The development and adoption of patient-relevant and -reported outcome measures are instrumental in accelerating R&D
Collaborative effort on adaptive design, statistical methods and acceptability of new methods in small population clinical trials
Exploiting existing data and knowledge is essential to identify new therapeutic targets and to repurpose drugs.
ICHPT: Core set of terms to be incorporated in any Human Phenotype Terminology intended to describe rare diseases.
PPRL aims to develop a policy for the generation of participant-specific identifiers across projects without revealing the participant’s identity.