Economic Burden of
With an aging population, from 2010 to 2030, there will be a 57% increase in the number of patients living with myeloma.12 A 2017 study published in Leukemia13 showed that the percentage of MM patients using novel therapeutics increased from 8.7% in 2000 to 61.3% in 2014. In a 2017 Journal of Clinical Oncology study,14 patients with MM were the highest percentage of patients taking targeted oral anticancer medications between 2010 and 2012 (26% of MM patients in 2012). That just includes lenalidomide and thalidomide, not ixazomib, which was approved in 2015.14
In addition to more diagnoses, patients are living longer due to more effective treatments. Patients diagnosed after 2010 had better survival outcomes and higher rates of novel therapy use than those diagnosed previously. The study found that the proportion of those diagnosed in 2012 with a 2-year survival rate was 1.25 times higher than those diagnosed in 2006. While the total costs for MM health care services increased, researchers found that drug costs remained fairly stable between 2009 and 2014.13 Cost increases are due to combinations of highly expensive agents, continuous treatment with maintenance, and improved survival leading to longer lifespans.15
Although treatments for MM can be costly, one study showed that direct cost to patients associated with oral drugs for MM can be significantly reduced through financial assistance.16 In a retrospective study, pharmacy claims were analyzed for patients with MM who received thalidomide, lenalidomide, or pomalidomide from a large specialty pharmacy in the United States between January 1, 2011, and December 31, 2013. Average direct cost to patients, per prescription, for any of these three agents was $227.23 prior to financial assistance and $80.11 after financial assistance, representing an average patient savings of $147.14 per prescription. With financial assistance, the percentage of all prescriptions with an average direct cost to patients of $50 or less increased from 57.6% to 86.2%.16
A different Journal of Clinical Oncology17 study showed that Medicare beneficiaries with MM who did not receive a low-income subsidy during 2007-2011 faced a substantial financial difficulty in accessing oral cancer medications. The median out-of-pocket cost for first IMiD prescriptions was $3178 for those without the subsidy, and the median out-of-pocket first year cost of IMiD therapy was $5623 for the same group. However, the patients treated with IMiDs had significantly fewer hospitalizations and emergency room visits compared with those who received only bortezomib. One-year survival rates and cumulative Medicare costs were the same with or without the IMiD.
The development of treatment guidelines and algorithms are a step in the right direction, said Dr Callander, but better data is needed to understand the relative value of different treatment options. “We’re operating to a certain extent without the best information we can have to say one treatment is more cost effective than another,” she said.
One study identified 2551 elderly Medicare patients with advanced MM from 2000 to 2009 who initiated novel agent-based therapy (bortezomib, lenalidomide, or thalidomide) or chemotherapy from the Surveillance, Epidemiology, and End Results-Medicare database.1 Twenty-month cost of care and OS related to MM were compared between patients treated with novel agent-based therapy and patients treated with chemotherapy. Overall, average 12-month MM total costs were 2.03 times higher for novel agent-based therapy ($144,665) than for chemotherapy ($47,750). Antimyeloma pharmacy costs represented about 31% ($45,095) of total MM costs for patients treated with novel agents but represented about 19% ($8921) of total MM costs for patients treated with chemotherapy. At the same time, 12-month survival rates increased significantly among patients receiving novel agents compared with patients receiving chemotherapy.1
A study in American Drug and Health Benefits18 estimated the annual costs—including administration, prophylaxis, adverse event monitoring, and adverse event treatment—for 7 common relapsed MM treatment regimens. The study found that total cost was highest for regimens that included lenalidomide (range: $126,000-$256,000), whereas treatments including bortezomib plus dexamethasone and the combination of panobinostat, bortezomib, and dexamethasone were less than $125,000 per patient (bortezomib plus dexamethasone was $90,616). While such analyses provide cost estimates, a balance between the cost and benefit from a regimen weighed with the adverse event profile and quality of life impact is very difficult discern for absolutely all regimens available.
These findings were contradicted by another retrospective study19 using 2006-2013 claims data from a large US database, which compared average patient monthly direct medical and pharmacy costs for patients who did not receive stem cell transplants. These patients received lenalidomide- or bortezomib-based treatment. The study tracked costs from treatment initiation to progression and initiation of second-line treatment, using time to next therapy (TTNT) to show progression. The study included 2843 newly diagnosed patients and found that total monthly cost for newly diagnosed patients was $15,734 initially, declining to $5082 at 18+ months after treatment. When second-line therapy was initiated, the total monthly costs were $13,876, declining to $6446 after 18 months. Patients on lenalidomide-based treatment had longer TTNT and longer periods of below-average costs compared with the bortezomib group. While treatment costs for both medications was similar, the lenalidomide group had mean monthly total costs at least $3200 lower than total costs for patients who received bortezomib as initial treatment. Cumulatively, that resulted in $120,000 in lower costs for lenalidomide patients.
The costs of adding the novel therapeutic panobinostat to the bortezomib and dexamethasone combination was published in a 2016 study.20 Panobinostat, the first histone deacetylase inhibitor, was approved for MM treatment by the FDA in 2015. The study estimated the economic impact of adding it to US health plan formulary as part of that treatment option for relapsed/refractory MM patients previously treated with a proteasome inhibitor and an IMiD. The study found that it was cost neutral and potentially cost saving compared with some newer treatments; commercial or Medicare plan costs increased by less than $0.01 per member per month, while PFS increased by 4 months compared with placebo.20 Such analyses are helpful but there are variations in how the cost-effectiveness analyses are conducted, making it difficult to obtain universal recommendation guidelines based on such analyses.