Costly Prostate Cancer Vaccine-Provenge-Likely to Be Covered by Medicare

The Centers for Medicare and Medicaid Services (CMS) say that the first-ever cancer vaccine, sipuleucel-T (Provenge) from Dendreon would be covered for FDA-approved usages of the treatment. CMS released a draft National Coverage Decision (NCD) on March 30th that would not change the current reimbursement situation for on-label use of Provenge, which currently allows all 15 subcontractors of Medicare to cover the vaccine for its approved indications. The reimbursement announcement was released in January 2010. 

For off-label, the draft coverage decision states that the decision to use the therapeutic prostate cancer vaccine would be left to the discretion of local Medicare claims carriers. The NCD stated that the main reason for not prohibiting off-label Provenge use was the hope that non-indication use would be confined to clinical trial studies as there is no current evidence to support additional indications. "If this turns out to be an overly optimistic viewpoint," states the draft, the agency may "reconsider this NCD to ensure that Medicare coverage is restricted to uses that are supported by robust evidence." Other reasons for allowing off-label use include enabling contractors to rapidly adapt coverage requirements as clinicians begin to gain experience with the vaccine. Leaving the possibility that the Provenge indication will be expanded, “we want our administrative contractors to have the flexibility to determine local coverage without the need to reconsider an NCD.”

The FDA approved the prostate cancer vaccine last April for men with asymptomatic or minimally symptomatic metastatic, castrate-resistant (hormone-refractory) prostate cancer. Provenge was approved based on three Phase 3 studies of 737 patients, including the pivotal IMPACT 512-patient study which extended median survival beyond 2 years, demonstrating a median improvement of 4.1 months compared to the control group (25.8 months versus 21.7 months). Provenge reduced risk of death from prostate cancer by 22.5% compared to the control group. 

The autologous vaccine is designed to induce an immune response against an antigen that is expressed in many prostate cancers, prostatic acid phosphatase (PAP). It is the first autologous cellular immunotherapy approved by the FDA. The vaccine is produced in a multi-step process: white blood cells are extracted from the patient’s blood sample, exposed to the PAP protein in order to “train” the blood cells to specifically target and attack the patient’s prostate cancer cells. The “trained” white blood cells are then administrated via autotransfusion in order to attack the prostate cancer cells in the patient’s body. The overall goal of this immunotherapy is to stimulate the body's natural defense system in a specific manner so that they attack and destroy, or at least prevent the proliferation of, cancer cells. The FDA-approved dosing regimen is three doses with each dose administered two weeks apart. The total 4-week treatment costs $93,000; $31,000 for each of the three infusions. 

As a condition of the FDA approval and part of the REMS (Risk Evaluation and Mitigation Strategy), Dendreon must set up a registry of a minimum of 1500 Provenge patients to track prognostic prostate cancer variables and cerebrovascular adverse events linked to treatment. 

Dendreon has since committed to collecting additional data on patients, a large portion of which is expected to be Medicare patients, according to the company. The new data collection commitment came after the November Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) meeting of presentations and public comment, and a subsequent meeting with the CMS.

The registry study protocol will be submitted to the FDA by June 30th and will be used to fill in the evidence gaps, including analysis on whether black men may benefit more from Provenge than other groups. Data on prior patient therapies will also be collected. The final NCD coverage will be published June 30th after an interim whereby it will accept public comments to aid in drafting the NCD.