In today's BMJ there is an open letter to the Secretary of State for Health from Professor Peter Lachmann, the retired professor of immunology from Cambridge University and past-president of the Academy of Medical Sciences. In it he makes this interesting suggestion:
About 60% if drug development is taken up by late stage, Phase III trials. Phase I trials ensure safety and phase II trials demonstrate efficacy. Both require small numbers of patients and are relatively cheap to run. Phase III trials are expensive to run and require large numbers of patients, but still cannot detect side effects that occur in fewer than one in a thousand patients.
He says that the population now believes that drugs have to be absolutely safe ans that if anybody comes to harm from them they should be able seek compensation from the courts, and that this has led to drugs being hugely expensive and very slow to bring into use. It is now estimated that to develop a novel drug costs several hundreds of millions of dollars and takes at least 10 years.
In his opinion there is a strong case for making drugs available after phase II trials have shown that they work and to rely on post-marketing surveillance for detecting rare side effects. This would make new drugs much cheaper and available much earlier. It is arguable that phase III trials save fewer lives from avoided side effects than they cause by delaying their availability.
Initially patients should be able to get these new drugs by signing a waiver indemnifying the manufacturer from damages should there be any harm from the drug.
In any case the practice of suing drug companies for adverse effects is pointless. It does the company no financial harm, since the cost is passed on to the consumer. Only lawyers benefit. Legal redress should be reserved for negligence of other malfeasance.
It is probable that many useful drugs are never developed because the potential market is too small and the financial risks too great.
I can see merit in this approach, but Lachmann misses out on the benefits of phase III trials. Phase II trials will show you that a drug has activity in a particular disease and by doing randomized phase II trials (which are a bit more expensive) you can even see if it is more active than the best available alternative, but without phase III trials you will never know whether the new drug gives you a better lifestyle or a longer life than what went before. And you certainly won't know whether it is worth buying.
2 comments:
There is truth to the argument that the increased costs of Phase III trials lead to increased drug development costs, but even these types of trials may fail to identify the types of adverse outcomes that may be noted when larger numbers of people use the drug. This, of course, depends to a certain extent on the size of the target audience. A disorder such as irritable Bowel syndrome with a large target population which is treated by a wide spectrum of physicians (FPs, GYN, GI specialists, etc) when offered a new therapy is more likely to result in nasty surprises than is likely to occur with a smaller population (eg, CLL patients) who have more to gain from any potential new treatment and who are treated by a smaller, more expert panel of physicians.
Perhaps some sort of compromise would be better...offer such new drugs as part of a large Phase III trial for which patients (and insurers) bear some cost and surrender some of their ability to sue for product liability. This scenario would be difficult to administer and trial lawyers would almost certainly have a field day anyway with any and all adverse effects that might accrue.
The two largest "swamps" are generated by the marketing that is done to sell the drugs (in effect maximizing the developer's profit by increasing the population's exposure to the drug) and the costs of product liability which are expensive.
Currently, eg, trial lawyers in the US advertise for people who have experienced adverse events with drugs to call them on toll free numbers to seek redress, often for problems which are uncommon and which involve drugs which have been widely used for more than 20 or 30 years!
I would not say that lawsuits are pointless. As an example, if you are in your 40s with a family and have an adverse reaction to a drug which results in you not working again, you have little recourse but litigation because you are in survival mode for your family.
TomD
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