FDA Sets Goals for Improvement in Drug Development and Approval

Jill Wechsler

A top priority for FDA leaders in the coming months is to maintain, and continue to improve, the efficient and effective drug development and review system the agency has established in recent years. It will be a challenge for FDA to match or exceed its success in 2018 in approving record numbers of innovative new medicines, but agency leaders aim to try. In January, FDA made it official: The Center for Drug Evaluation and Research (CDER) approved 59 novel drugs and biologics in 2018, surpassing previous high marks and improving on recent gains (see here,  for complete list of new drugs). Additional new products approved by the Center for Biologics Evaluation and Research (CBER) further boost the total.

While analysts may define innovative new products differently, the ability of biopharma companies to capitalize on important advances in science, plus strong regulatory support, have combined to move many important new medicines to market efficiently to provide more treatments for a range of critical conditions. The “class of 2018” includes 16 new cancer drugs, with several indicated for genetically defined conditions. There also was a number of new treatments for rare diseases, along with the first cannabidiol-based medicine and the first drug to treat smallpox. Nearly all the new drugs were approved in one review cycle, and three-fourths were approved first in the U.S.

Many of the new approvals benefited from one or more expedited development or review pathway, a trend that has prompted some observers to question whether FDA may be moving some experimental products too quickly to market. However, only 24% of the new drugs were designated as breakthrough therapies, and fast-track review applied to less than half.

Restructuring OND

An important goal for CDER director Janet Woodcock is to complete the overhaul of the new drug review process to better manage a growing volume of applications. She announced at the FDA/CMS Summit in December that the Office of New Drugs (OND) in CDER would be led by OND deputy director Peter Stein, and that long-time CDER guru Bob Temple will become OND senior advisor, positioned to address more controversial and difficult drug development and review issues. Woodcock hopes to finalize the OND reorganization by next summer, but it has been delayed by difficulties in gaining Congressional approval of a new user fee program for improving the regulation of over-the-counter drugs. And the recent government shutdown only delayed legislative action further.

Modernizing the review process will involve implementing new automation tools for managing drug applications, study data, and review documents under a “multi-disciplinary, issue-based review document” system. CDER also will continue to carry out provisions of the 21st Century Cures Act and reauthorized user fee programs to further advance patient-focused drug development, expanded use of real-world evidence, novel clinical trial design, and added authorities to hire more experts needed to carry out these multiple drug regulatory programs.

To continue advancing innovation in drug development, FDA Commissioner Scott Gottlieb recently announced plans to establish a new office of drug development science at the agency. The aim is to develop and authorize new tools and methods able to reduce the cost and uncertainty in testing and marketing new medicines. This Office of Drug Evaluation Science will be part of OND and will work to implement new methods for clinical outcome assessments, biomedical informatics, and biomarker development. Additional efforts will aim to devise more standardized tools for capturing and formatting safety data to achieve more consistent review of this information.

Limiting opioids, promoting quality

Amid advances in new drug development and review, Woodcock also recognizes the importance of addressing the nation’s deadly opioid epidemic as a top priority for the FDA and pharma companies. The immediate need is to reduce the over 200 million outpatient prescriptions for these drugs each year. Recently enacted legislation instructs FDA to develop evidence-based prescribing guidelines and to explore how manufacturers can provide medications in more secure packaging, Woodcock pointed out. At the same time, FDA will craft guidelines on developing non-opioid drugs for acute and chronic pain to improve treatment for patients.

Jill Wechsler is the Washington Correspondent for Applied Clinical Trials