FDA Looks to Consumers for Pre-approval Input
May 23, 2012 – At an FDA meeting held last week to explore more effective ways of including patient input in regulatory decisions regarding drug, device and biological products, the agency made it plain to patients, caregivers, patient advocates and patient advocacy groups that it wants patient perspectives to be included in the process.
“This initiative, led by Center for Drug Evaluation and Research Director Janet Woodcock, M.D., is the second of two ‘game changer’ proposals that put consumers and marketing at the center of the discussion, and potentially at the center of the drug approval and use processes,”
said Coalition for Healthcare Communication Executive Director John Kamp, who explained that earlier this year, Woodcock began a discussion about the creation of a third class of drugs where initial prescriptions and refills could sometimes be filled without a physician (http://www.cohealthcom.org/2012/04/30/proposed-otc-drug-distribution-would-expand-patient-access/).
“Together, these proposals mean that FDA is fully recognizing the power of the patient and putting patients at the center of health care in America,” Kamp said.
In a notice published before the May 18 Inaugural FDA Patient Network Annual Meeting, the FDA stated that “establishing a means for obtaining input from patients and patient advocate groups will allow FDA to further enhance its benefit-risk assessment in regulatory decisionmaking.”
Further, the agency said that “patients who live with a disease have a direct stake in the outcomes of the review process and are in a unique position to contribute to the weighing of benefit-risk considerations that can occur throughout the medical product development process.”
At the meeting, CDER’s Woodcock noted that in general, regulation is “the result of a societal consensus that limits the actions or speech of certain parties in society.” She added that although science can inform risk-benefit decisions, it “doesn’t tell us if the benefits outweigh the risks. That’s a value judgment.”
The FDA set forth junctures in the drug approval process where it believes that consumer/patient input would be most valuable: (1) the pre-Investigational New Drug (pre-IND) stage and (2) the pre-New Drug Application (pre-NDA) stage. Input during the pre-IND period is important, according to CDER officials, because such input would fill information gaps about treatment benefits for specific conditions. At the pre-NDA stage, patients could provide input regarding the new therapy’s effectiveness and tolerability.
It appears that patient input also would give the agency additional information about which treatments – drugs or other modalities – are available to and working for patients. This information could help the agency enhance its value judgment-making capability.
“Acetaminophen … very rarely causes devastating liver failure, and some people say that even a rare risk is unacceptable,” Woodcock said. She explained that knowing more about a drug’s impact on a population can help the agency with its decisions, citing a multiple sclerosis treatment that helped many patients but potentially could put certain individuals at risk of progressive multifocal leukoencephalopathy. “The MS community rose up to help keep this drug on the market,” she said.
The agency currently is preparing a list of 20 diseases/conditions for which it will gather patient input under the Prescription Drug User Fee Act V. Incorporating patient perspectives into the regulatory review process is a “major task for FDA over the next several years,” according to Woodcock.
“At this juncture, we need to work together,” she said. “The bottom line is to improve the diagnosis of illness and alleviate suffering.”
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