I just called Vertex to speak with them about doctor's prescribing Kalydeco off-label.
Here is what I learned~
Any doctor, any where can write a script RIGHT NOW for Kalydeco for any patient with CF regardless of mutation. They (vertex) do not require proof of mutation...that is only important if you have G551D and vertex can help get financial assistance for the drug. The form doc s sign is to help G551d get insurance coverage, that is all!!!!
Vertex is bound by legal obligation to only help (financially) patients who are G551D. There is actually very little they can say to a CF patient who is not G551D because of FDA REGULATIONS because the approval of the drug only covered G551D. They cannot tell you if it might be beneficial (even if it works great in vitro on your mutation) because it is against the LAW and they will get in trouble.
However, I was informed that if your doc thinks it is an important drug to try and might be helpful, they are absoltuely allowed to prescribe it off-label. At that point, your pharmacy will do what it can to get the drug covered by insurance.
If your clinic has a problem with that, have them call this number at vertex:
I have been keeping this info to myself because I didn't want to have a lot of "pressure" trying this new drug....and I really wanted to be able to keep a level head about improvements or no improvements....because, believe me, I really want to see improvements. I am Df508/621+1G->T
This second one used to be called a splice site mutation, but apparently vertex decided to make it sound much more ominous and call it NO SYNTHESIS/Premature stop codon mutation (Class I).
In November, I was put on IVs for an exacerbation and reached a wonderful low of 37% FEV1. They kept me on IV anti-fungals through the new year when I then caught the croup and plummeted. Normally, my fev1 has hovered in the low 50's (for many years), but these past few months I just really had a hard time. The croup led to another incarceration with the discovery of an old friend coming back to visit....m. abscessus. lovely.
When I left the hospital I was blowing in the 1.4 liter range on my home meter and that promptly plummeted to 1.27, 1.24, 1.29 after a week and a half or so at home. I tell all of this just so you see my trend. The consensus was to treat the myco (I've always self treated that with diet and silver and it never came back after culturing for 13 years....until now @6 years later). We decided to try the kalydeco to see if it might offer any help in my immune response.
The few days beofre I started I was taking my fev1 all throughout the days and they would not budge. I stayed in the 1.30, 1.31, 1.32 range.....beofre treatment, after treatment, etc.
I started on February 24 in the morning.
February 24, 2012 FEV1...1.31
March 2, 2012 FEV1..........1.48 (before morning treatment...holy!)
March 7, 2012 FEV1...........1.57 (beofre morning treatment...holy!)
March 14, 2012 FEV1..........1.55 (but my peak flow was the highest I have done...40 points higher than last week)
I will say that that first week I measured every single day and it fluctuated but never went back down below 1.31, it just slowly crept up...Of course, since I don't have a gating mutation I have pretty much accepted that I might not have all of the "other" great things...weight gain, etc. This is all new territory.
First DAY: My stomach started gurgling within 3 hours (my stomach is normally very quiet). My mom heard it sitting across the room. By late afternoon, I begin to feel moisture in my nose (exactly like I had just done a nasal rinse) By 7 hours, I experienced a HORRIBLE stomach ache which lasted exactly 1 hour and was so painful I couldn't think. Within 1.5 hours, the stomach ache was gone and I was fine. A few hours later at dinner I started having a wet cough, which surprised my friends and my husband. I then proceeded to cough up 2 huge mouthfuls of crap...again, not my normal at all.
Day 2: Felt lousy and coughed up some very old crap in my bedtime treatment session (two plugs that were crazy dark)
Day 3- Day 4: Nothing different. feeling a bit run down.
Day 5 : I woke up with energy...my hubby thinks he saw it on day 3 and 4, but 5 was definitely a turning day for me. I notice a slight increase in smells and maybe taste.
Day 6: 1 hour after starting the drug, my right lung which is my problem lung, just started to "release" It felt less tight and airy...
Day 8: I feel horrible. I feel like plugs are pulling away and are somehow getting lodged..junky...awful. My doc tells me he believes abscessus is making this a little trickier. He says to quit taking daily pfts and just do weekly and focus on moving out the plugs. That night I coughed up three of the darkest most horrific looking plugs I have seen ever come out of me.All of the second week I felt better, but not like I was getting tons better and was quite shocked when my FEV1 was up again. It was decided I was having a significant response from the drug by my CF team.
Friday the 16th will be my 21st day. I have to say I still am having random lung pains in random places and have had asthma-y symptoms, but there is no denying that my numbers are up and I feel better (but not great).
I don't want to give anyone false hope. Keep in mind, I stick to a very strick anti-inflammatory diet which I KNOW gives me an advantage with response to a lot of different meds. I think I wanted to have days of bringing old plugs up, but was reminded by my doc my bronchiectasis are terrible looking, but are completely void of mucus...I have very little plugging which is a praise God for the anti-inflammatory diet. So, who knows how many more plugs I will really move...
As far as getting the drug, we really believed we were going to have to fight to try it...we were prepared to do anything to get a one month supply. And then, I got a call from CF Services that insurance had paid for it!!! PRAISE the LORD! But, they were sorry to say I had a $50. co-pay.....unbelieveable.