As a practicing oncologist for eight years, I have come to recognize that the final stages of prostate cancer have been, historically, one of the most challenging tumor types to treat, making our goals, leavened by reality, modest. In 2004, docetaxel-based combination chemotherapies offered for the first time a survival advantage for patients with advanced disease. Unfortunately, docetaxel has serious adverse events that can limit its use in a frail elderly patient population, underscoring the need for finding additional effective therapies. Oncologists and patients alike were eager to find therapies that can extend survival, further improving on the modest, yet impressive, results with docetaxel.
Understandably, the oncology community closely tracked the long-awaited path for the FDA approval of Sipuleucel-T (Provenge), making it available to asymptomatic or minimally symptomatic metastatic castrate-resistant (hormone-refractory) prostate cancer patients.
Provenge received FDA approval in April 2010. It is the first approved immunotherapy for cancer. It is designed to teach the immune system how to target prostatic acid phosphatase, a ubiquitous protein that is over-expressed on prostate cancer cells. Provenge is derived from a patient's own immune cells, so it is tailor-made for every patient.
While Provenge wasn't the first rationally designed immune-focused therapy for cancer, it was the first to show success in the clinic; patients treated with Provenge in the Phase 3 IMPACT study experienced a median survival that was more than four months longer than patients in the control group. The Provenge Phase 3 IMPACT clinical trial results represent a significant successful advance in our fight against prostate cancer.
Many of my colleagues and many of my patients approached this as extraordinary news. While we are celebrating this first-in-class novel breakthrough, there has been much scrutiny over both the economics and necessity of such treatment. Media reports have questioned the price tag and the Centers for Medicare and Medicaid Services took the highly unusual step of asking whether immune therapies for cancer such as Provenge are “reasonable and necessary”--a question rarely asked by regulatory authorities when a survival advantage has been demonstrated.
If one looks critically at the cost of cancer care and the utilization of systemic chemotherapy, the economics of Provenge are in line with the economics of other cancer therapies. The total cost of Provenge does not exceed $93,000, because, as an immunotherapy, the entire treatment regimen is finite: three leukepheresis procedures followed by three infusions all given over a one month period. That makes this approach different from some of the other targeted-therapy regimens, which can be given in many cycles and for a prolonged period of time.
Accordingly, comparing the cost of Provenge to the cost of chemotherapy regimen that is used chronically could be misleading. For example, when evaluating the total cost of chemotherapy it is important to factor in the associated costs, such as in-patient admissions, emergency room visits, transfusions, growth factors, and concomitant therapies to address possible side effects that often occur during treatment. A recent report issued by Milliman, a consulting company, suggests that a cancer patient receiving chemotherapy incurred, on average, costs of approximately $111,000 a year.1
Regarding the question as to whether Provenge is “reasonable and necessary” for the treatment of advanced prostate cancer, I believe the following points demonstrate that it is both reasonable and necessary:
* The FDA approved Provenge as a treatment for asymptomatic or minimally symptomatic mCRPC based on the Phase 3 IMPACT clinical trial results.
* The survival advantage was demonstrated in two studies with Provenge showing consistency.
* The Provenge Phase 3 IMPACT trial results were published in the New England Journal of Medicine on July 29, 2010 and showed Provenge significantly improved patient survival with minimal side effects. The survival advantage was evident regardless of previous or future use of chemotherapy.
* Soon after FDA approval, the National Comprehensive Cancer Network Clinical Practice Guidelines (NCCN Guidelines) for Prostate Cancer added Provenge to the list of treatment options for patients with mCRPC as a Category 1 indication, supporting its use as a foundation of care treatment for this patient population.
* Lastly, according to a 2005 Us TOO survey, only half of the men suffering from late-stage prostate cancer would consider chemotherapy as a treatment option, the only available treatment at the time the survey was done, to delay progression because of quality-of-life concerns. These men need an alternative treatment option that is effective and does not impact quality of life.
* Many patients with mCRPC do not have symptomatic disease initially, suggesting that non-cytotoxic therapies are more attractive. Such patient populations would certainly benefit from Provenge.
As a practicing oncologist, I can share the following: Provenge is an eagerly welcomed and much needed treatment for patients who have few alternatives.
After years of frustration, care for advance prostate cancer is on the verge of a great leap, of which Provenge is a part. Our efforts would be better spent working together to get this therapy to the many patients who can benefit rather than questioning the economics and if it is reasonable.