TreasureGoddess
Member
occupyjapan, WOW AND YAY AND WOOHOO! Glad you got the meds so quick and super glad you're already feeling benefits. 
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Post Your Orkambi Updates
- Thread starter Aboveallislove
- Start date
Went to clinic yesterday (Kaiser in Los Angeles) and the doctor was ready to fax the Rx to the specialty pharmacy. I was not ready to pay a 10% copay. They did not have any of the Vertex GPA information so I have no idea what the copay assistance will be. I had brought these enrollment forms to the appointment... http://www.vertexgps.com/pdf/GPS_Enrollment_Form.pdf?0702
The social worker is going to work on it today so hopefully we will here something sooner rather than later.
I had called Vertex GPS prior to going to the appointment. They can't help until they received paperwork from the doctor because they have to confirm that you qualify for the drug before they can help you.
The social worker is going to work on it today so hopefully we will here something sooner rather than later.
I had called Vertex GPS prior to going to the appointment. They can't help until they received paperwork from the doctor because they have to confirm that you qualify for the drug before they can help you.
K
kenna2
Guest
Just wanted to give everyone a heads up. I went to my CF clinic today and asked about getting a prescription for Orkambi and my doctor, told me that because I have liver disease (brought on by CF) this isn't a med he would recommend for me at the moment due to the risk of more damage Orkambi has been shown to cause. He said most doctors he's talked to are very hesitant to prescribe this med until they can see what it actually does to patients.
occupyjapan
New member
It's the Ivacaftor (the same drug as Kalydeco) that can cause liver issues. Lumacaftor, as I understand it, does not due to being metabolized slightly different (easier to break down, I think). The dose of Ivacaftor is slightly lower in Orkambi than in Kalydeco. G551D patients aren't having that many issues with Kalydeco, so I can't imagine this will cause even as many problems, due to the lower dosage. They can also change the dosage (from 2 pills twice a day to 2 pills once and 1 pill second dose or even 1 pill per dose) based on your liver function.
S
stephen
Guest
2005CFmom,
From my understanding, as long as Orkambi and Kalydeco are prescribed on label, co-pay assistance should be available.
For those of us fortunate to be getting it off label, the co-payments however can be considerable. If it were not for dramatic results the drug is providing, some of us would not have been so willing to absorb the high monthly cost, (5% in my case).
Also, like you, I don't understand why any doctor is reluctant to prescribe Orkambi or Kalydeco, on or off label. Compared to other drugs we're taking, the side effects of these new drugs are not as prevalent or severe. Their positive results can be astounding, enabling many patients to eliminate, or at least reduce, the need for other more dangerous drugs.
Why not at least try to see what Orikambi or Kalydeco can do. From what I've been reading, patients are seeing results in a matter of days. If the drug is not helping, stop it.
I'm seeing more and more how all CF centers are surely not equal. Some are proactive, trying to get their patients drugs off label and via Expanded Access programs, if they think it would help. Fortunately, the three adult CF centers I've had experiences with in the New York area have been proactive. I know this can mean additional paper work on their part both in setting up programs with drug manufactures and in "persuading" insurance providers to cover the drugs. However, it's horrible to think that some CF centers might be too lazy, or uninformed, to provide their patients with the best care that is available.
Good luck in getting a positive response to both co-payment assistance to Orikambi!
From my understanding, as long as Orkambi and Kalydeco are prescribed on label, co-pay assistance should be available.
For those of us fortunate to be getting it off label, the co-payments however can be considerable. If it were not for dramatic results the drug is providing, some of us would not have been so willing to absorb the high monthly cost, (5% in my case).
Also, like you, I don't understand why any doctor is reluctant to prescribe Orkambi or Kalydeco, on or off label. Compared to other drugs we're taking, the side effects of these new drugs are not as prevalent or severe. Their positive results can be astounding, enabling many patients to eliminate, or at least reduce, the need for other more dangerous drugs.
Why not at least try to see what Orikambi or Kalydeco can do. From what I've been reading, patients are seeing results in a matter of days. If the drug is not helping, stop it.
I'm seeing more and more how all CF centers are surely not equal. Some are proactive, trying to get their patients drugs off label and via Expanded Access programs, if they think it would help. Fortunately, the three adult CF centers I've had experiences with in the New York area have been proactive. I know this can mean additional paper work on their part both in setting up programs with drug manufactures and in "persuading" insurance providers to cover the drugs. However, it's horrible to think that some CF centers might be too lazy, or uninformed, to provide their patients with the best care that is available.
