Evaluating the Benefits and Costs of Multiple Myeloma Treatment Options

Tampa—Although there is no cure for multiple myeloma (MM), the approval of several drugs in recent years has led to improvements in survival rates and quality of life for patients with the disease. People with MM live an average of at least 8 years post diagnosis, according to Joseph Mikhael, MD, consultant hematologist, deputy director of education, Mayo Clinic, Scottsdale, Arizona.

Still, Dr. Mikhael noted that it is expensive to treat MM, which is the second most prevalent hematologic neoplasm, with nearly 21,000 new cases diagnosed each year in the United States. For instance, the incidence of lung cancer is 11 times greater than the incidence of MM, but the costs associated with MM are >$100 million more than the costs associated with patients with lung cancer and metastatic bone disease.

Dr. Mikhael and others who spoke during a satellite symposium at the AMCP meeting discussed several of the new treatment options and methods used to manage the disease. The session was supported by educational grants from Celgene Corp., Millenium: The Takeda Oncology Company, and Onyx Pharmaceuticals, Inc.

Dr. Mikhael cited data from the 2012 American Society of Hematology annual meeting that found the 5-year survival rates for MM patients who are >65 years of age increased from 31% from 2001 to 2005 to 56% from 2006 to 2010. During the same period, the 5-year survival rates for MM patients who are ≤65 years of age increased from 63% to 73%.

The better survival rates are due to autologous stem cell transplants and new FDA-approved drugs, such as thalidomide, bortezomib, lenalidomide, carfilzomib, and pomalidomide, according to Dr. Mikhael. He added that the new medications have partial response rates of at least 90%, although the agents with the best complete response rates do not always lead to improved survival and better quality of life.

The costs of the new drugs range from $98,000 to $276,000 per year, according to Dr. Mikhael. The most expensive treatment is the combination of bortezomib, lenalidomide, and dexamethasone, while the cheapest option is thalidomide plus dexamethasone.

Comparative Effectiveness Research (CER)

When deciding the best treatment option for MM, healthcare professionals should consider utilizing CER, according Diana Brixner, PhD, RPh, professor, chair, department of pharmacology, University of Utah, Salt Lake City, Utah.

Dr. Brixner said the Institute of Medicine (IOM) defined CER as “the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care.” The IOM added that the purpose of CER is “to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve healthcare at both the individual and population levels.”

In addition to evaluating drugs, CER examines different approaches or methods of treatment, treatment outcomes, and better ways to reach the ideal treatment outcomes. Dr. Brixner noted that unlike randomized controlled trials, CER measures outcomes take place in real world settings, include nonadherent patients, and assess drugs after treatment failure and/or in combination with other medications.

As the costs of medications increase and payers are attuned to health outcomes, CER is becoming more popular, according to Dr. Brixner. As part of the Patient Protection and Affordable Care Act, President Barack Obama’s administration also created the Patient-Centered Outcomes Research Institute, a private, nonprofit health research organization that funds patient-centered clinical effectiveness research projects.

In January 2013, the AMCP released version 3.1 of its Format for Formulary Submissions and included an overview of the types of CER and study designs, such as Bayesian and adaptive trials, pragmatic clinical trials, prospective observational studies, retrospective observational studies, systematic evidence reviews, and modeling studies. AMCP has also partnered with the International Society for Pharmacoeconomics and Outcomes Research and the National Pharmaceutical Council to create a Web site (www.healthstudyassessment.org) that pharmacy and therapeutics committees can use to evaluate available CER studies when assessing drugs.

Dr. Brixner said head-to-head trials are ideal when comparing medications, but they are usually not feasible because they are expensive for manufacturers. She added that there is ample clinical evidence evaluating thalidomide, lenalidomide, and bortezomib in newly diagnosed and relapsed/refractory MM patients, although there is limited data and evidence on pomalidomide (FDA-approved in February 2013) and carfilzomib (FDA-approved in July 2012).

Although modeling studies have found the newer MM medications are cost-effective, Dr. Brixner said the results vary and the high costs associated the drugs should be taken into consideration before prescribing.

Benefit Designs and Managing MM

The increasing costs of medications and the growing trend of specialty pharmaceuticals has caused payers to change their attitudes toward managing oncology. Five years ago, payers never questioned the price of cancer medications and always covered them on their formularies, according to Jeffrey Dunn, PharmD, senior vice president at VRx Pharmacy Services, LLC.

Now, however, he said payers are paying more attention to the value of treatment options. In the coming years, he expects more formularies will have multiple specialty tiers to encourage people to receive less costly and more cost-effective drugs.

Dr. Dunn cited the Magellan Pharmacy Solutions’ pharmacy and oncology trend report that found 97% of the 21 payers surveyed in 2012 had a medical formulary in place for chemotherapy, up from 57% of the 28 payers surveyed in 2011. Furthermore, 48% of patients with MM were included on the formulary in 2012, down from 63% in 2010.

He also mentioned a 2012 report from Atlantic Information Services that surveyed 101 payers and found cancer was the top management priority followed by type 2 diabetes, asthma, hyperlipidemia, rheumatoid arthritis, depression, chronic pain, and hypertension. In addition, 60% of the payers said they would be or would likely be implementing clinical pathways within 18 months to help manage the rising costs associated with oncology.

Clinical pathway programs are usually evidence-based approaches to care developed by providers or national organizations that specify what interventions should be performed in what sequence. According to Dr. Dunn, the most common clinical pathway programs are for breast cancer, lung cancer, and colorectal cancer, and then MM.