FDA Clarifies Research Policies to Facilitate New Drug Development

This past year brought kudos to the biopharmaceutical research community, as manufacturers tested, and FDA approved, multiple innovative medical products, including important new cancer treatments, vaccines, cellular and gene therapies, and complex generics and biosimilars. Such advances have benefited from FDA efforts to streamline clinical testing methods, clarify regulatory policies, and accelerate application reviews to speed new therapies to market. FDA Commissioner Scott Gottlieb has emphasized the need to moderate the cost and time for developing new medicines to help bring down drug costs and spending. This objective has fueled agency efforts to update research policies, issue more draft and final guidances on investigative strategies, and to meet more often with sponsors and with stakeholders to advance development programs.

One visible result is a wave of new guidances outlining recommended approaches for testing certain types of drugs and for collecting research data. Recent draft guidances from the Center for Drug Evaluation and Research (CDER), for example, discuss endpoints for prostate cancer studies, meta-analyses for evaluating drug safety, methods for developing drugs to treat chronic hepatitis B, and use of certain markers in assessing metabolic malignancies (new CDER guidances listed here). There are advisories on using master protocols to expedite development of oncology therapies, on adaptive clinical trial designs, and on clinical pharmacology and toxicology issues. A recent listing of surrogate endpoints under development aims to help sponsors identify what markers might be useful in future R&D programs.

FDA also has held numerous public workshops and meetings to discuss models and approaches for advancing medical product development and to clarify regulatory procedures and policies. Training courses help advance the expertise of clinical investigators, and patients continue to provide valuable perspectives on developing innovative treatments to agency experts and advisory committees. A recent advisory committee examined whether FDA should require cardiovascular outcomes trials in developing new diabetes treatments. And agency experts discussed the qualification of biomarkers, animal models, and clinical outcome assessments at a December public meeting.

A main challenge for sponsors is to achieve timely enrollment in clinical trials of sufficient numbers of qualifying patients. FDA is assessing study inclusion and exclusion criteria to identify barriers to increased diversity in study populations, including women and elderly patients. One recent guidance advises on enrolling adolescents in adult studies as part of efforts to include more under-represented patients in trials. And to reduce the complexity of research requirements, FDA recently proposed to limit informed consent requirements for studies that involve minimal risk to participants, as determined by institutional review boards.

To expedite the review of applications for cutting-edge therapies, the agency launched a pilot for real-time review of oncology drugs. This allows early assessment of safety and efficacy data before the complete application is filed, as part of efforts to speed highly promising treatments for serious conditions more quickly to patients. CDER director Janet Woodcock hopes to expand the real-time review model to new treatments for additional serious conditions, and eventually to all new drug applications.

Further efficiencies are the goal of a reorganization of CDER’s Office of New Drugs (OND), which aims to be finalized in another year. The new OND will have more offices and divisions able to oversee more similar therapeutics that raise common research issues and can be managed more efficiently. Project management and policy staffs will move from review divisions to central OND offices with the aim of being more flexible and ensuring greater consistency in review decisions for all drug classes.

Jill Wechsler is the Washington Correspondent for Applied Clinical Trials