Industry, advocate pressure helps revive key 21st Century Cures exclusivity incentives
The sweeping 21st Century Cures FDA overhaul bill is steadily making its way through Congress. In fact, the pace of substantive changes to the bill in the House Energy and Commerce Committee, and the surprising confluence of overarching interests between the biopharma industry, patient advocates, and lawmakers on both sides of the aisle, is a sign that the legislation may become one of the 114th Congress' most consequential bipartisan achievements—one that would leave a lasting mark on the life sciences sector and American healthcare at large.
Yet another new discussion draft of the bill was released on Wednesday, prior to an Energy and Commerce subcommittee markup session scheduled for Thursday (BioPharma Dive will have you covered on what happens during that markup later this week). Earlier this month, the committee released a different discussion draft that nixed major drug marketing exclusivity provisions for orphan drugs, dormant therapies, and new chemical entities that were present in the original January draft (you can read our writeups of the first two drafts and the differences between them here and here).
Drug exclusivity makes a comeback
But now, the exclusivity measures for orphan drugs are back (although the others are still missing at this point). And pharma can thank intense lobbying pressure from stakeholders in the industry, as well as patient groups.
The extent to which interest groups have coalesced around the overall 21st Century Cures bill and the exclusivity provisions specifically was on full display during a PhRMA-sponsored event hosted by the The Hill on Tuesday. Sens. Richard Burr (R-NC) and Bill Cassidy (R-LA) spoke on the imperatives of driving U.S. pharma innovation by offering proper incentives during the session, dubbed NextGenCures.
Too often, patients have an option between "nothing and nothing," said Burr, and Congress would have to work at cutting away at a "thicket of bureaucratic red tape" and properly allocate resources in order to reverse that trend, including by rethinking the approval process and tinkering with when patients can use experimental drugs.
Later, a plenary session that featured Jeff Allen, executive director of Friends of Cancer Research; Margaret Anderson, executive director of FasterCures; Marc Boutin, CEO of the National Health Council; and Theresa Mullin, director of the Office of Strategic Programs at the FDA's Center for Drug Evaluation and Research, focused in part on the current status of 21st Century Cures and what could make the bill stronger. Several of the panel members weren't exactly coy about nudging lawmakers towards rethinking exclusivity provisions in order to spur drug development.
"Looking at incentives [is going to be crucial]," said Boutin. "That’s an issue that has plagued our system for decades. We need to drive the development of high-value small molecules. What is it we want to incentivize? Not all medicines have same value, and the incentives can change that dynamic."
Boutin didn't utter the phrase "exclusivity" specifically. But his meaning was quite clear: Give drug makers a reason to spend time and money on creating some of the hardest-to-develop therapies.
But are the reinstated measures good enough?
This isn't to say that every advocacy group or pharma lobbying force backs exclusivity. In fact, organizations like Public Citizen and the Generic Pharmaceutical Association (GPhA), and many Congressional Democrats, are staunchly opposed to the measures, arguing that they're giveaways to massive branded pharmaceutical corporations that will hike prices for patients and payers alike. Commerce Committee member Rep. Frank Pallone (D-NJ) in particular has been reticent to offer up excessive exclusivity for fears of "undermining" the Hatch-Waxman Act.
So what happened? "I think that what you're seeing here are the easier exclusivity provisions," said Craig Burton, director at Avalere Health, in an interview. "They have not included the Dormant Therapies Act that was in the January draft, and they have not included the language that was also in the January draft around what it called 'new therapeutic entities.'"
There's a chance that other exclusivity measures could eventually make it back into the bill (patient advocates have been pushing for the reinstatement of the Dormant Therapies Act in particular). In the meantime, although most of the industry is likely to applaud any sort of exclusivity, there's still a possibility it won't be enough to whet biopharma's appetite.
"I guess the ultimate question is, is this significant enough of an incentive to support investment in rare diseases?" asked Burton. "What's been included is, an additional six months of exclusivity for a label indication for a rare disease. But how big of a motivator, how big of an incentive, is that to manufacturers? And my guess is that's going to differ from manufacturer to manufacturer.
"The time and effort you're going to put into a difficult space, is that six months valuable enough? Is the juice worth the squeeze?"
Congress, with a dose of compassion
The power of the stakeholder is integral to a democracy. But sometimes, even the most organized interests can't persuade Congress to pursue difficult reforms that require tough choices and, more importantly, that cost money.
That's what has made the rapid rise of the 21st Century Cures bill so striking. "Every bill we draft, every vote we cast, we’re moving forward on the path to cures," said Energy and Commerce Committee chairman Rep. Fred Upton (R-MI) of the newest draft. "This is a major milestone for hope and health."
At the pace it's currently proceeding, there's a decent chance that a bill will clear the House before the end of summer. And Burr and Cassidy told NextGenCures attendees that it would be reasonable to assume the Senate could take up the legislation by the fall, meaning a final bill (if it clears all its various legislative hurdles) could head to President Obama's desk by the end of the year.
This is highly unusual for legislation of this magnitude. So what's going on?
"First of all, there is a human face to it," said Burton. "After a number of years where the political focus has been coverage... the pendulum has certainly swung back to also looking at the discretionary side of HHS to see what can be done to get cures faster. The patient face, the patient voice in this is critical."
In fact, as Avalere noted in an analysis of public comments for the legislation, "far and away the most comments have come from the patient community." That may have helped lawmakers from both sides of the aisle rise past partisan politics in order to get something substantial accomplished for patients lacking life-saving therapies.
"Both sides of the aisle have said this is a priority for us," said Burton. "And they've said it and they've carried it out from the member level on down. The members are making time for this, they're attending hearings, they're going to roundtables in their districts, they're going to roundtables in other members' districts to join them. And that—making it a priority, and that attending roundtables together—has built up I think a pretty good reservoir of trust, that folks are going to work seriously and in good faith together to get something done."