Bringing any treatment from concept to clinic comes with a unique set of challenges, but rare indications amplify the complexity.
Small patient populations make trial recruitment arduous
Limited natural history data complicates endpoint selection and trial design
Manufacturing specialized therapies on a small scale adds cost and strains logistics
To address these challenges and more, leaders in clinical development, translational science, and advanced therapy manufacturing collected their experiences into a free ungated eBook.
Expert insights from 200 rare disease development programs
Set your study up for success with practical (and actionable) advice:
Rob Maiale is a leading content strategist and creator with more than 15 years of experience in healthcare marketing and brand development. His specialties include pharma, diagnostics, med-tech, patient education, translational research and clinical development.