In the past few years, the world—or at least the portion of it dealing with cancer and its treatment—has gone gaga for oral drugs. Ever since the stunning success of Gleevec, a once-daily anti-leukemia capsule, patients, doctors, and drug companies have been wooed by the siren call of pill-based medicine. Numerous such drugs have been approved in the past few years for several major cancers, and at least one-quarter of new cancer drugs in the pipeline are for oral formulations.
These oral drugs appear to herald a new era for cancer as a chronic illness—not a deadly disease, but a pesky condition like high blood pressure that simply requires swallowing a few pills every week. The convenience of taking pills at home instead of sitting at the cancer clinic with an IV tube stuck in your vein for hours on end is obvious. In part, pill-based therapy is a consequence of medical advancement. Traditional chemotherapy, which attacks all fast-growing cells (tumors, hair, bone marrow), requires a period of recovery between doses. Most new drugs are designed to kill only cancer cells and their enablers. Doing so practically requires that the medicine be in a take-home pill form because it means your body needs constant exposure to the medication.
But oral cancer drugs are hardly the dream treatment that many believe them to be. A host of problems means their use could end up in calamity, causing a far bigger headache than lying in a chair at a cancer clinic ever could.
First off is the elusive question of adherence: How likely are cancer patients to follow their at-home regimens correctly? Failure to follow prescriptions is a perennial conundrum of modern medicine that no one seems to quite understand. It might seem that cancer patients, coping with such a severe diagnosis, would be immune to that particular problem. Not so. The difficult regimens many treatments require can easily lead to missed doses. One study found that even among patients prescribed Gleevec—just one pill a day, no muss, no fuss—only half took their pills exactly as instructed.
On the flip side, some patients may be too willful. Fixated on the idea that they will die if they don't take their medicine, they may push themselves to endure debilitating side effects that really call for at least a change in dose. A breast cancer patient with an "I can get through this" mentality might not report diarrhea to her doctor on Friday, continue to take Xeloda over the weekend, and by Monday face life-threatening dehydration that could easily have been prevented. The assumption that cancer treatment equals suffering—as suggested by TV shows, movies, friends' and family members' experiences—may also lead patients to postpone a call to the doctor. When a patient expects to feel miserable during therapy, it might even seem silly to inform the clinic about side effects.
All of which leads to the question: Are cancer patients able to doctor themselves? That is essentially what is happening for many oral-drug takers. Patients need to be carefully instructed about what side effects to expect, how to know whether or not they are serious, and what to do if they are. There also needs to be an infrastructure to ensure that patients taking treatment at home are safe and well-cared-for in the absence of a doctor's or nurse's careful watch. A recent study found that few of those safeguards are in place at cancer centers around the country. How those safeguards will get there is anyone's guess, since there are no budgets for installing such measures nor are such expenses reimbursed.
The ramifications of the pill trend also extend to the clinic. The extra counseling on the drugs and their side effects is time-consuming (although many pharmacists are relieved to serve a purpose beyond counting pills). But the real issue for clinics is with—surprise!—insurance. Unlike traditional chemotherapy drugs, oral drugs require prior authorization. The insurer needs to approve a prescription before it's filled, a task that regularly forces nurses, administrators, and even doctors to spend hours of nonreimbursed time on the telephone.
Also, many insurers require patients to fill their prescriptions through mail-order pharmacies, resulting in delays and botched shipments. For example, one kidney cancer patient suddenly experienced inexplicable disease progression on a drug that had been working for months. It turned out that her most recent refill, left on her front porch when she wasn't home to receive the delivery, had gone bad in the summer heat. Mail-order pharmacies also require large refills, sometimes up to 90 days, a completely impractical measure for a disease whose treatment requires frequent dose adjustments. That factor alone leads to thousands of dollars of medicine wasted.
On the plus side, many oral drugs are now distributed exclusively by specialty pharmacies, which help manage cancer patients taking treatment at home. Staffed by oncology pharmacists, these businesses serve as surrogate doctors, calling patients regularly to check about side effects, issue refill reminders, and help answer questions. But the extra middleman, while often useful, can just as easily lead to confusion and miscommunication. For example, a specialty-pharmacy caseworker might not know about a change in dose level made by the patient's doctor, causing problems with the next refill scheduled to be sent by the specialty pharmacy. Or a caseworker might neglect to report all the details of a conversation about an ongoing side effect. Motives come into question, too: Many specialty pharmacies provide data to drug companies about what drugs a doctor is prescribing to what patients, giving companies that restrict distribution of their drugs to specialty pharmacies a competitive edge over those who provide their pills through regular retail outlets.
Government insurance doesn't make things any easier. Oral cancer drugs are covered under Medicare Part D, but with a serious catch. Part D covers the first $2,000 with a 25 percent co-pay, followed by a $2,850 coverage gap known as the doughnut hole, for which patients are completely responsible. After patients emerge from the doughnut hole, benefits resume for the rest of the year. Because oral cancer drugs are extremely expensive, a Medicare-insured cancer patient will enter the doughnut hole after a single prescription and may not be able to afford a refill. Back to the clinic administrator, who then spends hours locating a patient-assistance program, a charitable organization set up to help cancer patients afford their treatments. Disturbingly, most patients who receive such assistance are probably getting money from an organization that gets donations from the very drug company whose pills they cannot afford.
Every single oral cancer drug is covered by Medicare Part D, giving pharmaceutical companies an extra incentive to focus on this approach. But doctors receive zero revenue for administering oral therapy. By contrast, traditional chemotherapy, covered under a different area of Medicare (without a doughnut hole and with generally lower co-pays), accounts for about 80 percent of the average oncologist's revenue. The eventual economic ramifications for cancer-care professionals are unknown.
Oral cancer drugs are an ideal option for vast numbers of cancer patients. These at-home regimens are convenient, often do have milder side effects than traditional chemotherapy, and herald a new era for those suffering from a horrible disease. For a patient who clearly comprehends what side effects are normal, which are not, and other complicated health matters, at-home treatment may make good sense. Clearly, though, hurdles abound, and many patients and doctors are proceeding under the misconception that pill-based therapy is a snap.
A recent report from the National Comprehensive Cancer Network, one of the main U.S. organizations for cancer-care professionals, states that it will probably be a decade before any cancer regimen is entirely oral-based. That should give plenty of time to work out the kinks.