Considerations for Use of Complex Innovative Designs in Rare Disease Drug Development
How complex innovative designs, meta-analytic predictive models and new statistical approaches can help overcome the unique challenges of rare disease clinical trials.
Rare disease drug development faces unique challenges, including small patient populations and heterogeneous treatment effects. Utilizing advanced statistical methods like Bayesian hierarchical models and meta-analytic predictive models promise to enhance trial efficiency, reduce costs and ultimately improve patient access to new therapies. By leveraging these new models in addition to external control arms and master protocols, there is the potential to transform rare disease clinical trials.
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