The US Food and Drug Administration (FDA) has several internal boards. Since 1997, it has been guided by its Science Board, which has from time to time recommended huge overhauls of the agency's operational approaches. More recently, it created a Medical Policy Board within its Center for Drug Evaluation and Research (CDER). Now a new proposal by the House Energy and Commerce Committee would establish something far more comprehensive: a Board of Directors for the entire agency.
The development, announced on 21 March 2013 as part of a package of proposed reforms for the agency, is in response to legislators' concerns that the agency isn't being receptive enough to patients. "Patients and innovators are on the front lines in the fight against diseases like Alzheimer's and cancer, yet their voices are not always heard by FDA," Reps. Fred Upton (R-MI), Joe Pitts (R-PA) and Michael Burgess (R-TX) explained in a statement. The representatives are all among the highest-ranking on the Energy and Commerce Committee and highly influential in the GOP-controlled House of Representatives.
An FDA 'Board of Directors'
Whether or their sentiments are actually true-and plenty at FDA, including Commissioner Margaret Hamburg, have vigorously contested those same sentiments in recent weeks-the perception could lead to huge changes at the agency if the legislators get their way.
"There are two ways Congress can address this problem," the legislators wrote.
The first recommendation, they said, was to create an "FDA Board of Directors" that would be comprised of patients, industry and scientific experts, all of which would be charged with evaluating the agency's ongoing performance and coming up with ways to improve its performance and regulatory processes.
"This will help in providing the oversight necessary to further correct FDA's current problems," the legislators explained.
Innovation: FDA's New Mission?
The second change, first proposed during the debate over the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012, would for the first time make the promotion of "innovation" a key part of FDA's statutory mission statement. Right now, that mission extends to protecting public health by ensuring that products are safe, effective and secure.
While FDA considers innovation to be a key part of its mission, frequently mentioning the concept in speeches, materials, on its website in the explanation of its mission and when discussing new product approvals, it is nevertheless not one it is mandated to consider when making regulatory decisions.
Legislators said that a report issued by a presidential panel of advisors, the President's Council of Advisors on Science and Technology (PCAST) supported this change. Their September 2012 report, "Propelling Innovation in Drug Discovery, Development and Evaluation," calls on the agency to make innovation central to its mission statement "to ensure a steady stream of new safe and effective medicines for unmet medical needs."
Similar measures were non-starters during the FDASIA debate, with some arguing that the novelty of a device should have no bearing on whether it is on balance safe and effective, and would only serve to complicate the regulatory process even further. In addition, most Advisory Committees used by FDA already have a patient and industry representative serving on them, as well as experts, and some argued the committees could already take innovation into account when making decisions.
Other Proposed Changes
The committee also raised the prospect of several other more general changes to the agency, all of which it said it will pursue over the coming term. Legislators said they would, for example, continue to protect mobile medical applications from "bureaucratic red tape," which they said could stifle innovation in the sector.
In addition, they said they would take steps to "empower the sickest patients to pursue available treatments [by taking] steps to improve patient education and doctor communication about potential hazards. This could be a sign that the committee is prepared to push FDA toward a less risk-averse regulatory paradigm, though the committee did not elaborate on the meaning of this particular language.
Legislators also said they wanted to expand upon their efforts, first taken under FDASIA's Generating Antibiotic Incentives Now (GAIN) Act, which gave new regulatory review incentives to companies with novel antibiotics, to bring new antibiotic treatments to patients.
"While the GAIN Act was a great first step, we will pursue additional ways to foster investment in antibiotics," they said. "We will encourage FDA to collaborate with innovators to use improved and transparent standards for approval to make sure that safe and effective drugs for the 21st century get to patients in a timely fashion. Further, incentives should be applied across technologies so that we do not unintentionally discourage life-saving treatments from getting into patient hands."