Dear Dr. Lippe,
I am the parent of an 8 yo. boy with Congenital Adrenal Hyperplasia. I often follow the discussions on the Yahoo Groups Peds-Endo chat line with interest. In reading about the recent 5 mg. cortef pill shortage, I was very interested to realize that, in addition to working for Pharmacia Upjohn, you are also a pediatric endocrinologist, who is now also president of the LWPES. I wonder if I might pick your brain, for a moment.
I often read a parents' Internet message board devoted to those with children with CAH. You are probably aware of it, as well.
Two issues have come up repeatedly in the past few months. The first is the need for a HC pill, in a 1 mg. size. Halving or quartering a pill, in order to fine-tune doses, is an exercise in frustration for most, not to mention inaccurate. Apparently, a pharmaceutical company in New Zealand is preparing to start manufacturing such a pill, as a result of inquiries from another parent with a CAH child. I am sure that Pharmacia Upjohn is a much larger and more sophisticated company than this New Zealand group. Being able to carefully titrate hc doses, which is something so crucial to the successful treatment of CAH, would be a big boon for patients and parents. What keeps PU from doing the same for CAH patients in the United States? Surely, the technology and ability to produce such a product is not an issue, I would guess?
The second product that would be incredibly beneficial to CAH treatment would be the development of a time-release pill, to coincide with the 3-4 AM surge of ACTH. As you know, promoting normal rates of growth is a major stumbling block to the successful treatment of CAH. Some of the fault must lie with the fact that, at best, current medication replacement regimes are inefficient in that rises in ACTH levels do not necessarily correlate neatly to normal wake-sleep hours. If a time-release medication could be made, treatment would certainly be improved for patients.
I searched briefly on the Web and found that the concept behind the idea of a time release preparation is essentially the encapsulation of the drug within a material that disintegrates over a number of hours. A time release HC pill would essentially need to remain inactive for about 6 hours before being released---say, from ~9 Pm till about 3 AM. Does such a material exist at this time, and if so, why could a time-release HC pill not be developed?
There was considerable interest recently among some parents on the CAH board to try to make these two things happen, but no one knew where to start and whom to contact first. I just happened on your name today, as I was lazily surfing around on the Internet. When I saw that you were not only President of the LWPES, but also employed at Pharmacia-Upjohn, the fact that you were the perfect person to contact about this issue was obvious. As a pediatric endocrinologist, you certainly understand what the issues are, and as an employer in the pharmaceutical company that currently produces cortef, you also understand them from the drug company's point of view.
Would you also be willing to consider the patients' point of view, on these two issues? I hope to hear back from you and look forward to hearing your opinion. Thank you for your time.
Yours Sincerely,
Carol _________
Dear Ms. _______:
I appreciate your concerns and issues as a parent who has to rely on a specific type and dosage of medication for maintaining the health of your child and of course I understand the issues in CAH quite well.
As a transplant from academics to the medical side of the pharmaceutical industry I am afraid that I am not an expert on the issues of drug formulation, dosage formulations, and production issues. What little I do know about Cortef tablets is that standards for producing each batch are set quite high by the FDA and batches that don't meet specifications do occur (resulting in the backorder situation that occurred recently). I also know that formulating very low doses is actually more problematic than larger doses forms. I have no idea what difficulties there could be from trying to get a very low dosage into a slow release preparation except to imagine how technically difficult it could be.
I am forwarding this email to those colleagues who might be able to give you a more specific answer and apologize for my inability to be anything but an advocate for doing the best we can, especially for our pediatric endocrine patients.
Sincerely,
Barbara Lippe MD