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Rare Disease advocates descend on Brussels to call for comprehensive policy action and expanded access to life-changing medicines

February 2023

Rare disease patient advocates from right across Europe have descended upon Brussels for Rare Disease Week 2023 – a four-day event hosted by EURORDIS–Rare Diseases Europe and supported by the EveryLife Foundation based in Washington DC.

Alongside empowering advocates with the knowledge and skills to effectively influence EU policymaking throughout their future careers, Brussels Rare Disease Week provides the opportunity for advocates to directly call upon policymakers to effect positive change on behalf of Europe’s rare disease community.

The week’s 44 advocates from 21 countries are already putting their knowledge and skills to practice, meeting with Members of the European Parliament, as well as representatives of the next Spanish Presidency of the EU Council. Over the week, our advocates will also meet representatives from the European Commission to give perspectives on how to work with the three main EU institutions.

Among the week’s activities will be a live-streamed conference on Wednesday 8 February on the European Commission’s highly anticipated Revision of the OMP Regulation.

The conference – hosted in the European Parliament by MEP Frédérique Ries in collaboration with EURORDIS – will explore how the EU can ensure more available, accessible and affordable treatments for rare diseases.

The advocates, assembled by EURORDIS, will present five key asks in particular when meeting with policymakers throughout Brussels Rare Disease Week:

  1. Advance the development of and access to orphan medicinal products (OMP).
  2. Improve and harmonise disability assessment practices across EU Member States.
  3. Recognise and act on the mental health needs of people living with a rare disease.
  4. Optimise the system for managing and using patients’ health data across Europe.
  5. Address the full breadth of the needs of people living with a rare disease by launching a European Action Plan for Rare Diseases.

“Isolation, exclusion, rejection. Unfortunately, these are often the words we hear from families affected by a rare disease,” says Yann Le Cam, Chief Executive Officer of EURORDIS, reflecting on the challenges and adversity faced by many Europeans with a rare condition and those close to them.

“Thanks to cooperation between EURORDIS and the EveryLife Foundation, Rare Disease Week helps spark a conversation about rare diseases between patient advocates and policymakers and fosters solidarity and hope among the wider rare disease community.”

Our advocates’ five asks

1. Advancing the development of orphan medicines

EURORDIS will be taking the opportunity of its Wednesday 8 February conference at Rare Disease Week to restate its proposed recommendations for the Revision of the OMP Regulation to attending policymakers from the European Parliament, the European Commission, the European Medicines Agency (EMA), and the upcoming Spanish Council Presidency.

The revision of the OMP Regulation is an unprecedented opportunity for the EU to revisit the current model for incentivising the development of medicines for rare diseases, and to reform the model so that success is truly driven by design.

Our advocates will be encouraging EU policymakers to define a research and development model for rare disease medicines that is centred on people’s unmet needs, and which includes patient participation in its establishment and implementation.

They will also be calling for the Revision to outline how Europe’s incentives framework can be evolved to situate Europe as a global leader in research, development and access, through a regulation that is attractive for developers, and competitive globally.

2. Improving disability assessments

To ensure all persons with disabilities, including those living with a rare disease, can exercise their right to free movement and residence across the EU, our advocates are calling for the proposed EU Disability Card to be coupled with: a) improvements to disability assessments and determination at national level, and; b) greater harmonisation of disability assessment principles and tools across Member States.

As discussed by Rebecca Tvedt Skarberg and Adéla Odrihocká – two Rare Disease Week participants who featured in our latest episode of Rare on Air – EU policy efforts to make sure that people living with a disability can equally and truly exercise their right to free movement will not be wholly successful without Member States establishing and sharing good practices on disability assessment.

Positive initiatives are being developed at the EU-level aimed at making sure people who live with a disability are empowered and able to move freely around the continent. But building a better and fairer society for people living with a rare disease and disability must be underpinned by adequate, expanded and harmonised disability assessments across the EU.

Adéla Odrihocká, Rare Disease Week participant from the Czech Republic

3. Acting on mental health needs

Experiences such as deficient medical support, a lack of medical treatment, delayed diagnoses, and social difficulties, like poverty, unemployment and stigmatisation, can all gravely impact the psychological impact of people living with a rare disease.

Our Rare Disease Week advocates are calling on people living with a rare disease to be recognised as a vulnerable population requiring specialised attention and support in the Commission’s Action Plan on mental health.

Personal experience provides the deepest understanding of the unique psychological impact that having a rare disease can have. During Rare Disease Week, I will be happy to share my experience with others and with policymakers in order to call on the EU to address the distinct mental health needs of people living with a rare condition and their families.

Velga Polinska, Rare Disease Week participant from Latvia

4. Optimising the European Health Data Space

Last year, the European Commission launched a proposal for a Regulation on the European Health Data Space (EHDS).

Smaller populations of patients affected by certain rare conditions mean that data sharing is particularly vital for learning more about rare diseases and their appropriate medical treatments. It should be no surprise, consequently, that 95% of people living with a rare disease are willing to share their health data. Within the rare disease community, data equates to hope.

Therefore, our advocates in Brussels will be insisting to EU policymakers this week that the opportunity of the EHDS is fully grasped.

Our advocates will be making the case for a EHDS which: harmonises and optimises electronic health records to facilitate cross-border healthcare; ensures the ethical use of secondary health data; engages rare disease patients to better understand what people living with a rare disease expect from research and data sharing, and; increases digital health literacy with educational programmes.

I am here at Rare Disease Week because advocating for a common health data space is a matter that I am really, deeply passionate about. A lack of data on specific rare diseases and a lack of joined-up health records were big obstacles to finding a diagnosis for my son. It should not have taken seven years for my son to receive a diagnosis. I want to prevent that experience for other patients.

Iuliana Dumitriu, Rare Disease Week participant from Romania

5. Launching a European Action Plan for Rare Diseases

Ultimately, each of our advocates’ asks would be most effective if included or incorporated into a comprehensive framework of EU actions – or a European Action Plan for Rare Diseases – that addresses the unmet needs of the 30 million people living with a rare condition across Europe.

Our advocates will this week be calling on the European Commission to deliver a goals-based Action Plan that ensures every rare disease initiative, across every Member State, works in the same direction, and achieves the overarching ambition of longer, more fulfilling, and better lives for people from our community.

A political consensus on the need for a European Action Plan for Rare Diseases has only continued to gather great momentum, officially endorsed by 21 EU Member States, MEPs from across the political spectrum, successive EU Council presidencies, and other EU bodies, such as the European Court of Auditors and the European Economic and Social Council.

While sharing their own or their family’s experiences of life with a rare condition, Rare Disease Week advocates will continue determinedly presenting to EU policymakers the strong and united message that this momentum toward a European Action Plan for Rare diseases must not be lost.

In Sweden, we do not have a national rare disease plan, and I believe that if we have a European one, then that will put pressure on our government to create and establish a rare disease platform for Sweden. Right now. The healthcare for rare diseases is fragmented at the regional level, and we need to have a national plan in Sweden. That’s why I’m here, calling for the impetus at the EU-level.

Mildred Lundgren, Rare Disease Week participant from Sweden

Follow developments from Rare Disease Week and our participants advocacy efforts in real time by following EURORDIS on Twitter, Facebook, LinkedIn or Instagram.

Julien Poulain, Communications Manager