WASHINGTON – The House Energy and Commerce Committee expects to send the 21st Century Cures Act up for debate in the House as early as next week, Clay Alspach, chief majority health counsel for the committee, said during Wednesday's Develop Innovate Advance (DIA) annual meeting here.

According to Alspach, one of the main goals of the 300-page legislation is to create an "innovation Eco system," which among other things would increase funding for the National Institutes of Health (NIH) and FDA so they can develop a better regulatory structure and provide a forum for collaborator engagement.

"There are over 10,000 diseases out there; we only have cures and treatments for 500, so there is a lot of work to do," he said. "We have to do better. This legislation is about what more we can do to help the innovation Eco system."

Alspach said the committee has been lobbying other members and engaging with additional stakeholders to make a few improvements to the legislation based on the additional input it has received. However, he added, it is just part of the process to make sure the bill they put forth to the House is in the best shape possible as soon as possible.

"The reason for that is patients can't wait," he said. "The innovation Eco system can't wait, and there are important provisions in the bill that need to happen. One of those provisions is the money that will go to FDA. That starts in October if we get our job done."

THE MOST TRANSFORMATIVE PROVISIONS

Alspach and other members of the panel discussed the most transformative provisions of the bill and the impact it would have on drug innovation. One key provision that all members touted was that the bill encourages and supports patient-focused drug development, which they said will drive even more patients into the drug development process than ever before.

Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), told attendees that patient-focused drug development has the most promise to be the most transformative piece of the legislation.

"If we can actually develop a structured way to gather information from a representative sample of patients with a disease about the burden of the disease so we understand what matters to them, then we can figure ways to measure that and then figure out a way to report it back to them so they can see how they are doing with their intervention," she said "I think this will be transformative in understanding what the medicines will and will not do. But this is a long-scale process, and we have to get it started if we are going to make this process happen."

Woodcock added that everything in the bill involves a consensus-driven process to get something official and that most people support.

"No one can dispute that innovation in drug development needs help," she said. "We are seeing a lot of drug development being based on molecular targets that are clear and understood, but there is still no doubt that this process takes too long, costs too much, and is way too unsuccessful. There are still only about two out of 10 drugs that will enter the clinic and come out the other end, and one of those two may not be that successful commercially so there is a lot of room for improvement in this area and I think this bill is going to see where legislation can help to move things along."

Another key issue the bill, as written, will help to address is the lack of funding for NIH and FDA. According to Woodcock, FDA is losing a lot of scientific expertise to other industries because the agency cannot afford to keep pace with salaries and other incentives to retain talent. (See story in this issue on budget preview.)

"There is a tremendous concern that young inventors are getting out of the game," she said. "Generally speaking, most of our mandates have been unfunded and that really stretches us thin and makes those mandates very difficult and for us to accomplish what people expect us to accomplish."

Woodcock added that the additional money called for in the bill would allow the agency to enlist other research and academic partners in developing guidance to help guide industry through the research and approval processes.

Ellen Sigal, founder and chairwoman of Friends of Cancer Research echoed Woodcock's concern about talent retention at the agency.

"[The FDA needs] to get the best and the brightest, she said. "The dollars for FDA and NIH are crucial because they have been underfunded. We can't get to innovation and science if we don't have research and discovery."