Precision Medicines: Implications for Clinical Trial Design, Speed of Approval, Physician Understanding, and Patient Consent

Background: The genetic targeting of precision medicines offers the promise of larger effect sizes, quicker outcome measures, smaller clinical trials, and more rapid market entry as compared to traditional drugs. 

Objective: To determine the clinical trial characteristics of precision versus non-precision medicines. 

Method: We evaluated all new drugs approve by the US Food and Drug Administration from January 2013 through June 2017. 

Results: Precision medicines benefited more often from special expedited development and approval programs and were associated with faster approvals based on smaller and fewer clinical trials. The FDA exercised its discretion in approving precision medicines, granting approval on the basis of trials that were less likely to be randomized, blinded, or concurrently controlled. 

Conclusion: Although precision medicines offer commercial benefits, the greater regulatory flexibility applied to precision medicines also raises questions about evidence quality, long-term clinical value, and patient understanding of the benefits and risks that precision medicines can be expected to provide.