The decades old Orphan Drug Act helped spur a renaissance in rare disease research, incentivizing development therapeutics that may otherwise be deemed unprofitable. However, factors like small, spread-out patient populations and funding considerations make running clinical trials for rare diseases complex and expensive. In a new PM Live article, learn about the factors underlying successful rare disease trials and how they can be improved.
According to Cameron Tew and Shauna Aherne, “Traditional trial design can limit effectiveness in rare diseases, so non-traditional trial models such as umbrella and basket trials should be considered. These models expand target populations without limiting the disease subtype being studied. Unfortunately, few companies have taken this route. A December 2021 analysis from Clinical Trials Arena found seven umbrella trials and 42 basket trials were initiated that year compared to 5,000-plus rare disease trials.”
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(Source: PM Live, March 24th, 2023)
