Setting the Stage
On Dec. 20, 2012, an advisory committee convened by the Food and Drug Administration (FDA) heard presentations and public testimony and discussed the evidence for the safety and efficacy of rintatolimod (Ampligen) for treating CFS. Background on the drug, its long route leading to this public meeting and the issues to be considered by the advisory committee and FDA before marketing approval is given are covered here on Research1st in a post titled, “FDA Set to Review Ampligen Application”: http://www.research1st.com/2012/12/04/fda-ampligen-review/. Background materials posted by the agency for FDA and the drug’s manufacturer, Hemispherx Biopharma, were described and linked from this Research1st post: http://bit.ly/FDA-background-materials.
The public session was the first such FDA advisory committee meeting to consider a specific treatment for CFS and the tightly structured format was new to most participants and observers from outside the agency. Seated at the front of the large room at FDA’s White Oak campus at a U-shaped table were members of the Arthritis Advisory Committee, including three regular voting members, 11 temporary voting members, one non-voting industry representative, five non-voting FDA participants and the designated federal official, an FDA employee. The temporary voting member of the panel appointed as the patient representative was Alaine M. Perry, MPH. Ms. Perry is known to the CFS community as the federal representative to the Department of Health and Human Services CFS Advisory Committee (CFSAC) from the Centers for Medicare and Medicaid Services. In her introduction, Ms. Perry made her first public disclosure that she has had CFS for more than 20 years and that she represented only herself on the panel and not the agency she represents at CFSAC meetings. To the left of the center table were two rows of representatives from Hemispherx Biopharma, the manufacturer of Ampligen. On the right, FDA staff. Members of the public were separated from these three groups by a red rope. There were about 100 people in attendance and the full session was broadcast live over the web by FDA. The meeting recording, minutes and a transcript will be archived by the FDA, along with slide presentations shown by the manufacturer and the FDA.
The morning session began with an overview by Dr. Theresa Michele of the FDA’s Center for Drug Evaluation and Research. She emphasized FDA’s recognition of CFS as a serious disease with no approved therapies and acknowledged the strong public interest in the day’s topic as evidenced by written testimony submitted to the record by more than 700 people. She reviewed the agenda and four questions that the committee would be voting on during the afternoon session. Dr. Michele noted the first question about drug safety was somewhat unusual and that it originated with agency’s concern about the reliability of the safety data submitted by Hemispherx.
Presentations and Discussion
Dr. William Carter, chairman and CEO of Hemispherx Biopharma, led the series of presentations that outlined the two controlled and seven open studies of Ampligen initiated by the company. He committed to “complete clarity and transparency” in presenting data about the safe and effective use of Ampligen by 589 unique persons with CFS. Dr. Carter’s information was supplemented by other speakers on behalf of Hemispherx, including Dr. Lucinda Bateman, a physician who participated in the “AMP 516″ controlled study of Ampligen and who administers the drug to selected CFS patients in her Salt Lake City clinic through a compassionate use program approved by FDA called AMP 511. Robert Miller, a well-known patient advocate who has had CFS since 1982, also presented information on behalf of the company, reporting the positive response to the drug that he experienced during two courses of treatment. The panel of speakers emphasized the safe use of a similar compound as an adjuvant in the human papillomavirus vaccine, the unmet medical need represented by CFS, the FDA’s transfer of the Ampligen application to five divisions over the 22-year development period and data showing Ampligen led to improved time on a treadmill test and reduced use of other medications compared to placebo. Questions from the committee to Hemispherx representatives followed; Dr. Carter was forceful in his responses that the company had met the burden of proof. [Note: Hemispherx's slide presentation can be viewed here: http://1.usa.gov/WAeOSJ]
Two members of the FDA’s internal review team then reviewed the agency’s analysis of the Ampligen clinical program. Their presentations drew attention to several areas in which the agency’s interpretation of data differed from the company’s. FDA speakers traced the regulatory review process beginning with the company’s first request to study the drug in 1990. They focused on missing or conflicting data submitted by the manufacturer, data analyses planned by Hemispherx (but not conducted) that reduced the differences between the group treated with Ampligen and those who received placebo, and concerns raised by study conduct issues documented at various time points. Again, committee members’ questions followed the FDA presentations. [Note: FDA's slide deck presentation can be viewed here: http://1.usa.gov/SMTU4J]
After a short lunch break, the committee heard compelling testimony from 30 members of the public, many of whom had received substantial benefit from Ampligen treatment or had family members who responded to the therapy. Others spoke to the desire among patients to get access to the drug in the absence of other primary therapies. Testimony was limited to three minutes per witness and time limits were strictly observed. Three videotaped testimonies were permitted for individuals unable to travel to the meeting. Only one witness, Dr. Sidney Wolfe of the Public Citizen Health Research Group, specifically requested that FDA decline the application for drug approval. The CFIDS Association’s testimony, which I delivered, focused on the unmet need for safe and effective treatment, the burden of illness imposed on the individual and the community and personal risk and benefit assessment made by every CFS patient every day in the absence of specific treatment.
