Closely Follows Draft Document
The Final Guidance closely adheres to the overall organization and content of the Draft Guidance. A line by line comparison between the two documents suggests that the changes made by the Agency in preparing the later version are more in the nature of clarifications than any outright reworking of the scope or structure of the earlier version.
Thus, for example, the Final Guidance reiterates that pharmacogenomic data must accompany investigational and marketing submissions when the data are used for decision making in the context of specific preclinical or clinical trials or to support scientific arguments to the Agency relating to issues of safety or effectiveness.
Unless the test results constitute a known, valid biomarker, in which case a summary will suffice, the sponsor shall submit a full report describing the test and the data obtained.
In the absence of any specific and comprehensive discussion of the public comments submitted regarding the Draft Guidance, the FDAs acknowledgement of and response to those comments might be gleaned from those clarifications.
Our earlier article regarding the Draft Guidance (see GEN, February 15, 2005, p. 18) summarized points raised in sometimes extensive evaluations of that document submitted to the public docket by several pharmaceutical and biotechnology companies. Key issues identified in those comments included:
the meaning and significance of a sponsors decision making or the FDAs regulatory decision making for purposes of identifying required submissions of pharmacogenomic data (e.g., several comments noted that pharmacogenomic data may be used through the discovery process to prioritize drug candidates without subsequently serving to address questions of safety or efficacy during the development testing of specific candidates);
the process by which a pharmacogenomic test would become recognized and accepted for regulatory decision making as a known valid biomarker;
confidentiality and control of intellectual property in the voluntary submission of pharmacogenomic data; and
whether submission of pharmacogenomic data could be in conflict with state and federal medical and genetic privacy legislation.
The first and second of these key issues are addressed in the Final Guidance, the first explicitly and the second implicitly. There are no observable changes in the Final Guidance that appear to respond to the third and fourth issues, although the new IPRG internal procedures do address the third issue.