Bloomberg.com has posted a particularly moving account of the struggles of US cancer patient Terence Foley in his battle with a rare form of kidney cancer.

One striking statistic – the total cost of his treatment was $$618,616.

The article raises questions about the economics of health care, the way highly-priced anti-cancer agents are selected for use against rare cancers and discussion of whether the cost was justified by the results.  Read this moving story here.

We often hear about the incredible efficacy of new anti-cancer agents when used against hard to treat cancers (e.g. kinase inhibitors, HER2 inhibitors, EGF-R inhibitors, etc.) but how well do the results in the real world compare with the Phase III approval studies?

Some believe there is very little correlation at all.  To quote Marcia Angell:

“Many drugs that are assumed to be effective are probably little better than placebos, but there is no way to know because negative results are hidden… Because favorable results were published and unfavorable results buried…the public and the medical profession believed these drugs were potent… Clinical trials are also biased through designs for research that are chosen to yield favorable results for sponsors. For example, the sponsor’s drug may be compared with another drug administered at a dose so low that the sponsor’s drug looks more powerful. Or a drug that is likely to be used by older people will be tested in young people, so that side effects are less likely to emerge. A common form of bias stems from the standard practice of comparing a new drug with a placebo, when the relevant question is how it compares with an existing drug. In short, it is often possible to make clinical trials come out pretty much any way you want, which is why it’s so important that investigators be truly disinterested in the outcome of their work… It is simply no longer possible to believe much of the clinical research that is published, or to rely on the judgment of trusted physicians or authoritative medical guidelines. I take no pleasure in this conclusion, which I reached slowly and reluctantly over my two decades as an editor of the New England Journal of Medicine.”

Taken from Marcia Angell (2009), “Drug Companies & Doctors: A Story of Corruption”, New York Review of Books, Vol 56, No 1; 15 January 2009.

Something for all our up-and-coming drug developers to think about.

John McCulloch

John provides assistance to life sciences entrepreneurs in business strategy, management, intellectual property, financing and licensing. See more…