Before committee discussion and votes, Dr. Michele reviewed the committee’s charge and the regulatory process. She again conveyed the agency’s understanding of the “life-destroying” nature of CFS and reiterated the readiness to engage with drug developers to foster safe and effective treatments for CFS. In response to questions from the committee, she described the meaning of “safe” and “effective” in regulatory terms as well as accelerated approval mechanisms that helped to frame the committee’s response to six topics defined by the FDA, four of which would be subject to a vote. She emphasized that the approval standard was “adequate well-controlled studies,” not individual accounts. She noted that approvals of immunomodulatory drugs are generally based on studies that involve thousands of participants, rather than hundreds.
Votes Cast
The discussion about the evidence presented to evaluate safety and efficacy centered on the company’s presentation of data analyses other than those planned as part of the study, exercise testing protocols used to measure improvement and side effects of treatment based on case reports and adverse events summaries filed by the company. Votes revealed ambivalence about the strength of the data and the conclusions that could be confidently drawn. The member-by-member voting tally is presented below. In all, the majority of committee members recommended that another larger study be conducted before approving the drug. Individual committee members suggested various enhancements to future study design to help clarify the drug’s effect, such as:
- Define “adverse events” and “serious adverse events” at the outset of the study so that differences can be detected between those treated with the drug and those receiving placebo, as well as the characteristic symptoms of the disease itself.
- Identify subgroups of responders to the drug and focus on them using “enrichment” study protocols.
- Select a different endpoint than time on treadmill in light of difficulties these studies had establishing a tolerable exercise testing protocol and which cutoffs were clinically meaningful.
- Choose a different endpoint than Karnofsky Performance Score. The 10-point improvement shown in only one of the two pivotal studies may be substantial to individual patients, but is a challenging statistical measure.
- Gain a better understanding of the effect of placebo in this population, a group of people that suffers isolation and medical neglect — being enrolled in a study and receiving regular medical attention as part of the study may have a therapeutic effect in itself that made the drug’s effect harder to demonstrate.
- Focus on securing approval for severely affected individuals, rather than the wider population that meets CFS criteria
The vote on the final question, “Has the applicant provided sufficient efficacy and safety data to support marketing of Ampligen for the treatment of CFS?” ended with five votes “yes” and eight votes “no.” Comments that followed the vote reflected struggle with the decision by most panel members. Those voting against the measure voiced concern about the weak data showing the drug to be effective and the lapses in data submitted to show safety. Members who supported approval indicated that post-marketing studies could be required to resolve outstanding concerns. After all the votes were tallied, the non-voting industry representative, Brian Kotzin, M.D., of Amgen, offered his perspective that the inadequacies of trial design and irregularities in the data didn’t measure up to the industry standard for showing safety and efficacy and that he hoped a subset of patients would be studied further. Dr. Michele was the last to speak before adjournment. She summarized the “very difficult” decision ahead of the agency and commended the panel and the public for their input which would be strongly considered in the final assessment of whether or not to approve Ampligen as the first treatment for CFS. She took the final opportunity to emphasize the agency’s active interest in working with sponsors to make safe and effective drugs available to CFS patients.
