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 | |
|---|---|---|---|---|
| USA | Pfizer |  | Katherine Beaverson (Chair) | Executive Director, Head of Patient Advocacy and External Engagement, Rare Disease Research Unit |
| USA | Illumina, Inc. |  | Adriana Huertas-Vazquez | Senior Director, Global Medical Affairs |
| USA | Sanofi Genzyme |  | Alaa Hamed | Global Head of Medical Affairs Rare Diseases |
| USA | Recursion Pharmaceuticals, Inc |  | Chris Gibson | CEO & Co-Founder |
| Italy | Chiesi Farmaceutici S.p.A. |  | Diego Ardigò | Head of R&D, Global Rare Diseases |
| Switzerland | Ultragenyx Pharmaceutical Inc. |  | Grania Crowley | Head of EU Patient Advocacy and Public Affairs |
| Belgium | European Federation of Pharmaceutical Industries and Associations (EFPIA) |  | Magda Chlebus | Executive Director Science Policy and Regulatory Affairs |
| USA | Takeda Pharmaceuticals |  | Madhu Natarajan | Head, Rare Diseases DDU Research |
| France | Lysogene |  | Marie Trad | Chief Medical Officer |
| China | Beijing Genomics Institute (BGI) |  | Ning Li | Director |
| France | InnoSkel |  | Samantha Parker | Chief Patient Access Officer |
| Switzerland | Roche | Waiting for the nomination of the new representative |