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Patients vs. Data

In a recent hearing on the reauthorization of PDUFA (Prescription Drug User Fee Act), concerns were raised about the FDA’s decision to revoke the approval of Avastin for use in the treatment of advanced breast cancer. Several congressmen gave heartfelt anecdotes of relatives or friends whose lives were improved or lengthened through the use of the drug, echoing the pleas of breast cancer survivors who testified at a previous hearing on Avastin.

Avastin is the world’s best-selling cancer drug and is prescribed for a slew of indications including colorectal cancer, lung cancer, brain cancer and macular degeneration. Side effects of the drug are severe and can include hypertension and the development of perforations in the nose, stomach and intestines. The FDA’s decision to withdrawal approval for Avastin was prompted by postmarket trials, which showed that it neither prolongs nor increases the quality of life of breast cancer patients.

What about the breast cancer patients who claim that the only thing keeping them alive is Avastin? Due to its many other indications, Avastin will still be on the market. However, a revoked approval likely means that insurance plans will no longer help cover the $90,000 annual cost of the drug—a prohibitive amount for the majority of patients.

In situations where neither data nor anecdote can disprove the other, decision makers are often at an impasse: who or what are they to trust? The clinical data or the stories of patients? Clinical data are backed by the credibility of blind trials and peer review whereas the stories tug at our heartstrings.

The utilitarian answer is to abide by the objective data instead of the subjective accounts of a handful of patients because the money that saved on this marginally effective drug can be put to better use. However, if Avastin is the last hope and patients are aware of the risk, is it not a valid argument that the drug should be made more available for them?

Han Zhong