The News:

Stanford University and the FDA announced a collaboration on January 12 that will lead to more efficient and effective methods of establishing the safety and efficacy of medications and devices.  This will, presumably, lead to the acceleration of devices–such as the artificial pancreas–to market.

What This Means for the T1D Community:

Whether you plan to drive hard to get yourself a first-generation AP or whether you think you’ll be content to continue injecting insulin until the technology has developed further, the notion of an artificial pancreas should be exciting.

Most importantly, this should be meaningful because a closed-loop system in which a continuous glucose monitor tells an insulin pump how to dose, means better–and far easier–management of T1D.

For this parent of a kid with type 1, the announcement is particularly meaningful.  I have been lucky enough to get to know Dr. Bruce Buckingham, who happens to be at Stanford, and who happens to be one of the leading developers of the AP and who has been instrumental in the acceleration of the AP through the FDA, and I believe that Dr. Buckingham is more than capable of getting this product working well and into our hands.

This is yet another sign of cooperation between the FDA and the academic centers where important device innovations are being made. It’s also another example of the impact JDRF’s grassroots efforts have had in terms of making our voices heard by the government.  This is good news, indeed!

If You Want to Read More:

Story on Stanford’s website >

Washington, D.C. — The JDRF today said it is encouraged that the Food and Drug Administration (FDA) has issued a draft guidance which appears to lay a foundation for accelerating the development and availability of artificial pancreas technology (AP) that will improve the lives of millions of Americans living with type 1 diabetes (T1D).

“Our initial review of the draft AP guidance indicates that the FDA has been responsive and listened to the recommendations of leading clinicians and researchers in the type 1 diabetes field,” said Jeffrey Brewer, President and CEO of JDRF. “It also appears that the draft guidance lays out a rapid timetable to move from inpatient trials to outpatient trials to prove the AP’s safety and efficacy, and if that turns out to be the case it is good news.”

Artificial pancreas technologies have the potential to be the most revolutionary advance in treating T1D since the discovery of insulin. They will allow the millions of Americans with T1D to maintain tighter control of blood glucose levels, thus significantly reducing the risk of both high blood sugar (hyperglycemia), which can cause long-term complications including heart disease, blindness and stroke, and low blood sugar (hypoglycemia), which can be life-threatening. It would also free them from much of the daily burden of managing the disease.

In 2006, the FDA added artificial pancreas technology to its Critical Path list, making a commitment to advance these systems. The draft guidance is the first step to getting the technology to those in dire need.

“To be sure, we have a ways to go before AP technologies are available to patients but this draft guidance suggests that Commissioner Hamburg and the FDA are committed to fostering innovation and being a global leader in bringing life-saving technologies for T1D patients to the U.S market,” said Brewer. “In the next few weeks, we will review the draft guidance in detail and provide detailed comments to the FDA so that the final guidance unmistakably lays out the pathway to put AP technologies in patient’s hands as soon as possible. In the end, that is what this is all about.”

About the Artificial Pancreas

The artificial pancreas (AP) is an external device which people with T1D could use to do what their bodies cannot-control both high and low blood sugar around the clock. It works by combining a continuous glucose monitor (CGM) and an insulin pump with sophisticated computer software to provide automatically the right amount of insulin at the right time.

One of JDRF’s top priorities is the development of the AP. JDRF-funded studies have shown greatly improved clinical outcomes from early trials of prototype AP systems. FDA’s guidance on artificial pancreas systems will allow for these systems to be further developed, tested in outpatient pivotal trials, and eventually approved for people with T1D.

An overwhelming amount of support from researchers, clinicians, policymakers, and patients called on the FDA to advance the development of an AP. Over 100,000 people in the diabetes community signed JDRF’s petition which urged the FDA to adopt clear guidance, and leading clinical organizations specializing in diabetes care urged the FDA to ensure that the development of an artificial pancreas is not delayed by unnecessary regulatory roadblocks. Furthermore, Congress demonstrated vast support for a reasonable and timely guidance from FDA, with 252 Representatives and 62 Senators having urged the FDA to act in the best interest of type 1 diabetes patients.

