Going Off-Label

There is a push among some cancer researchers to treat cancers based on genetic mutations rather than their area of origin. Because some breast cancers, for example, share mutations that appear in various lung cancers, a drug that is approved for breast cancer might prove effective for lung cancer as well -- but with the current system of FDA approvals, when clinical trials have not been completed for the lung cancer variant, it may be quite difficult for a lung cancer patient to access such a drug. The types of cancers in this scenario can readily be swapped with other cancers, too; the more that the genetic structures of cancer cells are understood the more ways are being discovered to effectively attack them.

Dealing with insurance approvals

Insurance companies, however, might be unwilling to pay for medications that are outside the strict approvals for the type of cancer being treated. This is a multi-tiered problem, with decisions being made within the insurance companies by people who are not necessarily experts in the medical condition being treated, as well as concerns over liabilities for treatments that have not been narrowly proven effective and safe through the clinical trial process.

Negotiating for a new medication requires a skilled member of the medical team acting on behalf of the patient. While there may be room to make a case with an insurance company, it often takes critical time that the patient may not have to squander. It seems like the only option sometimes is for the patient to pay out of pocket -- a prospect that, for most cancer patients, may well be out of the question considering the high cost of new cancer drugs.

Assistance programs & other resources can help with costs

But patients need not always be left without recourse. Off-label use of cancer drugs is becoming more common and accepted, even when insurance companies need a bit more cajoling. And when insurance denies a claim, some drug companies are helping to fill the gap by offering free medication to qualifying patients. To best take advantage of this, however, the patient and doctor should plan in advance, preparing various options in case the insurance poses a roadblock to treatment.

Certainly, there is an incentive for insurance companies to promote "proven" treatments whenever possible. Off-label use of medicine is often considered experimental, so there would be reason for hesitancy. But as the cancer narrative is redefined to revolve around the genetics that underly the disease, this should become less of an issue. Only in recent years, after all, have tumors routinely been sequenced to look for actionable mutations. Just a few decades ago, targeted therapies were still the stuff of science fiction. Now, however, the number of known mutations is growing exponentially. And taking advantage of research across the old cancer-discipline boundaries, it is possible to see how tumors originating in very different tissue types might still share unique vulnerabilities that can be exploited through new medicines.

A shift in the way cancer treatment is approached

Off-label use of drugs is nothing new. It has been used for years in treating issues like depression and pain management. But with the high costs and potential risks of cancer treatment, changing the approach of standard treatment will take time. It is all part of this new, shifting cancer narrative being led by rapid advances in the genetic understanding of this complicated disease.

Editor’s Note: We are extremely saddened to say that on October 21, 2018, Jeffrey Poehlmann passed away. Jeffrey’s advocacy efforts and writing continue to reach many. He will be deeply missed.