By Sarah Glass, PhD, Senior Director, Translational Medicine, Global Head of Rare Diseases; Shipra Patel, MD, Senior Medical Director, Medical Sciences, Global Head of Pediatrics; Altair Silva, Director, Integrated Solutions, Patient Recruitment Strategy Group; Parexel®
A clinical trial offers hope for individuals and families impacted by rare diseases, often in situations where there are no existing treatments. Early diagnosis and access to treatment provides the best opportunity for these individuals to experience the benefits, such as slowing disease progression and improving their quality of life. As an industry dedicated to accelerating the development of new treatments to address unmet medical needs, it is essential for every individual affected by a rare disease to be given the opportunity to participate in clinical trials that could offer promise for them and their families.
The biopharmaceutical industry has been rising to the challenge of developing treatments for rare diseases since the passage of the Orphan Drug Act in 1983 and now one-third of all drugs in active research and development worldwide target rare diseases. However, clinical researchers face unprecedented challenges with patient identification and recruitment for these clinical trials across all phases of clinical development.
Read how leveraging precision medicine, data-driven solutions and collaboration across the rare disease community are instrumental when developing a customized strategy for patient identification and recruitment.