Evolution of Clinical Trial Design

Challenge: The average cost of researching, developing, and introducing a new drug in 2000 dollars was $802 million, 3.5 times more than just 15 years ago. These increased costs may have resulted from changes in clinical trial design over the past decade that, among other factors, reflect:

• higher safety standards and special population impacts
• satisfaction of multiple regulatory authorities with a single multi-national trial
• multiple indications due to increased understanding of mechanisms of action
• payer demands for economic and population health impact

Some argue that these changes may hinder therapeutic innovation due to the increased costs and time delays entailed in conducting the resultant clinical trials. Others argue that these additional clinical trial complexities are necessary to assure the safety of approved drugs. Evidence regarding how clinical trial designs have changed over time, and how they differ across therapeutic areas is not available.

Approach:

• Quantify how the complexity of trials supporting approved FDA NDA/BLA submissions has changed over time, and by therapeutic area.
• Examine the practices, trends, benefits and costs of clinical trial designs with their concomitant impacts on the number and timing of new therapeutics

 

Principal Investigator and Co-Director: Ernst Berndt, PhD, Louis E. Seley Prof. of Applied Economics, MIT
Co-Director: Fabio Thiers, MD, PhD, Visiting Scientist, MIT

Collaborators: Food and Drug Administration

 

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