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505(b)(2) Approval Standards – Referenced Studies

Awareness of current FDA standards and those of its various divisions is important when preparing a new submission. A basic premise for a 505(b)(2) submission to the Agency is that the application contains full reports of investigations of safety and effectiveness. However, for 505(b)(2) applications, at least some of the information required for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained the right of reference. Often this referenced information is described in the label for a drug product that is a comparator for the subject of the 505(b)(2) submission; therefore, the quality of the study and the data have been scrutinized by the appropriate authorities previously. This type of information often forms the basis for satisfying some of the safety and efficacy requirements for the development program for the new version of a previously approved drug per 21 CFR 314.54.

It is also possible that the information in the public domain that is being referenced for approval of a 505(b)(2) program has not been included in a previous submission to the agency. In this case, depending on the quality of the data presented and the division performing the review, the agency has been known to make the following paraphrased statement:

…[W]hile it is possible to rely upon safety and efficacy data from the literature available in the public domain, the quality of that data would need to meet the current Agency standards. Thus, published studies for which original protocols and raw data are available for the Agency review would provide the most reliable information for assessing safety and efficacy, short of the sponsor conducting adequate and well-controlled trials on their own. It is also noted that it is unusual that data from the literature can meet this level of scrutiny; however, the information that is available might mitigate some of the burden of demonstrating safety and efficacy.

Keep in mind, when putting together information to support a 505(b)(2) submission, that the quality of the data will be scrutinized against current quality standards for safety and efficacy at the agency.