Off-label use of Kalydeco

Question

Dear physicians,
I am a mother with a 3.5-year-old son suffering from CF, living in Germany. My son has been diagnosed at the age of 2.5 years. Overall, his health condition is good. However, the illness affects markedly the gastro-intestinal tract. He has had a long-lasting colonization with germs and has even been underweight after this for a short time.
He has the very rare mutation G1244E and the more common one R553X. For the Mutation G1244E there is in my opinion the possibility, to treat him with Kalydeco. Until now, the drug is only licensed for children over the age of 6 in Germany, however on the 18th March 2015, the drug has been licensed for children under 6 years of age in the USA.
We as worried parents would like to treat him with the drug off-label, however our Center is denying the prescription off-label with the rationale, that there was no data about the dosage. They do not want to take resposibility for this. I am very despaired and want to do everything that my son can take the drug. I do not understand, why the physicians are refusing so vehemently to prescribe the drug, if even on the homepage of Kalydeco the dosage is written (50 or 75 g granulate).
Until now, we did not contact the health insurance however we talked about it in detail with Vertex and with a pharmacy in our home town. Both told us, that the health insurance could cover the costs for the drug, if a CF physician would prescribe it. We know another case of a child in Germany, in this case the father requested the off-label use for the drug and the health insurance agreed. The father posted a contribution on Ecorn. I was not successful in getting in contact with the parents of this child.
Could you help us further? I would be very thankful for an answer.

Answer

[The question & answer were originally asked on the German platform and therefore the answer refers to German circumstances]

Dear questioner,
there are a few possibilities:
1. Taking part in a compassionate use program (my personal opinion: it will be refused, therefore do not put effort in it).
2. In case of your request it does not deal with an off-label use, as the prescription of Ivacaftor is already licensed for children aged from 2-5 years by the FDA in the USA. Therefore, a prescription is not off-label anymore, what makes things easier.
If you thus do not want to wait some more months until the drug will be licensed in Germany, my proposal would be:

It has to be clarified with the health insurance in advance, that the cost are covered. With the respective justification of the physicians and with a reference on the official prescribing information (which I already have sent to your by mail) this should not be a problem.

Best regards,
Dr. Olaf Eickmeier

28.06.2015