Applied Clinical Trials
A new CDER “knowledge management” approach will see companies submit applications that can be transmitted to experts from multiple disciplines able to assess applications for new drugs and biologics in a timely and efficient manner.
Under a new “knowledge management” approach for the Center for Drug Evaluation and Research (CDER), sponsors will submit applications that present data in a structured format so that it can be transmitted to teams of experts from multiple disciplines able to assess applications for new drugs and biologics in a timely and efficient manner. The aim is to shift sponsors away from long, narrative documents filed in PDF format that are repetitive and obscure important issues. Instead, CDER assessment teams composed of medical reviewers, pharmacologists, statisticians, pharmacists and other specialists as needed will examine the data to answer specific questions relevant to the product under review.
CDER director Janet Woodcock is finalizing this process for assessing and approving new drugs in order to be able to handle a surge in submissions that reflects important advances in biopharmaceutical science, as well as the digital revolution that has expanded exponentially the data and information supporting biomedical research. Our understanding of clinical pharmacology, and the vast amount of data from gene sequencing, she commented, along with greater scrutiny of the value of new medical products, require “a new approach” for providing an assessment – not a review – of new drug applications (NDAs) and biologics license applications (BLAs).
FDA hopes that this process, moreover, will make new treatments more affordable. The aim is to find out early if patients will be better off or not from access to a new treatment, Woodcock observed at the recent Biostatistics Industry & Regulator Forum sponsored by the Drug Information Association (DIA) and FDA, acknowledging that greater scrutiny of drug value is currently a major topic of debate.
To be able to assess more applications for increasingly complex medicines, review teams will identify key issues during the IND stage to be able to focus on them efficiently and with appropriate expertise. An assessment document will present a benefit/risk evaluation, the drug development program, key issues and recommendations, aiming to provide a more transparent process and clarity for advisory committees. The aim is to finish this initial assessment midway through the review period to leave ample time for FDA and the sponsor to discuss post-market research commitments and the need for a REMS (Risk Evaluation and Mitigation Strategy) well before approval deadlines. This will lead to “high quality, more uniform work products,” Woodcock said, “with fewer surprises and last-minute changes.”
The new assessment process also involves a significant reorganization of CDER’s Office of New Drugs (OND). It will expand from six to eight offices to better provide patients and researchers with clearly identifiable experts in leading disease areas: neuroscience (including pain and addiction); inflammation/immunology (rheumatic, transplant therapies); internal medicine (diabetes, hematology); oncology; human reproduction (urology, OB-GYN, pediatrics, maternal health); over-the-counter (OTC) drugs; and other treatments (ophthalmology, medical imaging, compounding).
OND also will have a centralized project management group to ensure that all divisions and teams follow consistent processes and to help external stakeholders and other FDA operations identify appropriate contacts. And a new OND policy office will support the development of guidances and other documents.
These changes will form the basis for a more automated CDER work flow management system based on standard processes and protocol assessment templates that will result in more timely regulatory decisions. The aim is to move away from multiple, and often repetitive, individual staff reviews. OND is trying out this team approach on a few applications and hopes to have it in place by the end of this year.
Looking to the long-term, Woodcock envisions the data-based assessment process will provide a model to reform how CDER manages post-market safety issues. Eventually data submitted by sponsors may move to the “cloud” in standardized formats to facilitate data exchange and analysis. These innovations reflect a “big cultural change” that will take some time to accomplish, Woodcock acknowledged. But with the rise of patient-focused drug development, genetic targeting to treat rare conditions, demand for greater transparency in the regulatory process, and more startup biotech companies focused on innovation, she believes that FDA needs new approaches for dealing with these and future challenges.