Yann Le Cam | Chief Executive Officer

Yann Le Cam, Chief Executive Officer, EURORDIS

Yann Le Cam is a patient advocate who has dedicated 25 years of professional and personal commitment to health and medical research non-governmental organisations in France, Europe and the United States in the fields of cancer, HIV/AIDS and rare diseases.He has three daughters, the eldest of whom has cystic fibrosis. Yann is one of the founders of EURORDIS in 1996-1997 and its Chief Executive Officer since 2001. He has participated in the revision and adoption of European regulations having an impact on rare disease patients’ life, including the EU Regulation on Orphan Drugs, December 1999.He was one of the first patient representatives appointed to the Committee for Orphan Medicinal Products (COMP) at the European Drug Agency (EMA) where he served for 9 years and was its Vice Chairman for 6 years. He served on the Management Board and Executive Committee of the French HTA agency for 5 years, on the DIA Advisory Committee Europe for 3 years.He was the Vice Chairman of the EU Committee of Experts on Rare Diseases (EUCERD) from 2011 to July 2013, and he is nominated on the current Commission Experts Group on Rare Diseases  In November 2013, Yann Le Cam was elected Chair of the Therapies Scientific Committee of the IRDIRC – International Rare Diseases Research Consortium.


Day 2, Nov 8 @ 10:10

Recommendations on sustainable access to innovative therapies

  • Ensuring the sustainability of health spendingas well ascontinued innovation that meets patient needs
  • How does the current pricing system need to change to compliment this?
  • Are novel pricing & reimbursement mechanisms for gene therapy products necessary?
Followed by feedback from global stakeholders on value vs pricing and affordability
  • Are we paving the way to a fair, inclusive and on-going multi-stakeholder approach with the potential to generate sustainable, affordable and actionable improvements in patient access to rare disease therapies?
  • Balancing between recouping costs for R&D and incentives to support investment in future treatments that ultimately access to patients

Day 2, Nov 8 @ 16:00

IRDiRC: Enabling all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention. How do evolve to achieve target?

  • The IRDiRC goals, a community effort for a better future in rare disease research and clinical management and a call for international collaboration to an unprecedented scale
  • How are we going to achieve quicker diagnosis for 7000+ diseases and to solve unsolved diagnostic cases?
  • How are we going to deliver the best possible care and give access to all available therapies to rare disease patients?
  • What change in pace do we need to develop new treatments for thousands of rare diseases?
  • Summary of learnings from the congress and targets for next year
Followed by panel discussion on; how far we are from the IRDiRC vision and how much ambitious its goals are& what we need to change in the system to achieve these goals and vision
last published: 17/Oct/18 12:05 GMT

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