The IRDiRC Companies Constituent Committee (CCC) brings together representatives from the for-profit pharmaceutical and biotech members of IRDiRC.
The CCC identifies common – often recurring and resource-consuming – roadblocks to efficient execution of rare disease research in the company space and promotes collaborations on “zero” or pre-competitive aspects of research as well as development.
Within these broad areas, a number of potential activities were identified by the CCC, including the need for a natural history and registry platform for the collection of real-world evidence data to support more efficient therapeutics development.
In order to work towards the IRDiRC therapies and access goals of 2027, the CCC will also investigate how to transform science to accelerate clinical development and translate innovation into sustainable access to medicines.
| Country | Organization | Representative | Title | |
|---|---|---|---|---|
| Belgium | Sanofi |  | Vinciane Pirard (Chair) | Lead Scientific Advocacy and Insights - Global Medical Affairs Rare Diseases |
| USA | Illumina, Inc. |  | Adriana Huertas-Vazquez (Vice Chair) | Senior Director, Global Medical Affairs |
| USA | Pfizer | Denis Keohane | VP Global Global Medical Affairs in Rare Disease | |
| Italy | Chiesi Farmaceutici S.p.A. |  | Diego Ardigò | Head of R&D, Global Rare Diseases |
| Denmark | UCB |  | Gabriella Almberg | Global Head Policy & Public Affairs, Rare Disease Organisation |
| USA | Recursion Pharmaceuticals, Inc | Jeanene Fogli | Head of Patient Advocacy and External Collaboration | |
| USA | Biogen |  | Lewis Raynor | Director Epidemiology |
| Belgium | European Federation of Pharmaceutical Industries and Associations (EFPIA) |  | Magda Chlebus | Executive Director Science Policy and Regulatory Affairs |
| Switzerland | Ultragenyx Pharmaceutical Inc. | Marta Valente | Vice President Head of Medical Affairs Europe | |
| USA | Takeda Pharmaceuticals |  | Neta Zach | Head of Neuromuscular Disease Area Unit |
| China | Beijing Genomics Institute (BGI) |  | Ning Li | Director |
| France | ITALFARMACO |  | Samantha Parker | Patient Advocacy and Communication Lead Rare Disease Europe |
| Sweden | Alexion AstraZeneca Rare Disease |  | Wendy Erler | Vice President of Patient Experience, STAR and Advocacy |
| Switzerland | Roche | Waiting for the nomination of the new representative |