All I have to say about Atalurens update is my son who could of possibly benefited is again left out in the cold.  If and when it ever gets approved it will be too late for my son.

Michelle

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Everyone,

First, perhaps is a need for clarification.   Compassionate use actually means individual treatment IND.  If you remember from Dr. McNeil's talk -three things need to be in place for an individual treatment IND.  Company has to agree, patient has to agree and a physician has to agree.  

 The access programs areunder the vehical of treatment IND's and (at this time) the ataluren access program is  specifically for individuals who participated in the Phase II a and b studies.   The access program is not being done to appease anyone, rather  PTC agreed to the access program for participants in the Phase II a and b study because they believe it is the right thing to do as they seek approval for ataluren.

 At this time, it cannot be 'given' to everyone with a nonsense mutation.  It is not approved, therefore not available.  While there is an Individual treatment IND (sometimes referred to as compassionate use), this  type of approach is not sustainable and could not serve all of the patients with nonsense mutations around the world..   For this reason, the goal is to seek approval.

 

None of the organizations (big or small) has any ability to interfere with the approval process of ataluren or any other compound/biologic.  It is not a matter of attorneys and money.  FDA has an approval process in place.  It is long and painful and subject to barriers along the way.  We can all argue about the process, argue that FDA is risk adverse, that doing nothing is doing harm and that perhaps FDA needs to rethink benefit/risk and consider conditional approvals in rare disease especially. With some luck, some of this may be addressed as FDA legislation is up for revision and renewal in Fall, 2012.   There are any number of efforts and discussions underway focused on expediting the approval process and this will be included in our advocacy agenda for 2012.  At the moment, we have what we have in terms of FDA and have to work through the process.   I wish it was different.  

All of us want to see ataluren (and every other potential treatment -exon skipping compounds, utrophin upregulation, myostatin inhibition, replacement drugs for steroids, etc)  approved and access for patients with nonsense mutations. 

It is all frustrating and painful, but worth pursuing.  And we are doing just that.    All of us together.

Warm regards, Pat

 

Don't be too hopeful of that as an option, unless you're wealthy.  Off label use of drugs is not covered by some insurance policies, as it can be considered experimental.  Good discussion at this link relative to cancer.  Drugs developed with for rare conditions with limited markets are likely to be phenomenally expensive if and when they're approved for any disease. 

 

Insurance issues will be a huge hurdle to overcome.  Which highlights the importance of advocacy regarding health insurance reform, in addition to regulatory reform and NIH research budgets.

 

Bernardo A. Iriberri said:

I ask this because I have no doubt in my mind that if and when the drug is approved for any disease other than DMD I will do whatever it takes to get it as an off-label drug for my son and give it to him at the low dose that showed to be beneficial.

 

Thank you Keith. I can imagine that the off-label use is not going to be easy. Furthermore, I live in Argentina, so I don't even know if I would be able to get it here. I think that Genzyme's agreement with PTC for marketing ataluren outside the US is still valid for uses other than DMD. We'll see. Thanks again,

Bernardo

Keith Van Houten said:

Don't be too hopeful of that as an option, unless you're wealthy.  Off label use of drugs is not covered by some insurance policies, as it can be considered experimental.  Good discussion at this link relative to cancer.  Drugs developed with for rare conditions with limited markets are likely to be phenomenally expensive if and when they're approved for any disease. 

 

Insurance issues will be a huge hurdle to overcome.  Which highlights the importance of advocacy regarding health insurance reform, in addition to regulatory reform and NIH research budgets.

 

Bernardo A. Iriberri said:

I ask this because I have no doubt in my mind that if and when the drug is approved for any disease other than DMD I will do whatever it takes to get it as an off-label drug for my son and give it to him at the low dose that showed to be beneficial.

 

Thank you Pat and Sharon for your thorough replies.

First of all I want to point out that my first question ”where are the big organizations in the DMD-field” was addressed to everyone working for our boys – the FDA also.  

Ethic is a very popular word these days – in every situation involving human rights - and I think this is very real when it comes to Ataluren.  I do understand why Michelle is reacting the way she is: She is watching her son getting weaker and weaker, not allowed to use the drug that MIGHT help her son. A drug most researchers say will probably help best before the boy weakens too much.... For me, this is a question of ethic. I share Michelles frustration. A big hug to you Michelle. I think its harder for us who have older children to not  get access to the drug. Its heartbreaking, and some of the frustration goes out public.   

Having a boy with a stop, I have been following very closely the work PTC Therapeutics has done from day one.  Its my understanding that PTC went for full access of ptc 124 and that this is seldom done by the FDA without a phase 3 completed, which they did not do(they did 2a and 2b).  

I hope Pat or Sharon can help with some more info. Its always easier to relate to a case when  you know the facts, rather than speculation.

 

My questions are:

- What time in the process of a clinical trial, will it be possible to apply for ”compassionate use” ?(I thought it was when the safety data where good, and the drug showed benefit. (low dose Ataluren)

From Wikepedia: Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available. Drugs that are being scientifically tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. Being able to use one of these drugs when you are not in a clinical trial has many names, but is most commonly referred to as compassionate use.

 

-  Did PTC go for full access or for conditional access?  Or did they go for full access and were turned down due to lack of phase 3 data and then asked for conditional access. 

 

-  If so, why were they turned down?  The safety data is good so why was conditional access turned down? 

 

Best wishes to everyone

Berit 

I have posted that I have not seen a great improvement with Ataluren, not with intent to discourage, but with intent to encourage. Just wanted people to know that they are not missing out on a fabulous, amazing drug, but one that still has a long way to go.

In Ataluren's defense, Zach's decline became obvious when we went OFF drug for about a year after they terminated it. However, going back ON we have not noticed that it is slowing things down at all. Perhaps if he had stayed on the entire time things would be different. No one knows since the drug is still in experimentation and all boys progress differently.

My hope is that they will quickly approve the drug as is, and then PTC will have more freedom to "tweek" it or its dosages to make it work more effectively! That is the first step and then we will see what happens.

Truthfully it doesnt matter anymore whether Johnny received the Ataluren.  For him its too late and the damage is done.  Today he told me " I know I will die before you"  and I told him dont worry I will be with you soon after because I will not be able to stay without you. 

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