The relationship between pharmaceutical companies, patients and doctors is a complex one. It derives from a mish-mash of motives.
Let's start with the patient. He or she wants to be cured; if not cured, to have a longer life and a better one. Sometimes it's a choice between a longer uncomfortable life and a shorter more comfortable one. Most times neither the doctor nor the patient really knows what the outcome of treatment will be and decisions are made of a basis of statistics, false impressions and wishful thinking. Probably for most things the doctor knows best, but for other things the patient knows better. There is asymmetrical knowledge between doctor and patient. This is common in most professional relationships, but it does not make for a fair market.
When you are perplexed about whether you should buy a Ford or a Toyota there are magazines you can read to give you some insight into the choice. Should you get a Sony or a Samsung, an Epson or a Canon? Buy a "What Car" or a "What TV" or a "Which Printer" and become an instant expert. But it's not like that in medicine. A smooth talking physician may know nothing about your condition; a rude and brusque surgeon may know exactly what's right for you. How can you possibly tell? Trial and error may leave you dead or disabled. Market forces may have calamitous results.
The doctor has many motives. You might think his prime purpose is to cure as many people as possible, but if that were his motive pure and simple, he would be working in Africa and not many are. Why am I not working in Africa? Many reasons. I don't like the heat. I would miss all the mod cons. I don't speak the languages. I would miss all the diseases that are interesting to me and without the facilities I have in Britain I would be pretty useless. I need the money to support my family. And why lay it on a doctor's conscience? Africa needs engineers, accountants, businessmen, teachers and factory owners too.
So although a doctor wants to cure the sick, he wants lots of other things too. He certainly wants paying well. Not many doctors take poorly paid positions. He also wants the good wishes of the community; he likes to be well thought of. He may also want fame. Perhaps he would like the rewards that go with a successful academic career such as foreign travel, smart hotels, and lavish entertainment. Perhaps his real interest is in the science; ferreting out the mechanisms of disease.
Now let's consider the pharmaceutical company. Like any other company its main purpose is to make money. How do they make money? By providing the best product to their customers at the best price. Competition ensures that those who don't perform fail. That's how the market works.
That's how it ought to work, except that the market is deficient. For one thing, it is hard to know whether a particular treatment works or not. Patients often get better from some diseases without treatment. The mere presence of a sympathetic doctor and a pink pill will cure some things. In order to sort this out, we have regulators who apply an expert eye to this thorny question. They regard the randomized controlled trial as the only legitimate way of knowing whether a drug works.
However, even randomized controlled trials (RCT) can be manipulated. For marketing is not just about ensuring that the customer knows that your product is best, but about giving the impression that your product is best. If your marketing is good people can be induced to buy an inferior product.
In an RCT, the new treatment should be compared with whatever has been the best hitherto, not with something that used to be the best. Of course, a drug might have a niche market. There may be some patients for whom what is generally the best is not the best for them. An example of what I mean is this: probably the most effective treatment for CLL is FCR, but not for patients with del 17q, for whom it works very poorly. If a new treatment is worse that FCR for most patients, but better for these patients, then it should be licensed for this exception, but not for the whole gamut of CLL.
Lately, new drugs have been compared with chlorambucil. Now I think chlorambucil is a fine drug, but I do not claim it is the most effective drug in CLL. Until fludarabine came along, chlorambucil was regarded as the standard treatment, but the Rai et al trial in the NEJM in 2000 demonstrated that fludarabine was superior in terms of response rate, complete response rate and length of remission, though not in terms of overall survival. What nobody noticed at the time was that the dose of chlorambucil chosen as a comparator was on the low side - much lower than is usually used in the UK by CLL specialists, though comparable to the dose used by US oncologists who do not specialize in CLL (in other words the dose was a very safe one).
The CLL4 trial published in the Lancet last year demonstrated that fludarabine was no better than chlorambucil when chlorambucil is used in its usual UK dose, and fludarabine is more toxic than chlorambucil used at that dose. That's why fludarabine is not licensed in the UK for first line use in CLL. However, the same trial showed that the combination of fludarabine and cyclophosphamide is superior to both chlorambucil and fludarabine given separately. And the German CLL8 trial, although it has not reported formally, is believed to show that FCR is superior to FC.
Now there may be niches where chlorambucil should be preferred - older patients, frailer patients - and there is still no evidence that any other first line treatment improves overall survival, compared to chlorambucil, but the proper comparator should be FCR now, not chlorambucil, unless one is aiming for a niche market and trying to sell the new drug as an alternative to chlorambucil on the basis that it is less toxic (either short term or long term) or cheaper.
The difficulty anyone has in using FCR as a comparator is that the average length of remission with FCR is very long - perhaps greater than 6 years - and the patent would run out on a new drug before the trial was done. So the pharmaceutical companies try to hoodwink us, by choosing a feeble comparator; not just chlorambucil but chlorambucil in a half-hearted dose.
There is another way that the market is rigged. People don't pay for their own drugs. The cost is borne by the community; either directly by the state or through an insurer who passes the cost on to employers or individuals who don't have the disease. These third party payers have little inkling of whether they are getting value for money. The UK has set up NICE to answer that, but like all bureaucracies it is slow, ponderous and lacking in insight.
So what's the answer? It's not to nationalize the pharmaceutical companies. Leukaemia drugs do occasionally come out of communist countries. Bendamustine came from East Germany, and both ATRA and Arsenic came out of China, but most drugs have come from the big pharmaceutical companies of America, Britain, Germany, France and Switzerland. Paradoxically, my solution would be to extend patent protection for drugs. Currently it is 17 years. I would make it 35 years. This would give the companies long enough to do proper trials, and long enough to recoup their costs without inflicting unnecessarily high costs on third party payers.