FDA Needs Your Input on Islet Transplantation

The May 22, 2008 Edition of the Federal Register had another announcement that people with type 1 diabetes should be interested in. The U.S. Department of Health and Human Services, Food and Drug Administration's Center for Biologics Evaluation and Research (CBER) has issued draft guidance similar to the guidance I announced back in March on diabetes drug and biologic treatments -- the very same guidance that the American Diabetes Association thought was NOT important enough to comment on!

(See http://edocket.access.gpo.gov/2008/E8-11516.htm OR the regulations.gov/fdmspublic for the announcement and the guidance documents, search under the docket number, which is FDA-2008-D-0293-0001)

"Guidance for Industry "Considerations for Allogeneic Pancreatic Islet Cell Products", Docket #: FDA-2008-D-0293-0001.

Because it is about as succinct as possible, I will simply share the information from the Federal Register:

The Food and Drug Administration (FDA) is announcing the availability of a draft document entitled "Guidance for Industry: Considerations for Allogeneic Pancreatic Islet Cell Products" dated May 2008. The draft guidance document is intended to provide recommendations to manufacturers, sponsors, and clinical investigators involved in the transplantation of allogeneic pancreatic islet cell products for clinical investigations of the treatment of type 1 diabetes mellitus.

The draft guidance is intended to provide assistance by identifying the types of data and information obtained during investigational new drug studies that may be helpful in establishing the safety, purity, and potency of a biological product in a biologics license application (BLA).

DATES: Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit written or electronic comments on the draft guidance by August 20, 2008.

Submit written comments on the draft guidance to:

Division of Dockets Management (HFA-305)
U.S. Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852

Submit electronic comments to: http://www.regulations.gov

FOR FURTHER INFORMATION CONTACT:
Valerie A. Butler
Center for Biologics Evaluation and Research (HFM-17)
Food and Drug Administration, 1401
Rockville Pike, Suite 200N
Rockville, MD 20852-1448
Tel: (301) 827-6210

Initial Thoughts

In particular, one element I noted was that the FDA has provided guidance as to what an acceptable eligibility for C-Peptide count should be, insulin requirements, etc. and these may NOT be ideal. For example, by most counts, I would likely be considered eligible:

Subjects enrolled in trials of allogeneic islet cell products should have established Type 1 diabetes with a well-documented chronic history of severe metabolic instability. Subjects most likely to benefit from islet cell transplantation are those who cannot achieve acceptable metabolic control without experiencing multiple episodes of severe hypoglycemia, often with unawareness. Other eligible subjects may have lesser degrees of hypoglycemia, but still cannot be adequately managed with intensive insulin therapy alone. In screening subjects for clinical trials, we recommend that you document that such metabolic instability has persisted despite intensive diabetes management delivered by a qualified diabetes team for at least six months prior to enrollment.

However, thanks to their recommended requirement that subjects have a "stimulated C-peptide should be <0.3 ng/mL", I would NOT be eligible because my own stimulated C-Peptide count is 0.7 ng/mL, therefore I'm disqualified -- why? Because I've maintained sufficient control and therefore preserved the life of my few remaining beta cells, therefore that renders me as an ineligible type 1 diabetes patient? Give me a break. Is this really the best selection criteria? I don't know for sure, but I can tell you that I don't want a bunch of biotech industry geeks and drug company people making these determinations without ANY patient input as they have done historically, therefore I am commenting to make my concerns known.

Let me just close by noting that public comments are due on August 20, 2008 if you wish for your comments to be included in the FDA's evaluation!