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BPC Calls for Modernizing FDA’s Drug Development Process by Using Real-World Patient Data

Thursday, June 23, 2016

Washington, D.C. –  A new report released today by the Bipartisan Policy Center calls for modernizing the life cycle of drug development in the United States through the use of real-world evidence. It emphasizes that this data, which is available through electronic medical records, claims databases, and even data from patients themselves, has the potential to improve the Food and Drug Administration’s (FDA) process of bringing promising drugs and devices to market.

Policy experts speaking during today’s event, including Sen. Lamar Alexander (R-TN), chairman of the Senate Health, Education, Labor and Pensions Committee, agree that scientific discovery is moving rapidly, but the pace of advancing new drug discoveries to patients remains slow. With so many diseases and conditions still lacking effective treatments, the need to accelerate the search for tomorrow’s cures is greater than ever before.

“A new drug approval process that maintains the highest standards of safety and brings new medications to market faster will help tens of thousands of Americans desperately waiting for treatments and cures,” said Bill Frist, M.D., former Senate majority leader, and co-chair of BPC’s Advancing Medical Innovation effort. “Using real-world evidence -health data that reflects the actual experience of patients- to supplement traditional randomized controlled trial data, will enhance the safety and effectiveness of drugs in a wider population and benefit far more people.”

BPC recommends the following policy actions to expand the use of real-world data:

  • Improve Regulatory Clarity Regarding Use of Real-World Evidence
  • Improve Methods and Data Quality for the Generation and Use of Real-World Evidence
  • Improve Policies for Information Sharing to Support Clinical Research
  • Explore New Adaptive Pathways to Modernize Drug Development and Support a new Era of Personalized Medicine

Read details about the recommendations

Through its extensive research with experts and practitioners, BPC identified numerous benefits for using real-world evidence to modernize drug development. It can inform clinical study design and enable risk-benefit ratio stratification; improve the recruitment of patients to trials; reduce data collection and reporting burden; improve patient safety and adverse event reporting through real-time monitoring; and improve the generalizability of trials by including data from more diverse populations and practice settings.

For the past 50 years, randomized controlled trials have been the mainstay and “gold standard” of pre-approval drug research, and are designed to eliminate bias in outcomes. These trials are ordinarily conducted within small and narrowly defined populations without samples of patients with multiple diseases, concomitant use of other drugs, varying races and ethnicities, and different practice settings. This report demonstrates that real-world evidence has the potential to greatly enhance and complement findings from traditional randomized controlled trials by providing more data about the safety and effectiveness of drugs in the real-world. It is also expected to provide more relevant information to support evidence requirements in a new era of personalized medicine.

“Clinical trials are the most critical, expensive, and time-consuming phase of the drug development process,” said former Rep. Bart Gordon, co-chair of BPC’s Advancing Medical Innovation effort. “This phase takes nearly seven years and $1.6 billion of the nearly $2 billion spent on bringing a new drug to market. Using real-world data can significantly strengthen the evidence base for approval, safety, and effectiveness of new drugs and devices.”

“We need to give the FDA the authority it needs to advance real-world evidence in an incremental way across the drug development life cycle,” said Andrew von Eschenbach, M.D., BPC advisory committee member and former NCI director and FDA commissioner. “We also need to lay the foundation for the future of personalized medicine. Using real-world data can help explore a new paradigm for drug development.”

“Implementing the steps outlined in this report will enable the United States to make significant strides in leveraging real-world evidence to help get safe and effective drugs to market faster and more cost-efficiently,” said BPC Director of Health Innovation Janet Marchibroda. “Taking these actions will provide hope and help to patients, who are waiting for much needed cures and treatments.”

Leveraging real-world evidence to support medical product development and delivery was also one of the key recommendations of BPC’s 2015 report, Advancing Medical Innovation for a Healthier America.

BPC’s advancing medical innovation work is supported by the Jayne Koskinas Ted Giovanis Foundation for Health and Policy.

Learn more about BPC’s work on medical innovation.

KEYWORDS: BART GORDON, BILL FRIST, FOOD AND DRUG ADMINISTRATION, JANET MARCHIBRODA, LAMAR ALEXANDER, SENATE HELP COMMITTEE