and regulatory practices for pharmaceutical industries to conduct registry-based studies. This guideline aims to help those involved in registry-based studies to better define study populations and design study protocols. It also provides guidance on data collection, data quality management and data analysis to achieve higher quality evidence. This in turn will facilitate EU regulators’ assessment of the safety and effectiveness of medicines, for the benefit of public health.