by euthman
Fabry Patients Sue Genzyme Over Drug Shortage; After NIH Refuses To Allow Others To Make Fabrazyme
[Via Techdirt]
One of the more horrifying stories of patents costing lives out there is the situation with Genzyme and Fabrazyme. As we detailed, not enough of the drug is being made allegedly due to manufacturing errors by Genzyme (for which it was fined $175 million by the FDA), and its causing serious health problems — and even death — to the sufferers of Fabry disease, who are caught in the middle of this. The patients asked the NIH to allow others to make the drug, via a “march-in” order, so that there would be enough supply, but the NIH refused, basically because it said that the patients (you know, the people actually suffering) couldn’t make such a request. The end result, of course, is that lives remain at risk. Genzyme is apparently now rationing the drug, only giving 50% of the recommended dosage, despite a study suggesting that such reduced dosages are a problem:
Neither Genzyme nor Mt. Sinai has tested whether the reduced dosage is either safe or beneficial for treating Fabry disease. In fact, in November 2010, The European Medical Agency determined that the lowered dose of Fabrazyme resulted in patients having more strokes, heart attacks, renal disease and other symptoms including severe GI distress and pain and burning in their extremities (neuropathy). It also determined that the lowered dose actually accelerated the course of the disease in some patients. Europe recommended in October 2010 that all patients be given a full dose of Fabrazyme instead of the lowered dose. Genzyme rejected the European assessment and continued to provide only unapproved low doses to patients in the U.S.
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It is unlikely that another plant could get an approved manufacturing process done in a short time. It is not a matter of simply contracting the plant. The plant and process would also have to be approved to make sure they will not make the mistakes Genzyme did. This could take a lot of time and money. If Genzyme thought they could get the plant online sooner,then there is really no reason to sub-contract.
But there are two things worrisome here. One is the possibility that lowered doses could harm people. SImply rejecting the notion and providing unapproved doses opens them up to some real legal danger, it sems to me.
Say someone dies without getting the proper dose. They sue Genzyme for negligence, both because of the manufacturing problem and because of possible health risks of lower doses. It’s going to be Genzyme’s word against the European Medical Agency’s. Which way is a jury going to go? Hard to know. But could cost a lot to settle.
And the other is using orphan drug status to get exclusive control of a drug – seven years with no competition – and then not be able to manufacture it. There should be some sort of out-clause from the government when this happens. The problems with Genzyme’s manufacturing have been going on over a year now.There should be a real risk of losing the orphan status of the drug in order to make sure the companies place very focussed attention on manufacture.
This is a drug company’s worst nightmare. If things were going fine, they’d be saving people’s lives, overcoming an illness that no other company wanted to and getting richly compensated for it. They are the good guys. But by screwing the pooch with their plant – which also makes 4 other needed medicines – they are possibly ruining people’s lives and losing lots of money. Now they seem simply evil.
Going from good to evil can happen to any drug company if it loses focus on any part of the very complex process of getting a pharmaceutical to market.
Getting the science right is hard. But, the complexity of manufacturing for biologicals is, as we see here, just as complex and possible even more devastating when done wrong.
