Abigail Alliance

[Guest guest]

CBS Sunday Morning had a well balanced report Nov 13 called "On Trial", about the lack of access to clinical trials by many dying patients, as well as the extremely limited or totally lacking "compassionate access" programs for new drugs in development. The Abigail Alliance was the focus of this report. The group was formed by families following the deaths of cancer patients who were denied access to investigational drugs either through a clinical trial or compassionate access. The positions shared by the FDA, Pharma, and others (such as the American Cancer Society) in opposition to the Abigail Alliance included apparently reasonable concerns, such as about the ability to recruit patients for clinical trials if compassionate access were broadly available early on (after the Phase I dosing/safety trial was completed), ethical concerns ("first do no harm"), concerns about raising false hopes in dying patients, and the scientific sanctity of double blind placebo controlled studies.

The position of the Abigail Alliance is that dying patients, who have nothing to lose, should be allowed to decide if they wish to take a risk, even if the potential benefit may be a long shot.

I was particularly impressed by an oncologist at MD Cancer Center who spoke on behalf of the Abigail Alliance and addressed the scientific concerns of the opposition. He noted that the FDA (in terms of safety concerns during the drug approval process) makes no distinction between patients who are terminally ill and clearly have no reasonable life expectancy, and those who are suffering from possibly painful or debilitating, but not life threatening conditions. He argues that for those who are almost certainly going to die within months, the "risk/benefit" calculation is virtually meaningless. He also questioned the need for double blind. placebo controlled studies in situations where well documented terminal illnesses are involved where either the natural outcome, or the outcome with conventional treatments, is also well documented. In those cases, he argues, clinical trials should be able to be conducted where all the patients who chose to participate in the trial can

receive the investigation drug because it will be reasonably clear from the previous data whether the course of the patients being treated investigationally represents a significant improvement or not over those being treated with other therapies. And in terms of compassionate access for those who don't qualify to participate in the clinical trial, the decision to accept the risk through compassionate access should be left to the informed patient, assuming they have no other reasonable option, since they obviously are going to die soon anyway.

I thought about this issue in terms of AIDS drugs, and particularly vaccines. While AIDS is theoretically no longer the death sentence it once was, and we currently have many useful HIV drugs, they are not being distributed to most of those in the world who need them. In other words, most of those living with HIV, especially those with advanced disease, are still condemned to die. The issue with HIV drugs should therefore be mainly one of access to currently approved medications, although for those who have run out of drug options and are rapidly failing, the ability to access investigational drugs again comes into play. On the other hand, with millions still dying and not receiving drug therapy, therapeutic vaccine development has been, I think needlessly, slowed by "safety concerns". For example, I understand that the Phase I Dermavir trial have been delayed for three years for "safety concerns" (I believe the finally approved-for-trial version had the integrase gene removed, a

gene that was part of the original promising primate studies and which may potentially compromise its effectiveness in the human trials). We really do need a Manhattan Project for HIV/AIDS where every available avenue is being explored. Those who have clearly run out of options should be allowed to volunteer, with informed consent, for promising therapies, and with millions still facing death without treatment, vaccine development needs to proceed with much more urgency and less timidity.

The Abigail Alliance currently has a lawsuit pending against the FDA to expand compassionate access. The link to the Abigail Alliance is

www.abigail-alliance.org

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[Guest guest]

Yeah, I saw the program and loved it,. I sent them an email since I am also working on the same issues (see www.salvagetherapies.org)

Butch Kara wrote:

CBS Sunday Morning had a well balanced report Nov 13 called "On Trial", about the lack of access to clinical trials by many dying patients, as well as the extremely limited or totally lacking "compassionate access" programs for new drugs in development. The Abigail Alliance was the focus of this report. The group was formed by families following the deaths of cancer patients who were denied access to investigational drugs either through a clinical trial or compassionate access. The positions shared by the FDA, Pharma, and others (such as the American Cancer Society) in opposition to the Abigail Alliance included apparently reasonable concerns, such as about the ability to recruit patients for clinical trials if compassionate access were broadly available early on (after the Phase I dosing/safety trial was completed), ethical concerns ("first do no harm"), concerns about raising false hopes in dying patients, and the scientific sanctity of double blind placebo controlled studies.

The position of the Abigail Alliance is that dying patients, who have nothing to lose, should be allowed to decide if they wish to take a risk, even if the potential benefit may be a long shot.

I was particularly impressed by an oncologist at MD Cancer Center who spoke on behalf of the Abigail Alliance and addressed the scientific concerns of the opposition. He noted that the FDA (in terms of safety concerns during the drug approval process) makes no distinction between patients who are terminally ill and clearly have no reasonable life expectancy, and those who are suffering from possibly painful or debilitating, but not life threatening conditions. He argues that for those who are almost certainly going to die within months, the "risk/benefit" calculation is virtually meaningless. He also questioned the need for double blind. placebo controlled studies in situations where well documented terminal illnesses are involved where either the natural outcome, or the outcome with conventional treatments, is also well documented. In those cases, he argues, clinical trials should be able to be conducted where all the patients who chose to participate in the trial can

receive the investigation drug because it will be reasonably clear from the previous data whether the course of the patients being treated investigationally represents a significant improvement or not over those being treated with other therapies. And in terms of compassionate access for those who don't qualify to participate in the clinical trial, the decision to accept the risk through compassionate access should be left to the informed patient, assuming they have no other reasonable option, since they obviously are going to die soon anyway.

I thought about this issue in terms of AIDS drugs, and particularly vaccines. While AIDS is theoretically no longer the death sentence it once was, and we currently have many useful HIV drugs, they are not being distributed to most of those in the world who need them. In other words, most of those living with HIV, especially those with advanced disease, are still condemned to die. The issue with HIV drugs should therefore be mainly one of access to currently approved medications, although for those who have run out of drug options and are rapidly failing, the ability to access investigational drugs again comes into play. On the other hand, with millions still dying and not receiving drug therapy, therapeutic vaccine development has been, I think needlessly, slowed by "safety concerns". For example, I understand that the Phase I Dermavir trial have been delayed for three years for "safety concerns" (I believe the finally approved-for-trial version had the integrase gene removed, a

gene that was part of the original promising primate studies and which may potentially compromise its effectiveness in the human trials). We really do need a Manhattan Project for HIV/AIDS where every available avenue is being explored. Those who have clearly run out of options should be allowed to volunteer, with informed consent, for promising therapies, and with millions still facing death without treatment, vaccine development needs to proceed with much more urgency and less timidity.

The Abigail Alliance currently has a lawsuit pending against the FDA to expand compassionate access. The link to the Abigail Alliance is

www.abigail-alliance.org

Yahoo! FareChase - Search multiple travel sites in one click. Regards, VergelLecture Dates: Long Beach Aug 31, Philadelphia Sept 15, NY Oct 13, LA Sept 21 & 22, Salt Lake- Nov 5, AIDS Nutritionist Conf- DC Sept 15-17, El Paso Oct 10, San Francisco Oct 26, POZ Cruise- Miami- Oct 15-21, Houston Sept 29, Austin Nov 1, Baton Rouge Nov 9, New Orleans Nov 10, Mexico NATAF conf Nov 28-29DisclaimerThis information (and any accompanying printed material) is not intended to replace the attention or advice of a physician or other health care professional. Anyone who wishes to embark on any dietary, drug, exercise, or other lifestyle change intended to prevent or treat a specific disease or condition should first consult with and seek clearance from a qualified health care professional