Develop Drugs With Data

By Bill Frist and Bart Gordon

With continuing advances in science and technology, the pace of medical breakthroughs is accelerating. Today, we can treat or even cure conditions that significantly shortened life just a generation ago. The exciting promises of personalized medicine are now becoming reality.

But bringing new drug discoveries to patients remains a slow process. On average, it takes 10 years and $2 billion to bring a new medication to market. With 10,000 known molecular-based diseases, and approved treatments for only 500 of them, the urgency of finding the next generation of cures is clear.

Real-world data – data already amassed in electronic health records and other clinical software, claims systems and even in patients' personal health tracking devices – is a rich source of information that can play a key role in accelerating the Food and Drug Administration's approval process for promising drugs and devices to patients in need. While the use of real-world data in pre-market evidence development is not specifically prohibited by law, the lack of clarity regarding its use to support drug approvals has hindered its incorporation.

As co-chairs of the Bipartisan Policy Center's initiative on Advancing Medical Innovation, we have been working with experts and stakeholders to explore a range of ways to improve the discovery, development and delivery of cures for Americans. Last year, the center released recommendations in a number of key areas that we felt were ready for bipartisan action. One of those recommendations was to expand the generation and use of real-world evidence to enhance research and shorten the pathway to drug approval, while maintaining the highest standards of safety.

Over the past year, Congress has expended significant effort to move legislation forward that includes many of those recommendations, both in the 21st Century Cures Act passed by the House last year, and in a medical innovation package produced by the Senate Health, Education, Labor and Pensions Committee.

Clinical trials are the most critical, expensive and time-consuming phase of the drug development process, taking on average nearly seven years and costing about $1.6 billion. Drug companies have conducted clinical trials in essentially the same way for more than 50 years. These clinical studies are largely based on randomized controlled trials, in which carefully vetted and monitored patients are given the trial medication or a placebo, and the results are studied and compared.

But these tightly controlled trials aren't inclusive of the broad range of patients who may benefit from drugs. Patients with multiple conditions or those who take other medications are not generally eligible for trials. Clinical trial participants often don't reflect varying ages, races or ethnicities.

Real-world data from these patients can complement the findings from traditional trials by providing more information about safety and effectiveness. Using real-world data can help trial sponsors improve the design of clinical studies; ease patient recruitment; reduce data collection and reporting burdens; enable the development of new, adaptive approaches supported by close monitoring; enhance current post-market surveillance processes; and improve the generalizability of trials by including data from more diverse populations and practice settings.

To further integrate real-world data into the drug development process, several actions will be required. BPC's June 2016 report, "Using Real-World Evidence to Accelerate Safe and Effective Cures," discusses the challenges of using real-world evidence today, and recommends the following policy actions to move forward with the expanded 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; and
• Explore new adaptive pathways to modernize drug development and support a new era of personalized medicine.

America is a land of innovation. New, fresh ideas, combined with the hard work and confidence required to bring them to fruition, are the very stuff of the American spirit. To turn ideas into new treatments and cures requires a better, faster and smarter review process that adapts to today's realities, including the abundant availability of real-world data.

Patients are waiting for new treatments and cures. We owe it to them to use our best innovative thinking to combine scientific rigor and safety with efficiency, especially because so many of them don't have much time left to wait.

Bill Frist, a cardiothoracic surgeon who co-chairs the Bipartisan Policy Center's Health Project, served as Senate Majority Leader from 2003 to 2007.

Bart Gordon is a lawyer who served in the House from 1985 to 2011. He is co-chair of the Bipartisan Policy Center’s Advancing Medical Innovation Initiative.

Published in U.S. News & World Report on July 11, 2016.