The risky bet behind the first ‘artificial pancreas’ for diabetes patients

does

Twelve years dotcom millionaire stood at board meeting of an advocacy group patient and made an offer.

I’ll give you $ 1 million, he said. But only if they commit to get an artificial pancreas on the market.

This set challenge JDRF formerly known as the Research Foundation Juvenile Diabetes, a costly and risky campaign to recruit academic researchers, global companies, members of Congress, and even federal regulators to embrace the concept of a device that could take over most of the process of regulating blood sugar in patients with diabetes.

article continues after the announcement

The campaign worked :. The Food and Drug Administration last week adopted the first artificial pancreas, Medtronic, for patients older than 14 years Type 1 diabetes

long crusade JDRF reflects a trend in the world of patient advocacy: These groups are increasingly moving beyond traditional activism to fund research on drug and device companies

.

Those close ties with industry have generated some criticism – mostly because of the concern that advocacy groups will not be able to freely fight for patients if their finances are tied to the fate of a corporate partner. However, the expansion model can also help patients, advocacy groups if successful in driving the industry to get new treatments to market faster.

In the case of artificial pancreas, JDRF was taking a big chance.

Academic had been trying for years to develop algorithms for feeding a fully automated system for the regulation of blood glucose, a task over and over again proved impractical. device manufacturers were squeamish about letting a computer control system of insulin that could kill a patient if it does not work well at the wrong time. And even if someone could build an artificial pancreas, nobody knew what kind of evidence and data from the FDA require to be convinced it was safe and effective.

Yet the sunken JDRF. Has poured $ 100 million in support for academic research and has spent roughly $ 16 million to fund companies working in technology. (Some of that money went to Medtronic, although JDRF not invest directly in the newly approved device.) JDRF also pressured the FDA to issue a sheet basic route outline what would be necessary for companies to win approval of an artificial pancreas .

“I JDRF we have here a lot faster than you might have otherwise,” said Dr. Francine Kaufman, medical director of the Diabetes business of Medtronic.

A compelling vision

founded in 1970 and headquartered in New York City, JDRF has traditionally focused on finding a cure for type 1 diabetes When the group has that offer $ 1 million in 2004, search technology to make the disease easier to handle that was not part of his mission, much less a priority solutions.

The donor who issued the challenge, Jeffrey Brewer, had been involved with JDRF soon after his son was diagnosed with the disease. STAT told he was “appalled” by the amount of the burden of disease management fell into patients – and how dangerous it was for them to deviate from the routine. (Excess insulin can send blood sugar to low levels of life-threatening.)

Brewer quickly concluded that a computer system could help.

But back then, “no one was working on this problem, outside of a very small committed people who simply had no funding number,” Brewer, who later became president and CEO of JDRF he said and now runs Bigfoot Biomedical, a startup working on an artificial pancreas that connects with the smartphone of a patient.

JDRF was not really desperate Brewer of $ 1 million. The group has long been a formidable collection funds; last year rose $ 200 million and spent $ 145 million on research and education.

However, Brewer’s vision was compelling. The foundation accepted his challenge.

“The artificial pancreas project that represented us the nearest opportunity deadline to make a big difference for people with this disease,” said Derek Rapp, CEO and president of JDRF.

Playing your cards

In the following decade, JDRF made the artificial pancreas one of its main priorities – and to get patients “, played cards every step of the way,” said Margaret Anderson, executive director of FasterCures, based in Washington DC advocacy group to accelerate medical research.

JDRF initially focused on the funding of academic studies that could indirectly help device manufacturers.

The research showed that it would be possible to use a machine to partially automate the management, disease, developed algorithms to feed the process, and showed that patients who used what would become a key part of the artificial pancreas system – monitors continuous glucose – better results than their peers who do not.

JDRF also urged the FDA to clearly state their expectations for companies working on an artificial pancreas. But the agency did not move quickly. An FDA official involved in the approval last week said some FDA officials wanted to go slower to safeguard patient safety.

“At that time, the FDA was very, very difficult,” said Aaron Kowalski, who led the charge for the JDRF and has type 1 diabetes himself.

So JDRF down hundreds of Senators and Representatives to sign a letter on his behalf; the group even got some lawmakers introduced a press conference.

JDRF also made a public effort to issue type 1 diabetes is a dangerous disease an urgent need for better technological solutions. In 2011, the group carried out advertising in emotional newspapers featuring the face of a child with type 1 diabetes and the ominous warning that 1 in 20 patients as she will die from low blood sugar in the blood.

“Three million children, adolescents and adults with type 1 diabetes are counting on the FDA to do well,” reads the ad.

To the delight of JDRF, in 2012, the FDA finally issued a guidance for industry that reflects the group’s recommendations. Now companies know what is expected of them if they wanted to get approved artificial pancreas – and a flood of investment continued

.

Soon, a handful of device manufacturers were taking technology seriously – and not hurt that some of them were receiving funding from the JDRF. At the head was Medtronic who had been making small investments in technology for years, but now surged forward along the path that JDRF had cleared.

non-profit venture capitalist?

device manufacturers in financing, JDRF put your own spin on a model pioneered by the Cystic Fibrosis Foundation in the 1990s of high-risk investment that the foundation on experimental drugs paid off big: the drugs hit the market and the foundation was able to sell their property rights for $ 3.3 billion

.

Other groups of diseases have followed: the Leukemia and Lymphoma Society, for example, has invested tens of millions in different companies over the last decade

.

Increasing these alarms associations Dr. David Cornfield, a pediatric pulmonologist at Stanford who has been in close dialogue with the community of cystic fibrosis.

“There is a tension that develops between being able to effectively promote [for patients] and even being aware of the association that has developed [with industry],” he said, “and I have difficulty believing that these two issues can be fully separated from each other. ”

JDRF does not retain a stake in the background technology that helps, but it does require companies to commit to repay their research grants if the drug or device that is receding ago to market and becomes profitable.

JDRF not invest directly in the artificial pancreas Medtronic recently approved, so you will not get the money back there. But in the future we could get a refund of Medtronic related projects, including an experimental sensor glucose level that could be integrated into a next-generation version of the artificial pancreas.

Kowalski said JDRF is kept away from conflict of interest in making “very, very clear that we will never support a single company” and serves patients from “redistribution” investments recovered again in the most promising research.

However, for the corn field, the reimbursement model is particularly tense. “Normally when people are giving subsidies, there is no quid pro quo with regard to benefits,” he said.

JDRF has also accepted million in donations from firms operating systems artificial pancreas, including more than $ 5 million Medtronic since 2004. These corporate donations are often used for events and represent a small fraction of fundraising JDRF, most of which comes from individual donors, Kowalski said. He said that donations do not affect the group’s decisions.

The artificial pancreas approved last week is not fully automated; glucose levels in the blood have yet to be adjusted manually before meals. But experts say it is a true innovation that will make life easier for many diabetics because it can automatically control the level of glucose and deliver insulin as needed.

The device is expected to be available next spring.

Rapp said JDRF press for insurance coverage of the new Medtronic device and has urged the company to keep it affordable. (The company has not set a price yet.)

For JDRF, all this means pivoting to a new concern – how to ensure that patients have access to artificial pancreas- after more than a decade of struggle just to get on the market. The time came unexpectedly soon much faster than most industry watchers were expecting.

When the hard-fought approval came last week, just 103 days after Medtronic filed its application, “I almost could not believe,” Kowalski said.

Correction :. An earlier version of this story incorrectly in the year when the FDA came out with a guide JDRF fully supports

Add a Comment

==[Click 2x to Close X]==
Most Popular Today!