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Orphan Drugs: Interview With INSERM’s Ségolène Aymé

August 4, 2014

The 5th World Orphan Drug Congress Europe 2014 (November 12-14 in Brussels) is only months away and Total Orphan Drugs sat down with one of our key speakers, Ségolène Aymé (the Emeritus Director of Research at the French Institute of Health and Medical Research – INSERM) for a short interview.

Ségolène who won our European Leadership award at last year’s congress, will be addressing the challenges of orphan drug R&D from an academic perspective.  As one of the chairs to the WHO Topic Advisory Group for Rare Diseases, an Editor-in-Chief of the Orphanet Journal of Rare Diseases, and the project leader of “Support IRDiRC”, she has extensive expertise in the current orphan R&D challenges.

What are the biggest challenges for orphan drug R&D?

There are many challenges. One of them is the difficulty to access real data on patients due to the absence of specific codes in health information systems to code most rare diseases which is at the origin of a very limited knowledge on each disease: this has a huge impact at every step of the R&D process. Access to data is also limited by the absence of standards in the way signs and symptoms are coded, making multiple sources of information difficult to use, even if each of them is of good quality. An effort to agree on International standards is on the way and should be beneficial quickly.

What are some of the most pressing issues you are facing in doing this and how could this change in the future?

Many data are now produced through the sequencing approach but much of this will not be exploited, and will not be exploitable, if the genomics data is not linked to proper clinical data in a standardized way.  There are a few initiatives which could federate the research community at world level. I hope that the researchers will be wise enough as to join efforts.

Agreeing on International standards, deciding on shared standard infrastructures, requires a lot of effort, and it is not easy to obtain support for this as such initiatives are not seen as a real “research projects”. The International Rare Diseases Research Consortium initiatives are moving in this direction which is very fortunate.

What do you think will be the single biggest trend or change to the orphan and rare disease sector between now and 2025?

I believe that a lot of the research will be done in silico. Making the most of existing databases is the key factor of success for the future. Interfacing databases is feasible and all the infrastructures of this type should be considered as pre-competitive ones. It is why Industry should become involved in all the discussions concerning these infrastructures.

What would you like to gain from our congress?

I wish to meet the right people for a discussion about the precompetitive initiatives that can be developed jointly between Industry and Academia.

To hear more please visit our website to book your place so you can meet key speakers like Ségolène Aymé as they share solutions for orphan drug R&D.

Photo courtesy of Terrapinn.The Orphan Druganaut Blog is a Media Partner.

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