Good luck in getting a positive response to both co-payment assistance to Orikambi!
Kenna2, I’m not sure how severe your liver/lung involvement is and don’t mean to question your doctor’s decision; but I did want to make you aware of recommendations for those with hepatic impairment:Just wanted to give everyone a heads up. I went to my CF clinic today and asked about getting a prescription for Orkambi and my doctor, told me that because I have liver disease (brought on by CF) this isn't a med he would recommend for me at the moment due to the risk of more damage Orkambi has been shown to cause. He said most doctors he's talked to are very hesitant to prescribe this med until they can see what it actually does to patients.
Dosage Adjustment for Patients With Hepatic Impairment1
• No dose adjustment is necessary for patients with mild hepatic impairment (Child-Pugh Class A). A dose reduction to 2 tablets in the morning and 1 tablet in the evening (lumacaftor 600 mg/ivacaftor 375 mg total daily dose) is recommended for patients with moderate hepatic impairment (Child-Pugh Class B)
• Studies have not been conducted in patients with severe hepatic impairment (Child-Pugh Class C), but exposure is expected to be higher than in patients with moderate hepatic impairment. Therefore, use with caution at a maximum dose of 1 tablet in the morning and 1 tablet in the evening (lumacaftor 400 mg/ivacaftor 250 mg total daily dose), or less, in patients with severe hepatic impairment after weighing the risks and benefits of treatment
Did he mention what your Child-Pugh score was? Here’s a website that calculates your score based on different criteria.
http://www.fpnotebook.com/GI/Exam/ChldPghScr.htm
Maybe you could call your clinic and they can review this with you.
I also found this info. in prescribing information:
Among 6 patients with pre-existing cirrhosis and/or portal hypertension who received ORKAMBI, worsening liver function with increased ALT, AST, bilirubin, and
hepatic encephalopathy was observed in one patient. The event occurred within 5 days of the start of dosing and resolved following discontinuation of ORKAMBI [see
Warnings and Precautions (5.1, 5.2)].
Off Label for under the approved age?
Stephen,
Could Orkambi be prescribed off label for someone under the approved age? My son is 4 with double Delta and has learned to swallow pills already. We are very anxious for this new treatment to be available to him.
Stephen,
Could Orkambi be prescribed off label for someone under the approved age? My son is 4 with double Delta and has learned to swallow pills already. We are very anxious for this new treatment to be available to him.
2005CFmom,
From my understanding, as long as Orkambi and Kalydeco are prescribed on label, co-pay assistance should be available.
For those of us fortunate to be getting it off label, the co-payments however can be considerable. If it were not for dramatic results the drug is providing, some of us would not have been so willing to absorb the high monthly cost, (5% in my case).
Also, like you, I don't understand why any doctor is reluctant to prescribe Orkambi or Kalydeco, on or off label. Compared to other drugs we're taking, the side effects of these new drugs are not as prevalent or severe. Their positive results can be astounding, enabling many patients to eliminate, or at least reduce, the need for other more dangerous drugs.
Why not at least try to see what Orikambi or Kalydeco can do. From what I've been reading, patients are seeing results in a matter of days. If the drug is not helping, stop it.
I'm seeing more and more how all CF centers are surely not equal. Some are proactive, trying to get their patients drugs off label and via Expanded Access programs, if they think it would help. Fortunately, the three adult CF centers I've had experiences with in the New York area have been proactive. I know this can mean additional paper work on their part both in setting up programs with drug manufactures and in "persuading" insurance providers to cover the drugs. However, it's horrible to think that some CF centers might be too lazy, or uninformed, to provide their patients with the best care that is available.
Good luck in getting a positive response to both co-payment assistance to Orikambi!
S
stephen
Guest
jthomp,
I'm not a doctor, but age is definitely a legitimate reason for a doctor to not prescribe a drug off label.
My feeling is that if a patient was close to the approved age a doctor should consider prescribing it. Orkambi is approved for ages 12 and up. Maybe a doctor would stretch it down to 10. Four definitely seems much too low for now. Even if the approved low age was six, four still seems too young.
Everyone should keep in mind that if Orkambi is like Kalydeco, it may only be effective on lung problems, not digestive of sinus issues.
I'm not a doctor, but age is definitely a legitimate reason for a doctor to not prescribe a drug off label.
My feeling is that if a patient was close to the approved age a doctor should consider prescribing it. Orkambi is approved for ages 12 and up. Maybe a doctor would stretch it down to 10. Four definitely seems much too low for now. Even if the approved low age was six, four still seems too young.
