Has anyone considered contacting Vertex about this? They are very nice I just hesitate to do it myself because I don't want to take their time away from drug development. I'm just saying it may help to get their perspective before a lot of time is put into this. I personally always have to remind myself that things aren't always as they seem. And as much as we think we know everything, we don't. We have to remember to be appreciative of what Vertex is doing for the cf community. Not in a long time or maybe ever has a company invested in our community like they are. Always remember that from the day that Vertex patented V770, it has only 10 years to recoup its money before it goes generic. Also, the CFF VP who is in charge of legislation, Mary B. Dwight, may be a good perspective too. Perhaps they are endorsing this but have not sent an email blast yet.
Here's a little perspective, when people were discussing why doctors and possibly even Vertex would be discouraging off label use of Kalydeco, I asked my family member why this could possibly be so...wouldn't they want the additional customer, money, good publicity? My family member who works in the pharmaceutical industry told me that the reason was clear. It is because Vertex is so grateful (and that the CF community should be too) for how fast the FDA approved the medication and that to use the medication so quickly off label would be to go against what the FDA had permitted and most importantly the implications that this could have. That's there's a lot of dollars and sense since the process of drug dev is so expensive. That it's important to remember how fast a drug company can decide that development of a drug no longer makes financial sense and to never forget that we are only 70,000 people with regards to recruitment and usage after approval. The FDA approved it with the expectation that specifically DF508 would not use it. It considerably complicates the process when patients breaks the rules and uses a medication even though it is not approved for them. Therefore the FDA raises the bar to include population that wasn't originally designed for because they know that the cf community is likely to break the rules and acquire it anyway. I didn't have much of a response. Thanked the person for sharing their perspective and moved on.
The next week the person went to the American Chemical Society meeting in CA that included a talk by Vertex that said this very same thing.
Additionally, we can't forget that in very recent times CF clinical trials have had issues with recruitment. Hence Beth Sufian went to bat for people on disability to not have income from clinical trials count towards their annual income and disqualify disability payments. Proposing the Ph. 2 participants could stay on drug would disqualify from them from Ph. 3 trials as it is currently set up.
The next weekend I went to a CF education day where I talked with someone from Vertex. I asked the person why they could possibly not be encouraging the use of their medication in off-label-use especially if it was showing good results. The person explained that the process of FDA approval makes or breaks whether a drug gets to market 1) show to the FDA that the cf market still needs another drug that Kalydeco alone isn't enough and 2) that we can listen and follow rules, that Kalydeco was fast tracked and that we can only be hopeful that this could happen again with their future drugs.
The "shall do no harm" argument cannot be directed at this trial alone, since many drugs save lives. I know it's difficult to write without a bias. I just feel it's important when we speak of Vertex to be careful to show no ill will.
As someone who declined from the end of Vertex 770 Ph. 2 and the open label because I was one of the unlucky folks to get placebo in Ph. 3 I feel it was doing my part. Additionally, there was a lot of lag time between Ph. 2 and Ph. 3 for me. This is all part of getting medications to us. I have said I would be willing to lobby against cfers having to do placebo for Ph. 3 but with talking with the above mentioned people about this I realize it is unrealistic to believe that the placebo data could be collected from the patient population who is not taking the drug in as scientific a way to illicit the data the way the trial does. And placebo data IS needed in order to scientifically prove that a medication has a major impact on a person's health. Let's face it medications don't go to market if they only help a little; they need to show clinical significance.
If anyone finds out why the CFF hasn't endorsed Advancing Breakthrough Therapies for Patients Act I'd be interested in finding out. Or maybe they have but haven't sent out an email?
Sadly through having these discussions with my relative who keeps up with all thing cf research related, sees my decline and loves me very much and wants a cure for cf just as much as we all do...says now can understand why pharma. companies may want to shy away from drug dev for the cf market as our righteous behavior can be seen as a stumbling block to the process.
Our closeness, and small size makes us unusual - let's make sure all of our plays are smart and not nearsighted.
P.S. Mandi, I trust you won't take this personally. You are a very bright person and I know you appreciate my perspective. I hope it just gives you another side of the coin to consider in your project going forward. Keep us posted!
Here's a little perspective, when people were discussing why doctors and possibly even Vertex would be discouraging off label use of Kalydeco, I asked my family member why this could possibly be so...wouldn't they want the additional customer, money, good publicity? My family member who works in the pharmaceutical industry told me that the reason was clear. It is because Vertex is so grateful (and that the CF community should be too) for how fast the FDA approved the medication and that to use the medication so quickly off label would be to go against what the FDA had permitted and most importantly the implications that this could have. That's there's a lot of dollars and sense since the process of drug dev is so expensive. That it's important to remember how fast a drug company can decide that development of a drug no longer makes financial sense and to never forget that we are only 70,000 people with regards to recruitment and usage after approval. The FDA approved it with the expectation that specifically DF508 would not use it. It considerably complicates the process when patients breaks the rules and uses a medication even though it is not approved for them. Therefore the FDA raises the bar to include population that wasn't originally designed for because they know that the cf community is likely to break the rules and acquire it anyway. I didn't have much of a response. Thanked the person for sharing their perspective and moved on.
The next week the person went to the American Chemical Society meeting in CA that included a talk by Vertex that said this very same thing.
Additionally, we can't forget that in very recent times CF clinical trials have had issues with recruitment. Hence Beth Sufian went to bat for people on disability to not have income from clinical trials count towards their annual income and disqualify disability payments. Proposing the Ph. 2 participants could stay on drug would disqualify from them from Ph. 3 trials as it is currently set up.
The next weekend I went to a CF education day where I talked with someone from Vertex. I asked the person why they could possibly not be encouraging the use of their medication in off-label-use especially if it was showing good results. The person explained that the process of FDA approval makes or breaks whether a drug gets to market 1) show to the FDA that the cf market still needs another drug that Kalydeco alone isn't enough and 2) that we can listen and follow rules, that Kalydeco was fast tracked and that we can only be hopeful that this could happen again with their future drugs.
The "shall do no harm" argument cannot be directed at this trial alone, since many drugs save lives. I know it's difficult to write without a bias. I just feel it's important when we speak of Vertex to be careful to show no ill will.
As someone who declined from the end of Vertex 770 Ph. 2 and the open label because I was one of the unlucky folks to get placebo in Ph. 3 I feel it was doing my part. Additionally, there was a lot of lag time between Ph. 2 and Ph. 3 for me. This is all part of getting medications to us. I have said I would be willing to lobby against cfers having to do placebo for Ph. 3 but with talking with the above mentioned people about this I realize it is unrealistic to believe that the placebo data could be collected from the patient population who is not taking the drug in as scientific a way to illicit the data the way the trial does. And placebo data IS needed in order to scientifically prove that a medication has a major impact on a person's health. Let's face it medications don't go to market if they only help a little; they need to show clinical significance.
If anyone finds out why the CFF hasn't endorsed Advancing Breakthrough Therapies for Patients Act I'd be interested in finding out. Or maybe they have but haven't sent out an email?
Sadly through having these discussions with my relative who keeps up with all thing cf research related, sees my decline and loves me very much and wants a cure for cf just as much as we all do...says now can understand why pharma. companies may want to shy away from drug dev for the cf market as our righteous behavior can be seen as a stumbling block to the process.
Our closeness, and small size makes us unusual - let's make sure all of our plays are smart and not nearsighted.
P.S. Mandi, I trust you won't take this personally. You are a very bright person and I know you appreciate my perspective. I hope it just gives you another side of the coin to consider in your project going forward. Keep us posted!