Myself and Angie both talked to Bo Allen at Rare Disease Therapeutics today and he had a fair amount of information for us. The rundown in regards to the Falk foundation providing this drug and the whole insurance problem amounts to that Falk foundation will back the policy up in the case of a lawsuit if it is a result of treatment with Chenofalk for gallstones, not when it used as a treatment for CTX, this puts these pharmacies at huge risk.
The insurance policy is prohibitively expensive, Bo said that RTX cannot carry it, they can’t afford it. He said one of the proposals to the Falk foundation is a waiver scenario that we would all need to sign to absolve Falk/Ludwigs-Apotheke of any liability with the drug. If that happens, he will have RTX’s lawyers draw this waiver up for us. That is where it stands at this point. He did say that they will not let us go without this medication that they will do whatever it takes to get it here to us. He mentioned that Dr. Salen has some that could help to keep us supplied with it while this hurdle is overcome.
He did say that he is certain that the Falk foundation will not make this drug forever, which I have had in the back of my mind for some time. He figures we can get another 1-3 years of medication from them at which point we will have had to come up with an alternative. He mentioned that he is
in discussions with Dr. Salen to compile the findings of the research and start down the path of getting an IND(Investigational New Drug) for CTX. He said the process would likely be that of copying what Chenofalk is and classifying it as a new drug, conducting trials with it as compared to using Chenofalk, etc. This would ultimately result in it’s being available in a pharmacy under a regular prescription and eliminate all of the issues in dealing with drug imports. It’s an expensive laborious process not without it’s challenges but he figures that the only way to guarantee future availability of treatment is to go down this path.
He did mention Jim Bona at the FDA as well as a John Mccormick, saying they are good people and are fighting to get a solution to this problem.
Some other information that I got from him was that a Dr. Ken Setchell at the Cincinnati Children’s Hospital Center recently diagnosed 3 siblings that were under the age of 5 with CTX. I am going to send Dr. Setchell an invite to the yahoo board in hopes to foster some interest and buzz in the medical community around CTX and the treatment problems that we face, I will also encourage him to invite the parents to join as well so that they can communicate with some folks that are
dealing with this.
Again in my quest for numbers I asked Bo if he had figures on CTX affected patients, he said they have about 50 people who take Chenofalk in the US and Canada.
Bo and I also talked about lack of early screening and some other general rare disease topics. I feel that these are discussions that we should all have after we get through this current issue.
Angie and I have been talking a lot about this and we want to go public with this
issue, meaning big media. We feel that it might help garner the support that we would need to get something going in regards to getting a treatment approved and manufactured in the US.