About JDRF

JDRF is the leading global organization focused on type 1 diabetes (T1D) research. Driven by passionate, grassroots volunteers connected to children, adolescents, and adults with this disease, JDRF is now the largest charitable supporter of T1D research. The goal of JDRF research is to improve the lives of every person affected by T1D by accelerating progress on the most promising opportunities for curing, better treating, and preventing T1D. JDRF collaborates with a wide spectrum of partners who share this goal. Since its founding in 1970, JDRF has awarded more than $1.6 billion to diabetes research. Past JDRF efforts have helped to significantly advance the care of people with this disease, and have expanded the critical scientific understanding of T1D. JDRF will not rest until T1D is fully conquered. More than 80 percent of JDRF’s expenditures directly support research and research-related education.

For more information, please visit www.jdrf.org.

By Jeffrey Brewer, JDRF President & CEO

I’m pleased to share the news that on Thursday, December 1, the U.S. Food and Drug Administration (FDA) issued artificial pancreas (AP) draft guidance. While the draft guidance is still under review, our initial analysis suggests that it affirmatively addresses many of JDRF’s key issues, and, in so doing, it appears to lay a foundation for accelerating the development and availability of AP technologies that will improve the lives of millions of Americans living with type 1 diabetes (T1D).

But there is more to the story. The fact is that this encouraging outcome probably would not have happened without the contributions of JDRF, our community, and our allies. In fact, it is fair to say that without JDRF, there might not have been any guidance at all, or at least not in 2011. And that is a good place to start the story.

By 2010, it was clear that JDRF-funded research in academic, hospital settings showed that prototype AP systems could greatly improve glucose control, and with encouragement from JDRF, companies had committed to developing commercial products for people with T1D. But there was no defined regulatory pathway for AP outpatient studies or product review, nor were there any immediate plans for the FDA to develop these clinical guidelines, which threatened to greatly slow the research and development process. So JDRF decided to play a leadership role to short-circuit potential bureaucratic delays, and to ensure that the FDA had the most current, state-of-the-art scientific thinking on the AP.

In July 2010, JDRF convened a panel of leading clinicians and researchers to make recommendations to the FDA about the key clinical questions for AP studies, which were presented at a public meeting that November. In early 2011, JDRF incorporated those recommendations into a guidance document that we proposed to the FDA. At the same time, we intensified our efforts by making issuance of AP guidelines a key “ask” of the advocates at our annual Government Day in March. Their efforts, and the efforts of countless JDRF volunteers across the country, resulted in more than 250 House members and more than 60 U.S. senators sending letters to the FDA calling on the agency to produce timely AP guidance that takes into account the recommendations of leading experts. In these highly partisan times, securing such broad bipartisan support for this goal was truly remarkable.

We continued our efforts at Children’s Congress in June, where, at a Senate hearing on June 22, the FDA committed to releasing AP draft guidance by December 1. It soon became apparent that simply seeing the FDA issue guidance would not be enough. Indeed, when the FDA released unreasonable, highly problematic low-glucose suspend (LGS) system guidance—guidance covering a system that was already available and in use in more than 40 countries around the world—JDRF’s team recognized the very real risk that the AP guidance might be similarly flawed, setting back the development of AP systems by years.

Once again, we mobilized and increased our efforts. JDRF chancellor Pam Sagan testified at a House hearing on medical device regulation on July 20, when she highlighted the need for timely access to innovative, life-saving technologies to help better manage diabetes, citing LGS systems as a key example. In August, we launched our Promise to Remember Me Campaign, in which our volunteers held 250 meetings with members of Congress in communities across the country by the end of November to enlist them to encourage FDA action.

After Labor Day, we began a more intense campaign to encourage the FDA to not only issue AP guidelines by December 1, but to issue guidelines that, unlike the LGS ones, would not throw up unnecessary obstacles to the development of these systems. Of course, the centerpiece of these efforts was substantive, solutions-oriented engagement with the FDA’s scientific and device center teams. To their credit, members of the FDA staff not only were open to dialogue with JDRF’s AP team and the clinical community, they genuinely listened to our concerns.

