Enrich. Comment. Repeat.

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  • 01/17/2013

From the pages of Drug Industry Daily:

New FDA Drug Approval Pathway Aims to Promote, Ease Enrichment Trials

The FDA is considering a new drug approval pathway intended to boost development of treatments targeting small subgroups of patients with serious or life-threatening conditions.

The pathway would establish a new, speedier process for studying a new drug or biological product in a smaller subpopulation of patients with more serious manifestations of a disease.

Labels for drugs approved under the new paradigm would make clear the drug is narrowly indicated for use in limited, well-defined subpopulations in which the drug's benefits have been shown to outweigh the risks, the agency said in a Federal Register notice.

The FDA plans to hold a public hearing on the potential new approval pathway on Feb. 4 and 5.

The agency is allowed to "exercise its scientific judgment" under existing regulations to determine the level of data a sponsor must provide in support of a particular candidate. Such flexibility has spurred the advance of a wide range of orphan drugs for rare diseases.

But traditional drug development programs typically require very large study populations, which put subpopulations of patients with more severe conditions at a disadvantage in the clinical setting - a situation that often results in less timely availability of new drugs for those patients.

The new approval pathway "could involve smaller and more rapid clinical trials than would occur if the drug were studied in a broader group of patients with a wide range of clinical manifestations," the FDA said.

The pathway proposal comes on the heels of new draft agency guidance on strategies for developing enrichment trials, which prospectively apply a patient characteristic to select a subpopulation more likely to benefit from a drug.

"The two announcements coming together signal to the industry that the blockbuster model is slowly being replaced by the personalized medicine model," Peter Pitts, president of the Center for Medicine in the Public Interest, said.

The message to the investment community is that drug failures are often due to trial design difficulties and not just heavy-handed regulation, he added. The proposed approval pathway is not likely to allow for less-robust evidence, just a different kind of evidence, Pitts cautioned.

Public Hearing Questions:

The meeting at the FDA's White Oak Campus will address such issues as:

The pathway's necessity considering the existence of other expedited approval pathways;

The conditions and patient subpopulations most likely to benefit from the pathway;

The degree to which regulators should monitor drugs approved under the pathway; and

Whether the pathway should be used to help reduce antibiotic-resistant bacteria by limiting the drugs' use to those patients in whom use is "appropriate and necessary."

Comments are due to docket no. FDA-2012-N-1248 by March 1. View the Federal Register notice at

www.fdanews.com/ext/files/01-14-13 DrugApprovalPathwayMeetingNotice.pdf.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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