Advancing treatments for rare diseases together

Patient-centric approach

Patients are at the core of advancing our treatments for rare diseases

Individuals affected by rare diseases, their families, communities and advocacy organisations have increasingly been recognised as critical partners during drug development. Leveraging on the foundation's strategic alliance with a network of more than 1,200 disease advocacy organisations, EspeRare is uniquely positioned to work hand-in-hand with patient associations at each step of the development process. Every effort is made to drive the patients' interest first; insuring patients’ safety and maximising patients’ medical benefits, as well as ensuring patients' access to treatment and enhanced care.

Particularly in rare diseases, patient organisations are the ones creating the awareness needed to understand their diseases. Their critical understanding enables us to make the most therapeutic impact with our drug development efforts. They are key partners at each stage of the process.

Using an established network of patient organisations, experts and biomedical centres of excellence, the foundation supports all the development phases necessary to bring the therapy in an accessible way to the market.

EspeRare model

 

Patient organisations are key partners

  • > Are a link to biomedical expert networks
  • > Provide access to patient samples
  • > Support clinical trial recruitment and design
  • > Fundraise and participate in research and development
  • > Influence regulatory and reimbursement policies