On June 13, 2016, The Food and Drug Administration (FDA), in co-sponsorship with the American Association for Cancer Research (AACR), is announcing a public workshop entitled “Oncology Dose Finding Workshop.”
Since the approval of imatinib in 2001, the FDA has approved 26 small molecule kinase inhibitors for the treatment of oncology indications. Given the recent history of approvals based on the results of early phase trials driven by extraordinary efficacy data, the incentive for conducting rigorous dose-finding trials may not be overtly apparent. However, the increasing need for the development of combination therapy due to resistance to monotherapy and poor tolerance of approved dosing regimens underscores the need for a more efficient process of dose selection in the early stages of study design. Furthermore, the unknown efficacy in light of frequent dose reductions in the post-market setting begs the question of whether efficacy reported in early phase trials is accurate when applied to a real world population.
Objectives of the Workshop:
- To identify key “best practices” in the nonclinical evaluation of a compound, including, but not limited to: selectivity, pharmacology, secondary pharmacology and toxicology.
- To identify disease- and mechanism-specific nonclinical models better able to predict efficacy.
- To assess whether nonclinical information can be incorporated into the statistical assumptions of an adaptive dose finding trial.
- To discuss the “best practices” of integrating human pharmacokinetic and pharmacometric data, , including exposure-response analyses, into dose-finding studies.
- To assess how drug exposure can be integrated into the statistical assumptions of an adaptive dose-finding trial and to assess whether evolving exposure data can be adapted into an ongoing trial.
- To shift from conducting a large, single-arm drug trial with the MTD based on a 28-day window to identifying tolerable, biologically effective doses for confirmatory trials through prudent search of doses based on safety, efficacy and patient tolerability.
- To discuss potential regulatory implications of dose-finding studies, including but not limited to: product labeling of dose ranges, dose titration and post-marketing studies.
June 13, 2016
8 am - 5 pm
Walter E. Washington Convention Center
801 Mt. Vernon Place, NW
Washington, DC 20001
To register for this meeting, visit:
Registration will close on June 9, 2016
Who Should Attend
This workshop will be open to the public. The primary audience will include pharmaceutical scientists working with small molecule development whether from academia, industry, or government regulatory agencies.
This workshop is a follow-up to the successful FDA-AACR public workshop: Dose-finding of Small Molecule Oncology Drugs, which was held May 18-19, 2015. Full transcripts and select presentations from that workshop are also available.