Beyond this interaction with the FDA, some of our activities were visible, such as the letter from leading diabetes clinical organizations (AACE, ADA, AADE, Endocrine Society), the powerful full-page newspaper ads in The New York Times and The Washington Post, and the Capitol Hill press conference; and others were less visible, such as our ongoing outreach to key Congressional supporters, who directly communicated to the FDA how important it was to get the AP guidance right. Then there was our online petition, which so many of you signed, a petition that was eventually delivered to the FDA with more than 100,000 signatures gathered in five weeks’ time.

Which brings us back to Thursday’s announcement by the FDA.

The AP draft guidance clearly indicates that the FDA has worked hard to produce reasonable guidelines for artificial pancreas systems. The guidance appears to provide a rapid timetable to move from inpatient trials to outpatient trials, and its flexible requirements allow for clinical development in a manner that will advance AP trials while ensuring their safety and effectiveness. For this, the FDA deserves great credit. Furthermore, this draft guidance suggests that Commissioner Margaret Hamburg and the FDA are committed to fostering innovation and being a global leader in bringing life-saving technologies for people with T1D to the U.S. market.

We are still studying the details of this highly technical, 60+-page draft guidance. We know there will be issues and further considerations that we will want to address; we know there will be more dialogue with the FDA before final guidance is completed. But we also know that without the involvement of the clinical community; without the contributions of so many of our volunteers who testified, wrote op-eds and letters to the editor, signed petitions, and more; without the commitment of our Hill allies such as Diabetes Caucus co-chairs Susan Collins, Jeanne Shaheen, Diana DeGette, and Ed Whitfield; without the outpouring of signers to the petition; without our successful efforts to raise the profile of this issue in the FDA, the Congress, the White House, and the T1D community; without a regulatory agency with committed professionals pushing forward an innovative agenda; and without the extraordinary commitment and cooperation of so many JDRF staffers and departments, we would not be as close as we are to seeing this revolutionary technology reach the hands of people with T1D.

Thank you for your continued dedication.

Last week JDRF ran a large advertisement in The New York Times and in The Washington Post.  The ad shows a photograph a young girl.  Below her are the words: “Piper has type 1 diabetes.  One in twenty people like Piper will die from low blood sugar.”

Understandably, the T1D community was shaken.

Predictably, people who live with T1D called the ad “painful,” “sensational,” “unnecessary.”

The ad is painful.

Low blood sugar is the immediate danger with T1D.  For most of us, it’s the scariest part of the disease.

Low blood sugar is why people have worked so hard to train dogs who can sense hypoglycemia.

It’s why all of the jacket pockets and glove boxes and purses and backpacks in the Chisholm family have smarties and glucotabs and starbursts and why, when I glance down to check these items, I dread the moment when we need one of those things and it’s not available immediately.

The very real threat of hypoglycemia is why CGM’s are made to alarm and why our doctors suggest 2 a.m. blood glucose checks and it’s why I was so very relieved when a participant in one of last week’s many email exchanges about the ad made the point that 1 in 20 people dying from low blood sugar also means that 95% of people with this disease won’t.

Still, the advertisement is disconcerting.

It’s disconcerting and unsettling, but not sensational.

The statistic JDRF chose was the product of many studies by many researchers:  P.E. Cryer, T. Deckert, and W. M. G. Turnbridge as well as the Diabetes Control and Complications Trial, the very large, ground-breaking study run from 1983-1993.  These studies conclude that the number of deaths from hypoglycemia are between 2% to 10%.

Therefore, 1 in 20, or 5%, or the statistic JDRF chose to publicize, is actually a conservative estimate.

The ad is unsettling and frightening but it is not unnecessary.

As soon as the ad came out, my friend Mark wrote, in response to the alarm, about the four times his son has suffered severe hypoglycemia and how very scary each of those incidents was.

