Shauna Aherne, president of Med Evoke, shares her insights on the challenges that remain in rare disease clinical development, and what changes need to incur in clinical trials to bridge the rare disease treatment gap.
Key insights:
- Three hindrances remain in rare disease clinical development: trial design and trial protocol inclusion/exclusion, trial awareness and recruitment; and delayed stakeholder engagement and education prior to a launch.
- Defined endpoints allow for better focus to gain patients that meet trial standards without muddying the data that can negatively impact the outcomes being researched.
- Medical affairs can improve trial participation by working with patient advocacy groups that already have access to rare disease patients.
Even with the heightened interest in rare disease treatments, companies and researchers still find themselves searching for enough patient data. Interconnectivity/social media and greater technology have not lessened the challenges with bringing a drug to reality in disease that afflicts less than 10,000 people in the European Union and 200,000 in the United States.