Advancing treatments for rare diseases Together

Develop the Undeveloped

Once repositioning opportunities are identified, the foundation applies its pharma know-how to conduct preclinical and early clinical assessments of these therapeutic opportunities.

Using an established network of patient groups, experts and biomedical centres of excellence, the foundation progresses these rare disease programmes from preclinical validation through to clinical proof of concept.

The development operating model:

The Foundation

  • Engages patient groups at each step of the process
  • Leads research and development operations
  • Coordinates drug manufacturing activities
  • Manages alliance and stakeholder engagement
  • Formulates and applies the funding strategy
  • Scouts for late development and commercial partners

Access through patient groups

  • Are a link to biomedical expert networks
  • Provide access to patient samples
  • Support clinical trial recruitment and design
  • Favour fundraising for research and development Influence regulatory and reimbursement policies

Strategy through our Clinical & Regulatory advisory boards

A multi-disciplinary group of rare disease experts defines the programme development strategy:

  • Specific rare disease clinical experts and regulatory advisors
  • Statisticians with expertise in small cohorts
  • Disease and PKPD modelling and simulation scientists
  • Quality assurance resources

Execution through Preclinical & Clinical partners

Preclinical and clinical assessments of our programmes are conducted together with rare disease expert centres and organisations such as:

  • Research centres specialised in specific disease biology
  • Clinical reference centres
  • Specialised contract research organisations

What happens after translational validation?

After proof of concept in humans is validated and a conclusive data package is generated, compounds can go back to the originator of the drug or be developed by other biomedical partners who will carry out the later stage clinical trials, registration and commercialisation activities.