On December 11, 2013, I wrote a letter to the FDA’s Director of the Center for Drug Evaluation and Research, Dr. Janet Woodcock. I pleaded with her to use her considerable discretion and potentially accelerated procedures regarding the approval of drugs for life-threatening or severely-debilitating diseases that lack alternative treatments to finally approve Ampligen for the treatment of myalgic encephalomyelitis (ME). I asked Dr. Woodcock for the reason of the jarring unequal treatment of Bexsero and Ampligen. Bexsero is a drug that is not approved in the US and yet the FDA allowed its use in the vaccination of 8,000 students at Princeton University. Ampligen, on the other hand—a drug found to be safe by a majority of the FDA advisory committee and effective by a substantial minority of the committee—was denied approval in February of this year for ME, a severely debilitating disease without any approved drug treatment and none on the horizon.
One does not have to look far to realize that the preferential treatment of Bexsero over Ampligen is no exception. Below are further examples (not an attempt at a complete list) of drugs that have been approved by the FDA—one under an accelerated approval procedure—despite questionable safety and/or efficacy profiles and despite the fact that, in most cases, alternative drug treatments exist. In the case of the two weight-loss medications, there are also other treatments, such as improving one’s diet and increasing one’s amount of exercise. The latter is, of course, something ME patients would love do to, but are unable to due to the dire consequences most forms exercise would have on them, which adds to the irony.
We can all speculate as to the reason for the inequality. But see for yourself:
Type 2 Diabetes: Canagliflozin, sold as Invokana (Johnson & Johnson)
Canagliflozin, a new class of Type 2 diabetes drugs, was FDA-approved on March 29, 2013 despite serious concerns by the FDA advisory committee about elevated stroke risk and an increase in heart attacks and other cardiovascular problems. In addition, the drug raises LDL levels (so-called “bad” cholesterol) and increases the risk of side effects for patients with moderately impaired kidney function, a patient group for which the drug also isn’t as effective as it is for those with normal kidney function. Despite several committee members finding an unfavorable risk/benefit ratio for that patient group, the FDA approved the drug for patients with both normal and impaired kidney function. The reason cited by the majority of the panel members for their support of the drug was the unmet need “for new agents to treat the growing population of patients with type 2 diabetes.” Does “unmet need” ring a bell for ME patients? And I don’t mean unmet need for “new agents,” but for ANY agent.
Alzheimer’s: Aricept (Pfizer)
Mindy Kitei reported about the apparent double standard in the FDA’s denial of approval for Ampligen versus the agency’s refusal to ban the highest dose of the Alzheimer drug, Aricept, despite the lack of increased efficacy and in spite of the increased mortality rate of the higher dose of the drug compared to its lower doses.
Tuberculosis: Sirturo (Johnson & Johnson)
As Bob Miller pointed out during the FDA’s April 2013 Drug Development Stakeholder’s Meeting, the FDA approved on December 31, 2013,—under an accelerated approval program—Sirturo, a drug to treat multidrug-resistant tuberculosis that is five times more likely to kill patients than the standard drug treatment for the disease without proof of increased efficacy.
Weight Loss: Belviq (Arena Pharmaceuticals GmbH)
On June 27, 2012, the FDA approved the weight-loss drug, Belviq for the use in obese adults (BMI of at least 30) and overweight adults (BMI of at least 27) who also have at least one weight-related medical condition, such as high blood pressure, type 2 diabetes, or high cholesterol. The approval was granted despite the FDA’s advisory committee’s concerns about the potential risk of breast tumors and heart-valve problems and despite the proven increased risk of other serious side effects, such as serotonin syndrome (especially when taken together with medication for depression and migraines) and disturbances in attention or memory.
Weight Loss: Qsymia (Vivus Inc.)
