Memorandum

NIH initiative to fund up to $20 million in 2015 for outpatient artificial pancreas clinical trials – August 4, 2014

Executive Highlights

  • On July 21, the NIH posted a new request for application (RFA) entitled, “Advanced Clinical Trials to Test Artificial Pancreas Device Systems in Type 1 Diabetes.” NIH intends to commit up to a total $20 million in FY 2015 to fund between one and three awards.
  • The funding aims to support outpatient artificial pancreas trials that will generate safety and efficacy data able to satisfy regulatory requirements. Pilot/transitional studies will not qualify for this funding.
  • For-profit businesses, academic institutions, non-profits, foreign institutions, and governments are all eligible to apply. The application is posted here.

The NIH recently posted a new request for application (RFA) entitled, “Advanced Clinical Trials to Test Artificial Pancreas Device Systems in Type 1 Diabetes.” NIH intends to commit up to a total $20 million in FY 2015 to fund one to three awards. Notably, the funding aims to support outpatient artificial pancreas trials that will generate safety and efficacy data able to satisfy regulatory agencies. It was noteworthy to see that any entity is eligible to apply, including for-profit businesses, academic institutions, non-profits, foreign institutions, and governments. A letter of intent is due by March 15, 2015, and applications are due by April 15, 2015; studies cannot begin before December 2015. The application is posted here.

We’re not positive, but based on the RFA’s wording, it sounds like this funding is aimed at studies that would satisfy an artificial pancreas PMA application to the FDA. However, the criteria for proposed trials are fairly general (see below) and do not provide specific insight as to what these studies will look like – as a reminder, the FDA’s finalized Artificial Pancreas Guidance also looks very flexible at this stage and does not provide a detailed template for automated insulin delivery (that is what we are calling artificial pancreas and bionic pancreas projects) pivotal studies. We would assume studies to support regulatory approval will last at least a minimum of three or six months (most likely the latter) in at least 100 patients in the intervention group. As we understand it, pilot/transitional studies will not qualify for this funding. 

NIH has been a major funder of automated insulin delivery trials and related technology/research over the past decade (over $100 million), including in the academic setting (UVA, Boston University/MGH, Stanford, Sansum, among others) and through small business grants (Xeris, Thermalin, Biodel, Profusa, Biorasis, Glysens, Grover Instruments, Advanced Medical Electronics, Pacific Diabetes Technologies, SmartCells, Senseonics, and others). We will be particularly interested to see what mix of entities ultimately receive funding through this impressive initiative (academic vs. for-profit vs. non-profit). To date, the NIH has been hugely supportive of the artificial pancreas, most recently following Drs. Ed Damiano and Steven Russell’s publication in NEJM – at the time, the NIH published a dedicated press release and even had a blog post from NIH Director Dr. Francis Collins. Said Dr. Guillermo Arreaza-Rubín (Program Director, Division of Diabetes, Endocrinology, and Metabolic Diseases) at the time, “With promising results such as these, we plan to support larger multicenter trials of the artificial pancreas in the near future. Within the next few years, we hope these technologies will go beyond experimental trials and be available to benefit more people with type 1 diabetes.”

  • Any Clinical Trial proposed must meet the following criteria. We were particularly impressed by the requirement that said results must include a clinical diabetes measure, defined by either by an improved A1c “or decreased hyperglycemic and/ or hypoglycemic events” or both – it’s terrific to see ongoing encouragement of effective “time in zone” data even though there have not (yet!) been long-term trials establishing that increased “time in zone” reduces long-term complications like DCCT did for A1c.
    • ​It must be supported by strong clinical preliminary data that justifies pursuing the trial.
    • The trial must have a well-defined and clinically important outcome demonstrating improvement of management of type 1 diabetes. It must include a clinical diabetes measure (e.g., improved A1c, or decreased hyperglycemic and/ or hypoglycemic events).
    • It may also have improvement of quality of life as an important outcome using valid metrics for it.
    • A target population of patients with type 1 diabetes is required. The trials may include patients across the lifespan, or, if scientifically justified, may focus on a specific age group (e.g., children, adolescents, pregnant women or the elderly).
  • There are two other notable NIH requests for application: RFA-DK-14-014 and RFA-DK-14-015These complement the most recent one discussed above. NIH has ~$39 million (including small business grant projects) from the Type 1 Diabetes Special Program available to artificial pancreas projects in FY15, and some more from NIH's regular appropriation. In sum, that means that >$40 million could be provided by NIDDK to artificial pancreas and related technology proposals during FY15 (depending on the review success).  

 

Close Concerns Questions

Q: Will trials funded by this program support PMA application submissions to the FDA?

Q: Will the NIH work with the FDA to design studies that will support sufficiently robust PMA applications?

Q: Who is most likely to be funded – for-profit companies or academic institutions?

Q: What is the most likely pathway to market for artificial pancreas systems developed in academia – licensing technology to larger players, starting a new company, etc.?

Q: Is it a goal to have a mix of approaches funded?

 

-- by Adam Brown and Kelly Close