FDA-Patient Dialogue on the Unmet Needs in Diabetes
FDA-Patient Dialogue on the Unmet Needs in Diabetes
by The diaTribe Foundation team
Twitter summary: We provide our re-cap of the Nov.3 virtual discussion w/ the FDA here! Stay tuned for more information coming soon!
Update: You can find the complete transcript of the event at the FDA resource page here.
After hours of planning and anticipation, we were thrilled to see all of the hard work of the diabetes community culminate in the unprecedented patient discussion with the FDA on Nov. 3. It would not have been possible without the enormous help of the FDA staff who so generously agreed to partner with us, and the thousands of patients who have made their voices heard by taking the survey, spreading the word, and tuning in for the event.
Along with nine patient representatives (read more about their experiences below) we had the opportunity for an open discussion about the challenges that people with diabetes face every day, the tools we’d like to see, and what FDA reviewers should keep in mind as they consider new drugs and devices. The three-hour event included a patient panel, an FDA leadership panel moderated by Kelly Close, and perspectives from The diaTribe Foundation, JDRF, ADA, and the FDA.
Overall, we heard many moving patient stories and were gratified by the enthusiasm from the FDA on future discussions; we were also pleased by the focus on moving past static A1c numbers and placing greater emphasis on time in range. Unfortunately, the FDA's webcast system failed during the session due to the very high demand from the diabetes online community. Luckily, the diaTribe team has been able to piece together our own iPhone videos of the event (it's not perfect!) for anyone interested in the latter half of the day. Please see the full video here!
Opening Remarks - Our Meeting Goals
To start the day, Dr. Helene Clayton-Jeter (Director of the Cardiovascular and Endocrine Liaison Program in the Commissioner’s Office of Health and Constituent Affairs) outlined three major goals of the event:
Continue to open lines of communication between the FDA and patient community
Give FDA leaders a better sense of what it’s like to live with diabetes, what is difficult for patients, and what patients want in terms of new tools
Have this event serve as a “pilot program” for future FDA/patient meetings
Kelly Close (Founder, The diaTribe Foundation) then provided background on the meeting, which started nearly 18 months ago when she met FDA Commissioner Margaret Hamburg and spoke with her about the urgent needs in diabetes care. Commissioner Hamburg helped The diaTribe Foundation set up this meeting, where patients could speak directly to the FDA. “Diabetes,” Commissioner Hamburg noted, “is the biggest health problem of our time.” About 5,000 people are diagnosed with it each day, and we spend $250 billion annually on it in this country, yet most patients are not achieving their goals. In closing, Ms. Close presented the FDA with a 400+ page book comprised of 7,500 patient comments from dQ&A’s recent survey on the unmet needs in diabetes.
Lastly, Ms. Close shared a compelling video showcasing various voices from the diabetes community on what they’d like to see most for their diabetes management.
FDA Presentations – What the FDA Does in Diabetes
Four speakers representing the FDA elaborated upon the FDA’s role in diabetes.
Dr. Jonca Bull (Director of the Office of Minority Health, FDA) gave a talk on the increased burden of diabetes in certain demographics, including minority populations (particularly Hispanics and African Americans) and lower-income families. She said that while the burden is higher in these groups, they are often under-represented in clinical trials. For example, an FDA report found that only 5% of diabetes clinical trial participants were African American, despite that group making up 13% of people with diabetes. She explained that the FDA released a recent action plan with priorities to improve demographic data collection, to identify barriers to patient enrollment in clinical trials, and to make the data more available and transparent.
Dr. Courtney Lias (Director of the Division of Chemistry and Toxicology Devices, FDA) presented on the regulatory process for diabetes devices and highlighted that the FDA makes a concerted effort to work with device companies to avoid unnecessary burdens throughout the process. For us, the real takeaway was her acknowledgement of the tremendous need for patient input to the FDA. She mentioned several big themes the FDA has heard from patients, such as the need for more accurate meters, approval of an automated insulin system, remote monitoring technologies, and better test strips. She also highlighted an opportunity for patients at the FDA on November 13 that aims to address inter-device communication (e.g. a meter talking to a pump) and how patients and health care providers actually use insulin bolus calculators.
