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the news that on Thursday, December 1, the U.S. Food and Drug Administration (FDA) issued artificial pancreas (AP) draft guidance. While the draft guidance is still under review, our initial analysis suggests that it affirmatively addresses many of JDRF’s key issues, and, in so doing, it appears to lay a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.
JDRF advocates at the best anywhere. I recall years ago when we were working on another issue, a Senator saying to me "You know, it's hard to get 57 Senators to agree that today is Wednesday." You guys got 60. Remarkable, powerful and just plain cool.