Flashback to the Go-Go 90's: President Clinton is not having sex with that woman. The stock market is surging, unemployment is at record lows. Our biggest international concern is roving gangs of Paparazzi who cause accidents or fail to render aid, AKA Princess Di. Ah ... the innocence lost. Back in those days, a wonder drug of sorts for the treatment of obesity was all the rage. It was Fen-phen, a combo appetite regulator and appetite suppressant. The medication was relatively cheap and patients reported positive results, sometimes after just a few weeks of treatment. But in a few cases, serious cardiopulmonary side effects appeared. One of those side effects was
pulmonary hypertension or PHT.
PHT is not to be confused with routine high blood pressure for which a number of affordable medications exist. PHT occurs when the blood pressure in the pulmonary artery or the tiny blood vessels making up the vasculature structure in the lung rises to dangerous levels. In particular, arterial pulmonary hypertension (PAH) can result from heart disease, birth defect, medication interactions, and can sometimes even appear spontaneously in otherwise healthy people (See Kmiddle's comment).
In some cases the condition is progressive and terminal. PHT/PAH can lead to complications such as pneumonia, heart failure, or the victim may slowly suffocate to death. Prior to the advent of a new class of drugs, the only real treatment for these terminal cases was to postpone the inevitable as long as possible and then go with a heart-lung transplant. Needless to say, few patients are lucky enough to receive the double transplant and those that do face a life of endless medication and fatal complications.
One of the substances that relieves the effects of PAH is Prostacylin marketed as Flolan®. Prostacylin is highly effective, but it has a half life of only a few minutes--it requires a permanent central venous catheter. That kind of delivery system comes replete with a whole slew of other complications including sepsis and thrombosis. In addition, Flolan® is unstable, and therefore has to be cooled, near freezing, during administration. Because of the delivery system headaches, another, similar medication was developed which could be given subcutaneously. The most common is Treprostinil marketed as Remodulin®.
So far, so good. But PAH is a fairly rare condition. Only a few hundred new cases are diagnosed each year in the US. Severe forms of the disease only affect perhaps one in a million people. From a business standpoint, the market is small. That means the cost per patient must be high to recoup investment costs. Indeed, the cost is astronomical. A patient with severe primary PAH might spend almost $100,000 a year on Remodulin® and they'll have to take it for life.
It takes a lot of financial and intellectual resources to take a substance from the lab, through FDA trials, and on to the market. If the possibility of profit were decreased or eliminated, there would be no incentive for investors to shoulder the risk, and many drugs we take for granted today might not exist. Other, more advanced drugs for treating PAH are on the biotechnology horizon. And even more amazing treatments could be produced from stem cell research or nanotechnology. Who pays? And what happens if those who need the medication or treatment to stay alive can't pay?
If insurance companies take up the slack, it only means premiums for every one will have to increase, theoretically with no end in sight. Not to mention that insurance companies have a great deal of legislative influence and may well be able to have law written excluding coverage for expensive treatments on any number of contrived grounds. And what about the tens of millions of Americans that have no health insurance?
I chose PAH and Remodulin® for this illustration, but it could have easily been any hundreds of relatively rare diseases and treatments for them. The broader questions is how can we reasonably preserve the interests of all concerned as future treatments for increasingly rare disorders are developed? At times those interests are united into a single canvas of technology, capitalism, and individual innovation. But others times those hues are in direct, jarring conflict as in the work of Van Gogh.
I have no easy answer for this conundrum. The stakes could not be higher. We're talking about life itself; who will be granted that respite and who will not. Will new, life saving treatments be available for all? Or will the very breath of life be cut up into units and sold like butter only to those lucky few who can afford to go on breathing in a brave new world?