The planning and execution of rare disease clinical trials
involves unique considerations. Examples include the role of
patient advocacy groups, the value of outcomes registries
and complete natural histories, the challenge in recruiting
and retaining study participants, the special qualifications of
investigators and clinical sites, unsettled clinical endpoints,
and the variable availability of outcome assessment tools.
In this Watch page, Judith Ng-Cashin, MD, Chief Scientific
Officer, INC Research, underlines the importance of the
patient-centred approach in the conduct of clinical trials.