The past decade has given us several breakthrough treatment options for a complex and serious malignancy, multiple myeloma (MM). The next decade promises more breakthrough treatment options and may be the most important period for patients diagnosed with MM, as we learn more about this disease and offer treatment options that may lead to improved survival. We are currently on the cusp of this paradigm shift in the treatment of MM, and the excitement about newer therapies and combination regimens is palpable in the oncology community.
This publication provides an excellent summary of the 2012 American Society of Hematology meeting presentations on carfilzomib (Kyprolis), alone and in combination with other therapies. The proteasome inhibitor carfilzomib is one of the breakthrough drugs currently available for the treatment of MM that has progressed after treatment with bortezomib (Velcade) and an immunomodulatory drug (IMiD) (eg, thalidomide or lenalidomide). Although carfilzomib has not shown improved survival, the fact that patients have responded to this therapy after being treated with bortezomib and an IMiD is an important focus of future therapy for MM. It is known that MM almost always relapses, and remission durations shorten with each relapse.1 This is leading to the development of several additional treatment options for this malignancy, as well as to novel combination therapies, especially for patients at high risk for relapse.
Approximately 60 new medications are in development for the treatment of MM, with approximately 10 new medications in late-stage (phase 3 clinical trials) development.2 Although much attention has been given to one of the leading candidates in new drug development for MM, notably pomalidomide, other agents will likely gain similar attention in the near future, including oprozomib, an oral proteasome inhibitor that is a structural analog of carfilzomib.
Not all new drug development for MM is being met with excitement; some of it is being met with anxiety. That anxiety is notably evident with payers, as we continually see rising prescription drug costs at minimal clinical and, in this case, survival impact. Payers are excited to see advancement in the treatment of serious illnesses such as MM; however, the costs of these therapies alone—and especially in combination—is being viewed as cost-prohibitive. The published annual cost of therapy with lenalidomide plus dexamethasone and that of bortezomib as a single-agent therapy in the treatment of relapsed and/or refractory MM ranges between approximately $109,000 and approximately $130,000 per patient.3 The same article notes that the acquisition costs of lenalidomide and bortezomib were comparable; therefore, combination therapy with these 2 agents could result in the annual cost of therapy exceeding $200,000 per patient. This could be the future of the treatment costs for patients with MM, because of the high rate of relapse of this disease, and the growing interest in combination therapies for patients with relapsed and/or refractory MM.
Payers will respond to the continuing increase in the costs to treat MM, regardless of the excitement related to the progress being made in the treatment of this disease. It will not be uncommon to see the inclusion of MM in pay-for-performance programs, further adoption of treatment guidelines for MM, the adoption of end-of-life care programs, and common managed care strategies, such as utilization management and formulary management, over the next decade.
One of the strategies that will coincide with the burgeoning pharmaceutical pipeline for MM is medical management, rather than only focusing on pharmacy benefit management. As treatment options cross the pharmacy benefit and the medical benefit for payers, there is increasing interest by payers in managing treatment across both benefits. This is consistent with the growth in the use of oral therapies, and injectable therapies in combination, for MM. Many payers are relying on health plans, pharmacy benefit managers, and specialty pharmacies to assist with this strategy, which will grow in popularity as more treatment options become available, especially given the high cost of these therapies.
Another strategy that will help payers to address their concerns about the cost of therapy for MM, as well as other oncologic conditions and specialty disease states, is that of patient-centered oncology medical homes and accountable care organizations. As these care-delivery models gain in maturity and processes are streamlined, payers will again work with physicians and other caregivers to manage disease states across all benefits and sites of care.
The hope is that these models will establish a continuum-of-care approach to the treatment of patients with chronic, complex illnesses using a payment model that incentivizes physicians to follow the latest treatment guidelines and to use cost-effective methods to keep rising costs in check, while providing best-in-class care. This is especially important in the treatment of conditions such as MM, which is a very expensive disease to treat, given its rate of relapse and the cost of treatment options currently available as well as those in development.
Specialty pharmacies will continue to play an important role in the treatment of patients with MM. Regarding oral therapies, specialty pharmacies will continue to ensure that patients with MM begin therapy as prescribed, and that patients continue their therapy, as long as they are achieving a positive response while mitigating potential side effects. Specialty pharmacies will also continue to see some shift of injectables from a “buy-and-bill” approach to one where specialty pharmacies will dispense medications to the physician’s office for administration of the drug in the office setting. This will create a stronger bond between specialty pharmacies and oncologists in the treatment of MM.
The future for the treatment of MM is exciting, given the advances in understanding the disease and the development of therapies to successfully treat patients diagnosed with this condition. The use of various strategies to ensure that the right patient receives the right treatment at the right time will help to mitigate the anxiety of payers regarding the rapidly increasing costs of treating MM.
Author Disclosure Statement
Dr Kaddis has no conflicts of interest to report.
References
1. Rajkumar SV. Multiple myeloma: 2011 update on diagnosis, risk-stratification, and management. Am J Hematol. 2010;86:57-65.
2. Pharmaceutical Research and Manufacturers of America. Medicines in Development: Cancer. 2012 Report. www.phrma.org. Accessed January 23, 2013.
3. Borrello I, Binder G, Kahn ZM, et al. Total costs of therapy with lenalidomide or bortezomib in relapsed/refractory multiple myeloma (rrMM). J Clin Oncol. 2012;30(suppl):Abstract 18561.