Dr. Naomi Lowy (Medical Officer on CDER’s Professional Affairs and Stakeholders Engagement) gave a brief presentation on how diabetes drugs go from research trials to patients. Dr. Lowy pointed to the FDA website’s info graphics on this process, which provide a clear overview of the multiple steps necessary for drug approval. She also provided resources for patients to learn about diabetes drugs (National Diabetes Information Clearinghouse, PubMed Health, the Drug Informational Portal), but stressed the best resource is a patient’s own health care provider.
Dr. Patricia Beaston (Medical Officer and Endocrine Consultant at the Center for Devices and Radiological Health, FDA) gave a talk on how the FDA considers the risk/benefit trade-offs of new diabetes products. Dr. Beaston pointed out that while many patients want devices to be more convenient, improving convenience often comes with diminished accuracy and reliability. For example, smaller insulin pumps – while more convenient – are likely to affect the accuracy of insulin delivery and decrease the product’s overall usefulness. To close, she invited feedback on what patients would consider an ideal “consumer report” for diabetes products, e.g. what safety, accuracy, durability, etc. information do patients want to see most?
Perspectives from The diaTribe Foundation, JDRF, and ADA
The diaTribe Foundation’s Adam Brown spoke on the complexity of diabetes and the key issues affecting patients. Mr. Brown explained that A1c does not tell the full story of a patient’s experience, and that time in range, hypoglycemia reduction, and quality of life are important measurements as well. He also remarked upon the struggles of managing diabetes when over 22 factors can affect blood sugar on a daily basis. Mr. Brown called for increased FDA focus in a few specific areas, including:
Creating an approval pathway for prediabetes therapies – given 86 million Americans have prediabetes today, this population has no guidance for new therapies and needs greater focus.
Getting more drug and device options for type 1 diabetes – going beyond A1c inferiority/superiority as markers for therapy effectiveness in clinical trials and really looking at improvements in overall quality of life.
Understanding the patient perspective of accuracy/convenience trade-offs for devices – Mr. Brown specified that patients want accuracy “to a point,” but that major gains in convenience can often be more important to some patients for overall satisfaction.
Re-examining the basis of cardiovascular outcome trials – these trials cost billions of dollars and enroll hundreds and thousands of patients each year, with little shown benefit for patients or health care providers.
Mr Brown also interviewed Francisco Estrada, who has been living with type 2 diabetes for five years, on his experiences after being diagnosed and on the need for better visibility of clinical trials and increased patient participation.
JDRF’s renowned (and type 1 patient) Dr. Aaron Kowalski gave a compelling presentation on some of the fundamental issues affecting type 1 patients. Echoing Adam Brown, Dr. Kowalski stated that diabetes is more than A1c, it’s more than blood glucose control, and it affects more people than just the person living with diabetes. He discussed that while current therapies and technologies can help diabetes management, they aren’t cures, and the majority of patients still don’t meet the ADA recommended guidelines. According to Dr. Kowalski, “Highs and lows happen almost every day, and it can still be life threatening…we need better therapies.” Dr. Kowalski called upon the FDA, industry, and the diabetes community to work together to ease the burden of this disease.
In one of the most moving moments of the day, Dr. Kowalski spoke with Angie Platt (Board of Directors, LA JDRF Chapter, and mother of a son with type 1 diabetes), who shared the story of her son Jonathan’s diagnosis at age 6. Ms. Platt dealt with the shock, surprise, guilt, and hopelessness. She went on to describe their family’s daily routine, which begins at 5:30 a.m. for Jonathan’s first blood sugar test while he’s still asleep. While overnight lows were her biggest concern at first, now the family worries more about the extreme highs and lows throughout the day. She says that by lunchtime, Jonathan’s blood sugar can vary by over 100 mg/dl, even though he has the exact same thing for breakfast. One of the hardest parts, for her, is her desire for Jonathan to feel like any other kid, who can go on vacation, eat chicken nuggets and French fries, and not constantly have to worry about diabetes.
