A new European policy framework: eight recommendations to leave no person living with a rare disease behind

A new policy framework is the only way to ensure that progress made for the 30 million people living with a rare disease in Europe is not left to luck or chance during the next decade.

The eight Rare 2030 recommendations, the result of the two-year participative Foresight Study led by EURORDIS-Rare Diseases Europe, set the roadmap for the next decade of rare disease policies to leave no one behind.

While great progress has been made in the last decade and rare diseases are recognised as a public health and human rights priority, for people living with a rare disease in Europe, huge challenges remain.

  • Only 6% have access to a treatment for their disease.
  • 52% of patients and carers say their disease has a severe or very severe impact on daily life.

COVID-19 has further exacerbated the vulnerability of people with rare diseases. It has shown the need for more resilient healthcare systems, while demonstrating the possibilities that arise from international research collaborations, and rapidly streamlining procedures and regulatory activity.

Actions at Member State level alone, or legislative changes in only specific areas are not enough. We need a new European collective strategy for rare diseases to bring Member States’ commitment to rare diseases under a common umbrella and mark a step forward in the post-COVID world.

We have a rare opportunity to bring the policy in step with the scientific, technological and societal shifts. Only by implementing new National Plans and policies fit-for-purpose for this decade, aligned with the Europe’s strategy, can we actively achieve the preferred future, one prioritising patient-led innovations and social justice, and prepare for the shortcomings of other possible scenarios which may include innovations led by the profits or one that prioritises individual gains over solidarity.

“We must reincentivise Europe’s strategy for rare diseases with measurable objectives for unmet needs. We must renew our strategy to make equal access to health a priority and a distinct value of Europe. We must coordinate across research, digital, healthcare, and social welfare to harvest the synergies of European Collaboration,” – Terkel Andersen, President of the Board, EURORDIS-Rare Diseases Europe.

The Rare 2030 recommendations

The proposed recommendations align with Europe’s commitment to delivering the Sustainable Development Goals (SDGs) by 2030, adopted by all UN Member States in 2015. The recommendations cover improved diagnosis, treatment, and care to better research, data management, and European and national infrastructures.

They bring forward innovative concepts and models to support the research, development and access to affordable medicines and care and achieve universal health coverage. For the first time, they present measurable goals that every country in Europe should be working towards.

Rare 2030 Foresight Study

Over the past two years, EURORDIS has led the Rare 2030 Foresight Study. With the participation of over 250 experts and almost 4000 patients and patient advocates, this study followed four steps:

  • Establishing knowledge base
  • Identifying trends and wild cards
  • Preparing potential scenarios
  • Developing policy recommendations

This participative and iterative multi-stakeholder study gave consensus for a future defined by equality: a future where the place of birth does not affect one’s access to the best diagnosis, care and treatment; a future where innovations across the pathway are needs-led; a future, where every person living with a rare disease is valued in society.

Read the recommendations and use as a basis for your advocacy.

But whether these recommendations are implemented is not just in the hands of policy makers. We each have a role to play in making sure the call for a new policy framework inspired by these recommendations is heard.

Contact:

For more information on how you can take part contact [email protected]