Value Propositions – March 2015
CLL Therapy Named “Cancer Advance of the Year” by ASCO
In its 2015 Annual Report Against Cancer, for the first time ever the American Society of Clinical Oncology (ASCO) named the advances made in a specific tumor type as “cancer advance of the year.” That cancer is chronic lymphocytic leukemia (CLL). This new designation highlights the recent FDA approval of 4 new drugs in a span of 12 months for the treatment of patients with CLL, the most common type of leukemia in adults.
“This has truly been a banner year  for CLL and for clinical cancer research as a whole. Advances in cancer prevention and care, especially those in precision medicine, are offering stunning new possibilities for patients,” said ASCO President Peter P. Yu, MD, FACP, commenting on the announcement. “It’s also remarkable that almost one-third of the year’s top studies were made possible by federal research dollars. We cannot underestimate the importance of federal investment for answering critical cancer care questions, particularly in rare, understudied cancers.”
The 4 new drugs include the first 2 immunotherapies to receive FDA approval for leukemia—ofatumumab (Arzerra) and obinutuzumab (Gazyva)—and 2 targeted therapies, idelalisib (Zydelig) and ibrutinib (Imbruvica). Ofatumumab and obinutuzumab are both anti-CD20 antibodies indicated for use in combination with chlorambucil for treatment-naïve patients with advanced CLL. These immunotherapies delay the progression of CLL by approximately 12 months.
Idelalisib and ibrutinib are 2 targeted therapies, and each of them is a first-in-class oral agent for CLL. Idelalisib is the first PI3K-delta inhibitor approved by the FDA for patients with relapsed or refractory CLL for use in combination with rituximab (Rituxan).
Ibrutinib is the first Bruton’s tyrosine kinase inhibitor approved by the FDA for the treatment of patients with CLL and a deletion in chromosome 17, a marker of poor prognosis in CLL.
“These new therapies fill an enormous need for thousands of patients living with CLL,” said Gregory Masters, MD, FACP, FASCO, Co-Executive Editor of the ASCO annual report. “For many older patients, especially, these drugs essentially offer the first chance at effective treatment, since the side effects of earlier options were simply too toxic for many to handle.”
Approximately 120,000 Americans have CLL, and approximately 16,000 new patients are diagnosed with the disease annually.
Cancer Network; January 21, 2015
Novel Test Can Identify Apoptosis from Cancer Drugs in 16 Hours
A new test developed by researchers at Dana-Farber Cancer Institute may offer a new direction in personalized or precision medicine. The test, called Dynamic BH3 Profiling (DBP), can predict which drug among a variety of current treatments will be most effective for a specific tumor type. DBP is designed to detect the early signs of apoptosis in a patient using a cancer drug, and the test can assess the efficacy of individual drugs or drug combinations. The DBP usually can begin to detect the self-destruction of cancer cells within 16 hours.
“This measurement can be made in less than a day. It turns out that those drugs that push cancer cells closer to the threshold of apoptosis even over this short time frame are the drugs that eventually kill the cancer cells best, both in the laboratory and in mice, and even humans,” said Anthony G. Letai, MD, PhD, Associate Professor, Harvard Medical School and Dana-Farber Cancer Institute, Boston.
The researchers have tested cancer drugs for a variety of tumors, including melanoma, breast cancer, leukemia, lymphoma, prostate, colon, and ovarian cancer. According to Dr Letai, the new test is 80% to 90% accurate in predicting which drug is most effective for a specific tumor.
“We can take early pick to see if the cancer cell is being pushed toward death. What makes this especially powerful is that we’re not restricted to using one drug at a time; it can test the effectiveness in combinations,” said Dr Letai. “This is functional precision medicine, getting the right drugs to the right patient.”
FoxNews.com; February 26, 2015
Filgrastim-Sndz First Biosimilar Approved by the FDA
The FDA approved the cancer drug filgrastim-sndz (Zarxio; Sandoz/Novartis) as the first biosimilar agent to ever be approved in the United States. Filgrastim-sndz is biosimilar to filgrastim (Neupogen), and is approved for the same indications as filgrastim, which include several hematologic cancers and severe neutropenia. A biosimilar must demonstrate the same safety and effectiveness that were shown with the original (reference) biologic drug; a biosimilar, however, is expected to be sold at a lower price than its reference biologic drug.
“Biosimilars will provide access to important therapies for patients who need them,” said FDA Commissioner Margaret A. Hamburg, MD. “Patients and the health care community can be confident that biosimilar products approved by the FDA meet the agency’s rigorous safety, efficacy and quality standards.”
The Biologics Price Competition and Innovation Act of 2009, which was passed as part of the Affordable Care Act in 2010, created a pathway for the FDA to approve biosimilars in the United States. Based on that act, biosimilars can be approved if they can provide sufficient evidence to show that their efficacy and safety profiles are biologically similar or interchangeable with an FDA-approved biologic agent, which is known as the “reference” drug. The biosimilar must demonstrate that it has the same mechanism of action, route of administration, and dosage form/strength as the reference drug, and it can only be approved by the FDA for the same indications for which the reference drug is approved.
US Food and Drug Administration; March 6, 2015
Type of Health Insurance Affects Clinical Outcomes in Patients with Brain Tumor
According to researchers from the University of Florida Health, the type of health insurance—private versus public—can affect the clinical outcomes of patients with brain tumor. The researchers found that patients with brain tumor who are uninsured or are covered by Medicaid are hospitalized for longer periods and have more complications than patients who have private insurance. In part, this could be related to the late diagnosis that is more typical for this patient population; these patients have less access to care than those with private insurance. And as in most cases of cancer, a late diagnosis is associated with a poorer prognosis than an early diagnosis.
“When private-insurance patients start to have a problem, it gets picked up really fast. They go to a primary doctor, who makes a quick referral to a neurologist or neurosurgeon,” said lead investigator Kristopher G. Hooten, MD, Resident, Department of Neurosurgery, University of Florida College of Medicine, Gainesville. The results of the study were published in February in Neurosurgery.
“It’s both an access-to-care and a quality-of-care issue before patients are admitted. [Uninsured or Medicaid patients] come in when their brain tumors are more advanced,” Dr Hooten said.
The uninsured and patients covered by Medicaid were at increased risk for developing a new medical condition in the hospital, and their risk for death during hospitalization was 25% greater than those with private insurance. They were also more likely to end up in a nursing home, a rehabilitation center, or hospice than patients with private insurance. These results are based on an analysis of 2002-2011 National Inpatient Sample data, the largest healthcare database in the United States. The researchers collected information on 566,346 hospital admissions for brain tumor.
“This type of research is important from a global standpoint to understand what goes into quality assessment, how hospitals are ranked based on quality and which patients are potentially high-risk. The true benefit is identifying areas of improvement and making things better for patients,” suggested Maryam Rahman, MD, Assistant Professor, Department of Neurosurgery, University of Florida.
University of Florida press release; March 3, 2015