Helmsley and JDRF: Envisioning the Future
By Jeemin Kwon, Adam Brown, Brian Levine, and Kelly Close
Discussion with David Panzirer and Dr. Aaron Kowalski at JDRF’s 2018 Mission Summit
JDRF and the Helmsley Charitable Trust (Helmsley) are two enormously influential organizations in the diabetes community. At JDRF’s 2018 Mission Summit in San Francisco, Dr. Aaron Kowalski (Chief Mission Officer, JDRF) and David Panzirer (Trustee, Helmsley) spoke at length about the history and future of the partnership between these two organizations and on far broader topics relevant to those living with type 1 diabetes.
Below are select Q&A’s from the evening, organized by topic:
Ms. Margaret Anderson: Can you talk about the creation and launch of the JDRF T1D Fund? (The JDRF T1D fund invests in early-stage T1D therapies.) It seems significant not just for type 1 diabetes, but for the entire ecosystem.
Dr. Kowalski: One of the biggest concerns is that the type 1 diabetes marketplace is not very healthy. It has dramatic impact on our ability to move programs forward. The dissolution of Animas should be concerning for everyone in this room. It's a huge problem that no pump company has ever turned a profit besides Medtronic… Before the JDRF T1D Fund, support was very, very difficult.
Mr. Panzirer: We sometimes hear the T1D market described as the valley of death. If we don't pave the way, companies are not incentivized to play. This is another vehicle to pave the way. Both JDRF and Helmsley have a little bit of a halo effect - that gives investors a degree of confidence.
Dr. Kowalski: David, how do you think about philanthropy and people supporting the Fund and JDRF Research?
Mr. Panzirer: I think of this as a business. We want to deliver a drug or device or therapy to people. In order for that to happen, the FDA must approve it, an insurance company must pay for it, and a company has to believe it will make them money so they can do the manufacturing and distributing of it.
Sean Doherty (Chairman, JDRF T1D Fund): For us at the T1D Fund, if we were able to decide to raise an extra $100 million, what should this organization do over the next 1-3 years?
Dr. Kowalski: We focus on devices, but as I always say, 'no one without diabetes wears an insulin pump.' I still don't like the tube coming out of my pocket, ripping out on a door, the rash on my arm, I don't like any of that, but we need to push. I love prevention, but I'm still in this for my brother and me too. We will not lose sight of that. Glucose-responsive insulin, cell therapy, microbiome, we're going to push like hell on them and try to figure it out.
Dr. Kowalski: One of the areas I'm most fond of is prevention. Obviously, I want my brother and me to be cured, but I also worry a lot about my kids getting diagnosed. They're at genetic risk, at about 15 times the risk of the general population. I'm proud of the role JDRF has played in prevention. Helmsley just announced a massive investment in prevention, can you talk about that?
Mr. Panzirer: Like anyone, I'd cut my right arm off to get a cure for my kids, but that's not happening anytime soon. As a private foundation that does not have to fundraise, we believe we have an obligation to think differently and take more risk and fund longer-term projects such as primary prevention. Simply put, primary prevention would be to vaccinate against type 1 diabetes, although in reality it will not be that simple.
We just launched a very large primary prevention program over in Europe. It's general population screening, and we're going to screen about 300,000 newborns. We have started in Germany and will add four other countries. The goal is to see who has the genetic risk for T1D and intervene prior to autoimmunity.
Almost all of the interventions tried today are on people who already have autoimmunity. Once you have autoimmunity (more than two antibodies) I would argue you already have the disease. In T1D, (a) There's nothing to treat it with and (b) there's a stigma to diagnosing early, especially a child. Helmsley believes we have to intervene prior to autoimmunity. We're trying large doses of oral insulin in people who have no autoimmunity.
Dr. Kowalski: I mentioned today that I was Looping; can you talk about Morgan's experience on Loop?
Mr. Panzirer: Loop is not for everybody; you have to be a bit of tinkerer. But I believe that to be impactful in type 1 diabetes, technology is the way. If you asked me what the single biggest advancement in diabetes is, I would say it's CGM. If we could only have one tool to work with, it's CGM. The way I see the future, automated insulin delivery is going to be important, and hopefully it will catalyze more people to get on pumps.
