I am a woman who had breast cancer last year and had a mastectomy, followed by adjuvant chemotherapy with Herceptin, Taxol and carboplatin. The cancer came back and is now in my bones and my doctor recommended Tykerb but the insurance company refuses to pay because the drug is not FDA indicated in this situation. What advice can you give me?
First, let’s discuss the medical aspects of your situation. There are many options for your treatment and Tykeb is certainly one reasonable option among them. Lapatinib(Tykerb) is an oral drug that in trials has been shown to have activity in breast cancers that , like yours, have a molecule on the cell surface called HER. Specifically, Tykerb is effective in patients with metastatic breast cancer, with inflammatory breast cancer, and possibly, with brain metastases. An ongoing clinical trial and another anticipated clinical trial will investigate lapatinib as adjuvant treatment after surgery in early-stage disease.
Lapatinib is FDA indicated for the treatment of metastatic HER2-positive breast cancer, both in combination with the drug capecitabine in patients who have received a taxane, anthracycline and Herceptin, and in combination with letrozole for postmenopausal patients with hormone receptor- and HER2-overexpressing breast cancer.
Not infrequently one encounters HER over-expressing patients who had not been treated with both antracyclines, such as Adriamycin, AND taxanes in the past, as the FDA requires. Taxol is one of the taxane drugs; the others are Taxotere and Abraxane. What about Tykerb for women who did not receive BOTH a taxane and an anthracycline in the past? As Adriamycin use declines, this situation has become more and more common. You were not given Adriamycin after surgery because the Taxol/carboplatin regimen was considered to be as effective and less toxic by your providers, a wholly reasonable medical decision.
What can you say to your insurance? To understand how an off label use, a use of a drug outside of its FDA indication, can still be acceptable and medically appropriate, one must recall that FDA usually, and certainly in the past, based its approval on randomized prospective studies, especially what is called Phase III studies. These are studies that actively compare two different treatments or a treatment and a placebo. More recently, under growing public pressure the FDA has become more flexible and began to accept less compelling evidence as sufficient to approve drugs in special situations. Because such studies are expensive and time-consuming, once a drug is approved, manufacturers do not often attempt to obtain additional indications. Because studies submitted to the FDA were in patients previously exposed to the anthracyclines and taxanes, the approval was for only such patients; however, Tykerb in other HER positive patients is well supported by a variety of phase II and other studies.
What you should do is to enroll your doctor in an appeal process, which most insurers allow. You may also consider engaging an oncologist who is familiar with how insurers think and do things. After all, you only have one chance to reverse the denial and you are more likely to succeed if you “play” their game. A doctor who understands how insurers make decisions and deal with appeals will review the policy and is more likely to successfully argue your case. With thousands of dollars potentially at stake, it may be worth the investment. A physician like this can be located through an URAC approved review organization, or we are happy to help. You can contact us at Cancer Treatment Today.
If the insurer’s policy specifically denies any non-FDA approved therapy, and only minority of them do, you might be facing a difficult, though not an impossible challenge. You might consider an option of considering an alternative approach, which the insurer will accept and which can produce similar results. Talk to your oncologist about that.
You also have an option of engaging an attorney, although in my experience it is a more expensive and less productive option, unless you live in one of the states with specific laws that disallow denying care based on FDA indication alone.
E. Frampton Lapatinib: a review of its use in the treatment of HER2-overexpressing, trastuzumab-refractory, advanced or metastatic breast cancer. Drugs. 2009 Oct 22;69(15):2125-48.
O’Shaughnessy J, Blackwell KL, Burstein H, et al. A randomized study of lapatinib alone or in combination with trastuzumab in heavily pretreated HER2+ metastatic breast cancer progressing on trastuzumab therapy. Program and abstracts of the 44th American Society of Clinical Oncology Annual Meeting; May 30 – June 3, 2008; Chicago, Illinois. Abstract 1015
Gillick, M. Controlling Off-Label Medication Use. Annals of Internal Medicine. March 3, 2009 vol. 150 no. 5 344-347
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