Starting on October 1, Medicare will cover more of the $178,000 cost for a standard course of treatment with the new immunotherapy, blinatumomab (Blincyto; Amgen), which was approved by the FDA in December 2014 for the treatment of patients with Philadelphia chromosome–negative precursor B-cell acute lymphoblastic leukemia (ALL).
The high cost of new cancer drugs continues to be a major concern for patients, providers, and society at large, with prices continuing to climb with each new FDA approval. A recent poll from the Kaiser Family Foundation released in July showed that 94% of Democrats and 84% of Republicans are in favor of allowing the government to negotiate with drug manufacturers to get lower prices on medications for Medicare beneficiaries.
Although the initial decision of Medicare was not to pay the extra cost for blinatumomab because the data did not show that it substantially improves outcomes for patients with ALL, a later decision to cover more of the cost of the drug came after additional information from the drug manufacturer suggested that the drug “represents a substantial clinical improvement over existing treatment options,” according to a New York Times (NYT) report from August 8.
This decision potentially indicates a change by Medicare and a willingness to absorb more of the cost of promising therapies, especially the new targeted therapies.
The article cites several cases of patients with ALL whose disease was in remission after 28 days of receiving blinatumomab. “It was amazing to me that it could work so well so quickly,” Ms Jane Wirth told the NYT. “I had just spent a month going through standard chemotherapy, which did not make the cancer go away. It seemed so hopeless.”
Discussing the benefits of blinatumomab, Anthony S. Stein, MD, of City of Hope National Medical Center in Duarte, CA, said, “Its mechanism of action is totally different from that of any other approved drug.”
Source: New York Times; August 8, 2015.
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I. Ixazomib First Oral Proteasome Inhibitor for Patients with Relapsed or Refractory Multiple Myeloma
Takeda Pharmaceuticals announced that the FDA granted priority review for the New Drug Application (NDA) of its investigational drug ixazomib, the first oral proteasome inhibitor intended for the treatment of patients with relapsed and/or refractory multiple myeloma.
“We are encouraged that both the U.S. and European regulatory bodies have determined that the ixazomib applications qualify for an expedited review, underscoring the importance of new treatment options for patients with relapsed/refractory multiple myeloma,” said Melody Brown, Vice President of Regulatory Affairs, Takeda.
“Our ixazomib program is designed to evaluate whether sustained therapy with an oral proteasome inhibitor improves the outcomes of patients living with multiple myeloma. There is a significant unmet medical need in multiple myeloma and we look forward to working with the regulatory bodies to bring ixazomib to patients.” The European Medicines Agency also recently granted ixazomib accelerated review.
The NDA submission for ixazomib was based mainly on the results of the first prespecified interim analysis of the pivotal phase 3 trial, TOURMALINE-MM1. This international, randomized, double-blind, placebo-controlled clinical trial of 722 adults with relapsed and/or refractory multiple myeloma compares the efficacy of ixazomib plus lenalidomide (Revlimid) and dexamethasone (Decadron) with placebo plus lenalidomide and dexamethasone. The patients continue with treatment in the trial until disease progression to assess the long-term outcomes of ixazomib plus lenalidomide and dexamethasone.
Source: Takeda Press Release; September 9, 2015.
II. Alectinib for Patients with ALK-Positive Lung Cancer
A day earlier, Genentech announced that the FDA accepted its NDA for alectinib, an investigational oral anaplastic lymphoma kinase (ALK) inhibitor, and granted it priority review for the treatment of patients with ALK-positive, locally advanced or metastatic non–small-cell lung cancer (NSCLC) whose disease progressed with crizotinib (Xalkori).
The FDA granted the priority review based on results from 2 phase 2 studies, NP28761 and NP28673, which showed that treatment with alectinib led to objective response rates of 47.8% and 50%, respectively. The median durations of response were 7.5 months and 11.2 months, respectively. The safety results were consistent with the results of previous studies.
“Alectinib was granted Priority Review by the FDA based on results from two studies showing the medicine shrank tumors in people with ALK-positive NSCLC that progressed on crizotinib,” said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development at Genentech. “There is a need for new treatment options in this patient population, especially because the disease often spreads to the brain at progression.”
In June 2013, the FDA granted alectinib a breakthrough therapy designation for the treatment of patients with ALK mutation NSCLC whose disease progressed on crizotinib. The FDA is scheduled to make its decision on approval by March 4, 2016.
Source: Genentech Press Release; September 8, 2015.
A team of economists suggests that the current drug development system promotes research for cancer drugs that treat late-stage cancer, because they are cheaper to develop than drugs used in early-stage disease. The study was published in the American Economic Review by economists from the University of Chicago and the Massachusetts Institute of Technology (MIT) who say that it is less expensive to develop drugs for use in late-stage cancers, mainly because the duration of clinical trials for these drugs is shorter, as a result of the life span of the enrolled patients being shorter. Therefore, the drug manufacturer has a longer life span under the patent protection than drugs being developed for early-stage cancer, because patent protection starts when a patent is filed, not when the drug is approved by the FDA.
“There is a pattern where we get more investment in drugs that take a short time to complete, and less investment in drugs that take a longer time to complete,” said Heidi Williams, PhD, MSc, MIT economist and coauthor of the study. Dr Williams, together with Benjamin N. Roin, MBA, MS, PhD, of MIT, and Eric Budish, PhD, of the University of Chicago, estimate that the current system’s pattern of encouraging late-stage drugs has resulted in the loss of many lives.
“We investigate whether private research investments are distorted away from long-term projects. Our theoretical model highlights two potential sources of this distortion: short-termism and the fixed patent term. Our empirical context is cancer research, where clinical trials—and hence, project durations—are shorter for late-stage cancer treatments relative to early-stage treatments or cancer prevention….We document several sources of evidence that together show private research investments are distorted away from long-term projects. The value of life-years at stake appears large,” Dr Budish and colleagues wrote.
The team suggests that a change to the current patent system that will start the clock when the drug gets to market rather than when the patent application is submitted, to even the playing field for drugs that take longer to develop, will encourage more drug developers to focus on early-stage disease, when more lives can be saved.
Dr Roin, a patent law and biomedical innovation expert, said in a statement that changes to this policy are “extremely realistic,” although he believes it would take time. The team also suggests that because public funding does not involve pressure from the private sector to produce return on the investment, more public funding should be poured into early-stage cancer drug development.
Source: Budish E, et al. Am Econ Rev. 2015;105:2044-2085.
