The Aplastic Anemia and MDS International Foundation (AA&MDSIF) has initiated a Patient Advisory Committee for Clinical Trials (PACCT+) to bring the patient voice to industry and academic researchers developing clinical and health services research for rare bone marrow disorders. Patients with these diseases are eager to propel research, but their input in design of rare disease research is minimal. AA&MDSIF will identify, select, and provide basic research training to a representative cross-section of patients with bone marrow failure disease, along with their parents and caregivers. PACCT+ will be available for researchers to solicit non-binding recommendations, demonstrating inclusion of meaningful patient input in design and implementation of research. PACCT+ will address health based studies within bone marrow failure disease that would benefit from front-end patient input, clinical trials, health services research and quality of life studies.
The projected outputs from this project are the creation of a review process for a patient committee to provide meaningful and practical information and insight to clinical and health services researchers as they design trials; development of an educational and training process for committee participants with the assistance of rare disease clinical researchers and educators; and creation of a patient advisory committee to provide input to bone marrow failure disease studies conducted within academia and industry.
Project collaborators include patients and caregivers from our disease areas (aplastic anemia, myelodysplastic syndromes [MDS] and paroxysmal nocturnal hemoglobinuria [PNH]); academic clinical and health services researchers; industry representatives; physicians; and pharmaceutical industry representatives.
Project Resource: Review Committee Recruitment Progress Report