The ADA’s Dr. Maria Mupanomunda then gave a brief presentation on the high prevalence of diabetes and prediabetes in the US, noting that despite improvements in care, diabetes is still the leading cause of blindness and end-stage renal disease, and many patients are not meeting their goals. She advocated for the community to work to lower A1c without increasing hypoglycemia or weight gain. Dr. Mupanomunda spoke with well-known patient advocates Rebecca Killion and Anna McCollister-Slipp on their daily struggles – our favorite part was Rebecca’s powerful statement on coping with diabetes. “I tried to convince my doctor that I would cure my diabetes with my sheer force of will,” she said, “but I have yet to do that. My therapy failed me and I needed insulin; that’s when I realized I would need to do this every day in order to have a life.”
Perspectives from the Amazing Patient Panelists
Of course, the day could not have been so successful without the perspectives given by the brave patient panelists who went over and above to share their stories with the FDA. While each panelist has SO much to say, below are a few key quotes from each member of the panel about their experience with the FDA discussion (comments from remaining panelists coming soon!). For the panelists' full bios, please see this link.
Francisco Estrada (type 2 diabetes, 5 years): “Fighting diabetes is crucial to the well-being of the nation and the American people. It is winnable if everyone does his or her part. The FDA should encourage companies to create diabetes support programs (nutrition education and physical activity programs) in the workplace and to provide nutrition and cooking classes as part of work life programs to support employee health along with stress management. It should encourage greater physical activity programs, from daily walking to hosting yoga, tai chi, or other exercises that can be done at work, and also promote better health by providing time off, monetary rewards, or other recognitions to employees who are able to improve their health (and by extension their productivity) during a period of time. Lastly, it should use more PSA (Public Service Announcements) to increase awareness of this silent disease and to encourage people to get themselves checked out earlier and to manage their diabetes.
The FDA should also think about the level of effort and costs associated with managing the disease and its impact on economic productivity. With around 30 million people (and growing) in the US with diabetes, its impact on worker productivity (loss time, days, etc.) hinders the nations ability to provide goods and services and thus lowers GNP. It decreases governmental revenues and increases national healthcare costs.”
Rebecca Killion (type 1 diabetes, 17 years): “I have great respect for the FDA, but what I would like the overarching message to be is we’re all in this together. A point that was made earlier is we [patients] rely on the FDA to help us ensure we don’t have fraudulent products, that they meet the claims they say they will, etc. But again, we – as patients and caregivers - are an enormous source of passion and perspective, and I know that together with the FDA we will achieve so much more.”
Howard Lee (type 2 diabetes, 46 years): “I have been a diabetic for 46 years and after having this experience of communicating with the FDA for the first time, it is comforting to know diabetes may be elevated to a higher priority level. I left the meeting feeling optimistic about the new FDA interest in trying to find better ways to expedite the approval process for both equipment and drugs to enhance better management. Now that the diabetic community has a foot in the door, I expect great strides towards fast responses from FDA to the needs of the diabetes community.”
Cherise Shockley (latent autoimmune diabetes, 10 years): “People ask me, ‘How are you doing?’ and I’ll respond, ‘Oh I’m doing fine.’ And if you take look at me, I look like I’m fine, but I’m not. There are 30 million people in America just like me. Please move with urgency when approving tools and devices, so you [FDA] can help improve the quality of life of myself and the 30 million others like me.”
Brian Cohen (type 2 diabetes, 10 years): “We had an opportunity to share things that are difficult in our lives because of diabetes. And through this we could share those things that should be considered during the FDA deliberations, but have traditionally not been. Patients talked about quality of life, issues with hazards and side effects and the need to look beyond simplistic measures of effectiveness like A1c. The panel, which consisted of 11 people, was exactly on point. I don’t think I’ve seen nearly a dozen people take part in a 45-minute panel and be so clear.
I always thought of the FDA as being a closed off regulatory agency that just talked to industry, researchers and medical professionals, but that has changed. The FDA is now recognizing that it is important to hear the patient voice, the patient has a key role in defining effectiveness, safety and the context of risk and benefit. FDA representatives are really starting to get it… I came away feeling like the FDA is listening patients, something I’m not sure I would have necessarily believed before seeing it. I think many of us, as patients, feel vulnerable at times. We aren’t always sure who is looking out for us, and who is looking to make a buck. I came away feeling much better that the FDA has my back.”
