Bill to speed drug evaluations advances to House
Washington — A bill that would overhaul how the Food and Drug Administration reviews and approves new medical treatments and products won unanimous committee endorsement Thursday to advance to the House floor, led by U.S. Rep. Fred Upton, R-St. Joseph.
The 51-0 vote followed a one-day delay to build consensus among lawmakers around how to pay for the bipartisan legislation, which provides $550 million to the FDA over five years to reform and modernize programs and $10 billion for the National Institutes of Health.
"We have all said too many early goodbyes to people we treasure," said Upton, chairman of the Energy and Commerce Committee. "We can and must do better."
Rep. Joe Barton, R-Texas, a former chairman who has served on the Energy and Commerce Committee for 20 years, emphasized the rarity of having no dissenting votes on a major piece of legislation. "That, I say, Mr. Chairman, is a major accomplishment."
Upton said the spending offsets would "fully pay for the cost of the bill."
The cost of the legislation is paid in part by drawing down and selling excess crude oil from the strategic petroleum preserve; adjusting the timing of pre-payments to prescription drug sponsors through Medicare Part D; and limiting Medicaid reimbursement rates for durable medical equipment. The bill's architects are Upton and Rep. Diana DeGette, D-Colorado.
Besides trimming red tape, the House bill would streamline clinical trials and provide incentives for companies to research and repurpose drugs approved to treat rare diseases. It also encourages more patient input during drug development and evaluation.
Currently, patients have a single vote on the FDA panel that determines whether a drug or medical device is safe or effective enough.
"That's all the way after a 15-year development program and after the clinical trials are done," said Kim McCleary, director of strategic initiatives for the nonprofit FasterCures, which wants patients' experiences and preferences to be considered earlier in the process.
Upton wants to get the bill on the president's desk by year's end, but a parallel Senate effort led by Republicans Richard Burr of North Carolina and Lamar Alexander of Tennessee isn't as far along.
Burr said last week he's more concerned with accuracy than speed when drafting the Senate bill.
"We only get one shot at getting this right," Burr said at a symposium on biomedical innovation. "What drives me right now is that many Americans, when they get sick, have a choice between nothing and nothing."
Advocates say there are too few treatments for 10,000 or so known diseases. It can cost billions and take more than a decade to develop and test a drug and get it to patients. Still, many promising therapies fail.
Rep. Jan Schakowsky, D-Illinois, said in the future she'd like to see the FDA requiring companies to report the total cost to research and develop a drug or product, and what portion of that cost was covered by NIH dollars.
"We keep hearing that companies need to be incentivized to produce more drugs, but I'm not convinced," Schakowsky said.
The actual cost of production of a particular drug is "hidden from view," forcing policymakers to rely on estimates of the average cost of drugs, she said.
"That is no longer good enough. If drug companies are going to make the argument that they need more exclusivity, we need to have the data to prove that. We continue to see drugs come to market to address critical health problems that are simply unaffordable for most people."
She cited as an example the new hepatitis C drug Sovaldi, which costs $84,000 for a course of treatment.
Rep. Bobby Rush, D-Illinois, proposed a revision incorporated into the bill that calls for increased inclusion of individuals from minority communities in clinical trials.
Upton has said his work on the bill was inspired by sisters Brielle and Brooke Kennedy of Mattawan, ages 7 and 6, who have a rare, genetic muscle-wasting condition called type II spinal muscular atrophy, for which there is no known cure or treatment.
"I can't help but think of the patients who are sitting across from their doctors, about to get the news that will change their world," Upton said at a hearing Wednesday.
"It's not just the disease that makes them feel powerless and vulnerable — it's the very system that's designed to help them has not kept pace with scientific advances. They need the next generation of treatments and cures, but they do not have until the generation to wait."