The FDA rejected the recommendation of its advisory panel, which last December voted 5-4 against the drug, because the benefit in slowing tumor growth wasn't believed to be worth the added risk of serious side effects, including high blood pressure and death. Avastin killed more patients than the control arm.
This mirrors the statin debacle. Drug companies didn't have to prove that lowering cholesterol did anything positive for patients, just that it lowered cholesterol. So we're back to square one again. Instead of showing a drug can increase survival, all they have to do is show that it can shrink a tumor. Didn't we just come from that neanderthal moment?
I'm all in favor of the FDA approving any and all the drugs they want. The needed change in the war on cancer will not be on the types of drugs being developed, but on the understanding of the drugs we already have. The system is overloaded with drugs and underloaded with the wisdom and expertise for using them.
Pre-testing cancer cells before treatment, whether with chemotherapy or targeted therapies, would be more advantagous. Laboratory screening of samples from a patient's tumor can help select the appropriate treatments to administer, avoiding ineffective drugs and sparing patients the side effects normally associated with these agents.
Even these "smart" drugs, like all other chemotherapy drugs, do not work for everyone, they often have unwanted side effects, and they are extremely expensive. Patients, physicians, insurance carriers, and the FDA are all calling for the discovery of predictive tests that allows for rational and cost-effective use of cancer drugs.
Proof of efficacy of a cancer treatment such as Avastin requires a randomized trial in which it has been shown that the group treated with it experienced significantly increased survival when compared to that of an untreated group. Most claims for the efficacy of a chemotherapeutic agent comes from trials showing shrinkage of tumors.
Unless tumor shrinkage is accompanied by evidence of increased survival, the treatment cannot be claimed to be effective. Tumor shrinkage should not be the criteria for approving cancer drugs. Pharmaceutical companies are not concenttrating on solving the problems of metastasis, they focus on devising drugs that shrink tumors. As the executive director of The Breast Cancer Action says, the FDA has lowered the bar on the approval of breast cancer therapies.
Another case of lowering the bar is the validation standard that private insurance carriers is accepting from molecular profiling tests, "accuracy" and not "efficacy." No longer will it be essential to prove that the use of a diagnostic test improves clinical outcomes, all they have to do for these molecular profiling tests is prove that the test has a useful degree of "accuracy." However, at the same time, the validation standard they want for "cell-based" profiling tests is "efficacy."
The cell-based profiling tests have the same entitlement to be judged by the same validation standard as molecular profiling tests. The combination of measuring morphologic (structural) effects and metabolic (cell metabolism) effects constitutes measuring the "profile" at the whole cell level. It must be noted that both types of dignostic tests are just that, "tests" and not treatment.
We are witnessing too much hypocrisy in the cancer medicine system (a.k.a. Provenge). Their pockets are not deep enough.