Lawmakers Respond To Advocates’ Concerns About Innovation Initiative

July 17, 2014

Lawmakers appeared to respond to concerns that the House Energy & Commerce Committee is receiving a one-sided, industry-backed patient perspective as part of its 21st Century Cures Initiative by soliciting comments July 11 from other groups and calling for a balance between the push for more expedited approvals and public health safeguards.

The National Center for Health Research recently criticized the committee for primarily working with patient groups focused on expediting approvals and excluding others that seek establishment of better safety and efficacy standards (see FDA Week, July 11).

During a Friday Energy & Commerce health subcommittee hearing, Rep. Michael Burgess (R-TX) suggested interested groups contact members about the 21st Century Cures Initiative. “I know there are many people who may be watching and thinking I would like to be involved with committee staff, so that’s the way to do it,” he said.

Rep. Frank Pallone (D-NJ) also made it clear that he supports expediting patient access to innovative treatments but is also concerned about the safety and efficacy of medical products. Lawmakers should be cognizant of the risks that come with speed, he said.

Further, in his testimony Cystic Fibrosis Foundation President and CEO Robert Beall pressed for a balance between efforts to advance the development of innovative treatments and those to ensure patient safeguards.

“As policy options are considered, it is important to find a balance between accelerating drug discovery and innovation, while also ensuring that patients have safe, effective treatments,” he said. “There must be clear communication about the benefits of current therapies so that patients can make informed decisions…as new therapies are developed.”

Rep. Jan Schakowsky also echoed women’s health advocates’ concerns about the possibility that the smaller clinical trials being pushed as part of expedited approval could work against efforts to collect more clinical trial data for women and demographic subgroups. NCHR has predicted that as clinical trial populations grow smaller they will also become less diverse.

Leonard Lichtenfeld, deputy chief medical officer of the American Cancer Society, agreed that there was a risk of excluding important patient populations but advised against using that possibility to argue against the expedition of innovative product approvals. There is the other side of the coin, he said, noting that some treatments can elicit a response from the vast majority of patients.

“When you suddenly see moments like that no one would want to hold back,” he said. “Develop the data, yes, but don’t hold back the opportunity.”

Still, the National Women’s Health Network said it remains concerned about what it sees as the committee’s focus on expediting product approvals as the best way to speed patient access to innovative treatments. “The idea that you can get treatments more quickly to patients by only accelerating the FDA process is ridiculous,” said Kate Ryan, NWHN’s senior program coordinator.

She said she would like to see the committee put more emphasis on the need to invest in diversifying scientific research to provide patients with more options. But she noted the group has talked with the office Rep. Diana DeGette (D-CO) — DeGette leads the committee’s innovation initiative alongside Chair Fred Upton (R-MI) — and feels there are lawmakers that share those priorities and concerns about women’s health.