My friend Shelly, mother of a three and a five year old, wrote about almost having died of hypoglycemia but for a “good Samaritan” who recognized her symptoms and came to her aid.  Shelly, when her husband is traveling, goes to bed with her blood glucose purposely high because of the “gruesome” specter of the severe hypoglycemic nighttime “event” that might leave her children “fending for themselves for days until her husband returns.”

Aaron Kowalski, Ph.D. and Research Director for JDRF, said:

“JDRF did not publish this ad to raise money. We ran this ad to tell the FDA that lives can be improved and even saved. We ran it because we want the FDA to understand that tools do exist to do this! JDRF-funded research has shown that predictive algorithms can minimize hypo by up to 80%. We can prevent many of these deaths. It is unacceptable to JDRF and it should be unacceptable to you that the United States is pretty much the only country in the world that hasn’t approved the Medtronic Veo pump that suspends insulin delivery when a person is low and non-responsive. This ad says to the FDA that they must get this guidance right.”

One in twenty is terrible.

But JDRF ran the advertisement with the express purpose of getting the FDA to approve technology—like the Medtronic VEO—that could LOWER THAT NUMBER.

JDRF and the T1D community need to create a sense of urgency.

We need even more of what the advertisement created:  a public outcry.

We need to recognize facts in ways that are not overblown or exaggerated but that will spur people to action.

We need to sign the petition urging the FDA to accelerate its review of the artificial pancreas (do so HERE! It takes two seconds!).

We need to educate the public and advocate for change and reimbursement and better treatments.

We need to fund research for new drugs and for the safer, more accurate, more impactful devices that will better treat this disease.

We need the regulatory agencies in this country to allow people to use them.

JDRF Urges Changes to FDA Policy to Enable Patients to Have Access to Lifesaving Diabetes Technology Sooner

Washington, D.C., September 21, 2011 – Yesterday, JDRF submitted comments to the Food and Drug Administration (FDA) on its draft guidance for the testing and approval of a Low Glucose Suspend System (LGS). JDRF commended FDA’s efforts to provide guidance in this area, but strongly warned that the guidance is unreasonable and will further delay the availability of the lifesaving technology to patients who need it. An LGS system is a version of an insulin pump which suspends insulin delivery when a monitor indicates a person with diabetes has or is projected to have dangerously low glucose levels. These systems are the first step toward an artificial pancreas, a device that could transform the lives of individuals with type 1 diabetes.

“This lifesaving technology is already available in over 40 nations around the world, and unfortunately this proposed guidance by the FDA will only further delay access to this system in the United States,” said Jeffrey Brewer, President and CEO of JDRF. “We urge the FDA to resolve three core issues in the guidance to enable people with diabetes to have access to this lifesaving technology as soon as possible.”

JDRF’s three core issues are as follows:

• The proposed clinical study pathway is excessively burdensome, and would further delay patients’ access to the technology. Without changes, this guidance will require multiple clinical trials (inpatient and outpatient) involving a large number of subjects in order to show statistically significant differences in preventing hypoglycemia. This would be an excessive hurdle in order to make available to patients a simple but important feature which shuts off insulin when someone has or is near severe low blood sugar (hypoglycemia). Instead, the LGS systems should be approved based on data showing safety and equivalent glycemic control. Clinical effectiveness data among larger populations could be collected in post-market studies.
• There is a lack of clarity in the use of continuous glucose monitors (CGM) in LGS studies. JDRF is pleased the guidance allows the use of CGM data in evaluating the safety and effectiveness of LGS systems. The use of any other outcomes would be considered impractical by the diabetes research community. FDA must express a commitment to the use of CGM data, rather than indicating it may change its standard later after studies have begun.
• The proposed guidance requires that substitution of substantially equivalent components in the LGS system would be allowed only if additional separate clinical studies of the system for each component variation are conducted. This requirement would severely limit choice for patients and discourage the development of technologies serving a critical public health need. FDA needs to adopt more efficient means to allow sponsors of clinical investigations and holders of approved premarket approval (“PMA”) applications to utilize multiple versions of components or make modifications to their LGS and artificial pancreas systems.

