Patient advocacy groups (PAGs) play a vital role in supporting clinical research for rare diseases. By collaborating with researchers at institutions, PAGs can increase the thoroughness and efficiency of clinical trial readiness and natural history studies. A case study of Cure VCP Disease and Nationwide Children’s Hospital (NCH) demonstrates the value of this partnership.
Cure VCP Disease commissioned a functional measures study with NCH researchers to assess clinical trial readiness for Valosin-containing protein-associated multisystem proteinopathy (MSP1), previously known as inclusion body myopathy with Paget’s disease of the bone and frontotemporal dementia (IBMPFD), a rare, dominantly inherited disorder with multisystemic involvement. Cure VCP Disease collaborated with researchers by providing patient insight and perspective, organizing a proof-of-concept pilot project, and providing funding to support key research initiatives.
Cure VCP Disease also drove recruitment efforts via their network and registry; provided travel stipends to patients; organized and shipped standardized equipment kits for remote assessments to participants; conducted technology and equipment training with participants; and provided feedback on study logistics and execution.
As a result of this partnership, the study was able to start in less than three months with both in-clinic and remote measures. Over forty patients (including three international) were enrolled to participate in the study. Researchers were able to detect meaningful changes in the participants. The study also yielded meaningful data for conducting assessments remotely.
This case study could be a model of how authentic partnerships between institutions and PAGs can accelerate clinical trial readiness in rare diseases.