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Thread: Expanded Access it is law...for those in dire need of an investigational drug....

  1. #1
    Join Date
    Mar 2003

    Expanded Access it is law...for those in dire need of an investigational drug....

    Read how you may qualify from the CFRoundtable newsetter!

    For instance, in 2007 it became law that when a drug company is registering a clinical trial on, they are required to specify whether there is an expanded access program (EAP) for the investigational drug available for those who do not qualify for enrollment and how to obtain information about such access.

    Salt and Light,


  2. #2
    Super Moderator
    Join Date
    Sep 2016
    Thanks for posting Imogene.

    I wrote the article I just wanted to clarify that it's not the law to provide expanded access but to make information about the compassionate use/expanded access programs offered readily available to the public. In December 2016, President Obama signed the 21st Century Cures Act into law which states that a pharmaceutical company of an investigational drug must now make public and readily available its policies on evaluating requests for expanded access, such as posting such policies on a publicly available Internet website. The company must include a hyperlink or other reference to the clinical trial record containing information about the EAP.

    Vertex has had an expanded access program available for certain residual function mutations available for years but it wasn't known about my most CF doctors until this past October. Sadly, even with the passage of this law, Vertex isn’t complying and fails to make public the EAP available for residual function mutations. Although it appears they are….

    Appearances can be deceiving. This past February, in response to this law, Vertex added an expanded access section to their website. It gives you general information about expanded access programs. It also provides a link to and gives you instructions on how to find out about the EAPs they are offering. When you follow Vertex’s instructions, it will bring you to a dead end—an EAP that hasn’t been available in 5 years--since 2012. There is no mention of the residual function Expanded Access Program they currently have for those with residual function mutations. I’m in the process of following up with Vertex’s compliance department in regard to this matter. It sickens me to think people have suffered unnecessarily or even lost their lives because of lack of transparency of a compassionate use program that has been available for years.
    45 y/o with CF (R334W and DF508)

  3. #3
    Super Moderator
    Join Date
    Aug 2016
    I can't over emphasize how this program can really benefit some of us with off-label Residual Function mutations.
    Over the past years I've spoken with CFers who are unable to get Kalydeco.

    For some it was their insurance provider. Others had CF centers that didn't even want to try!

    If this program was more publicized maybe this would be different.

    I showed the article to my CF doctor last week. She was familiar with the program and told me they were in the process enrolling one of their patients. They have 10 patients with unapproved Reaidual Functions. They have been successful in getting Kalydeco for seven. The others have insurance issues. This program could obviously be of great help.

    The only reason I can think of why Vertex is so secretive about the program, is tat they may lose some potential study participants.

    Jeannie, keep up the good work!
    Last edited by stephen; 05-17-2017 at 05:02 PM.
    76 Y/O with CF (D1152H and G542X) and Broncheiectesis.
    FEV1 Low 40s%.
    Started Kalydeco March 2014, Switched to Symdeko March 2018 - and doing very well.

  4. #4
    Thanks JRicci and Stephen. You have both helped me so much and I am so grateful. Stephen alerted me to the possibility of getting Kalydeco and because of him I've been on it for 2 years! Just recently Kalydeco was approved for 23 different Residual function mutations, including mine.

    Thanks also to JRicci for the amazing article she wrote for CF Round Table. I had no idea that this was available and am disappointed to hear Vertex is not helping patients get VX-661 and other drugs early. Let me know if there is anything I can do.

  5. #5
    Super Moderator
    Join Date
    Sep 2016
    Ladybird- so good to hear from you! It's been awhile. I'll let you know what I find out about this whole EAP issue. I've been in contact with Vertex's Chief of Compliance and Senior Medical Director that deals with their EAPs. I've also been in touch with the FDA. I'm getting some answers but still waiting for a few things to be clarified.
    I'm so happy that the FDA approved Kalyceco for some residual function mutations!! Getting to the bottom of why the EAP wasn't publically available isn't as pressing of an issue since these patients will soon have access to Kalydeco, but I still want answers. CF research is ahead of the curve in precision medicine and is setting a critical precedent for the future of rare disease clinical trials and drug development. This is an issue that needs to be addressed so that it doesn't happen again in the future for CF and other serious and life-threatening diseases. Patients deserve to advocate for themselves and transparency of a compassionate use program is critical and may be life-saving.
    Last edited by jricci; 05-24-2017 at 11:31 PM.
    45 y/o with CF (R334W and DF508)

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