“Millions of people are in desperate need of the LGS system to help manage this disease and prevent life-threatening complications. Nighttime is a particularly dangerous time for a person with diabetes, because an individual’s blood sugar level can drop while they are sleeping, unaware that their levels are too low and unable to do anything about it, leading to seizures, coma, or death,” Brewer stated.

Low glucose suspend systems were a focus of a July 20th hearing in the U.S. House of Representatives Energy and Commerce Committee, which included testimony from a Bay Area mother of a young woman with type 1 diabetes who has been hospitalized many times from dangerous low blood sugars. A copy of that testimony can be found here.

This OpEd is published in the San Francisco Chronicle. Read Pam’s entire OpEd here. Pam also testified before Congress on this topic. Read a transcript of her testimony here.

For more than 20 years, my daughter Piper has lived with the constant, frightening, deceptive and malicious disease called type 1 diabetes. Piper has always been prone to the kind of hypoglycemic – low blood sugar – life-threatening attacks that come on hard, fast and without warning. She almost drowned as a youngster after becoming unconscious from low blood sugar. In college, she went into hypoglycemia while she slept and didn’t wake up in the morning. Fortunately, she was discovered and emergency care saved her life.

Unfortunately, the Food and Drug Administration has been dragging its feet on technologies that could revolutionize diabetes care and make these kinds of episodes a thing of the past. Key trials are on hold and it looks to be years more before these proven, life-saving technologies are available for patients in the United States. Meanwhile, kids are dying.

Every hour of every day, individuals with type 1 diabetes have to balance insulin, food and activity to try to prevent low and high blood sugars, and the devastating and costly complications: seizures, comas, kidney failure, heart disease, blindness and amputations. The human cost is incalculable; the economic cost isn’t: Diabetes costs our nation more than $174 billion a year and $1 in $3 of Medicare spending goes to care for people with diabetes.

Read more: http://www.sfgate.com/cgi-bin/article.cgi?f=/c/a/2011/07/26/ED7H1KEPU9.DTL#ixzz1TKfWkQhV

Pam Sagan is Vice President of Government Relations for JDRF Bay Area’s Executive Board.

This week, we asked you to call your Members of Congress and ask them to sign a letter to FDA Commission Hamburg concerning the artificial pancreas technology. The House letter closed on Wednesday, and we are happy to report that, in addition to both California Senators, 240 Representatives have signed on!

That number is well over a majority for that chamber, and we could not have reached that goal without the actions of advocates like you!

Seeing as it is National Volunteer Week, we are especially grateful for your efforts.

Original message:

Contact Congress to Support the Artificial Pancreas

We need you to email your Members of Congress today.

The artificial pancreas has demonstrated remarkable results in the hospital setting, and now trials need to be conducted in the outpatient or “real world” setting.  However, the FDA needs to provide clear and reasonable guidance for outpatient trials to begin.

It is important that you email your Members of Congress to encourage them to sign a letter to FDA Commissioner Hamburg concerning the need for clear and reasonable guidance on the artificial pancreas.

CLICK HERE or on the link below to EMAIL your Member of Congress about this issue NOW.

All we need is your name and signature and JDRF has taken care of the rest.  Take a look >

Experts Present at FDA-NIH Workshop

WASHINGTON, D.C., Nov. 10, 2010 — Diabetes experts at a meeting convened by the U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH) took the next step in advancing efforts toward the development of an artificial pancreas: putting forth clinical recommendations to ensure the safe and effective testing of artificial pancreas technology in real-life situations. We are pleased at today’s meeting there was a strong consensus among leading clinicians, researchers and industry leaders regarding the path toward outpatient studies for both low-glucose suspend and artificial pancreas systems.

Even with treatments available today, tight blood sugar control remains a challenge and daily struggle for those living with type 1 diabetes. In fact, the majority of people living with the disease are not achieving recommended target levels. “An artificial pancreas, essentially a device that would both measure blood sugar and dispense appropriate amounts of insulin to keep levels in optimal range, would take much of the guesswork out of daily management of the disease,” said Dr. Aaron Kowalski, Assistant Vice President of Treatment Therapies at JDRF. “In the long-run, controlled blood sugar levels will help to lessen or avert the devastating complications from type 1 diabetes.”