Manny Hernandez (latent autoimmune diabetes, 12 years): “At the end of the day, what good is a product or a drug if we don’t have access to it after it hits the market? I was super inspired when I heard Dr. Courtney Lias speak a few weeks ago about how there’s a creative and intentional movement towards looking at the economic studies as well as safety and efficacy analysis, so by the time a drug moves through the pipeline, it’s closer to reimbursement reality. I would love to see more of that. I would love to see agencies and payers becoming more involved, because at the end of the day it’s great moving these products through, but it’s also critical that they be accessible.”
Angie Platt (caregiver, parent of a son with type 1 diabetes): “Two summers ago, my son Jonathan went away to a diabetic summer camp. And it was the first time in years that my husband and I actually got to miss our son – just miss him as a person, because we weren’t monitoring his diabetes for 24 hours a day. I’m so thankful for the FDA for keeping us safe, I truly am. And as technologies and treatments come through the pipeline, I’d like you all to remember that A1c is important, but just as important is keeping families together, and keeping families happy, and keeping marriages together. The ripple effect of living type 1 diabetes, it affects all of those aspects of a family – not just A1c.”
Adam Brown (type 1 diabetes, 13 years): “We have enough drugs on board to kill us at every minute, and that’s a really big risk we that we as patient take every day. And one of the biggest errors that patients make ever day is in carb counting. And you ask diabetes educators, 'How many carbs are in this?' and they get it wrong every time – not even the experts can figure it out. So please appreciate the risks that we take every day.
[on the topic of product labeling] Most patients don’t look at package inserts or labeling information - the print is small, it’s hard to unfold the map that is packaging labels. When we test new products at diaTribe we apply this test: ‘Can I set this up without an instruction manual?’ Because that’s how easy products should be. We live in the world of iPhones and iPads where we can take a product out of a box and turn it on and a 5-year old could get it up and running. And that’s how our medical devices should be; they should be that easy. It’s really hard from a patient perspective to read 300-page manuals, because none of us want to spend more time on diabetes – we’re already doing it 24 hours a day.”
Rubin “RJ” Scott (type 2 diabetes, 19 years): “I've had low blood sugars twice as a diabetic. That terrifies me. I go to sleep, but it always wakes me up when my blood sugar starts to jump. But what happens when I don't wake up? My biggest fear is that I’ll take insulin before I go to bed, and it will interact with other medications and then I’ll go low. Will I wake up or realize it's dropping in time to counteract it? The longer I'm diabetic, the longer it takes for me to react. When I first was diagnosed, I still reacted, but sometimes it takes a little longer now before I know what is going on and then it's too late. One night it went as low as 37.
In the community, people are fed up. They’re tired of being sick and tired. We need to get information in a way that we can understand it, so that there aren’t as many complications. People I work with don’t want to go to the doctors – they don’t want to hear the words, ‘You’re diabetic,’ or, ‘You’re prediabetic,’ or even, ‘You have to take a daily shot.’ People ask me everyday how I do it, and I say, ‘Well, I have to live with it if I want to live.’
I'm glad we had the opportunity to talk to someone who can actually implement what we're saying. I was almost in tears after hearing Angie and Anna - their stories really got to me. To live like that every day because you are diabetic and living with complications, that's just scary. The experience was great, and I hope to do it again.”
Anna McCollister-Slipp (type 1 diabetes, 28 years): “In terms of other important diabetes measurements aside from A1c, time in range is a particularly helpful alternative measure. But quality of life is also absolutely critical. Additionally, non-glucose related hormones that are affected by diabetes – there are a variety of satiety hormones that are impacted. We have very significant increases in eating disorders, particularly in girls and adolescents with type 1 diabetes. My fear is that there are so many treatments that could be advanced that aren’t being advanced. Because if you’re a pharmaceutical company and deciding which drug to license, you ask yourself, ‘Am I going to make a drug that impacts A1c, or do I want to license a drug that perhaps has a more difficult FDA regulatory pathway ahead?’ And you go with the safer bet, and as a result we’re living without potential cures that could be advanced in clinical trials.
We as patients want to support you [the FDA] in your desire to be our advocates, so please reach out to us and contact us. We are your asset, so let us be that asset.”
Additionally, below please find a few great resources from the meeting:
#DOCasksFDA - Diabetes Patient Voices (video below!):
The diaTribe Foundation perspective by Adam Brown - The Complexities of Diabetes (powerpoint slides)