There are concerns that the US Food and Drug Administration (FDA) is failing to consider the views of patients who need access to life-saving drugs, including those treatments that carry some risk, a Congressional leader has said.
Moreover, a "lack of predictability and certainty" at the agency appears to be "stifling American innovation, costing American jobs and hurting American patients," added Republican Congressman Fred Upton, chairman of the House Energy and Commerce Committee, as he opened the first of a series of Congressional hearings on reauthorisation of the Prescription Drug User Fee Act (PDUFA).
The current PDUFA legislation - under which the drug industry pays user fees to the FDA, which in turn commits to performance goals - expires on September 30, 2012.
Rep Upton wondered if the FDA is striking the right balance on the "delicate issue" of risk/benefit analysis, and was also highly critical of certain provisions of the FDA Amendments Act (FDAAA), including those affecting advisory committees and Risk Evaluation and Mitigation Strategies (REMS).
"The rigid and unrealistic conflict-of-interest provision has prevented FDA and its advisory committees from utilising some of science's best minds and left advisory committee slots unfilled. We must also look at implementation of FDAAA's REMS provision and whether it has caused delays in the approval process," said Rep Upton.
The hearings are being conducted by the House panel's health subcommittee, whose chairman, Joseph Pitts, told the meeting that he plans to have the PDUFA reauthorisation legislation signed into law by June 30, 2012; "PDUFA is too important to leave to the last minute," he said.
Witnesses at the first hearing included Dr Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research (CDER), who was questioned by the panel on risk/benefit issues, and specifically on the agency's decision that Genentech/Roche's Avastin (bevacizumab) will no longer be approved for the treatment of metastatic breast cancer.
Dr Woodcock told the legislators that studies with Avastin had failed to show a better quality of life, or longer life, for patients, but she added: "that does not mean that Avastin is not an active drug perhaps for some women, but we do not know what women."
However, Republican subcommittee member Michael Burgess told her that, among the list of people on the advisory panel who made the decision to withdraw this indication, "I don't see anyone that would have had the ongoing daily treatment of breast cancer patients under his or her control. You had a lot of experts and a lot of oncologists, but I didn't see a specific specialist in the speciality of metastatic breast cancer," Rep Burgess told Dr Woodcock.
Marc Boutin, chief operating officer of the National Health Council (NHC), pointed out that a patient with a life-threatening disease with limited treatment options is likely to have a much higher tolerance for "riskier" drugs than people who are not using medicines to manage their daily lives.
He called on the FDA to develop and implement a risk/benefit framework in the drug review process "that places the drug's intended recipient into the equation." When evaluating a drug's risk, the FDA must take into consideration the size of the population affected, the range of existing treatment alternatives available to those patients and the risks of living with the condition, said Mr Boutin, adding: "for patients with a rare or incurable condition, especially those with few or no treatment options, restricting access to a new drug is potentially devastating."
Rep Pitts told the hearing that “the American people expect - and have a right to expect - that the federal government is doing everything possible to ensure that drugs on the market are safe and effective. They also have a right to expect that applications for life-enhancing and life-saving drugs are not languishing on a reviewer's desk at FDA." He added that he hoped the new agreement "balances both of these considerations."
Details of the drug industry/FDA agreement on PDUFA reauthorisation will be published on September 1, 2011, and the agency will send its final recommendations by January 15 next year.