The FDA, which has successfully reduced the review times for new medications, wants to help biopharmaceutical companies lower the rising costs of clinical trials by streamlining some of its processes and working more closely with industry.
That’s the proposal Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research (CDER), outlined to senators on the Health Education Labor and Pensions committee last week.
“Each time a new drug is tested, the process is repeated, at great expense, only to dismantle the infrastructure when the study is completed,” said Woodcock. “We believe that there are ways to greatly improve clinical trial efficiency, such as widespread use of clinical trial networks and master protocols, and we would like to work with you to examine those possibilities.”
The FDA’s proposed efforts follow the U.K.’s National Institute for Health Research, a clinical research network that recently restructured and supports numerous trials each year.
Woodcock cited President Obama’s $215 million Precision Medicine Initiative, which aims to arm clinicians with the new tools, knowledge and therapies that will work best for each patient. In addition to master protocols, Woodcock cited the need to identify and evaluate the utility of biomarkers and enhancing other scientific tools to predict and evaluate the effects of proposed drugs before clinical testing.
She cautioned that while biomarkers are important tools for predicting and evaluating the effects of potential drugs, they have been slow to come into clinical use due to a lack of validity. However, that can change, she said, and new tests could speed evaluation of drug performance, including drug safety and prediction of effectiveness.
“Similar to the problems with clinical trials, the scientific infrastructure for evaluating the validity of new biomarkers has not kept pace with the need for this activity,” said Woodcock.
She cited CDER’s recently established Biomarkers Qualification Program, which can evaluate “qualified biomarkers” for a certain use during the regulatory process by any developer.
Still, there is confusion about how new biomarkers can be qualified, along with a perception that many biomarkers are “stalled” in the qualification process. Woodcock explained the biomarker qualification process still is in the evidence-gathering stage, adding that it may take considerable more development work within the scientific community.
Expanded use of biomarkers continues to play a significant role in personalized or precision medicine, in which new drugs often are targeted to a genetically determined subset of people with a disease, such as cancer, cystic fibrosis or hepatitis C.
“As targeted therapies become ubiquitous, advances in standardizing and increasing our understanding of the biomarkers that enable use of these therapies will be necessary,” said Woodcock.
Another source of information about drug effects is evidence from clinical experience, often called real-world evidence or big data, which has prompted the FDA to develop Sentinel Initiative, a national electronic system that tracks the safety of drugs and biologics once they reach the market.
“There are other areas in which we hope to work with you as well, including modernizing drug manufacturing, encouraging the development of new antibiotics and improving the process for FDA review of dug/device combination products,” concluded Woodcock. “We look forward to working with Congress to address these challenges in ways that will serve patients, and pharmaceutical innovation as well.”