But there is going to be a group that says, 'I don't want both devices on my body.' When you pair form factors like Libre and the future Dexcom/Verily sensor - the sensor that looks like an M&M, which is pretty compelling - and you couple that with connected insulin pens, you begin to see the pathway clearly towards decision support/AI. What decision support or AI means to me is: Let's take all this data and turn it into something actionable. That is not that far off.
Ms. Kelly Close (Founder, The diaTribe Foundation): I worry deeply about the doctors and nurses trying to take care of all of us with diabetes with so few resources and so much administrative burden. How do you think about this important population, healthcare providers, and what might be possible to help them be more successful, perhaps partly with digital health and telehealth, where the Helmsley Charitable Trust has so much experience?
Dr. Kowalski: We have a massive problem looming in the US with a lack of endocrinologists, pediatric endocrinologists, diabetologists, and funding for folks – the pipeline and resources to help them do their jobs. This is why the policy work we've been doing with Helmsley and NIH and everyone is so important.
Mr. Panzirer: It's like a train wreck happening in slow motion. We need to get the clinician out of the middle - the majority of clinicians are actually a barrier to the adoption of technology. With the endocrinologist shortage, most patients see a primary care provider who doesn't know anywhere near enough about type 1 and has no incentive to learn due to the of a lack of reimbursement. We've piloted health models to get doctors to see more people - telemedicine, group appointments, the easy layups. But in the long term, we see the need for a different model, one in which the CDE, who does 95% of the heavy lifting in a clinic anyway, writes the prescription and becomes the core of the care team.
Audience question: I'm heartened to see the movement away from just A1c, and also the move to primary prevention. But how do you turn that into billing codes? That's a fundamental problem in the field.
Dr. Kowalski: What's really motivated me over the past 10 years is the model for what I think is the future: Diabeter. This is a center in Holland. The word means "better diabetes" in Dutch. Hank Veeze had this idea to go to the payers. He said, "I want reimbursement for X% less than you pay per year, wherever and whatever you're paying. For example, I'll take 75% of what you're paying, and then get out of my way. He then completely flipped the model.
Mr. Panzirer: I went to that Diabeter clinic … this clinic, which is now owned by Medtronic, receives two to three times the amount of the reimbursement of the city clinic. When patients do better at Diabeter, they get more reimbursement. That is a model we need to follow here in the US. Let's go at-risk. If you give the right care, put your money where your mouth is.
Dr. Kowalski: There are not a lot of $6 billion charitable trusts out there. Talk about Helmsley and how you've championed type 1 diabetes - with other trustees there, how does it work?
Mr. Panzirer: Helmsley’s philanthropic giving is focused almost exclusively on helping to advance health and medical research. In fact, we've been winding a couple of programs down to narrow that focus further. I was lucky enough to meet with the first CEO of the Bill and Melinda Gates Foundation Patty Stonesifer, and she told me two things I'll never forget: (i) We are the Gates Foundation, and even with our resources, we can't truly solve anything. I thought, "jeez, you're 10-times the size of us, I might as well go home." (ii) With each dollar, you have to be laser-focused; ask yourself what the impact will be. It was really humbling to hear this.
Dr. Kowalski: Helmsley was an important initial investor in our automated insulin delivery project. Dr. Sanjoy Dutta (Assistant Vice President, Research & International Partnerships, JDRF) and Campbell Hutton (JDRF Senior Director, Regulatory Affairs) were brought in because of support from Helmsley. To date, we're probably approaching $100 million that JDRF and Helmsley have co-funded together. Talk about the decision to keep your type 1 program separate from JDRF.
Mr. Panzirer: I started out really naïve and really cynical, now I'm less naïve and still really cynical. My cynical thought was that JDRF should be working towards a goal that would put themselves out of business, and if they haven't done that yet, then they must be doing something wrong. As I said, this is a completely naïve and cynical point of view. Clearly I was wrong. As I began to study the landscape and understand it, at the end of the day, if Helmsley and JDRF have two separate funnels of ideas, each with its own separate staff to compare and contrast, that’s got to be better. We have our own, they have theirs, and we compare and share ideas.