Everyone should keep in mind that if Orkambi is like Kalydeco, it may only be effective on lung problems, not digestive of sinus issues.
2005CFmom,
From my understanding, as long as Orkambi and Kalydeco are prescribed on label, co-pay assistance should be...
...Good luck in getting a positive response to both co-payment assistance to Orikambi!
Thanks Stephen.
I spoke with Vertex GPS today and they are in the process of contacting insurance to figure out what the copayment will be. I should know by early next week what their assistance will be.
Aboveallislove
Super Moderator
Stephen,
Could Orkambi be prescribed off label for someone under the approved age? My son is 4 with double Delta and has learned to swallow pills already. We are very anxious for this new treatment to be available to him.
Just wanted to add to Stephen's thoughts on this as our son is 6 and like you we're very anxious. Drugs can be scripted off-label for age or "use" (i.e., mutation). In fact many of the CF drugs are off-label for age (i.e. Pulmozyme) because they can't get studies on lung function for young age and/or can't get study participants (our doctor explained this to use in scripting stuff the first time). With the Vertex drugs, though, they are so new the doctors might not be comfortable doing off label with age when it is a big disparity especially given the issues that have come up re cataracts for young ones. I had originally intended to request off label for our son as soon as approved but because they don't know the dosing for that age (that's what the study for the younger group is), and because of the newness, we are waiting for the study results. They are testing 6-11 now and that will be done in October. Not sure if results will be announced then but definitely early next year. Vertex is also starting a 2-5 study soon.
So you could try to get in the 2-5 study or once approved for 6-11, then I think the doctor would be open to scripting off-label (especially if your little one is 5 and/or close to 5 and especially if close to the weight in the 6-11 study--I just pulled and the study weight says: "Subjects who weigh > 15 kg without shoes at Screening Visit"
https://clinicaltrials.gov/ct2/show/NCT01897233?term=vx809&rank=5
With Kalydeco I know doctors were scripting for those under 6 when it was approved for 6-11. But there would be no co-pay assistance from Vertex so it would depend if you have insurance that covers (i.e. do you have a $60 mail order copay or a 20% copay which could be $$$$$).
AboveAllIsLove
Thanks Aboveallislove. It's encouraging to hear that the 6-11 yr. old study will be completed so quickly. Like you, most of Nathan's meds the doctors have him on (albuterol, pulmozyme) have not been studied under age 6. If it's coming soon, we can wait. In the back of my mind as a parent, I am constantly thinking that if I can just keep his lungs healthy enough until the new meds or "cure" is found, maybe he won't have to face the lung decline and struggles that those before him did. You hope help comes soon, before too much damage is done. He's currently in a clinical trial for those that have cultured pseudomonas, and has cleared it already. You just never know what CF is going to throw at you.
Thanks Aboveallislove. It's encouraging to hear that the 6-11 yr. old study will be completed so quickly. Like you, most of Nathan's meds the doctors have him on (albuterol, pulmozyme) have not been studied under age 6. If it's coming soon, we can wait. In the back of my mind as a parent, I am constantly thinking that if I can just keep his lungs healthy enough until the new meds or "cure" is found, maybe he won't have to face the lung decline and struggles that those before him did. You hope help comes soon, before too much damage is done. He's currently in a clinical trial for those that have cultured pseudomonas, and has cleared it already. You just never know what CF is going to throw at you.
Just wanted to add to Stephen's thoughts on this as our son is 6 and like you we're very anxious. Drugs can be scripted off-label for age or "use" (i.e., mutation). In fact many of the CF drugs are off-label for age (i.e. Pulmozyme) because they can't get studies on lung function for young age and/or can't get study participants (our doctor explained this to use in scripting stuff the first time). With the Vertex drugs, though, they are so new the doctors might not be comfortable doing off label with age when it is a big disparity especially given the issues that have come up re cataracts for young ones. I had originally intended to request off label for our son as soon as approved but because they don't know the dosing for that age (that's what the study for the younger group is), and because of the newness, we are waiting for the study results. They are testing 6-11 now and that will be done in October. Not sure if results will be announced then but definitely early next year. Vertex is also starting a 2-5 study soon.
