Table 2.

Requirements for changes in study design and review for progress-centered regulation of clinical research in lethal diseases

Current stateWhere we need to go
Extensive/costly preclinical toxicologyOnly LD10 in rodents
Extensive/costly preclinical pharmacologyOnly oral bioavailability and Cytochrome p450 interactions
Study review: obstructive, frustratingStudy review: fast, facilitating through redesign processes used by industry
Consent form: long, complex, multiple changes to wordingShort, simple, standardized, with patient access to additional online information
Study approval and activation: multiple individual institutions review/approval/activationSingle site approval
Multiple site “just-in-time activation” if eligible patients seen
IDE/CLIA: Technical validation of a predictive test, then assess itNo IDE/CLIA: Assess predictive test, then perform technical validation if it is useful