Potential Outcomes
FDA will render a decision on Hemispherx’s request for marketing approval on or around Feb. 2, 2013, six months from the date the manufacturer’s application was accepted by FDA. FDA is not bound to act in accordance with the advisory committee’s recommendation, although it is unusual for the agency to depart from advisory committee guidance. The presentations by Hemispherx representatives and public testimony seemed to make a strong case for the patient community’s willingness to tolerate risks of side effects that mimicked the disease symptoms themselves, but the committee did not seem persuaded that the trials demonstrated clear and sufficient benefit across large enough numbers of patients to warrant approval for at least one million CFS patients and other patient groups that might try to gain off-label access for other conditions.
If the FDA follows the advisory committee’s recommendation and does not approve the drug for marketing, the next move will be Hemipsherx’s. The company can try to raise capital to conduct a third controlled study, shaping its study design to address concerns expressed by FDA in the briefing documents and by committee members through their votes and discussion. In a press release issued the day after the meeting, Hemispherx stated that it “will seek to do everything necessary to confirm in a scientifically rigorous manner that Ampligen® is a safe and effective treatment for CFS.” Regardless of the FDA’s decision on this application, hopefully access to the drug will be provided by the company to those who have benefitted from it.
Media Coverage
In the days leading up to the public meeting and vote, news stories focused on the stock value and prospects for investors to gain or lose based on the critical assessment of data reflected in the FDA’s pre-briefing materials.
- “FDA questions Hemispherx’s fatigue drug,” by Jennifer Corbett Dooren, Wall Street Journal (Dec. 18, 2012)
- “Doubts raised by chronic fatigue drug,” by David Pittman, MedPage Today (Dec. 18, 2012)
- “Hemispherx lied to investors and SEC about Ampligen,” by Adam Feurstein, The Street (Dec. 18, 2012)
- “Hemispherx’s unsurprising 45% plunge,” by David Williamson and Max Macaluso, The Motley Fool (Dec. 18, 2012)
- “Hemispherx chronic fatigue drug scrutinized ahead of panel review,” by Brett Chase, Minyanville (Dec. 18, 2012)
- “Hemispherx faces FDA day,” by EnhydrisPECorp, Seeking Alpha (Dec. 19, 2012)
- “Bronstein, Gewirtz & Grossman LLC announces investigation of Hemispherx Biopharma,” press release issued Dec. 19, 2012
- “FDA panel considers first drug for CFS,” by Steven Reinberg, HealthDay/U.S. News & World Report (Dec. 20, 2012; before meeting)
After the meeting, news stories focused on the vote and the mixed data on efficacy that failed to win the committee’s endorsement for effectiveness and overall approval.
- “FDA panel nixes Hemipsherx CFS drug,” by David Pittman, MedPage Today (Dec. 2o, 2012)
- “Panel rejects drug for CFS,” by Jennifer Corbett Dooren, Wall Street Journal (Dec. 20, 2012)
- “Hemispherx soars then plunges as FDA panel votes against Ampligen approval,” by Scott Rubin, Benzinga (Dec. 20, 2012)
- “FDA panel nixes Hemispherx’s Ampligen drug,” by John George, Philadelphia Business Inquirer (Dec. 20, 2012)
- “FDA panel recommends against first drug for CFS,” by Steven Reinberg, HealthDay/U.S. News & World Report (Dec. 21, 2012)
- “Rintatolimod for chronic fatigue gets panel’s ‘no’ vote,” by Miriam E. Tucker, Medscape Today (Dec. 21, 2012)
- “Panel rejects experimental CFS drug,” by Heidi Ledford, Nature News (Dec. 21, 2012)
- “FDA advisory gives negative opinion on Hemipsherx’s Ampligen,” The Pharma Letter (Dec. 27, 2012)
- “Effective therapy needed for CFS patients,” by Nancy Klimas, M.D., released by NOVA Southeastern University to News-Medical.net (Jan. 25, 2013)
(Note: The article posted to Medscape Today provides a good overview of the issues raised at the meeting and the outcome. Free registration may be required to access it.)
[Update: In the weeks since the advisory committee meeting, interested individuals have launched a petition seeking approval of Ampligen and are conducting other activities to influence the final outcome. Press releases and financial and legal sector media outlets have reported that some Hemispherx stockholders are considering or have taken legal action against the company.]
