title: Improving the operational efficiency of Phase 2 and 3 trials.
abstract: The period toward the end of patients' participation in late stage blinded clinical trials is highly resource intensive for the sponsor. Consider first a Phase 3 trial. If the trial is a success, the sponsor has to implement the next steps, which might be filing for approval of the drug with the US Food and Drug Administration (FDA). To shorten the time interval between trial completion and submission of the package to the FDA, sponsors front-load as much work as is possible at risk. The approach is efficient if the trial succeeds but is inefficient if it fails. For a failed trial, the sponsor is unlikely to proceed with the plan that assumed success. Phase 2 trials are also at risk of being inefficient. Many activities, such as planning for drug interaction studies, thorough QT studies, or site selection for Phase 3 trials, are set in motion prior to completion of the Phase 2 trial. The work going on in parallel is wasted if the trial fails. The proposal to improve the efficiency is to let an independent entity provide the sponsor critical information at an earlier time necessary to reevaluate activities ongoing in parallel and external to the trial.
authors.affiliation: Global Blood Therapeutics, South San Francisco, CA, 94080, USA. jganju@globalbloodtx.com.
authors.forename: Jitendra
authors.initials: J
authors.lastname: Ganju
mesh_terms: D017322:Clinical Trials, Phase II as Topic; D017326:Clinical Trials, Phase III as Topic; D006801:Humans; D014481:United States; D014486:United States Food and Drug Administration
keywords: Blinded clinical trials; Risk reduction; Unblinding