hard enough. However, the vast majority may not be helped, and many might even be harmed by side effects. The risk to benefit ratio for any medication is evaluated based upon the seriousness of the disease being treated. Society is willing to tolerate more serious side effects in a cancer medication than a cold remedy, but these still need to be outweighed by the benefits. And since most patients can’t afford to pay for these medicines out of pocket, then insurance companies must bear the cost. This is ultimately passed along to consumers in the form of higher premiums. Insurance companies are not in the habit of paying for drugs that are ineffective, have serious side effects, or both, which is exactly what the FDA concluded in their review of this treatment for breast cancer. There is a longstanding historical rationale for having the FDA serve as a societal shield that protects consumers from dangerous and ineffective drugs.
I’ll suggest a potential solution for those rare patients who might be helped by the drug, but whose insurance companies won’t pay for it. Why not set up a system where patients that may benefit from the drug get to take it on a trial basis? Their insurance plan will pay for its use in this indication under strict guidelines. If the patient shows no clear benefit from the drug in a specified period of time, then the drug’s seller will reimburse the insurance company (or the patient, if they paid for it out of pocket) for the expense.
Such an approach, though admittedly complicated, has several benefits. Patients will gain “off label” access to a drug that their doctors feel may benefit them, and have the expense covered by their insurance if it works. Insurance companies only pay for the drug if it works effectively; otherwise the manufacturer reimburses them. The drug company’s potential pool of patients will be expanded with a proviso that their sales are directly tied to the ability of the medicine to benefit this population. This reimbursement approach links company profits from “off-label” sales to how frequently the drug actually works in this population, which would help promote more effective medicines.
This idea is not as radical as it sounds. A very similar approach is already being employed in England. Britain’s drug oversight group, the National Institute for Health and Clinical Excellence (NICE), originally rejected covering the multiple myeloma drug bortezomib (Velcade) for patients in the U.K. Andrew Dillion, the head of NICE, put forth a “money back” proposal to the drug’s seller “If the drug’s manufacturer accepts the proposals . . . it will mean that when the drug works well the NHS pays but when it doesn’t the manufacturer should bear the cost. All patients suitable for treatment will get the chance to see if the drug works well for them.” Janssen-Cilag, the drug’s manufacturer, accepted the proposal under which NICE would now recommend the drug (which costs $49,450 per year). Patients that show a minimal response, or none at all, will have their treatment stopped and the drugs costs will be refunded. Why not bring such a system here to the U.S?
This isn’t to say that all drugs should be sold on a refundable basis if the patient derives no benefit. While a merchant can readily establish that a toaster is defective or a pineapple is bad, proving that an expensive drug failed to work would likely require costly and
