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.
The campaign worked: The Food and Drug Administration last week approved the first artificial pancreas, from Medtronic, for patients over age 14 with type 1 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.
When will we STOP treating this Horrible and Robbing Disease and pool our Scientific Teams together and find a Cure. OH WAIT A MINUTE, I forgot this Industry is Money Driven. What then would become of ALL the RESIGNED TO A DIABETIC CAREER do then for employment. WAIT A MINUTE AGAIN, here comes another Cure Breakthrough and Just Around the Corner Cure Update, OOPS READY IN ANOTHER 5 YEARS. Watching my Daughter endure this Daily is HEARTBREAKING. Unending Funding and Continued False Hope. Shameful. Updated pump, this Device DOES NOT mimic a Pancreas.
STOP CALLING IT ARTIFICIAL PANCREAS! Anyone who has first hand on experience with managing Type 1 diabetes and some common sense would tell you that until somebody invents a faster (much faster) acting insulin and stable liq. glucagon this is just a glorified insulin pump. Just another expensive toy with great profit margins for Big Pharma that may or may not save you from some low blood sugar episodes if you don’t mind keeping it in unhealthy 6-12 threshold. What a joke.
You hit the nail right on the head Natallia! There is no way they can call this an Artificial Pancreas! I can not believe how any T1D can be excited about an algorithm controlling your insulin pump… The pump systems I have used for over 12 years work just fine. I’m waiting for an insulin that works faster, and a CGM that is more accurate and far less expensive.
That will be news worthy.
Thanks for saying this Natallia! I’ve had so many people ask me how happy I must be thinking Medtronic had “cured” diabetes. I’m a bit disappointed in the Globe for not doing a bit more of a fact check with an actual endocrinologist and just relying on the language in a press release. Putting artificial pancreas in parenthesis without explaining that this is a very tiny step for Type 1s does us all a disservice. I’m scared to think of how many folks will now shuffle their donation dollars elsewhere since Type 1s have a “cure” for their disease. (And I do realize that the above article is focused on funding, but it’s the second day in a row the Globe hasn’t fact-checked the Medtronic press release with an actual Type 1 specialist…or even bothered to find out that even though there were no DKA incidents in the 123 patients studied, that there were indeed quite a few highs.) Not a cure. Just a tiny step in the right direction when it comes to FDA approval of linking CGMs to pumps.
I have two 22 year old grand-daughters with type 1. One diagnosed at 8 and one at 15. I have prayed that someone would come to the forefront to help my grand daughters and millions of other children and adults make their lives easier and longer. THANK GOD SOMEONE HAS A SUBSTANTIAL START.
“He said donations never impact the group’s decisions.”
Anyone in a position of authority who receives or whose organization receives money or other benefits from donors who says this should be reminded that human beings simply don’t work that way, and all of us would do well to learn that, and structure systems and decisions in a way that acknowledges it.
In July of 2015, it was discovered that I had type 2 diabetes. By the end of the month, I was given a prescription for Metformin. I stated the ADA diet and followed it completely for several weeks but was unable to get my blood sugar below 140. With no results to how for my hard work, I panicked and called my doctor. His response? Deal with it. I began to feel that something wasn’t right and do my own research. Then I found Rachel’s blog http://curediabetespro.gq/ . I read it from cover to cover and I started the diet and by the next morning, my blood sugar was 100. Since then, I have a fasting reading between the mid 70s and 80s. My doctor was so surprised at the results that, the next week, he took me off the Metformin. I lost 30 pounds in the first month and lost more than 6 inches off my waist and I’m able to work out twice a day while still having lots of energy. The truth is we can get off the drugs and help myself by trying natural methods.
Glad you pulled it together with diet and exercise. Being a Type 1 diabetic nothing gets me more annoyed than a Type 2 diabetic complaining about their diabetes. Type 2 diabetics have a chance to reverse their diabetes that Type 1 diabetics do not have. Lucky for you this article serves no relevance unless you continued with a bad diet and no exercise then end up with Type 1. Type 2 is the slow boat for many lazy/uneducated people to Type 1. Good for you pulling it together but Type 1’s really have no use for your comment. Most Type 1’s don’t develop diabetes because of bad lifestyle choices. Your link only helps those with Type 2 Diabetes. This article about the artificial pancreas solution is for Type 1 diabetics not Type 2. Your comment seems to be simply for marketing purposes as it doesn’t relate to Type 1 diabetics.
Type1, you’re probably just replying to a paid spammer/promoter of that blog.
That’s great for you but you can’t compare a type 2 to a type 1 diabetic. There are no “natural methods” for a type 1. That’s why they are called insulin dependent. The artificial pancreas is a blessing for type 1 diabetics and their parents. You have no idea what it is like checking on your child during the night and every morning hoping you don’t walk into your worst nightmare. So please don’t compare the two.
Apples and Oranges. If I only had a nickel for every Type 2 who lectured my brittle Type 1 Diabetic son the dangers of eating too much sugar and losing weight (he is 17 and 110lbs). Educate yourself. Your disease, while it is serious and potentially deadly, is nothing compared to the day to day struggle of an insulin dependent diabetic who will die without a steady dose of insulin…..or perhaps die from an insulin imbalance.
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