So you could try to get in the 2-5 study or once approved for 6-11, then I think the doctor would be open to scripting off-label (especially if your little one is 5 and/or close to 5 and especially if close to the weight in the 6-11 study--I just pulled and the study weight says: "Subjects who weigh > 15 kg without shoes at Screening Visit"
https://clinicaltrials.gov/ct2/show/NCT01897233?term=vx809&rank=5
With Kalydeco I know doctors were scripting for those under 6 when it was approved for 6-11. But there would be no co-pay assistance from Vertex so it would depend if you have insurance that covers (i.e. do you have a $60 mail order copay or a 20% copay which could be $$$$$).
As far as the doctors that are reluctant to prescribe it - I went to clinic yesterday and my doctor said this drug has been very polarizing. Some doctors are very excited and think this should be an immediate new standard drug; others are unimpressed with the clinical trial results, basic science and insane price and are more reluctant. She seemed to be more in the middle and we both agreed that it was at least worth trying to see what happens. The side effects, as others here have said, are no more serious really than in any other drug we take. The bigger issue is the drug interactions - there are a lot. But still, some have had huge benefit and on average everybody got some benefit so it does seem worth trying if insurance will cooperate.
Diana Blue
New member
We are in Sonoma County, CA. Just had call this morning from CA Children's Services (state insurance) that Dr. Jill Abramson is reviewing Orkambi and will update us in 2-4 weeks. Wrote a letter to her this morning.
Always waiting. ugh.
Diana
Mom to Lawson, 18 dd 508, MAC
Always waiting. ugh.
Diana
Mom to Lawson, 18 dd 508, MAC
Someone from Vertex GPS called me earlier this afternoon to verify info on enrollment form. I'd filled out our portion on Thursday morning and our clinic sent the completed form to them and me a copy. The rep said he wasn't our normal rep, but was helping out because of overwhelming response from patients. Indicated they'd send the info to our insurance company and hopefully they'd respond in 1-3 days. So we're waiting.
Someone had asked me to relay my experience as someone on medicaid, so my update. I got a call last Monday saying they had to contact my provider and all that and that they'd call that in 2-3 days. A week later I hadn't gotten a call so I just called them myself. Turns out the drug is currently a plan exclusion, not surprising with the price and the fact that it was just approved. Vertex sent a prior authorization request to my doctor to see if they could get an override on that and set it up for appeal if necessary. So that's where it is now.
Heard back from Vertex GPS this morning. Was pleasantly surprised to hear it was approved by our insurance. Insurance's preferred pharmacy is not one on the list of distributors, thank goodness. We've been assigned Walgreens Specialty Pharmacy and they're supposed to contact me within a week. We're supposed to call Vertex GPS is the copay is more than what they quoted ($15/per script)
The Vertex rep did tell me they signed us up for copay assistance and due to the script charge, which I thought was odd being we've met our out of pocket max for the year. This is because it's a specialty drug that is noncovered and nonformulary according to the insurance company's drug list. Odd because it IS listed on as a specialty drug, no PA required. The rep said the reason for the $15 copay is due to the noncovered/nonformulary status and originally they were trying to charge it under those terms with a 50% copay, which would've been about $10,000. Yikes!
So hopefully we'll hear back soon from the pharmacy. Unfortunately the state I live in only allows for privately owned pharmacies, so we don't have name brand pharmacies. So it can either be shipped to our home/office, or I can go over the river to another state (just a few miles), which has a local Walgreens pharmacy.
So, pleasantly surprised as I expected more of a struggle to get approval.
The Vertex rep did tell me they signed us up for copay assistance and due to the script charge, which I thought was odd being we've met our out of pocket max for the year. This is because it's a specialty drug that is noncovered and nonformulary according to the insurance company's drug list. Odd because it IS listed on as a specialty drug, no PA required. The rep said the reason for the $15 copay is due to the noncovered/nonformulary status and originally they were trying to charge it under those terms with a 50% copay, which would've been about $10,000. Yikes!
So hopefully we'll hear back soon from the pharmacy. Unfortunately the state I live in only allows for privately owned pharmacies, so we don't have name brand pharmacies. So it can either be shipped to our home/office, or I can go over the river to another state (just a few miles), which has a local Walgreens pharmacy.
So, pleasantly surprised as I expected more of a struggle to get approval.
Aboveallislove
Super Moderator
Yippe, thanks for sharing..personally I'd l Be any and all little tidbits of any changes you notice once started!
Yippe, thanks for sharing..personally I'd l Be any and all little tidbits of any changes you notice once started!
Plan to do so. Just figured I'd update our process of getting the drug. Quite frankly it seems like we're playing a game with our insurance in which we're expected to know the rules and the rules keep changing.