(139) This could be taken a step further to require an adaptive Phase II/III trial and then either merge the Phase I trial into the Phase II/III structure, creating one large trial with different "stages," or start over and recreate the clinical trial process.
(147) A blanket rule requiring regulations resembling Phase II trials indicates that if all Phase I trials are governed by efficacy-driven regulations, then every single trial is a new Phase I trial and does not only test for safety.
(148) See ClinicalTrials.gov, A Phase 1/2 Clinical Trial of an Alphavirus Replicon Vaccine for Influenza, http://clinicaltrials.gov/ct/show/ NCTO0440362:jsessionid=FD76BCC991FFDB566E7FBE84EEFF3E31?order=46 (utilizing healthy volunteers in a Phase I trial for a nonterminal illness).
Historically, Phase I trials solely tested toxicity and dose-responses ("Traditional Phase I Trials").
Section II of this Note discusses the shift in design and purpose of Phase I trials, as illustrated by oncology trials utilizing terminally-ill patients to test drug efficacy, and by the FDA's accelerated approval processes through which seriously and terminally-ill patients obtain access to investigational drugs prior to FDA approval.
In Phase I trials, clinicians introduce an experimental drug to humans for the first time.
Traditional Phase I Trials stand in stark contrast to the remaining phases of clinical trials.
The traditional concept of Phase I trials, however, is no longer accurate.
Phase I Trials Now Test Efficacy and Therapeutic Benefit Instead of Just Safety
Phase I oncology trials illustrate the shift from Traditional Phase I to New Phase I trials revealing that New Phase I trials have more in common with Phase II trials than points of differences.
In contrast to Traditional Phase I trials, which utilize healthy volunteers, researchers in modern oncology trials can and frequently do enroll patients that have cancer and have exhausted all standard treatment options.
To ensure that only INDs showing promise of providing a therapeutic benefit are used in Phase I trials, the FDA recently promulgated regulations that allow testing of INDs prior to, and sometimes in lieu of, Phase I trials.