Sooner or later, as the nation struggles to contain health care spending, we may need to devise measures to determine whether very high-priced drugs provide enough medical benefit to warrant paying the bill.
Neither the Food and Drug Administration, which decides which drugs can be marketed, nor Medicare, which decides which treatments to cover, considers costs.
Last week, the Centers for Medicare and Medicaid Services decided that Medicare would pay for the use of Provenge, made by Dendreon, for men whose prostate cancer has spread beyond the prostate gland, is no longer responding to hormone therapy, and is causing few or no symptoms. The drug, tailor-made for each patient to spur his immune system to attack the tumor, costs $93,000 for a course of treatment, far more than most patients can pay. In the critical clinical trial, it extended median survival time by only four months compared with a placebo.
In a second case, Medicare said it would pay for an expensive drug that provides almost no benefit for the typical woman with advanced breast cancer. The drug, Genentech’s Avastin, costs $88,000 a year. In clinical trials, when combined with other drugs, it delayed the median time at which tumors started to grow worse from one to 5.5 months. But it failed to extend the lives of patients or improve their quality of life, and in some patients it caused severe side effects, like gastrointestinal perforations and hemorrhaging.
Based on these results, an advisory committee to the F.D.A. recommended rescinding approval for use of Avastin in breast cancer. The final decision will be made by the F.D.A. commissioner. Whatever the F.D.A. decides, Medicare will keep paying for Avastin for those with advanced breast cancer, and some private insurers may do so as well. Some women believe the drug has prolonged their lives even if it has failed to do so for the typical patient. Whether the drug is worth its high price is a question our health care system is currently unprepared to answer.