Sanjiv S. Agarwala, MD
Professor of Medicine
Temple University School of Medicine
St. Luke’s Hospital and Health Network Bethlehem, PA
The US oncology community applauded the FDA’s approval of ipilimumab (Yervoy) in March, and the European Commission supported the use of the drug for patients with previously treated advanced melanoma among European Union member nations in July.
In the United Kingdom, however, the National Institute for Health and Clinical Excellence (NICE) has issued a draft recommendation against its approval, stating that clinical trial results thus far do not demonstrate sufficient benefits to justify its costs, and that biomarkers are needed to identify patients who would benefit. The agency is scheduled to make a final decision in February.
Sanjiv S. Agarwala, MD, chief of Oncology/Hematology at St. Luke’s Hospital and Health Network in Bethlehem, Pennsylvania, is enthusiastic about Yervoy but believes NICE’s points are well taken.
“It certainly is practice-changing,” said Agarwala of the drug, noting that increasing overall survival in patients with melanoma has been quite difficult.
Agarwala agrees that focusing more efforts on genome mapping and biomarker identification research is critically important in order to deliver a more personalized therapy for melanoma patients.
At the same time, he said, the costs of the drug are certainly worthwhile to those who would benefit.
“There are about 8000 metastatic melanoma patients in the United States,” said Agarwala. “That’s a relatively small number of patients who can be helped by the drug, but if it increases their survival, then it will be worth the cost to them.”
Yervoy, which Bristol-Myers Squibb developed, is the first drug that has been shown to help patients with advanced melanoma live longer. The FDA’s approval was based on a clinical trial in which patients were randomized to receive Yervoy in combination with the investigational peptide vaccine gp100, Yervoy alone, or gp100 alone.
Patients who received Yervoy either alone or in combination had a median overall survival (OS) of approximately 10 months, compared with a median OS of 6 months for those in the vaccine-only arm. Participants had been previously treated with 1 or more of 5 drugs.
Sir Andrew Dillon, chief executive of NICE, said in announcing the draft guidance in October that “the results did show the drug could potentially be very effective for a small percentage of patients.”
He said, however, that the trial did not compare Yervoy with currently used therapeutics, and it likely would improve survival for only about 30% of patients, with about 10% of those patients experiencing long-term benefits.
In its economic analysis, NICE said the incremental cost-effectiveness ratio (ICER) would be at least £54,000 to £70,000, and possibly significantly higher, per quality-adjusted life year (QALY). In the United States, a complete course of treatment consisting of 4 infusions of 3 mg/kg doses during a 3-month period carries an estimated cost of $30,000 per infusion, or $120,000 for the series.
NICE makes recommendations to the National Health Service that apply in varying ways in England, Wales, Northern Ireland, and Scotland. The type of guidance ultimately issued determines whether a drug or device will be covered under a mandatory funding requirement.