Member-by-Member Vote
(R = regular voting member of the Arthritis Advisory Committee; T = Temporary voting member of the Arthritis Advisory Committee)
|
|
Vote 1 |
Vote 2 |
Vote 3 |
Vote 4 |
|
Member |
Is there substantial evidence of efficacy? |
Has safety been adequately assessed? |
Is the safety profile adequate for approval? |
Do the safety and efficacy data support approval? |
|
Lisa Gualtieri, PhD, ScM (R) |
No |
No |
No |
No |
|
Robert Lahita, MD, PhD (R) |
Left early; did not vote |
|||
|
I Jon Russell, MD, PhD (R) |
No |
Yes |
Yes |
No |
|
|
|
|
|
|
|
Larry Borish, MD (T) |
Yes |
No |
Yes |
Yes |
|
Lenore Buckley, MD, PhD (T) |
No |
No |
No |
No |
|
Ralph D’Agostino, Sr., PhD (T) |
No |
No |
No |
No |
|
Jacqueline Gardner, MPH, PhD (T) |
No |
No |
Yes |
No |
|
Sean Hennessy, PharmD, PhD (T) |
No |
No |
Yes |
Yes |
|
Anthony Komaroff, MD, MA (T) |
No |
No |
No |
No |
|
Gailen Marshall, Jr., MD, PhD (T) |
Yes |
Yes |
Yes |
Yes |
|
Alaine Perry, MPH (T) |
Yes |
Yes |
Yes |
Yes |
|
Matthew Rudorfer, MD (T) |
Yes |
Yes |
Yes |
Yes |
|
Elizabeth Unger, PhD, MD (T) |
No |
No |
Yes |
No |
|
James Ware, PhD (T) |
No |
No |
No |
No |
|
FINAL VOTE |
No: 9 |
No: 9 |
No: 5 |
No: 8 |
K. Kimberly McCleary is the CFIDS Association of America’s chief staff executive.
This article was updated most recently on Jan. 25, 2013.
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Thank you for the excellent report on this important meeting.
While it is disappointing to learn that the search for the first CFS treatment will probably heve to continue, it is gratifying to see that the FDA is approaching it’s decision with plenty of sunlight on the process. I hope the final decision follows the science and doesn’t drift off into politics.
Thank you, Phillipe. Regardless of the outcome, the meeting focused a great deal of attention on the severity of CFS, the impact the disease has on the lives of every patient and their loved ones, and the need for more effective treatments than are currently available.
Thanks Kim.
I agree completely.
By the way, I note that several of the news articles at the end of the report gave the Advisory Panel vote as 8 to 5. Perhaps someone changed their vote from no to yes? Can you clarify the official number?
Thanks for catching the error, Phillipe. I’ve corrected the post to reflect the actual vote on the final question — 5 in favor of approval (Borish, Hennessy, Marshall, Perry and Rudorfer) and 8 against (Gualtieri, Russell, Buckley, D’Agostino, Gardiner, Komaroff, Unger and Ware).
I am conserned for Seniors over 60 with 20 or more years with disease me/cfids and yes the extreme effect on the lifestyle and losses both mentaly and physically. Each uniquely tornapart, diagnosed (mis), treated desperately by well meaning physicians to very small good account and at times wildly causing perhaps a kind of pts along the way. This drug will it be understood by clear standards aplicable to weight size and gender and case history for past and present breakdown of physiological conditions? Each within this age group must surely have, as I have now, added many break downs of various systoms with in and overall body. How can human aging process and certain life experience (unique to each) not of course have reacted to the extreme “free radicals” because of the weakened immune system. This is only one or two thoughts that I have on the matter. Perhaps you can already protect us from being put through the trails of experimenting once again on all of the population as though with some cookie cutter type attack on the whole. It is exciting to read the serious trials and intense consideration being taking on our behalf. So, thank you and may God
be involved in the final decision making process. Supernatural inclusion just adds to the fruits of your and my own success! Once again THANK YOU. Sincerely Patricia anne Pope, Orange, CA 92867
Hi Patricia Anne, One of the things that was reviewed by the advisory committee reviewing the Ampligen data was the lack of usual dosing studies to estabish a clear dosing regimen, incuding the time over which each infusion was given. So, questions about the ideal dose for weight, gender and baseline physical condition of treatment recipients still need to be addressed. If the drug is approved, FDA has the authority to require post-marketing studies that could better inform dosing regimens. A decision was expected by today; here’s the latest: http://bit.ly/WlMXbu
My daughter was devastated & has lost all hope at hearing this result after having this illness since the age of 14, 29 long horrible years. Ampligen is the ONLY light at the end of the tunnel. I hope the many PhD’s who voted against this have seen many many people with this awful disease & will know just what their vote will mean. When a medication to grow eyelashes with the side effects, of seizures, blindness, strokes, heart attacks & death gets through the FDA, it really makes one wonder what the FDA truly represents & I hope the arthritis advisory committee really looked long & hard at their testimony & know how much heartache this result will bring to so very very many people who have no life at all.
