Lobbying groups hungrily eye fast-moving FDA overhaul bill

WASHINGTON—Why is the nation's capital suddenly obsessed with biomedical innovation?

And why is the fruit of that obsession, the 21st Century Cures Act, moving like a greased pig through the usually deadlocked Congress—even as some experts warn it could undermine the Food and Drug Administration's ability to protect the public from unproven and potentially unsafe new drugs and medical devices?

Last week, the House Energy and Commerce Health Subcommittee unanimously approved the marked-up bill, sending it to the full committee, which is scheduled to consider it this week.

“This is a product deserving of the committee's support,” said Rep. Frank Pallone (D-N.J.), who led Democratic opposition to the bill only a few months ago.

Energy and Commerce Chairman Fred Upton (R-Mich.) says he wants to pass the bill later this year.

But key senators say they are working on their own bill, which won't be ready until at least the fall. There is no agreement yet on how to pay for the legislation's costs or on whether to boost FDA funding to help the agency handle its new obligations.

There also are divisions over whether to grant drugmakers longer market exclusivity for drugs treating rare diseases.

The sweeping bill would overhaul federal regulation of prescription drugs and medical devices. It also would provide significantly more funding to the National Institutes of Health, a move that brought Democrats onboard.

The overhaul bill would give the NIH $10 billion in additional mandatory funding over five years, as well as yearly increases, hiking the agency's budget by $8 billion by 2018 over current congressional appropriations.

Observers say nearly every sector of the healthcare industry has gotten involved in lobbying the bill.

According to the Senate's lobbying database, the number of groups listing the 21st Century Cures Act as a lobbying issue grew from 62 in the second quarter of last year to 222 in the first quarter of this year.

“It became a Christmas tree,” said Billy Wynne, of Thorn Run Partners, who has lobbied the bill. “Many people see it as the moonshot opportunity for lots of stuff,” another lobbyist said.

Consumer advocacy group Public Citizen called the NIH funding a horse trade “providing perks to the pharmaceutical and medical-device industries to approve medications and devices faster based on weaker evidence.”

The feeding frenzy has attracted groups such as the Natural Products Association, which lobbies on dietary supplement issues. Its CEO, Daniel Fabricant, said the group hopes to gain coverage for its members' products under tax-sheltered health-savings accounts and flexible-spending accounts.

What has given the bill legs is the enthusiastic support of groups representing patients with particular diseases.

There's a perception that biomedical innovation is moving too slowly, said Gregory Daniel, head of the Brookings Institution's pharmaceutical and medical-device policy group. “We're making a lot of benchtop, pre-clinical breakthrough discoveries,” he said. “But we don't see a lot of breakthroughs translating into therapies.”

Under the bill, the FDA would be required to strengthen its consideration of “real-world evidence” about the efficacy of drugs.

Manufacturers would be able to expand a drug's indications based on observational data or registries indicating how it performs in the field. The FDA also would be required to develop more surrogate endpoints to allow shorter clinical trials.

For the device industry, a “breakthrough device” program would provide a faster path to market for devices that might substantially raise the standard of care.

Dr. Margaret Hamburg, who stepped down as FDA commissioner in March, has called the bill's approach misguided. She cautioned that placing additional requirements on the FDA will overstretch the agency's staff. Pallone and other Democrats have complained that the bill lacks additional funding for the FDA.

But some members of Congress argue that more regulatory flexibility is needed. Sen. Richard Burr (R-N.C.) said that for too many patients, their treatment choices are between “nothing and nothing.”

The Senate will develop its own bill that “collapses” the FDA's product review times and the CMS' time to approve payment for products, Burr added.

Pallone expressed concern about proposed longer market exclusivity incentives for drugmakers, but noted that those provisions were limited. Drugmakers would get six additional months of exclusivity for a drug with a new indication for treating a rare disease. But, Pallone said, “I do not believe companies need more protection from competition to invest in the development of novel and innovative drugs.” Other Democrats warn that longer exclusivity could boost healthcare costs.

Some experts question the need for revamping the FDA approval process. Ameet Sarpatwari, a research fellow at Brigham and Women's Hospital in Boston, said the FDA has already sped up its approvals, and now acts more quickly than European regulatory agencies.