BIO 2015 Dispatches: Can expanding FDA's 'breakthrough' program be an answer to high drug costs?
FDA's "breakthrough therapy" program should be broadened in a way that helps increase market competition and drive down costs for first-in-class medicines, some stakeholders suggested at the Biotechnology Industry Organization's annual convention in Philadelphia June 16.
During a panel discussion entitled "Paying for the 21st Century Cure," Dan Durham, acting CEO of America's Health Insurance Plans, and American Enterprise Institute Resident Fellow Dr Scott Gottlieb made the argument for moving beyond the current product-based approach to granting breakthrough status and, instead, extending the expedited pathway's benefits to subsequent treatments that target the same indication.
"When you have that breakthrough therapy that gets an expedited approval, you have several other products in the pipeline," Mr Durham said. Allowing these subsequent products to enjoy the same advantages as those on the expedited regulatory pathway benefits the consumer by bringing more competitors into the marketplace more quickly, he said.
Mr Durham also suggested a safety benefit that would come with more competition sooner for breakthrough therapies.
"If there is a problem with the first drug to market, you then have a second and third that can go to those patients as well," he said.
Looking to 'Cures' bill to make the competition argument
The breakthrough program was created through the FDA Safety and Innovation Act to expedite the development and review of drugs that treat a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.
The program has proven enormously popular with the biopharma industry, far more so than was ever anticipated. Through the end of May, FDA had received 308 requests for designation, 90 of which were granted.
The health insurance industry has been far less enthusiastic about breakthrough, pointing not only to the treatments' high costs but also to the more limited clinical dataset upon which they are approved.
AHIP's focus is clearly on the cost-savings that result from greater competition.
Mr Durham pointed to what he called the "poster child" for high-priced breakthrough treatments: Gilead Sciences hepatitis C drug Sovaldi (sofosbuvir), which entered the market with a price tag of $1,000 a day.
The market entry a year later of another breakthrough hep C treatment, AbbVie Viekira Pak (paritaprevir/ritonavir/ombitasvir/dasabuvir), gave health plans leverage to negotiate price in the category, Durham noted.
Mr Durham said AHIP has advocated for the extension of breakthrough to later-in-class drugs in the context of the 21st Century Cures legislation.
Such a provision has not been included in the measure now making its way through the House. However, similar legislation is under development in the Senate, and Mr Durham said AHIP officials are continuing to talk to lawmakers about the importance of competition in the marketplace.
Helping to ensure later-in-class agents reach market
The idea drew strong support from Dr Gottlieb, a former FDA official, who said that taking an indication-based approach to awarding the designation would help ensure that later-in-class products actually reach market.
"What we've seen I think over the last number of years, probably since [Medicare] Part D, is the drug makers really getting away from being second, third, fourth in class," Dr Gottlieb said. "Even in some of these higher reimbursed classes, if a drug maker thinks that they're going to be third to market, sometimes they will close down the program … and that's not a good thing."
FDA Office of New Drugs Director Dr John Jenkins has also lamented the decline in so-called "me too" drugs in primary care settings.
"It would be a very simple change," Dr Gottlieb said. "Rather than giving breakthrough designation on the basis of a specific molecule, you'd grant the designation on the basis of a therapeutic indication, just like we do with orphan drugs."
He noted that already some products that are second or third in class for a condition with high unmet need are designated as breakthroughs or receiving benefits under FDA's other expedited pathways.
Any effort to expand the program's breadth seems likely to meet with strong objections from FDA as well as some innovator companies and patient advocacy groups, who can be expected to argue that the program's benefits will be diluted if they are opened up to a much broader group of drugs than currently qualify under the existing designation process.
FDA, which has cited the resource demands of having to respond to so many designation requests that are ultimately denied, likely would make the case that such an expansion demands new and dedicated funding.
Dr Gottlieb said he believed the additional resources that FDA would need to implement a breakthrough expansion would likely be small relative to the projected savings resulting from earlier competition and lower prices.
Panel moderator Dan Mendelson, Avalere Health's CEO and founder, said he supported the idea but suggested such an expansion could undercut existing incentives to expedite development of novel, innovative therapies.
"The idea that if you were able to do that, you were able to bring in more competition, it strikes as a kind of a free market-oriented approach," he said.
"I think what's on the flip side of that is if you want to set up a system that really incents getting that first drug to market, are you starting to take away the incentive for that innovator, the Gilead, to kind of push things through really rapidly, and get that drug out there more rapidly," Mr Mendelson said.
Sue Sutter is with The Pink Sheet. Our daily BIO 2015 Dispatches draw on the combined forces of The Pink Sheet and Scrip Intelligence staff, reporting from the Philadelphia meeting.