Hi Ann, I too have lost all hope now. My depression was lifted for a few months w/ the idea of hope, but now it has returned worse than ever. I have been sick so long and honestly don’t think I can continue, I do not want to suffer and be tortured anymore.
Hi Chris,
The outcome of the meeting is disappointing since a vote to approve Ampligen would have boosted chances for the first FDA-approved medication for CFS. I hope you’ll reach out to close family and friends and seek support to help you though this time of despair.
Hi Chris,
We still have to stay strong & hope that clearer minds will prevail at some point. At this time Ampligen is the only medication with hope but the patients who are all too weak & sick to rise up have family & friends who are now joining the ranks to demand some help from the FDA to find & push through medication to help. Ampligen may not be the panacea hoped for but after almost 30 years of researching I hope that one day I will be able to have my daughters at least try it.I have two who fell ill after a `flu`virus, both exceptionally bright, one hoped to be a neurosurgeon the other a veterinarian & for almost 30 years have been house bound/ bed bound with this wretched isolating misunderstood illness. They still hope for a productive life.
This is extremely disappointing. While I understand the FDA decision, it is not 1 million who suffer this horrible, deblilitating and life changing disease. As I said in my testimony submitted to the FDA for this meeting, Please give us as individuals the opportunity to try this drug as it is are only hope. I will pray the company continues its trials and that some other miracle for this disease becomes available soon. I want my life back.
Maybe my english is not the best, but if someone votes no to the question “Has safety been adequately assessed?”, she/he should vote no on “Is the safety profile adequate for approval?” as well? If safety has not been assessed adequately, how could it be adequate for approval? This is inconsistent voting behavior in my view.
And while I remain doubtful, yet on the fence whether Ampligen should be approved, I think this panel did not help to shed light on the matter…
To paraphrase Wellington at Waterloo: Give me better data or give me Lipkin.
Thanks for the up date and assistance. So so disappointing. My daughter is severely ill with ME as defined by the international Consensus Criteria. To ill to leave her room, be washed, wash her hair, talk to us, watch TV, read, listen to music. It is heartbreaking. She was made much much worse by taking medical advice and trying to push through this disease. Please let us try Ampligen for the severely ill anything that helps them will help the rest of the ME community by default. How many years of a living death must she suffer and we have our hearts broken by watching her suffer and caring for her.
There comes a point in human suffering when acceptable risk is most appropriately determined for oneself, not by government. There comes a time when being alive does not equal living a life. Numerous cancer patients willingly suffer side effects of chemotherapy and radiation for a chance to live. CFIDS patients have been given a different kind of life sentence but are also fighting for there lives — give us a chance.
Hi Suzy,
Very well said, I concur.
Suzy, what you said was so great that I used it in a call for people to email the FDA to get Ampligen approved in an article on Phoenix Rising:
http://phoenixrising.me/archives/15031
Thanks for making the point with such force and clarity.
While it is understandable that patients and clinicians are disappointed in the outcome, the FDA is doing their job. That other committees have approved drugs since then proven to be risky or ineffective does not support the argument that Ampligen should also be given a pass. Quite the opposite.
Hemispherx BioPharma was well acquainted with the requirements of the FDA and chose not to meet them. This is highly unfortunate given the severity of the disease, the lack of effective treatments and the long standing strain on families and patients.
Tony mentions Dr. Lipkin. Something that Dr. Lipkin insists on is very carefully selected patient groups – and the FDA committee members made it clear also – Hemispherx needs to tighten up studies to focus on the subgroup of patients that do appear to respond to Ampligen. Until then Ampligen may continue to be a good drug in the wrong hands.
