21st Century Clinical Trials for 21st Century Cures

21st Century Cures is about embracing technological and scientific advancements to get treatments and cures to patients more quickly. It’s a forward-looking approach to health care, using new strategies to fight disease rather than doing things the way they’ve been done in the past.

When a new drug or device is developed, it must go through a clinical trial to test its safety and effectiveness. The gold standard for how these trials are conducted was developed decades ago, and a lot has changed since then. Modernizing clinical trials is essential in achieving our health innovation goals.

Clinical trials are slow and expensive. In fact, their cost and time to completion are at all-time highs. While there has been a number of promising advancements, our current regulatory structure does not fully embrace the potential of personalized medicine. Throughout our yearlong listening tour we heard time and again that addressing clinical trials is vital if we are going to get lifesaving and improving medical products to market more quickly.

“Clinical trials need to be modernized for the molecular age.” So explained Dr. Roy Herbst, Chief of Medical Oncology at the Yale Cancer Center and co-chair of the Lung-MAP Trial, which is an innovative and personalized clinical trial for lung cancer. He’s right. Our approach to health research has advanced thanks to innovations at the molecular level, but our approach to health product regulation has not kept pace.

William Murray, President and CEO of the Medical Device Innovation Consortium, summed up the potential benefits of reform: “We believe that clinical trial innovation has the potential to improve the safety and effectiveness of products being introduced into the market, reduce clinical trial timelines and costs, and give U.S. patients earlier access to beneficial innovative technologies.”

Better treatments for patients is reason enough for reform, but we cannot ignore the economic cost of an outdated approach to clinical trials. Mike Carusi, testifying on behalf of the National Venture Capital Association, explained during a June 2014 hearing, “A small, venture-backed company typically spends $500,000 to $2 million per month in operating costs as it conducts clinical trials and awaits regulatory approval.”

What can we do about it? For starters, let’s ditch the unnecessary paperwork. It wastes time and money.

Next, we can make it easier to find patients who could benefit from the research. Clinical trials for children can be some of the most difficult to conduct, and at least part of the reason why is because it’s hard for sick children and pediatric researchers to find each other. The 21st Century Cures initiative establishes a Global Pediatric Clinical Trial Network to cut time from the process by helping developers identify patients much more quickly than if they had to recruit participants on their own. Providing hope to these children is what drives this effort.

More flexibility in the design of clinical trials is another opportunity for reform. This includes use of biomarkers, biostatistics, and adaptive trial designs — all of which are tools to customize a trial based on the unique characteristics of patients most likely to benefit. The rise of personalized and precision medicine means treatments can be significantly more targeted for a patient’s individual genetic makeup or illness. Embracing these tools will help us better identify individuals eligible for trials and ultimately get the most effective treatments and data sooner.

Ultimately, clinical trials are about the patients who raise their hand and say, “I want to try a new therapy. I want to test a new treatment. I want to help you learn not only would could help me, but many others like me.” Clinical trials are a chance to better understand diseases so we can eventually beat them. Even if a clinical trial fails, the data and lessons learned contribute to someday finding cures.

Clinical trials are a linchpin in the discovery, development, and delivery cycle for cures. Health breakthroughs are meaningless if we cannot test and prove them in real patients. So let’s make clinical trials better. Let’s make them faster, safer, and more personalized. We owe it to the patients who are on the front lines trying new treatments, and those who will follow and benefit when they work.

To learn more about the exciting 21st Century Cures initiative please click here.