Previously, I discussed the basic concept of randomized controlled trials, but didn't go into all the complexities. Actually, I can't go into all the complexities without writing a very big book, and that's not the book I want to write. However, there are some important points I'd like to cover here.
First, there's the question of what drugs (or possibly other kinds of interventions) even get into clinical trials in the first place. In order to conduct trials, the sponsor needs to get approval of what's called an Investigational New Drug Application from the FDA. This requires a reasonable expectation of safety and evidence of pharmacological activity, usually from trials in animals. Note, however, that this is not required for a trial of an already approved drug for an off-label use, since the safety profile of the chemical is already known and it's legal to prescribe it. This is unusual, however, because the only value to the manufacturer would be approval to market the drug for a new purpose and that doesn't often promise sufficient profitability to make the trial worthwhile economically.
Because trials are enormously expensive, companies will not undertake them if they don't a) have a reasonable expectation that the stuff will be safe and effective and b) have an expectation that it will be profitable enough to justify the expense of the trial. These calculations depend in part on the laws about how long they will have exclusive rights to sell the drug, which are a bit complicated and I'll get to them later. They also obviously depend on the size of the potential market for the drug, and how much they can get away with charging for it.
What all this means is that the decisions about what drugs to develop and try to get approved don't necessarily align well with what might offer the most benefit to public health. Drug companies are in business to enrich their executives and shareholders, not to benefit humanity. One of the lowest risk investments the companies can make is so-called "evergreening." If they have a profitable drug for which they are about to lose exclusive rights, they can test a slightly different formulation, get brand new rights to it, and then try to convince doctors that the new formulation is sufficiently better that they will prescribe it rather than prescribing a generic version of the old drug. That threat may discourage companies from making a generic version of the old drug so it actually ceases to be available.
There is also a lot of money to be made developing treatments for terminal diseases that only prolong life for a short time, because people are desperate and they're willing to pay a lot for those drugs -- or more precisely it's politically imperative for Medicare and private insurance to pay for them. So cancer treatments that only prolong life by a few months and cost $100,000 or more will be paid for because of the "death panels" argument. Note, by the way, that is not true in the UK where there is a cost-benefit requirement for drug approval. That's another complicated issue I'll have to expand on later.
The last thing I'll say today is that there are many problems with translating clinical trial results into actual benefit for patients. Clinical trials happen in a very different universe from the real world in which a drug will actually be used. Participants are selected specifically not to have certain comorbid conditions that are common in the general population. They may be restricted by age. They are also generally monitored to make sure they are taking the drug as prescribed, which many people don't do in reality. And there is heterogeneity of treatment effect. Just because a drug has a net benefit on average in a sample doesn't mean it's benefiting everybody, and it might even be harming some people. But that is very difficult to sort out and establishing it requires a whole new expensive trial, which the companies have no obvious incentive to do. (You can't just go back and look at how sub-groups did in your original trial because that violates the rules of inference. That's an important philosophical question which I may discuss at the risk of boring people.)
To be continued.
4 comments:
Who decides that you're too old for treatment "A" or drug "B"?
Who decides that this drug is worth developing and that one isn't?
In your world, who is that person?
WTF do you mean in my world? We live in the same world. The question of whether someone is "too old" for a treatment is between that person and the doctor, assuming there isn't a contraindication associated with age.
Who decides that a drug is worth developing is, generally speaking, pharmaceutical executives, although there are some special NIH programs for drugs they wouldn't ordinarily pursue, called "orphan" drugs, because the market is too small.
You've critiqued how drugs are developed, etc., but have not offered up any alternative solution.
That was my point. So, I'm asking if not by market need, then what?
Woody, I have a little thought experiment for you:
Suppose a researcher at a pharmaceutical company discovered a molecule that could easily and permanently cure one of the chronic diseases that plague the first world. Say, diabetes, asthma, arthritis, or one of the STD's. It would be easy to make, and one dose taken orally would obliterate the condition.
I understand that this isn't realistic since most pure research is actually done at universities using public funds, that is, paid for by your taxes.
So, our hypothetical researcher excitedly tells his superiors about his discovery. Assume further that the company already sells a popular and profitable prescription drug that must be taken regularly to control this same condition. What do you think the company will do about it?
They could develop and sell it at an astronomical price, at least until an Indian pharm company finds a way around their patents and undercuts them, but that would kill the golden goose that has been one of their main cash cows. (Biologically impossible but metaphorically apt.) You and I both know that the odds of that happening are less than the odds of buying a winning lottery ticket.
Instead they'd do everything in their power to bury this information so deep that no one else could ever find it. That researcher would either end up a very, very rich man, committed to a mental institution, or dead; depending on the ethics of the corporation. There's no way to show that something like this hasn't already happened. Several times, even.
The fundamental Economic Good of a corporation is necessarily its financial self-interest. In my limited understanding of Economics "Good" is left as an undefined term. Its up to the parties involved to decide what is Good for them. (" 'I just make them go up, I don't make them come down, / that's not my department', says Werner von Braun." - Tom Lehrer)
When it comes to health, and life, I certainly don't think that money is the primary Good involved, and I doubt that you do either; you're way too intelligent for that.
So how about looking at other domains where Money is not the primary Good -
criminal justice, the military(maybe), primary education, the Post Office, streets and highways, for example.
It seems pretty clear that a capitalist solution in these areas would at least be inefficient, if not disasterous.
Perhaps the ways these areas are managed might offer us some clues. ...
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