Agency will issue guidance for speeding up approval of competitive products.
The drive toward evidence-based medicine may be having an unexpected and undesired impact. While most people would agree that new drugs should provide treatments that are not already available, there is a benefit to having a choice of medications for an indication that work by a similar mechanism of action – competition.
In a recent report to a congressional committee, US Food and Drug Administration (FDA) Commissioner Scott Gottlieb noted that branded drugs that operate by a new mechanism of action, known as first-in-class drugs, face less competition today than they have in the past.
Between 1991 and 2000, the percentage of first-in-class drugs to treat non-Orphan conditions to face competition within five years was 41%, and from 2001 to 2010 just 18%. Over the same time periods, the rate of -in-class therapies facing competition within five years for non-cancer Orphan drugs fell from 26% to 13%.
“When a novel sole source drug wins approval it faces no competition from other drugs in the same class. Follow-on drugs and biologics to compete with the first in class have been arriving more slowly,” Gottlieb said.
To address the problem, the agency is promoting the use of master clinical trial protocols and “seamless trials.” FDA will issue guidance on the “design and conduct of first-in-human clinical trials with multiple expansion cohorts intended to efficiently expedite the clinical development of cancer drugs,” according to Gottlieb. FDA has also published a list of surrogate endpoints to support accelerated and traditional approvals, including those that sponsors have used as primary efficacy clinical trial endpoints for approvals and those that FDA anticipates could be appropriate for use as a primary efficacy clinical trial endpoint for approval of a drug or biologic therapy.