Patients Shouldn't Be Used As Hostages, Vulnerable To Sudden Drug Price Increases

Martin Shkreli is not the only bad player in the game of hiking up drug prices (which is not a game). Recently, the New York Times ran a story on Valeant Pharmaceuticals, a company that's similarly profited by buying old drugs and jacking up the prices. Others here at Forbes have explored that particular business.

The problem is more general, and widespread: Patients and their families are essentially being held hostage to firms that raise prices of medications they need to stay alive, or pain-free, or without sores, or symptoms like fatigue or tremor or jaundice, to be productive and well.

The Times tells of a man in his fifties who has Wilson Disease. As reported, Valeant recently raised the cost of his medication, Cuprimine, to about $35,000 per month. Most will be covered by Medicare. The patient, who was paying around $366 out-of-pocket per month, just last May, now must cough up some $1800 per month for the same pills.

Wilson disease is quite rare. The NIH says it affects 1 in 30,000 people. The condition is named for a physician, Samuel Alexander Kinnier Wilson. Around 1912 he first described patients with a distinctive pattern of neurological and liver problems. It results from copper deposits in cells due to impaired copper metabolism. In Mendelian terms, it's an autosomal recessive condition; most people with this disease have inherited mutations from each of their parents. Modern science links Wilson disease to abnormalities in the ATP7B gene.

Cuprimine is the brand name for an old drug called penicillimine. Patents for this copper-binding compound go back at least to 1976 – a half century ago. It is occasionally prescribed tor patients with other conditions such as rheumatoid arthritis (for which there are now many other treatment options). In patients with Wilson disease, penicillimine helps by keeping copper from accumulating and damaging organs, like the liver and brain.

A doctor like me is more likely to have seen Wilson's disease, as we called it, on a board exam than in a patient. We were tested on characteristic Kayser-Fleischer rings, findings from copper build-up that might be observed in the eyes of a person with neurologic and liver abnormalities – a clue to the disease.

But while all this may be interesting to physicians, medical historians and a few others, it's not helpful to patients if they can't afford the price of the medication.

What I'm saying – and I do intend to go meta with this Cuprimine example – is there's little point in all this progress: identifying a disease, finding its cause, examining its molecular underpinnings, and developing medications to treat it if, in the end, the price of the drug is so high that people can't be helped.

Medical research would be little more than an academic exercise, a hobby (in extreme terms) if society does not somehow protect patients from medication price-gauging.  This applies to drugs for treating parasites, like Daraprim (generic: pyrimethamine) now sold by the venomous Turing Pharmaceuticals. And it could soon apply to many other, no-longer patent-protected drugs which frequent the FDA's medications shortage list.

Unless the policy (or lack of policy, or regulation of drug prices, which perhaps might be considered along the lines of utilities) changes, the pattern will diminish the long-term value to the public of newer drugs, like antibodies and other novel meds, as those approach their patent expiration dates, too.

I'll close by noting that the recent announcement of the 2015 Nobel Prize in medicine recognizes painstaking work, years ago, against parasites like roundworm and malaria – key advances in the same category of infectious disease as Daraprim counters. But science, in itself, nor profit can't be the most valued outcome in drug development: It's how these efforts help patients that motivates doctors and scientists working in the field, and what ultimately matters.