More New Drugs and Faster Approvals

Print 23 December 2014
Jill Wechsler, BioPharm International

FDA set several milestones in approving more new, important drugs and biologics in 2014. Breakthrough drug designations went through through the roof, speeding more new therapies for cancer and critical conditions to patients. FDA approved 35 novel new drugs, up from 27 in 2013, reported FDA commissioner Margaret Hamburg in December 2014. Hamburg noted that the tally includes a record 15 orphan drugs and 15 first-in-class therapies, and that there has been some progress in developing new antibacterial drugs: three new medicines to treat skin infections are on the 2014 approval list.

A related gain, commented 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. Two-thirds of novel drugs last year were first approved in the United States. All these accomplishments confirm the success of OND’s “program” for making the drug approval process more efficient and effective in meeting Prescription Drug User Fee Act (PDUFA) user fee goals, Jenkins observed at the December 2014 FDA/CMS Summit in Washington, D.C. That program involves more FDA-sponsor meetings during development to discuss clinical trial approaches and ensure that applications are complete.

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 develop policies that encourage broader use of Bayesian statistics and adaptive clinical trials designs; more efforts by sponsors to study 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.

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