Back in the Go-Go 90s, 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 vascular structure in the lung rises to dangerous levels. In some cases the condition is progressive and terminal. Prior to the advent of a new class of drugs, the only real treatment for those 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 drug developed to relieve PHT is Prostacyclin marketed as Flolan®. Prostacylin is highly effective, but it has a half life of only a few minutes and requires a permanent central venous catheter to administer. In addition, Flolan® is unstable, and therefore has to be cooled to near freezing during administration. Because of the delivery headaches, another, similar medication was developed which could be given subcutaneously. The most common is Treprostinil marketed as Remodulin®.
So far, so good. But PHT 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. The cost of Remodulin® is astronomical. A patient with severe primary PAH might spend almost $100,000 a year on 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 PHT are on the biotechnology horizon. And even more amazing treatments could be produced from stem cell research or nanotechnology.
I chose PHT and Remodulin® for this illustration, but it could have easily been any hundreds of serious diseases and treatments for them. The broader question is how can we reasonably preserve the interests of all concerned as future treatments for life threatening disorders are developed? In other words, who pays?
Insurance companies already know the answer. It involves the Law of Large Numbers and the power of bulk buying. With a large, diverse, number of insured, insurance companies can work both sides of the equation, spreading risk from the healthy and fortunate to the unlucky and sick, while negotiating with drug makers and healthcare providers for the lowest prices possible for services rendered. They even do it with enough profit left over to pay their top execs wages that would make a Saudi oil Prince blush. But commercial insurance companies have an awful conflict of interest between providing care and making money. Sick people are expensive. With their legal and lobbying power, they've managed more than just care, they manage their exposure. Mostly by relentlessly nibbling away at their customer roster, clearing out expensive cases, often by denying care to those who need it most by hook or by crook. Healthcare horror stories, even for those who are insured, are legendary and universal; just about anyone in America over age 21 has one. The consequences for those who lose insurance or can't afford it in the first place can be nothing short of tragic.
With healthcare reform making headlines every half hour and millions of aging American joining the ranks of the uninsured, the stakes could hardly be higher. We're talking about life itself; who will be granted that respite and who will not. Will life saving drugs and treatment be available for those who need them? Or will the very breath of life be cut up into units, and sold like a pound of butter only to those lucky few who can afford to go on breathing in a brave new world?