Figure 1

Three model clinical development programs used to estimate costs. The top program shows a typical abbreviated two study clinical program as a starting place for analysis. The two study surrogate endpoint study uses the same pre-approval studies but adds a post-approval Phase 4 study. The third program assumes that a single clinical study would be conducted preapproval and that a Phase 4 confirmatory study would be conducted. Timelines begin once a drug has been developed and shown to be effective in an animal model; a 2-year preclinical time period follows, during which pharmacology-toxicology studies and clinical drug production are conducted, at a cost of $5 M/year. Clinical trials then occur with time allotted for study startup, enrollment, and discussion with regulatory authorities, followed by a six month priority review approval process. IND: Investigational New Drug, FPI: First Patient In, LPI: Last Patient In, LPO: Last Patient Out, NDA: New Drug Application.