番茄社区

LAS VEGAS -- The oral BRAF inhibitor vemurafenib (Zelboraf) increased overall and progression-free survival in patients with metastatic melanoma, but the cost may not justify its use in a state Medicaid program, giving rise to many ethical questions, a budget impact analysis suggested.

Assuming an incidence of 11 cases of metastatic melanoma in the MassHealth program over 1 year, conventional treatment with dacarbazine would cost $31,873 per year, whereas a year of treatment with that agent plus vemurafenib would carry a price tag of $314,347, according to Amber King, who is a PharmD student at Northeastern University in Boston.

"Given the inexorable progression of late-stage melanoma, these findings have important ethical implications, such as what price do you put on one patient's life and how treatment decisions are made," King told 番茄社区 during a poster presentation at the midyear clinical symposium of the American Society of Health-System Pharmacists.

During 2012, more than 75,000 Americans were expected to be diagnosed with melanoma, with 4% developing poor-prognosis metastatic disease.

In Massachusetts, an estimated 2,190 cases were expected in 2012.

Before the approval of vemurafenib in 2011 for patients whose tumors have BRAF mutations, the sole approved agent for metastatic melanoma was dacarbazine, but response rates have been reported at only 7% to 12% with this agent.

In their analysis, King and colleagues first summarized the known clinical efficacy data, and found that in a phase III study, median progression-free survival was 5.3 months for vemurafenib compared with 1.6 months with dacarbazine.

Overall 6-month survival rates for the two treatments were 84% and 64%, respectively.

In their budget impact analysis for the 1.3 million patients enrolled in MassHealth over a 1-year period, with progression-free survival of 5.3 months, the base-case scenario estimate was an additional $9,995 per month for each patient treated with vemurafenib.

In contrast, a month of treatment with dacarbazine would cost $1,811 per person.

In a favorable-case scenario, which also factored in efficacy and low costs associated with adverse events and oral administration for vemurafenib, the first year of treatment would cost a projected $536,139, compared with $6,559 with dacarbazine.

Arguing in favor of the use of vemurafenib were factors such as an increase in patient self-sufficiency, decreased institutional resource use, and improved quality of life, according to King.

But she noted that this analysis was limited by a paucity of long-term data, and advised that further experience will be needed to better clarify the clinical and economic implications of the use of vemurafenib.

Given the budget constraints facing MassHealth, stakeholders including physicians, policy-makers, and patients need to carefully consider these implications, King noted. "There will be lots of conflicts and questions."