Sabrina Tavernise, New York Times: April 30, 2015
WASHINGTON — Legislation that would have accelerated the pace of federal drug approvals in a way that critics said threatened to erode patient safety was formally released this week, in a scaled-back version with many of the most controversial provisions left out.
The draft bill presented at a hearing in the House on Thursday represents a less aggressive streamlining of the drug approval process, critics of the earlier draft said, and seems to have secured strong bipartisan support.
The legislation, called 21st Century Cures, has been in the works for months. Its lead sponsor, Fred Upton, Republican of Michigan, said it would speed the pace of drug cures by removing unnecessary hurdles from the regulatory process.
Critics, including top officials at the Food and Drug Administration, had expressed concern that the changes would risk patient safety — for example, by potentially permitting shorter clinical trials and letting drug companies use alternative measures of health as evidence of a drug’s effectiveness and safety.
The bill’s supporters said it was a work in progress that had been assembled in one of the most collaborative, transparent processes Congress has seen in years. The sponsors held eight hearings and more than a dozen round-table meetings in districts across the country to gather comment.
“We have a chance to do something big, and this is our time,” Mr. Upton said.
His co-sponsor, Diana DeGette, Democrat of Colorado, said the lawmakers had made “tremendous progress,” recalling “that hokey video” that she and Mr. Upton made to promote the effort last year.
The draft — at about 200 pages it is half its former size — seemed to allay fears among some experts that it would fundamentally rewire the way drugs are approved. Earlier proposals to give drug companies broad powers to promote their products for uses other than the approved ones and to market brand-name drugs for a longer time without generic competition were not included.
The F.D.A. has defended its record for drug approval speed, saying that in 2014, it approved the most new drugs in almost 20 years, and that it moves faster than its counterparts in other wealthy countries. Experts note that pathways exist for expedited approval, and that the F.D.A. is held to relatively strict timelines under other rules set by Congress. Instead, they say, the bottleneck for new drugs is often a matter of the years of research it takes to find them.
Diana Zuckerman, the president of the National Center for Health Research, said earlier versions of the legislation had “considerable micromanaging of the F.D.A. and enormous power given to industry.”
She added, “Most of that is gone.”
But she cautioned that the bill still held numerous provisions she believed put people at risk, including ones she described as lowering standards for the approval of medical devices and antibiotics. The bill would increase the work the F.D.A. must perform, but not the money it receives, and Dr. Janet Woodcock, an agency official who testified Thursday, said that was a concern.
“We are very stretched in our resources,” she said.
One point that is likely to concern the F.D.A. is a provision that would reduce its authority to regulate some software associated with medical devices.
An earlier version of the bill would have let drugmakers use quicker measures for a drug’s effectiveness during testing — for example, changes in blood sugar level instead of a more final outcome, like development of diabetes. The new draft instead suggests ways the F.D.A. could use those alternative measures, called biomarkers.
The F.D.A. says it already uses them in a substantial share of drug approvals, and Dr. Woodcock told the lawmakers Thursday that the agency did not need additional authority on this count. Progress has been slow in adopting more biomarkers, she said, because they often did not produce enough evidence to allow scientists to draw firm conclusions.
“You have to know those biomarkers are reliable before you can take a chance on a human life,” she said.
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