The Rare Disease Consortium

The Rare Disease Consortium (RDC) is a joint undertaking of Pfizer and six leading UK universities, known collectively as the Global Medical Excellence Cluster (GMEC). The RDC provides a framework for world-leading academic investigators from GMEC, who have unrivalled scientific insight into specific diseases, to collaborate with scientists at Pfizer, who are often also disease experts and who have extensive experience in developing medicines for patients with rare diseases. The resulting collaborations between GMEC and Pfizer have the potential to accelerate the development of new treatments for patients suffering with some of the most debilitating and often life-threatening illnesses.

Collectively, rare diseases affect more than 3.5 million people in the UK, yet less than 5% of these diseases have any approved treatments. This unmet medical need is in part due to the vast number of diseases and the diversity of underpinning biochemical pathways. The RDC aims to address this by facilitating collaborations in early stage discovery, focussing on rare hematologic, neuromuscular, and pulmonary diseases.

For GMEC academics, participating in the RDC presents the opportunity to potentially translate their hard work and biological expertise into a real benefit for patients. Approximately 70% of the estimated 7000 rare diseases have a monogenic origin, and the RDC is seeking to build on recent discoveries linking diseases with specific genetic defects to drive forward drug discovery.

GMEC comprises six leading UK research institutions (University of Oxford, University of Cambridge, UCL, King's College London, QMC, and Imperial College London). Academic researchers from these universities are invited to submit project proposals to the RDC, and successful proposals are fully funded by Pfizer, including staff and research costs. Pfizer also provides access to their molecules, tools, drug discovery platforms, and/or expertise in an effort to accelerate the translation from research to patient benefit.