Other Lecture or Seminar

From Product Decision to Regulatory Submission (and beyond!)

Dr. Bill Huster
Eli Lilly & Company

Start Date and Time: Mon, 12 Nov 2001, 4:30 PM

End Date and Time: Mon, 12 Nov 2001, 6:00 PM

Venue: LAEB B222

Abstract:

The product decision to regulatory submission interval of the drug development timeline is usually called Phase 3 - the "confirming" part of the "learning-confirming" drug development model. Phase 3 clinical trials are designed to confirm hypotheses about the efficacy of a new compound suggested by results from the earlier phases of drug development. As competition within the pharmaceutical industry has increased, there has been consequent pressure to shorten all phases of drug development - including Phase 3. An opposing force is the need to meet stringent regulatory requirements to register the compound.

Issues which must be considered in the design of Phase 3 clinical trials and the role of the clinical statistician in addressing these issues will be described. Clinical statisticians are instrumental in developing innovative and efficient clinical trial designs that meet regulatory requirements. One relatively recent innovation has been the use of interim analysis. Interim analysis has been used to shorten drug development; however, there are statistical and operational biases associated with an interim analysis. Statistical methods for "spending" type I error and the statistician's role in Data Monitoring Boards will be discussed.

The regulatory submission requires an intense, inter-disciplinary effort to pull together all known information about the new compound in a concise and understandable fashion. Although there have been efforts to harmonize the contents of regulatory submissions around the globe, variations still exist. The statistician plays a key role in determining how the efficacy and safety information from the clinical trials will be integrated and summarized for submission to the various regulatory agencies. Statistical methods and principles for the integration of such data will be discussed.

Regulatory review occurs after submission. The reviewing regulatory agency may consult with academic experts and scientific thought leaders to solicit their opinion concerning the adequacy of the trials and the results. The statistician's role in anticipating and addressing both regulatory and academic review of the submission will be discussed. Commercialization efforts of the sponsor are gearing up at the same time as regulatory review. As a result, the statistician needs to be able to balance addressing regulatory issues with designing Phase 4 (commercialization) studies.