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Value Propositions - June 2015

June 2015, Vol 6, No 5

In This Article

Value-Based Drug Pricing a New Target for a Large PBM Company in Its Negotiations with Pharmaceutical Companies

Express Scripts, a large prescription benefit manager (PBM) company that is handling the pharmacy benefits for many health plans and employers, is negotiating with various pharmaceutical companies to link the pricing of high-cost drugs to their true value, or how well they perform in the real world, according to a recent report in the Wall Street Journal.

With the rising cost of drugs, especially for many cancer drugs with an annual cost of >$100,000, value-based pricing or reimbursement continues to be a strong concern for many in healthcare, and the pressure to curb costs continues to be a top issue for insurers and PBMs.

In May 2015, Express Scripts said it was working with drug makers to come up with a payment plan that will tie the cost of their drugs to their performance based on the specific indication in each tumor type, reported Steve Miller, MD, Chief Medical Officer at Express Scripts.

“One of the big frustrations has always been people paying top dollar for drugs that aren’t always giving them the best response,” Dr Miller said in an interview with the Wall Street Journal. “If pharma is truly sincere about wanting value-based reimbursement, we now have the sophistication to do that.”

Dr Miller did not specify which drugs Express Scripts is looking for this type of indication-specific pricing. The company hopes this new arrangement will go into effect in 2016.

Drug pricing has been “very hard for the payers to do anything about,” said Steven Pearson, president of the Institute for Clinical and Economic Review, a nonprofit company in Boston that evaluates cost-effectiveness in medicine. “Now they’re starting to think very hard about it, to look for practical ways to have more of an influence on pricing.”

The Wall Street Journal; May 26, 2015

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Prenatal Blood Test Can Also Detect Cancer in Pregnant Women, Potentially in Other Populations

Previous studies have suggested that using noninvasive prenatal testing (NIPT) by scanning for cell-free DNA (cfDNA) circulating in the blood may have value as a prognostic tool for diagnosing malignancies. Now a new study from Belgium shows that using this new method and cfDNA can indeed reveal the presence of cancer in pregnant women before any symptoms become apparent. The study was presented at the European Society of Human Genetics Conference in Glasgow in June and was simultaneously published in JAMA Oncology (Jun 5 2015; Epub ahead of print).

Frederic Amant, MD, PhD, of the University of Leuven, Belgium, and colleagues used the prenatal blood testing from 4000 pregnant women to perform a cfDNA analysis for any signs of cancer. Their analysis revealed that among the 4000 pregnant women, 3 showed signs of cancer, including 1 woman with stage IV-A ovarian cancer, 1 woman with stage III follicular lymphoma, and 1 woman with stage II Hodgkin lymphoma.

The 3 women then underwent whole-body magnetic resonance imaging, which further revealed the presence of a tumor in all 3 instances. A biopsy performed in each of the 3 women confirmed the number of copy variations initially found in the original prenatal screening.

“We show that plasma DNA profiling allows for presymptomatic detection of tumors in pregnant women undergoing routine NIPT,” the investigators wrote. “We aim to further investigate the potential of NIPT for cancer detection, not only in pregnant women, but also in the general population,” they added, suggesting that this method could be applied to a population-wide cancer screening using “genomic analysis of plasma DNA.”

This study was funded by grants from the University of Leuven Center of Excellence in Computational Biology, the Belgian Ministry of Health, and the Belgian Science Policy Office Interuniversity Attraction Poles program.

European Society of Human Genetics Conference/JAMA Oncology; June 5, 2015

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Myxoma, a Virus Found in Rabbits, Prevents GVHD in Bone Marrow Transplant While Destroying Cancer Cells

According to researchers from the University of Florida, the myxoma virus, which is found in rabbits, can simultaneously help to kill cancer cells while also eliminating a common complication of bone marrow transplants, a treatment used in patients with hematologic cancers (especially in multiple myeloma and acute myeloid leukemia).

Although bone marrow transplant is used to treat hematologic cancers, it also increases the risk for graft-versus-host disease (GVHD), in which the newly transplanted T-cells attack the host tissue, causing skin rash, shortness of breath, abdominal pain, jaundice, and muscle weakness. These common complications can be fatal in severe cases. The risk for GVHD increases when there is no full match between the donor and the patient’s blood marrow.

Christopher R. Cogle, MD, lead investigator and Associate Professor, University of Florida College of Medicine, Division of Hematology and Oncology, said that the myxoma virus can prevent these transplant-related side effects, as well as help to destroy the patient’s cancer cells. The rabbit virus could be especially helpful to patients with recurring disease who are unable to find a bone marrow donor with a perfect match, Dr Cogle said.

The risk for GVHD from bone marrow transplants of partially matched donors is approximately 80%, which can be eliminated by the use of the rabbit virus.

“Myxoma is one of the best strategies, because it is effective but doesn’t affect normal stem cells,” Dr Cogle said. This is especially important in African-American patients and in elderly patients, who are less likely to find perfectly matched donors.

The myxoma virus originates in rabbits in Australia and parts of Europe and is benign to humans.

This is the first time that a virus has been shown to simultaneously prevent GVHD and help to kill cancer cells, according to the researchers, who suggest that this process could one day have broader applications for other kinds of cancers. The investigators hope to be able to raise the money to start a clinical trial within 1 year.

University of Florida press release; June 5, 2015

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First-in-Class Inducer of the KLF4 Gene Receives FDA Designation as Orphan Drug

The FDA granted an orphan drug designation to APTO-253, a first-in-class inducer of the Krüppel-like factor 4 (KLF4) tumor suppressor gene for the treatment of patients with acute myeloid leukemia (AML).

“AML is a particularly challenging cancer of the blood and bone marrow for which there are currently few treatment options,” said William G. Rice, PhD, Chairman, President, and CEO of Aptose Biosciences Inc, a company developing new targeted oncology drugs. "APTO-253, with its unique mechanism of action, has the potential to emerge as an entirely new therapeutic approach for this patient population, and receiving orphan drug designation is a key regulatory milestone along the path.”

APTO-253 is currently in a phase 1b clinical trial in patients with relapsed or refractory AML, high-risk myelodysplastic syndrome, and other hematologic malignancies involved in suppressing the KLF4 gene.

Suppression of the KLF4 gene has been reported as a key feature in the development of AML. APTO-253 is a targeted inducer of the KLF4 gene and has shown a good safety profile, with no evidence of suppression of normal blood cells. Preclinical studies with APTO-253 have demonstrated its strong activity in killing AML cells as a single agent, as well as a potent synergy when used in combination with other drugs.

Aptose Biosciences press release; June 2, 2015

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