I’m heartbroken!! I am now 60 years old! I am going to a grandma in March. After waiting for 27 years for something that helps this awful nightmare of a life. I have now all but completely given up. I’m sure the panel hasn’t seen the complete hell one goes through with CFIDS/ME. At this point I probably will never experience wellness.How long is one supposed to wait for a life. Life is so short anyway. Hemispherix should of waited until they had clear evidence of improvement on the drug! It’s amazing the drugs that are on the market that have many dangerous side effects. Ampligen has almost none! Why can’t they see how desperate people are. We are pleading for any kind of relief!!!
Mary I read your comment with such heartache as I feel just as you. What is wrong with this pharmaceutical company, don’t they know how to run a trial. I will say thought it seems that the FDA is making requirements of them that are beyond those in other trials as I know how clinical trials are performed. I too have no life and have waited for 22 yrs now. I have only a glimpse of who I use to be and of knowing what it is like to feel just one day of energy…I might get a little a couple hours a day for a few than crash and burn. The symptoms of this disease are like a mountain and the longer you have it the less chance of any recovery. I will pray for all of us that they reconsider their decision. I just want the choice! I want a chance of atleast part of my life back as you do. Don’t give up!
Please continue to fight for approval of Ampligen by joining the petition and writing emails. See the two links:
http://www.ipetitions.com/petition/ampligen/signatures
http://www.facebook.com/events/121302214705174/
I too was devastated at the AAC vote, I sat there, like many of you for eight hours in disbelief of this panel who has our life in their hands. Can someone please tell me that one of the Doctors/Persons on the panel did NOT say “What about the patients that get well from this”? Did I hallucinate that? IF that was said, that DR./Person should be removed from that panel, because clearly they do not have a clue about this disease. I cried so hard the morning after, just overwhelmed and hopless, like many of you. I did sign a petition on phonix rising to encourage the FDA to do the right thing.
Dr. Komaroff had NO BUSINESS participating as he used to work for HEB. No wonder he was so hostile. Shame on the FDA for inviting him there? You have to wonder which of the other invited doctors had alterior motives? Or is it the FdA that has alterior motives?
Hi Amy,
Dr. Komaroff has been on staff at Brigham and Women’s Hospital and Harvard Medical School since 1982. All members of the Arthritis Advisory Committee are reviewed for conflicts of interest under this FDA guidance: http://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM125646.pdf
Hi All,
what you need is public!
You can’t win without public power!
It’s nobody here with a friend who have connection to a radiostation and/ or TVstation?
Show the world all the pain, tell the world about the petition about the e-mail action,
tell the world what happend at the adcom!
A lot of public pressure!
That’s your only chance!
Otherwise you loose, that’s for sure!
Good Luck!
I am a almost 70 now. I had to close my business, Tate’s Auto Service after 25 years because of CFS. Bare in mind that we all could have something different that causes our symptoms but we DO have one thing in common. A compromised immune system. My problems started with flu-like symptoms in 1983 and CFS (I will use that term) has ruined my life. I have severe food allergies now, migraine headaches about 2 times a week, memory problems, extreme fatigue, of course, joint aches and when I am sick as I am right now, with the flu, it takes FOREVER to get well. I still hope for a break-through but time is running out. Ampligen has been studied and researched for years now and I wonder if it would be effective ans I doubt that I could afford it anyway. I had a really hard time getting on disability because no single doctor would go to bat for me because i had been to so many doctors. I won’t say here how I finally did get disability, when so many people are on disability that don’t deserve it seem to get disability so easily. May God Bless all of you with this problem. You can friend me on Facebook. Just look for me with my 2 sweet grandboys and you can also Google adobekid1 which is my hobby when I am able to function. Kim and the association have my e-mail address and anyone could contact me through Kim. She has my permission to do that.
I WAS ONE OF MANY AFFLICTED AND NEGATIVELY IMPACTED BY AMPLIGEN DURING THE FDA PHASE III TRIALS.
