| Abstract: | The history of drug/vaccine development has included major advances guided primarily by risk/benefit analyses concerning the
innovative agent, not by evidence-based clinical trials (Phase I–IV). Because the approval for new drugs is hindered under
the present process, the system requires restructuring.
The Phase I/II study period should be more flexible, using the “environment of knowledge” about the new agent, plus risk/benefit
assessments. Phase III, as presently constructed, does not add new adverse events data, it provides a narrower profile of
drug efficacy than properly done Phase II studies, and placebo-controlled trials continue to raise unresolved ethical and
social issues. Phase III studies should be abandoned for most drugs, and substituted with properly powered Phase II doseranging
studies plus careful post-marketing surveillance. Phase III should be a penalty for poor drug development, not a regulatory
requirement.
To accomplish efficient drug development, greater cooperation between pharmaceutical companies and governments in developing
clinical trials is needed rather than over-regulation. These changes will synchronize the drug development and regulatory
process with the current rapid drug discovery process, reduce drug development time and cost, and improve patient care.
The author is Adjunct Professor of Medicine, Weill Medical College of Cornell University, New York, New York, USA. |