In September of 2012, less than three months after approving Belviq, the FDA approved a second weight-loss drug, Qsymia, for overweight people despite concerns that the drug causes an increased heart rate; a condition called metabolic acidosis, which can lead to hyperventilation, fatigue and anorexia; and birth defects, such as cleft lip and cleft palate and despite the fact that the FDA advisory committee initially voted against the approval of this drug. The FDA and the drug maker both acknowledged that the clinical trials designed to assess the drug’s safety and effectiveness did not properly assess cardiovascular risks. Qsymia most likely has to be taken long term, a factor that seemed to weigh heavily against Ampligen in the FDA’s decision not to approve it.
Bottom of the barrel for ME/CFS patients . . . grrrrroan 😦
About diabetes type 2: exercise + diet are the PERFECT remedy for getting it under control (!);
This is all so so so mindblowing.
My father is a perfect example of a person who’s got his diabetes type 2 under control by sheer discipline of eating differently and walking every day a fair distance.
So, a disease that CAN be managed with EXERCISE and DIET (of course the patient needs to be able to exercise every day), gets approved yet another drug.
While patients with ME who are totally unable and even get severe flair-ups of their illness by having to exercise do not get Ampligen approved.
Money, pharma influence and total ignorance about the severeness of ME seem to prevail time and time again.
That is so ‘right on,’ elsvh, about how exercise and a proper diet get diabetes under control; then drugs are not needed–diabetes is a disease where exercise ‘helps’. My gosh, if only we p/w ME/CFS could exercise and be active or even vacuum (domestic activity).
I certainly do not mean to demean diseases like diabetes–yet, must point out that there is a complete difference as to how a person with diabetes lives their life as compared to being ‘housebound’ in debilitating illness with ME/CFS. Our uncle has had diabetes for years; it is under control, he feels well and he is out and about constantly living life to the fullest . . . now in his late 70’s. CONTRAST that to ‘this’ life . . .
The MIStreatment–BOTH intentional and out of ignorance–of p/w ME/CFS is INhumane; it is human cruelty by the ‘powers that be’ in a democratic society that ‘oppresses the seriously ill’.
I think one of the most hypocritical comments by the FDA was about sub group analysis. The FDA wrote:
“The sponsor states that this trial shows benefit of Ampligen
on ETT in a post-hoc responder analysis and a post-hoc subgroup analysis. In addition to
the usual concerns related to post-hoc analyses, the responder analysis was found to lack
benefit when performed with a pre-specified cut off of 20%, and the subgroup analysis was
found to lack scientific justification, raising issues as to the validity of these analyses.”
However, the FDA admitted that if you changed the responder analysis cutoff to: 25%, 30%, 35%, 40%, 45%, 50% and on… benefit is clearly found. Hemispherx guessed wrong at 20% and is apparently being penalized for it. Despite ME being finally considered a “Serious Lie Threatening Disease”, – the lack of this designation was cited as why Hemispherx did not receive accelerated approval after 5 requests – once again Hemispherx is denied any help in the form of accelerated or other approval pathways, other than a costly and lengthy additional phase III trial, for a 6th time. It’s astounding.
I agree, Johhny. When you go through the FDA meeting materials, you’ll find a bunch of stuff that makes it clear that the agency just did not want the drug approved. For example, a case of pre-existing cancer was found to be a serious adverse advent. It doesn’t get worse than that.
And they claimed that some of the symptoms of mecfs were “side effects” of the drug. How disengenuous!
yes, that was another “good” one.
Jeannette: Thank you so very much for yor letter and emails to the FDA. This FDA has been a decades long trail of tears. I finally just gave up somewhere around 2007 after my children were all settled on their own. I crashed through several years helping with my then seriously autistic Grandson. We worked hard and Chris is doing very well. He is a wise and interactive young man.
I would bash through a million years for Chris if needed, Ampligen would have helped so much. After the kids left I found I could not do much of anything anymore. My kids were o.k.
I now find I have anger at the fda so deep it is bottomless. Thank you for taking them to task. Izola