A bipartisan initiative in the U.S. House of Representatives to modernize the drug development and approval system held the first in a series of exploratory hearings  today. The so-called 21st Century Cures initiative, launched by two senior lawmakers on 30 April, will spend several months soliciting feedback from scientists, regulators, patients, and industry experts. The lawmakers may then propose legislation aimed at more quickly moving potential treatments from the lab to the clinic.
The initiative is the result of collaboration between Representative Fred Upton (R–MI), the chair of the Energy and Commerce Committee, and Representative Diana DeGette (D–CO), one of the panel’s senior Democrats. In an online announcement of the effort , the pair expressed concern that getting drugs to market is increasingly costly and time-consuming. Upton lamented “a major gap between the science of cures and the way we regulate these therapies,” and DeGette, an outspoken supporter of human embryonic stem cell research, vowed to “take a comprehensive look” at how Congress might intervene. Upton, a conservative who often parts company with DeGette on policy issues, wrote in a recent U.S. News and World Report op-ed  that “U.S. regulatory policies remain stuck in the Sgt. Pepper era.” Upton is in a powerful position to act on these concerns. He has at least 2.5 years remaining as chair of his committee, which helps oversee the Department of Health and Human Services and the Food and Drug Administration (FDA), among other agencies.
On 6 May, the pair kicked off the initiative by hosting an informal round table. Guests included National Institutes of Health Director Francis Collins and Janet Woodcock, the director of FDA’s Center for Drug Evaluation and Research. They discussed what are likely to be recurring buzzwords in the ongoing conversation: promoting “clinical trial networks” that could oversee multiple drug trials; identifying “surrogate endpoints” for clinical trials—short-term markers that a patient is benefiting from a drug that might speed its approval; and encouraging FDA to make broader use of its “accelerated approval” pathway, historically limited mostly to approving cancer and HIV treatments.
At today’s health subcommittee hearing, the experts invited to testify echoed many of those suggestions. The panel included five contributors to a President’s Council of Advisors on Science and Technology report on drug innovation , which in 2012 offered a list of recommendations  for improving the drug approval process. Sara Radcliffe, executive vice president for health at the Biotechnology Industry Organization, said she’d like FDA to further clarify how a drug trial might introduce a new endpoint to measure its efficacy, and how medicines targeted at small populations might better take advantage of the expedited approval process that already exists. Frank Sasinowski, speaking on behalf of the National Organization for Rare Disorders, repeated that suggestion. Questions from committee members also focused largely on FDA, and many were critical of what they perceived as a risk-averse culture at the agency.
The emphasis on FDA reflects an area where the committee has focused its legislative energies in the past, Jeff Allen, one of the witnesses at the hearing and executive director of Friends of Cancer Research in Washington, D.C., told ScienceInsider. He noted that the panel played a big role in shepherding into law a 2012 measure—the FDA Safety and Innovation Act (FDASIA)—which created a new “breakthrough therapy" designation to speed the approval process for certain treatments for seriously ill patients. The new effort is a way to look beyond FDASIA, he believes. “That law was squarely focused on the FDA, and perhaps the 21st Century Cures [initiative] will allow an examination of the totality of the system, rather than just the point of market entry.”
Press releases and white papers from the initiative hint that it might take a more holistic view. The most recent , for instance, asks patients to send the panel comments on the current state of research, funding, and available treatment for their diseases.
Not surprisingly, many researchers and patient groups watching the new initiative say funding for basic research that identifies new drug targets is a key issue. But spending levels aren’t something that Upton’s committee controls; that’s largely up to the appropriations panels. Still, Radcliffe told ScienceInsider that although today’s hearing produced nothing she hasn’t heard before, there were “some really interesting proposals.” It will likely be many months, however, before advocates learn how Upton and DeGette plan to respond to what they learn.