FDAMA Section 113 requires registration of clinical trials conducted under an investigational new drug (IND) application for a drug to treat a serious or life-threatening disease or condition and also tests effectiveness. (Guidance for Industry Information Program on Clinical Trials for Serious or Life-Threatening Diseases and Conditions; Section III A.) Although not all trials carried out under 21 CFR part 312 test effectiveness, FDA considers all phase 2, phase 3, and phase 4 trials with efficacy endpoints as trials to test effectiveness. (Guidance for Industry Information Program on Clinical Trials for Serious or Life-Threatening Diseases and Conditions; Section III E.) Note that FDAMA does not specifically exclude Phase I studies. If a Phase I study has efficacy endpoints (eg, Phase I trial of a drug [under an IND] to test the effectiveness of Drug A on tumor progression in patients), it could qualify for registration.