To date, artificial pancreas devices have been successfully tested in controlled inpatient or hospital settings, demonstrating the potential for this technology to improve blood sugar control. Now it must be tested safely in real-world conditions. And clear and reasonable regulatory guidelines must be established to ensure that the upcoming studies advance the technology to reach patients as soon as possible.

“We believe a safe and effective first generation artificial pancreas system is possible with today’s technology, even as we continue to encourage development of improved devices. Experts at today’s FDA workshop outlined a clear path forward to safely speed the development and delivery of artificial pancreas systems to patients,” said Jeffrey Brewer, President and CEO of JDRF.

To help advance these efforts, JDRF formed a Clinical Panel of internationally renowned leaders in the diabetes field to make recommendations to FDA on key clinical steps and issues critical to the advancement of studies of these systems outside of the hospital. Panel members speaking at today’s workshop included David Nathan, Director, Clinical Research Center and Diabetes Center at Massachusetts General Hospital and Professor of Medicine, Harvard Medical School; and William Tamborlane, Professor and Chief of Pediatric Endocrinology and Diabetes, Yale University School of Medicine.

The panel developed a series of clinical recommendations that were shared at today’s meeting. They were based on key areas addressed by the FDA, NIH, JDRF, clinicians and industry. First, the recommendations addressed questions on how should studies on artificial pancreas systems move safely from inpatient (hospital) settings to outpatient (real-world) testing. Second, the panel identified which subset of patients should be considered when testing artificial pancreas systems. The third area focused on how to ensure the safety of patients participating in the studies and eventually for everyday use. Lastly, the panel identified what outcomes should be measured in studies to demonstrate the safety and effectiveness of the device.

A summary of the panel’s recommendations is available here. A full report by the panel will be forthcoming.

According to panel chair Robert Sherwin, M.D., Yale University, “The panel believes, with certain safeguards, artificial pancreas systems can be safely tested in real world settings.”

“The incidence of type 1 diabetes is on the rise. Today’s tools to manage the disease are insufficient. We have the technology at our disposal to make an artificial pancreas work. Now it’s time to move forward quickly to define the regulatory pathway so final studies can be completed and better technologies can be made available to adults and children struggling with this difficult disease,” added Mr. Brewer.

About JDRF’s Artificial Pancreas Project

JDRF launched the Artificial Pancreas Project in 2005 to speed the development of automated diabetes management systems. A self-regulating system, the artificial pancreas would be able to sense sugar levels continuously and automatically release the right amount of insulin at the right times – eliminating the need for multiple blood tests, insulin injections and therefore lifting the daily burden associated with managing diabetes.

Since that time, JDRF has supported a number of initiatives that have advanced progress toward the development of an artificial pancreas. This has included the formation of the Artificial Pancreas Consortium, a group of university-based mathematicians, engineers, and diabetes experts to develop the computer algorithms that are needed to connect the devices needed to form a closed-loop system.

In addition to the consortium, JDRF has collaborated with several industry partners to develop a first-generation artificial pancreas system, as well as better and faster-acting insulin products, a key component of developing a safe and effective artificial pancreas system.

More information about JDRF’s Artificial Pancreas Project can be found online at www.jdrf.org/artificialpancreasproject. The site includes information for people with type 1 diabetes about research leading to the development of an artificial pancreas, as well as interactive tools, project timelines, chats with researchers, and access to information about clinical trials.

August 14, 2009

The FDA has issued an alert about the possibility that people with type 1 diabetes who take certain medicines that contain non-glucose sugars might get falsely elevated readings from home blood sugar monitors if they use a specific kind of test strip.  These false high readings could be dangerous, because they might mask low blood sugars or cause people to take excessive amounts of insulin.  The test strips in question are GDH-PQQ glucose test strips, and there is a list of medications that might result in incorrect readings – as well as additional information – on the FDA website.