October 30th, 2009

Rare diseases at the forefront to address the healthcare challenges of the future

Personalized Medicine

The European Union and its Member States are faced with the challenge of an emerging field in medicine: personalised medicine. Therapeutic interventions, and in particular medicinal products, are increasingly intended for well targeted and smaller patient populations.

These interventions are usually intended for the prevention, diagnosis, treatment, cure or long term management of severe, debilitating, life-threatening diseases. They are using high value innovations, made possible thanks to the progress of medical research such as: pharmacology, gene therapy, cell therapy, tissue engineering, gene testing and other sophisticated diagnostic tools, including medical imaging and high-tech medical devices. Bringing these innovations to patients, as well as guaranteeing the best possible care and medical practices, while optimising the use of healthcare resources, requires both a higher concentration of expertise and an increased collaboration at the EU level.

This challenge is one more reason for policy makers at Member States level as well as at European level to make rare diseases an always higher priority in the EU 8th Research Framework Programme and the 3rd EU Health Policy Programme, both covering the period 2014-2020. Rare diseases are the laboratory to prepare the future of healthcare. Rare diseases are already at the forefront to help shape the future of healthcare.

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About the author

CEO, Rare Disease Europe (EURORDIS)

Yann Le Cam is a patients’ association advocate who has dedicated 20 years of professional and personal commitment to health and medical research nongovernmental organisations in France, Europe and the United States in the fields of cancer, HIV/AIDS and rare diseases. He is one of three patient representatives appointed to the Committee for Orphan Medicinal Products (COMP) at the European Drug Agency (EMEA) and served as its Vice Chairman from 2000 to 2006. He is a member of the DG Sanco Task Force on Rare Diseases and other European Commission working groups. He has three daughters, the eldest of whom has cystic fibrosis.

2 comments for “Rare diseases at the forefront to address the healthcare challenges of the future”

  1. Great idea and great job! I’m looking forward to reading more and participating to the discussion!

  2. caroline carr-locke

    write your comment here… Can anyone tell me if idiopathoic axonal peripheral neuropathy falls into the category for rare disease None in Scotland/UK can find the cause of my illness which is muscle degeneration
    below the knees and after a year i can hardly walk I am desperate as none can help me and my neurolgist said it will be decades before anyone can help me how can this be so in 2010 Can stem cells help ?

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