Good news for the shareholders in the mega pharmaceutical company, Bristol Myers Squibb: the FDA just approved the drug ipilimumab, also know as "Yervoy", for use in metastatic melanoma.
Bristol-Myers Squibb Co shares rose 3.2 percent to close at $27.29 after the Food and Drug Administration approved its drug under the brand name Yervoy for patients in the late stages of the disease. Analysts see sales reaching $820 million in 2015, according to Thomson Reuters.
The average "dose" for this drug is four infusions. At a cost of $30,000 per dose, that makes the average four dose cost $120,000. Cost $30,000 per dose for Yervoy
If you respond to the drug, of course, you may need multiple doses.
Good news for BMS, good news for BMS shareholders, good news (perhaps) for patients with the brutal and highly resistant to most treatments stage IV melanoma.
My late friend, Will, had stage IV melanoma.
When he was diagnosed in the fall of 2008, his disease was at stage III. It had been found in one lymph node in addition to the site of origin. His oncologist was a melanoma specialist - there aren't that many of those, but in Chicago, there are now three - and like many melanoma specialists, this man was a "mud/phud" - an MD/Ph.D - who ran clinical trials.
Clinical trials, of course, are the process by which new drugs get tested and possibly approved for use by the FDA. They are usually run by drug companies, sometimes in conjunction with the National Institute of Health.
Getting FDA approval for a drug, especially a drug that treats an otherwise swiftly fatal illness, is a license to print money. So drug companies are what you might term "highly motivated" to get FDA approval.
It's a complicated process, of course, as it should be. Until the drug is approved for use, the manufacturer, in this case Bristol Myers Squibb, owns and controls it. There are different "stages" of clinical trials, as they test the drug to see if it has a positive impact on the disease process and ultimately, survival time.
But back to Will. It was September of 2008. He'd just found out he had stage III melanoma, and his oncologist, as luck would have it, was running a clinical trial using this new drug, ipilimumab (now Yervoy). Recruiting patients for clinical trials is a tricky thing. People, naturally, prefer approved drugs - they have a track record, obviously. And many clinical trials have a "control" arm, using a less effective drug or even a placebo. You are randomized into these arms. You may or may not know - depending on whether the trial is "double blind" - which arm you are in.
Getting into a trial can also be tricky. In the word of melanoma clinical trials, you are already dealing with people who are sick - they have one of the most aggressive and lethal cancers existent. But you don't really want people who are TOO sick; they tend to die fast and mess up your research statistics. So you triage people with a system designed to weed out those who are most likely to die anyway. In other words, to get into a clinical trial for melanoma you have to be sick, but not too sick, and you have to pass numerous tests to even qualify for participation. You may need to have particular genetic mutations. Your bloodwork has to fall within accepted parameters. And your "performance status" - the degree to which the disease has impacted your ability to carry on with your normal life - has to be acceptable to the trial coordinators.
It sounds like a lot of work, getting into these trials. It is. But you do it, if you have melanoma, because you just don't have many other options. The average survival rates are grim - often less than a year. And it's frequently a disease of the young. Will was 43 when he was diagnosed. But he was still "healthy" (except for that lethal cancer thing) and passed all the tests for getting into the clinical trial using this new drug, ipilimumab. There was one small complication. This was a double blind placebo trial. Half the patients would get absolutely nothing in their infusion except saline water. They had to sign a 17 page consent form stating they understood this and accepted the risks (and the risks were that your cancer would spread uncontrollably, and you'd die a pretty awful death). Will, who had mild developmental disabilities, signed the consent form because his doctor recommended it.
No where in the form did it state, nor did anyone explain, that if this drug proved effective and you were in the placebo group, you couldn't get access to the drug.
And that's what happened to Will. He had no side effects at all from this treatment, which, given the generally profound side effects of this drug, was a clear signal that he was in the placebo group. His cancer progressed. Subsequent attempts at treatment failed. And three oncologists that we consulted said "ipilimumab is his best chance."
So we asked Bristol Myers Squibb to release the still-unapproved but now found to be effective and fastracked-to-be-approved drug to Will under the "compassionate use" program.
They refused. He had been in a trial, and to release the drug to him might compromise their data. They wouldn't give him access to the drug until it was FDA approved. He'd be dead by then, I pointed out. Sorry, they said, these things happen.
I went to the state's attorney general. They expressed interest in trying to help me get BMS to release the drug to Will. I told BMS this. They contacted Will's oncologist and said that if I proceeded - I was arguing that he could not possibly have given "informed consent" as the consent did not explain the drug would not be available should he receive the placebo and there was no chance Will, who read with difficulty at about a 2nd grade level, could have understood the 17 page form he signed - Will could be banned from ALL clinical trials.
And in the US, drug companies are not subject to anti-trust laws. So theoretically, they could conspire together to do such a thing. There were really no more options. I gave up, Will fought the disease as best he could, suffered a lot, and died in October 2010 from liver failure, brought on by a combination of liver metastases and the toxicity of the chemotherapy drugs we were using to try and arrest the disease until Bristol Myers Squibb got FDA approval.
That approval came yesterday, and it came in part due to people like Will, who took a chance and risked their life for science. And profits. Dying for monopoly capitalism - it's a noble thing, I guess.
I don't know if this drug would have extended or saved Will's life. But he deserved a chance to find out. Bristol Myers Squibb had a moral obligation to make a drug they now knew was effective available to those who participated in earlier trials and got a placebo. But to do so might have compromised their fast profits, they feared ( though with some effort on their part, that could have been averted) and that was what mattered most. When I tried to fight this, they said, in essence, "Nice friend you've got there. Be a shame if anything happened to him."
He deserved better. We all do. The entire US health care system operates, on a macro level, like organized crime, from insurance companies to for-profit hospitals to the multinational pharmaceuticals. Let's turn it all upside down.