The Rare Disease Puzzle: Bringing the Picture to Life

In the past two decades we have identified the pieces that address the needs of the rare disease community. The challenge remains to combine those pieces into a picture that drives action forward with clarity and energy into a coherent and sustainable strategic plan.

The Executive Summary for ECRD 2014 Berlin is now available and  can be downloaded here.

None of us are protected by statistics.

The fact is that while a disease might be labeled as “rare”, the number of persons in Europe suffering from a rare disease is estimated at over 30 million. Rare diseases do not only affect those diagnosed, but their families, friends, carers and society as a whole. While one rare disease may affect as few as 1 in 50,000 people, rare disease patients collectively comprise 6 to 8 % of the EU population. These statistics do not seem as comforting.

The European Conference on Rare Diseases and Orphan Products is rare. It is the one event where everyone from patients, to policy makers, healthcare professionals, industry, payers, regulators, researchers and academics are given the opportunity to meet, exchange information and ideas and join together in the fight against rare diseases. This event provides a unique platform comprising all rare diseases, across all European nations. With over 100 speakers and countless professionals in attendance, this biennial conference covers the latest research, developments in new treatments and information regarding innovations in health care, social care and support at both the European and national levels.

ECRD allows you to be on the front lines in the fight against rare diseases, whether you are a patient, family member, healthcare professional, policy maker, researcher, or simply an interested citizen. The diseases are rare, but the support doesn’t have to be. 

The Basics:

What is a Rare Disease?

A rare disease is classified in Europe as being a condition which affects less than 1 in 2,000 individuals. Because rare disease patients represent the minority, there is often a lack of public awareness. Common issues facing patients and families living with rare diseases include delayed diagnosis, access to care and medication and a feeling of isolation. This is true across different countries, cultures and all rare diseases.

What are Orphan Products?

Orphan products are medicinal products intended for the diagnosis, prevention, and treatment of rare diseases.

What is ECRD?

The European Conference on Rare Diseases & Orphan Products (ECRD) is the unique platform/forum across all rare diseases, across all European countries, bringing together all stakeholders: patients’ representatives, academics, health care professionals, industry, payers, regulators and policy makers.

ECRD covers research, development of new treatments, healthcare, social care, information, public health and support at European, national and regional levels.

ECRD provides the state of the art of the rare disease environment, monitoring and benchmarking initiatives.

Why attend ECRD:

  • Network with all stakeholders in the rare disease community
  • Learn about the current landscape of policy development on orphan products and rare disease therapies
  • Contribute to addressing the challenges of healthcare pathways
  • Understand the opportunities in rare disease collaborative clinical research
  • Hear about challenges and experiences in disease specific registries
  • Contribute to the discussion on how to improve gene testing, genetic counselling and newborn screening at the European level
  • Get insight into how to sustain attractiveness for companies to invest in rare diseases
  • Hear about the challenges of off-label use and access
  • Learn about the discovery process of a medicinal product from the point of view of industry and regulators