[LIVE FROM WODC USA] Medicines Adaptive Pathways to Patients (MAPPs)

In Orphan Drug Congress, Orphan Drugs Live by Freya Smale

Yann Le Cam, CEO, EURODIS presented the afternoon keynote on “Medicines Adaptive Pathways to Patients (MAPPs): Europe’s focus on fixing the process rather than new legislations to get more, better, faster, cheaper orphan drugs”.

Session highlights included:

  • How is ’significant benefit’ assessed at time of orphan drug designation and at the time of market authorization? Why is it so important in the European orphan regulation?
  • Importance of having proper clinical development to show a ‘significant benefit’ at the time of marketing authorization
  • What has Europe learned from international collaboration on orphan drugs and contributed to the international discussions?

Yann started off by speaking about the growing but stable European legislative framework that will be changing over the next few years. The current framework is being pushed. There has been an increase in designation assigned in Europe though still less than the number of designations in the U.S.

Yann stressed how there is a trend of seeing products for the same rare diseases, which in turn creates competition but that does not address the economic needs. Many are using the regulatory process and adapting it to fit into the pathway to get approval. There is also a push to strengthen support for medicines that target an unmet need.

The gap between innovation and access needs to be closed according to Yann. The way this can be done is with not only the safety of clinical trials, but also the effectiveness. There is also an end to the high price orphan drugs model in Europe.

Yann’s “new deal” model for Orphan Drugs includes:

  • New R&D model with more flexible clinical trials
  • New evaluation process with early dialogue
  • New healthcare delivery (blurring the lines between healthcare and research)
  • Flexible pricing and differential pricing across Europe

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.