IntroductionThis is the first in a series of blog posts VOI Consulting will publish based on high-level examination of the company’s new insiteinvestigator database which contains information on design, endpoints, populations, size, placement, timelines, efficacy outcomes, and safety results for every pivotal trial supporting a cancer drug approved by the U.S. FDA from 2005 through 2011. Each article will examine a specific topic to include: cancer drug approval trends, approval requirements, pivotal trial design, size, location, and key timing elements such recruitment periods and patient accrual per site within specific cancer types.
Cancer Drug Approval Trends
From the start of 2005 through the end of 2011, 48 different drugs were approved by the FDA for 83 different indications, of which 38 were new and 45 were supplemental. One hundred pivotal trials supported these approvals, an average of 1.2 trials per indication. (Note that there is some latitude in what constitutes a pivotal trial. For our purposes, we have defined a pivotal trial as a study that was extensively relied on by the agency for approval purposes. This means extensive discussion in regulatory materials surrounding the approval and, generally, inclusion on the approved prescribing label.) As shown in Figure 1, 60% percent of approved indications were for solid tumors while the remaining 40% were for hematologic malignancies. Of 22 different conditions with approvals, leukemia, breast cancer, and Non-Hodgkin lymphoma saw the highest level of activity; together, these three cancers represented over one-third of all drugs approved during the period. Forty-one percent of all approvals had previously received orphan drug designation. Although more approvals were for solid tumors, the majority of orphan drug indications were for hematologic malignancies.
Figure 1- Cancer Drug Approvals by Cancer Type 2005-2011
The rapid advance in cancer research is clearly evident in Figure 2. As shown, traditional anti-cancer agents such as chemotherapeutics and hormonal drugs accounted for two-thirds of approvals in 2005. By 2011 this had shrunk to less than 15% as new generation treatments such as biologics, targeted oral drugs, and vaccines have come to dominate the field. Although not shown on the chart, the trend towards new generation therapies has been driven mostly by the kinase inhibitor class. The first of these therapies, imatinib, was approved just a little over ten years ago but the category, which also includes erlotinib, nilotinib, sutininib, and several others, accounted for one-third of all approved indications over our total time period and reached 50% of all approvals in 2011.
Figure 2 - Approvals by Drug Type 2005-2011