It’s a well known secret that many medications work only for a fraction of the patients who take them. This is especially true for two common diseases: certain cancers and Alzheimer’s disease, where the percentage of patients benefiting from costly drugs is very small. So the “system” pays billions of dollars for therapies that bring no benefit at all, except in small proportion of patients where the benefit is very small or transient. It is a subject of great debate (often taking on religious and philosophical dimensions) whether prolonging the life of a terminally ill patient by 2-3 months is justified for an expensive drug in an over-burdened health-care system, but I suspect most people will reach different conclusions depending on whose life is being discussed.
Given that there is no choice that will please everybody, the main options are to ration treatments (that is, withhold payments for questionable treatments), shift most of the financial burden of such treatments to the family, cap drug costs at much lower levels or pay for the drug only if a particular patient actually benefited (using criteria that will need to be developed for this purpose). To ask developers of new drugs to show superiority over existing therapies (so as to get higher prices) is also logical. These measures have been used to a different degree in different situations and countries.
Recently, Medicare agreed to pay for off-label uses for some cancer therapies (that is, using the drug to treat a different type of cancer than the one approved by the FDA, and for which usually there is no good proof of efficacy). I happen to believe that for life-threatening conditions (at a late stage) the FDA should lower the bar significantly for approval. In return for the easier access to market, pharmaceutical companies should reduce costs and accept a much lower payment (or no payment) when the drug didn’t work at all or caused greater harm. I realize, of course, how tricky this is (on many levels), but common sense solutions are never easy to implement.
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