FDA: More “Me-Betters” and Focused Breakthroughs

FDA once again approved more innovative new drugs this past year, beating a near-record set in 2014. Yet, John Jenkins, director of the Office of New Drugs (OND) in the Center for Drug Evaluation and Research (CDER), is concerned if this pace of development can continue.

CDER approved 45 new molecular entities (NMEs) and biotech therapies, including more than 20 orphan drugs. In the process, the agency met all review timeframes and goals set by the Prescription Drug User Fee Act (PDUFA V). Almost all NMEs were assessed in the first review cycle, Jenkins reported at the FDA/CMS Summit in December 2015, and more new drugs reached the market first in the United States. Jenkins noted that expectations are high for continued gains, as reflected by increasing requests from sponsors for breakthrough drug designations, a program that has led to the approval of some 30 innovative products over the past four years.

The Center for Biologics Evaluation and Research (CBER) also approved more than a dozen new biologics, vaccines, blood products and diagnostics. This last item supports the drive for personalized medicine, which relies on the development of innovative in vitro diagnostics and combination products. FDA is pursuing initiatives to better coordinate oversight of drugs, biologics and medical devices, with particular attention to reducing redundancies in manufacturing inspections and data requirements.

Seeking “Me-Betters”

One concern for Jenkins is that sponsors tend to shy away from developing the third or fourth drug in a class, which can limit patient choice in important, widely-used drug classes such as beta blockers, anti-inflammatories and statins. He would like to see investment in more “me-better” drugs-as opposed to “me-too” medicines-to expand treatment options for patients and to boost competition among manufacturers.

“Choice is a good thing,” Jenkins commented, as later drugs in a class often enhance safety or efficacy for certain individuals. The move away from “me-too” drugs, he said, is “not necessarily good for public health.” He acknowledged, though, that the development of “me-better” therapies may be challenging and costly due to requests for extensive safety data. Further guidance from FDA, he said, should help sponsors by making clear where to set the bar for information and by not asking for more studies than are really needed.

A continued rise in new drug approvals, moreover, may fall short, said Jenkins, due to a fairly flat rate in submissions to the agency. Even though FDA oversees some 7,000 commercial INDs for drugs and biologics, Jenkins pointed out that actual submissions generally total only about 35 to 40 applications a year. CDER has about 30 NMEs under review at any given time, he said, but it “can’t approve more NMEs every year if it doesn’t get more applications.”