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In swansong, US FDA chief defends drug approval process
[WASHINGTON] In her final speech after six years as commissioner of the US Food and Drug Administration, Dr Margaret Hamburg defended the agency's drug approval standards against critics seeking weaker regulatory standards. "The great leaps forward in evidence-based medicine of the last 50 years have come in large part as a result of the high standards for product approval that Congress put in place after a series of disasters involving unsafe and ineffective medical products," said Hamburg, 59, said on Friday at a National Press Club lunch in Washington, DC.
Her comments come amid a push by some lawmakers for faster approvals of experimental drugs. A provision in a draft House of Representatives bill known as 21st Century Cures, for example, would allow the FDA to approve drugs designated as "breakthrough therapies" based on preliminary clinical data.
Only after the products were on the market would companies be required to conduct the type of longer-term safety and efficacy studies typically required for a drug's approval. "Breakthrough therapy" designation is currently given when early data shows a product may confer substantial improvement over an existing therapy. The products are reviewed particularly quickly and companies receive more intensive FDA drug development guidance.
But they are not approved based on the preliminary data alone. Under the proposed bill they could be. Last year the FDA received 96 breakthrough therapy requests and granted 31.
Ms Hamburg noted that the FDA typically approves new drugs faster than any other developed nation. Last year it approved the most new drugs in 20 years.
She said she disagrees with critics who argue that FDA regulation is the principal hurdle to the development of innovative new treatments. "In the race for the newest treatment we must remember the point that innovation doesn't matter if the product doesn't work," she said.
Under pressure, the FDA has proposed allowing pharmaceutical companies to distribute medical literature showing that a drug may work for a condition for which it has not been approved. It also has proposed allowing companies to distribute literature showing the risks may be lower than shown on a drug's label.
Ms Hamburg said that while the FDA supports "responsible"communication of scientific information it does not support an approach that undercuts the incentive for studies to be done. "History has shown that patients have been harmed by physician reliance on preliminary or incomplete information regarding unproven uses," she said.