An expensive novel agent for acute lymphoblastic leukemia, blinatumomab (Blincyto, Amgen), will be reimbursed after all by the Centers for Medicare & Medicaid Services (CMS), but two experts are questioning this decision.
Not only is the drug costly but its "efficacy is unknown," assert Sanket S. Dhruva, MD, from the Robert Wood Johnson Foundation Clinical Scholars Program and Yale University School of Medicine, New Haven, Connecticut, and Vinay Prasad, MD, MPH, from Oregon Health and Sciences University, Portland.
It appears that the CMS has bowed to industry pressure and reversed a previous decision regarding reimbursement, the pair write in a Viewpoint article published online December 30 in JAMA Oncology.
CMS will now authorize an additional payment to hospitals under Medicare's new technology add-on payment (NTAP) program for blinatumomab, for use in patients with relapsed or refractory acute lymphoblastic leukemia (ALL).
The NTAP was established to soften the financial blow hospitals take when they use expensive new technology. It provides supplementary payments to hospitals to offset additional costs associated with the adoption of innovative technologies and interventions.
Dr Dhruva and Dr Prasad question the latest CMS decision, pointing out there is "no reliable evidence" that suggests blinatumomab will benefit the Medicare beneficiary population (ie, 65 years and older) and argue that for "a number of reasons the drug may not."
Blinatumomab is the first of a novel class of drugs known as bispecific T-cell engagers and is designed to direct cytotoxic T cells to CD19-expressing cancer cells. CD19 is a protein expressed on the surface of B-cell-derived ALLs and non-Hodgkin's lymphomas.
The drug was approved in December 2014 by the US Food and Drug Administration (FDA) for patients with Philadelphia chromosome-negative precursor B-cell ALL who have relapsed or who were refractory to previous treatment.
Some experts have greeted the drug with enthusiasm, believing that along with other new agents, the treatment of hematologic cancers can be revolutionized.
Very High Price Tag
However, similar to many of the new oncologic agents that have entered the marketplace during the past decade, blinatumomab has a high price tag; in fact, it is priced at about $178,000, making it one of the most expensive.
The FDA labeling also states that as many as five cycles of blinatumomab may be administered, which will significantly boost the cost, the authors point out. Hospitalization is recommended for the first 9 days of cycle 1 and the first 2 days of cycle 2 because of the need to monitor the patients for toxic effects, which include tumor lysis syndrome, neurologic effects, febrile neutropenia, and the cytokine release syndrome.
"These last 3 effects occurred at any grade in 52%, 28%, and 2%, respectively, of the patients in the registration trial," they write.
The approval was also based on the findings of an uncontrolled, open-label, single-arm phase 2 study, Dr Dhruva and Dr Prasad write. The primary end point was a surrogate consisting of complete remission with or without partial hematologic recovery, and although that was achieved in 41.6% of the cohort, the benefits were short lived; the median time from response to relapse or death was 5.7 months.
The authors also emphasize that in the registration study, the Medicare population was not well-represented. The median patient age was 39 years, and only 25 (13.2%) of 189 patients were older than 65 years. Because of their age, Medicare beneficiaries may have more comorbid conditions which in turn can reduce the effectiveness or tolerability and could increase serious adverse events. In addition, the frequency of stem cell transplant among those older than 65 years was not reported in the registration study, and transplant is challenging in this patient population.
"Without consolidation through transplant, blinatumomab appears to be a costly and toxic drug that can generate only modest and transient responses with no clear improvement in survival or quality of life," they write.
Does Not Meet NTAP Criteria
Another issue they highlight is that certain criteria must be met for the NTAP program, and the authors argue that blinatumomab does not fit the bill.
First, it must be a new technology, and although CMS initially deemed that it was not, they changed that opinion after an appeal from the manufacturer.
Second, the current Medicare diagnosis-related group severity must be inadequate, which Amgen has argued is the case.
Third, and the most important one for patients, note the authors, is that the technology should represent "an advance in medical technology that substantially improves, relative to technologies previously available, the diagnosis or treatment of Medicare beneficiaries." On the basis of current data, the fulfillment of this point is very unclear, they argue.
CMS is prohibited from considering cost when it makes coverage decisions, and this includes the NTAP program. The agency also has little opportunity to oppose cancer drugs with marginal or questionable benefits because it is required to pay for any drug that is recommended in one of several compendia, no matter how "unfounded the recommendation."
But the NTAP program provides an opportunity for CMS to say no to additional payments for blinatumomab. This is not because the drug is costly, "but because its efficacy is unknown," the authors write. "Regardless of cost, NTAP for blinatumomab should be questioned."
"As prices for cancer drugs continue to rise, serious reforms are needed to allow the CMS to make impartial decisions devoid of industry pressure," Dr Dhruva and Dr Prasad write. "Although changes may seem politically intractable, the current system with endlessly escalating cancer drug prices and payers unwilling to oppose them is a runaway train heading for a cliff."
The authors have disclosed no relevant financial relationships.
JAMA Oncol. Published online December 30, 2015. Extract
Medscape Medical News © 2015 WebMD, LLC
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Cite this: Medicare's NTAP for Blinatumomab Should Be Questioned - Medscape - Dec 30, 2015.