Twelve years ago, a dotcom millionaire stood at a patient advocacy group’s board meeting and made an offer.
I’ll give you $1 million, he said. But only if you commit to getting an artificial pancreas on the market.
That challenge set JDRF, formerly known as the Juvenile Diabetes Research Foundation, on a costly, and risky, campaign to enlist academic researchers, global companies, members of Congress, and even federal regulators to embrace the concept of a device that could take over much of the process of regulating blood sugar in patients with diabetes.
JDRF’s long crusade mirrors a trend in the patient advocacy world: Such groups are increasingly moving beyond traditional activism to fund research at drug and device companies.
Those close ties with industry have sparked some criticism — mostly out of concern that advocacy groups won’t be able to freely fight for patients if their finances are tied to the fate of a corporate partner. But the expanding model may also help patients, if the advocacy groups succeed in pushing industry to get new treatments to market quicker.
In the case of the artificial pancreas, JDRF was taking a very big chance.
Academics had been trying for years to develop algorithms to power a fully automated system for regulating blood glucose, a task that again and again proved unfeasible. Device makers were squeamish about letting a computer control an insulin delivery system that could kill a patient if it malfunctioned at the wrong time. And even if somebody could build an artificial pancreas, no one knew what kind of tests and data the FDA would require to be convinced that it was safe and effective.
Still, JDRF plunged in. It has poured $100 million into supporting academic research and has spent roughly $16 million more to fund companies working on the technology. (Some of that money went to Medtronic, although JDRF didn’t directly invest in the newly approved device.) JDRF also lobbied the FDA to issue a crucial roadmap outlining what it would take for companies to win approval for an artificial pancreas.
“I think JDRF got us here a lot faster than we could have any other way,” said Dr. Francine Kaufman, chief medical officer for Medtronic’s diabetes business.
A persuasive vision
Founded in 1970 and based in New York City, JDRF has traditionally focused on finding a cure for type 1 diabetes. When the group got that $1 million offer in 2004, finding technological solutions to make the disease easier to manage wasn’t part of its mission, much less a top priority.
The donor who issued the challenge, Jeffrey Brewer, had gotten involved with JDRF soon after his son was diagnosed with the disease. He told STAT that he was “appalled” by how much of the burden of managing the disease fell on patients — and how dangerous it was for them to stray from the routine. (Too much insulin can send blood glucose to life-threateningly low levels.)
Brewer quickly concluded that a computerized system could help.
But back then, “nobody was working on this problem, outside of a very small number of committed people who just didn’t have any funding,” said Brewer, who later became JDRF’s president and CEO and now runs Bigfoot Biomedical, a startup working on an artificial pancreas that links to a patient’s smartphone.
JDRF wasn’t really desperate for Brewer’s $1 million. The group has long been a formidable fundraiser; last year it raised $200 million and spent $145 million on research and education.
Still, Brewer’s vision was persuasive. The foundation accepted his challenge.
“The artificial pancreas project has to us represented the nearest-term opportunity to make a great difference for people with this disease,” said Derek Rapp, JDRF’s current CEO and president.
Playing their cards
Over the next decade, JDRF made the artificial pancreas one of its top priorities — and to get it to patients, “they played cards at every step of the way,” said Margaret Anderson, executive director of FasterCures, a Washington D.C. group that advocates to speed up medical research.
JDRF initially focused on funding academic studies that might indirectly help device makers.
The research demonstrated that it would be possible to use a machine to partly automate disease management, developed algorithms to power the process, and showed that patients who use what would become a key part of the artificial pancreas system — continuous glucose monitors — fare better than their peers who don’t.
JDRF also urged the FDA to clearly lay out its expectations for companies working on an artificial pancreas. But the agency wasn’t moving quickly. An FDA official involved in last week’s approval said some FDA officials wanted to go slower to safeguard patient safety.
“Back then, the FDA was very, very difficult,” said Aaron Kowalski, who led the charge for JDRF and who has type 1 diabetes himself.
So JDRF pressed hundreds of senators and representatives to sign a letter on their behalf; the group even got some lawmakers to show up for a press conference.
JDRF also made a public push to cast type 1 diabetes as a dangerous disease in desperate need of better technological solutions. In 2011, the group took out emotional newspaper ads featuring the face of a little girl with type 1 diabetes and the ominous warning that 1 in 20 patients like her will die of low blood sugar.
“Three million kids, teens, and adults with type 1 diabetes are counting on the FDA to get it right,” the ad read.
To JDRF’s delight, in 2012 the FDA finally issued guidance to the industry that reflected the group’s recommendations. Now companies knew what they would be expected to do if they wanted to get an artificial pancreas approved — and a flurry of investments followed.
Soon a handful of device makers were taking the technology seriously — and it didn’t hurt that some of them were getting funding from JDRF. Leading the pack was Medtronic, which had been making small investments in the technology for years but now surged forward down the path that JDRF had cleared.
Nonprofit or venture capitalist?
In funding device makers, JDRF put its own spin on a model pioneered by the Cystic Fibrosis Foundation in the 1990s. That foundation’s high-stakes investment in experimental drugs paid off in a big way: The medicines came to market and the foundation was able to sell its ownership rights for $3.3 billion.
Other disease groups have followed: the Leukemia and Lymphoma Society, for example, has poured tens of millions into different companies over the past decade.
The rise of these partnerships alarms Dr. David Cornfield, a Stanford pediatric pulmonologist who’s been in close dialogue with the cystic fibrosis community.
“There’s a tension that develops between being able to advocate effectively [for patients] and still being mindful of the partnership that one has developed [with industry],” he said, “and I have a hard time believing that those two issues can be completely divorced from one another.”
JDRF doesn’t retain an ownership stake in technology it helps fund, but it does require companies to pledge to refund its research grants if the drug or device it’s backing makes it to market and becomes profitable.
JDRF did not invest directly in Medtronic’s newly approved artificial pancreas, so it won’t be getting any money back there. But in the future it could get a refund from related Medtronic projects, including an experimental glucose level sensor that could be integrated into a next-generation version of the artificial pancreas.
Kowalski said JDRF steers clear of conflict of interest by making “very, very clear that we will never support just one company” and serves patients by “redeploying” recouped investments back into more promising research.
But to Cornfield, the refund model is particularly fraught. “Usually when people are giving grants, there is not a quid pro quo in regard to deliverables,” he said.
JDRF has also accepted millions of dollars in donations from companies working on artificial pancreas systems, including more than $5 million from Medtronic since 2004. These corporate donations are often used for events and represent a small fraction of JDRF’s fundraising, most of which comes from individual donors, Kowalski said. He said donations never impact the group’s decisions.
The artificial pancreas approved last week isn’t fully automated; blood glucose levels still have to be manually adjusted before meals. But experts say it’s a genuine innovation that will make life easier for many diabetics because it can automatically monitor their glucose and deliver insulin as needed.
The device is expected to be available next spring.
Rapp said JDRF will push for insurance coverage of Medtronic’s new device and has urged the company to keep it affordable. (The company hasn’t set a price yet.)
For JDRF, all this means pivoting to a new concern — how to ensure patients have access to the artificial pancreas— after more than a decade of fighting to simply get it on the market. The moment arrived unexpectedly soon, far faster than most industry observers were anticipating.
When the hard-fought approval came last week, just 103 days after Medtronic submitted its application, “I almost couldn’t believe it,” Kowalski said.
Correction: A previous version of this story misstated the year that the FDA came out with guidance that JDRF fully supported.