n complications of multiple myeloma include bone pain, kidney dysfunction, bone loss, impaired immunity, and anemia.
Although the overall incidence of multiple myeloma continues to increase, the mortality rates associated with this malignancy have declined during the past years.
Specifically, the advent of novel therapy options for multiple myeloma, as well as improvements in high-dose therapy and supportive care have contributed to extended survival for patients with multiple myeloma.
New anticancer drugs and novel combinations have emerged in part because of improved understanding of the bone marrow microenvironment and the biology of multiple myeloma. Immune modulators and proteasome inhibitors now represent the cornerstones of initial treatment for multiple myeloma based on their ability to enhance the overall response rates and survival.
Because novel agents have had a considerable impact on the US healthcare, understanding their relative cost-effectiveness is important for ensuring efficient use. Overall, recent eva luations of the economics of new agents in multiple myeloma resulted in similar conclusions.Using claims data from more than 2600 patients with multiple myeloma, one study showed that the 1-year cost of bortezomib-based therapy was similar to the cost of older drug combinationswhereas the costs of thalidomide- and lenalidomide-based regimens were significantly higher than older combinations. In addition, patients taking thalidomide and lenalidomide had higher out-of-pocket costs because of Medicare Part D coverage gaps.
The second study modeled the cost-effectiveness of novel agents combined with melphalan and prednisone in patients with newly diagnosed multiple myeloma who were ineligible for a transplant.
The researchers concluded that adding bortezomib to melphalan and prednisone was more cost-effective than adding thalidomide or lenalidomide to the same drug combination.
Despite strides in the treatment of patients with multiple myeloma, patients will experience disease relapse after initial treatment, and multiple lines of therapy are typically required. Considerations for patients with relapsed or refractory multiple myeloma include the duration of response to previous treatment and the risk for toxicity. There remains a marked need for additional therapeutic options for this patient population.
Ixazomib Approved for Relapsed or Refractory Multiple Myeloma
On November 20, 2015, the US Food and Drug Administration (FDA) approved ixazomib (Ninlaro; Takeda Oncology) capsules in combination with lenalidomide and dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma who received at least previous therapy.Ixazomib is the first oral proteasome inhibitor approved by the FDA for this patient population.
The safety and efficacy of ixazomib were demonstrated in the TOURMALINE-MM1 study, an international, phase , double-blind clinical trial.More than 720 patients with relapsed and/or refractory multiple myeloma were randomized to ixazomib plus lenalidomide and dexamethasone or to placebo plus lenalidomide and dexamethasone.After the first prespecified interim analysis, treatment with ixazomib plus lenalidomide and dexamethasone significantly extended progression-free survival (PFS) compared with placebo plus lenalidomide and dexamethasone.
Richard Pazdur, MD, Director of the FDA’s Office of Hematology and |
