More New Drugs, Faster Approvals

FDA set several milestones in approving more than 40 important new drugs and biologics in 2014. FDA commissioner Margaret Hamburg reported in early December that approvals were up to 35 new molecular entities (NMEs), an increase from 27 in 2013; the total reached 41 new drugs by year-end. Hamburg’s tally included a record 15 orphan drugs and 15 first-in-class therapies, and she noted some progress in developing new antibacterial drugs: three new medicines to treat skin infections are on the 2014 approval list. Breakthrough drug designations went through through the roof, setting the stage for more more new therapies for cancer and critical conditions to reach patients in the coming year.

A related gain, according to John Jenkins, director of the Office of New Drugs (OND) in FDA’s Center for Drug Evaluation and Research (CDER), is that more submissions are moving through the review process in one review cycle, saving time and resources for sponsors and for all OND offices – not just cancer therapies.  And two-thirds of novel drugs approved last year were first approved in the U.S. All these accomplishments confirm the success of OND’s “program” for making the drug approval process more efficient and effective in meeting PDUFA user fee goals, Jenkins observed at the December FDA/CMS Summit in Washington,  D.C.. That process involves more FDA-sponsor meetings during development to discuss clinical trial approaches and ensure that applications are complete.

Sure, there’s room for improvement. Jenkins explained that FDA has been hit with a large number of breakthrough designation requests that fall far short of meeting basic standards, but which still have to be reviewed by OND staffers. FDA plans to issue further guidance to clarify the “bar” for breakthrough requests, an issue that will be discussed at a workshop with the Brookings Institution in April 2015. Jenkins also noted that manufacturing and scale-up difficulties more often are responsible for delays in bringing critical drugs to market – compared to a lack of clinical data.

CDER director Janet Woodcock would like to see more drugs studied in children, more anti-microbials, and additional efforts to streamline clinical trials, she said in presenting a long list of CDER goals for the coming months at the Summit conference. Although her top priorities center on new initiatives to ensure drug quality and to implement new drug compounding and supply chain security programs, Woodcock hopes to make progress on many fronts, including a re-evaluation of drug advertising regulation as it relates to first Amendment issues, managing the development of new Ebola therapies, encouraging new abuse-deterrent opioid formulations, and continuing to build FDA IT systems to manage all these activities.

Woodcock also cited numerous opportunities to improve drug development and clinical testing.  She hopes to issue policies that encourage broader use of Bayesian statistics and adaptive clinical trials designs; sponsor studies on additional indications for cancer therapies; a benefit-risk assessment template; more guidance on specific conditions such as Alzheimer’s disease; evaluation of the impact of FDA’s patient-focused drug development program; and advancing the work of the more than 20 consortia that involve CDER staffers.