Thread by @BasedMikeLee

🧵 1. @US_FDA often stalls progress on otherwise-promising treatment options for people with type-1 diabetes. #T1D

2. For example, I’ve long criticized FDA for tight grip it holds over glucose monitors (CGMs) and other diagnostic devices. Its approach benefits a few market incumbents and hurts would-be competitors.

3. I’ve also long held that at least some of the price spikes we’ve seen in insulin are the product of regulatory problems created by FDA, which artificially constrict the market for insulin. This is good for a few pharmaceutical companies but bad for all others.

4. But here’s one that makes me especially mad: FDA is treating pancreatic islet cells as “drugs” rather than what they are—organs. Consequently, it’s taking forever—just as it does with any “new drug”—to move this new treatment option forward. But it’s not a drug!

5. The FDA is nonetheless stifling innovative islet transplantation by regulating islets as new drugs.

6. Islets (pronounced “eye-lets”) are small organs (termed “microorgans”) found in the pancreas, with each one comprised of about 2,000 cells. Islets secrete insulin. In people with type-1 diabetes, they stop making insulin—likely because of an auto-immune response.

7. Type-1 diabetics therefore must take insulin injections or have an insulin pump, while monitoring their blood sugar continually using finger-stick test strips and CGMs.

8. Islet transplantation presents a more sustainable solution than the traditional treatment of injecting insulin and using continuous glucose monitoring to determine the appropriate dose of insulin.

9. By essentially giving type-1 diabetes patients the organ that produces insulin for them, islet transplantation has the potential to replace insulin and CGM, improving the lives of patients with type-1 diabetes.

10. Current developments are promising. Clinical trials have found that patients with T1D who receive islet transplantation are able to go without injected insulin for much longer periods of time.

11. Moreover, because the islets are so small, they can usually be transplanted through less-invasive means.

12. Islet transplantation isn’t a perfect solution. Like any other organ transplant, islets are sometimes rejected. However, recent advances in islet production have allowed scientists to develop islets from stem cells with a higher acceptance rate.

13. Allowing doctors and patients to weigh the benefits and risks of this option, rather than preventing it entirely through regulation, would increase consumer choice and competition.

14. Currently, the FDA regulates islets as new drugs, which triggers clinical trial and manufacturing requirements. The U.S. is unique in regulating islets as drugs. Many other countries, including Canada, the UK, France, Italy, Australia, and Japan, regulate them as organs.

15. As a result, islet transplantation has become the standard of care in other countries, whereas they are currently performed only in the U.S. at a single transplantation center at the University of Chicago.

16. According to the Dr. Piotr Witkowski of the University of Chicago, islet transplantation has passed all three phases of clinical trials to demonstrate safety and effectiveness. However, manufacturing regulations pose the main obstacle.

17. Universities are not equipped to comply with pharmaceutical manufacturing regulations.

18. Consequently, 10 islet transplantation centers have already shut down because insurance companies do not cover the procedure, so there is no steady funding stream. Currently, procuring an adequate number of islets for transplantation costs between $30,000 and $50,000.

19. We could solve so many problems for people with T1D by treating pancreatic islets as what they are—organs, not drugs.

20. I’m working to file legislation on this in the near future to fix this. Once we get it passed into law, this technology will have a meaningful chance to usher in a brighter, healthier future for Americans with type-1 diabetes.