I was one of 25 subjects in San Diego CA during the 1999-2000 FDA Phase III drug trials for Ampligen. The trial was “overseen” by Dr. Anthony Mercandeti of San Diego; Dr. Mercandeti was seldom present, visible or available during the drug trial. There were very few drug trials for Ampligen.
Of the 25, at least 4-5 were immediately, negatively and severely impacted during the drug administration portion – I was one of those. I had an immediate major relapse of a well-documented 20 history of CFIDS which had been mostly in remission over many years. While it was not known who received a placebo and who received Ampligen, those of us who had a severe and immediate negative reaction knews. We, the afflicted, withdrew from the drug trial.
The process was a farce, a sham, and poorly supervised. The bi-weekly reports were trivial and mostly relied on the “Karnofsky” scores which were superficial and almost irrelevant. The medical staff not only did not want to hear of any negative impacts, they showed the afflicted as “dropouts” with no notations of negative symptoms or impacts. They refused to record our complaints and to show us our drug trial records (so much for HIPPA and FOIA). The FDA did receive complaints directly from those afflicted, but quickly shelved or disregarded those reports.
I now find it interesting that the FDA moves forward, albeit over 10 years later, despite known information that Ampligen has some serious negative side effects and is in fact counter-productive for many.
How is it that those of us WHO WERE THERE AND AFFLICTED do not show up in the review and approval process?? We KNOW FIRST HAND; everyone else gets second and third hand laundered and redacted info.
John, Having followed the studies of Ampligen and long regulatory process since early 1991, I have met people for whom Ampligen is a life-changing drug enabling them a much higher level of function that they had before it. I’ve also known people whose experiences were quite similar to the one you describe. The complete-ness and accuracy of safety reports was a specific (and unusual) discussion at the Dec. 20 advisory committee meeting and two separate votes on safety data were taken, with the committee ultimately judging the drug was safe enough (8-5) in spite of its judgement that the safety was inadequately assessed (9-4).
Kim:
The difference is that I WAS THERE AND EXPRERIENCED FIRTS-HAND the Ampligen FDA drug trials. I did not “study” it; I lived it. I KNOW how the FDA drug trial for Ampligen was (mis)conducted.
True, some Ampligen drug trial patients had mostly minor improvement. However, the negative impacts were more severe and more frequent.
I plainly state here and elsewhere, that the results were “doctored”, rigged, and manipulated to hide the severe and frequent negative side effects of Ampligen. I lived that also. There are others in my AMpligen drug trial who will attest (and have, to the FDA and elsewhere) to what I state re Ampligen.
Absolutely, John. The same avenues for giving testimony and input into the FDA policy decisions are available to you that others have used to communicate a different view. That’s why it’s important for all to advocate from their own experiences. You might wish to share your views in the comments to the Research1st post about the FDA’s decision to decline Ampligen: http://www.research1st.com/2013/02/04/fda-ampligen-decision/
I REPEAT: those of us who WERE NEGATIVELY IMPACTED AND AFFLICTED IN THE FDA Ph. III TRIAL FOR AMPLIGEN **DID** CONTACT AND NOTIFY THE FDA, and we were completely ignored after the FDA heard what we experienced and had to say. The FDA has that information yet never acknowledged it, acted on it and apparently did not make it available or known.
So I ask you: why should we believe the FDA would act any different later in the process. I hear you state “policy” but I am stating experience, truth and fact – which is much more than the FDA has done. I also ask you Kim, did you as an authorized review panel member EVER receive the communication or information I speak of here???
We gave up on the FDA long ago, and for good reason.
I have had a major case of CFIDS for almost half my life now (it has ruined me financially and maritally), and I clearly understand the hopes and desires of our fellow sufferers for some relief and treatment. But the FDA, Hemispherx and many well-meaning but under-informed organizations and individuals have raised false hopes. Those still hoping re Ampligen know not all the relevant facts – still.
Hi John,
I was not a member of the FDA panel that reviewed the data on Ampligen. You can find a list of the members of that panel here: http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/ArthritisAdvisoryCommittee/UCM332507.pdf
The FDA declined Hemipsherx’s application to market Ampligen for CFS, so perhaps your experience and others’ testimony about harm you experienced as a result of therapy was factored into that decision. Here is FDA’s public statement about the decision: http://1.usa.gov/11GaXcT