Rare disease protocol design, vendor selection, patient recruitment and retention, and site and patient qualification can be easily undermined. Learn why the research community must use its influence to ensure trials are designed and executed in a way that considers each patient’s situation and overall well-being.
Biopharma organizations can inspire and motivate investigators and sites by showing them in both word and deed that their contributions are valued, resulting in a trickle-down effect: well-supported physicians and sites are better equipped to provide optimal patient care and to execute trials with greater efficiency, accuracy, and enthusiasm.
A traditional regulatory pathway requires being able to accurately describe the normal clinical course of the disease. This can be difficult with rare diseases because existing academic data is incomplete and/or poorly controlled. Moreover, data rarely exists that is relevant to specific, high-sensitivity endpoints needed for a gene therapy study.
From a scientific research perspective, finding a way to proliferate or silence (as needed) the protein of interest equals success. But once medical scientists become involved, the translational bridge must be constructed — arguably the hardest step in gene therapy development. How do we apply that science and make it meaningful?