Leading leukemia experts: High leukemia treatment costs may be harming patients

(Press-News.org) (WASHINGTON, April 25, 2013) – The increasing cost of treatments for chronic myeloid leukemia (CML) in the United States has reached unsustainably high levels and may be leaving many patients under- or untreated because they cannot afford care, according to a Blood Forum article supported by nearly 120 CML experts from more than 15 countries on five continents and published online today in Blood, the Journal of the American Society of Hematology (ASH). Blood Forum articles are a new feature in the journal that present well documented opinions on controversial topics and provide a sounding board for current subjects of importance to the science and practice of hematology.

CML was selected as the focus of the piece because it is now considered a highly curable disease, thanks to the emergence of powerful, targeted CML therapies known as tyrosine kinase inhibitors (TKIs) that allow patients to manage their disease with few symptoms by taking a well-tolerated pill. Since the introduction of TKI therapy more than a decade ago, the annual mortality of patients with this disease has declined from 10 to 20 percent in the early 2000s to just 2 percent today and the estimated 10-year survival of CML patients has increased from 20 percent to more than 80 percent. Patients with CML, who were once told at diagnosis that they had a grim prognosis, are now enjoying close to normal life spans as long as they receive and adhere to prescribed treatments. The management of CML has become similar to that of chronic disorders such as diabetes and hypertension, yet a key difference remains in the extremely high cost of CML drugs.

"Patients with CML have a much better outlook today than ever before, thanks to advances that have greatly improved survival rates. But these patients now face dire financial struggles as they try to maintain their treatment regimen with the drastically inflating cost of care. And this issue likely extends to patients with other types of cancer who require ongoing treatment to maintain therapeutic benefit," said corresponding author Hagop Kantarjian, MD, chairman of the leukemia department at The University of Texas MD Anderson Cancer Center.

In the Blood Forum article, Dr. Kantarjian and colleagues note that newly approved CML treatments in the U.S. are priced substantially higher than older options, and the trend is consistent among other cancer types. For example, of the 12 drugs approved by the U.S. Food and Drug Administration for various cancer indications in 2012, 11 were priced above $100,000 per yeari. Monthly cancer drug prices today (more than $10,000 per month on average) have almost doubled from just a decade ago, when they averaged $5,000 per month. The overall cost burden on families is significant, as out-of-pocket cancer care-related costs comprise approximately 25-30% of an average annual household budget. Cancer care-related costs contribute heavily to the unprecedented cost of health care in the U.S., now estimated at 18 percent of the U.S. Gross Domestic Product, compared with just 6-9% in much of Europe.ii

"A major question we need to answer is how to determine the 'right' price for these drugs. In many cases, it makes sense to let the market govern the price; however, when a product is directly related to a patient's survival over a period of years, it is critical to set a price that allows companies to profit and ensures that patients can afford their treatment," said Dr. Kantarjian. "Since CML treatments must be taken on an ongoing basis, we are concerned that the surging prices are potentially harming patients."

Research suggests that up to 10 percent of patients in the U.S. fail to take prescribed drugs, largely because of cost concerns.iii And while U.S. CML survival rates have improved in the last decade, the estimated survival remains at roughly 60 percent, suggesting that a portion of the population is not receiving adequate treatment, which may be related in part to the high cost of these therapies. By comparison, in Sweden, where costs are managed and compliance rates are high, CML survival rates are at least 80 percent.iv

"We believe that lowering the prices of CML drugs might improve accessibility to treatment and increase treatment adherence, effectively expanding the population of patients who live longer by continuing their TKI therapy," said Dr. Kantarjian.

The authors advise that advancing a long-term solution for the high price of these drugs will require the participation of and collaboration among many invested parties, including treating physicians, patients, advocacy groups, and pharmaceutical companies, as well as government entities, insurance companies, and pharmacies. Collaborations will require agreement from a cooperative group on how best to manage the research process to control treatment costs, how the community can balance those investments, and how newly approved products are priced in the market; similar to established processes in other countries around the world.

"Identifying better ways to manage the cost of cancer care will require an evolution in thinking about current pricing-related policies and regulations, including those that limit price negotiation for Medicare coverage of treatments, as well as patent-related laws that limit the introduction of more affordable generic drugs," said Dr. Kantarjian. ### Blood, the most cited peer-reviewed publication in the field of hematology, is available weekly in print and online. Blood is the official journal of the American Society of Hematology (ASH), the world's largest professional society concerned with the causes and treatment of blood disorders.

ASH's mission is to further the understanding, diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems by promoting research, clinical care, education, training, and advocacy in hematology. blood® is a registered trademark of the American Society of Hematology.

i "New Drug Approvals Hit 16-Year High In 2012." Chemical and Engineering News. 91 (5): 15-17. 2013. Accessible at : http://cen.acs.org/articles/91/i5/New-Drug-Approvals-Hit-16.html iiNational Health Expenditure Projections 2011-2020; Accessed from: http://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/NationalHealthExpendData/Downloads/Proj2011PDF.pdf (Accessed: 10/2012). iii "The costly war on cancer-New cancer drugs are technically impressive. But must they cost so much?" The Economist: May 26 2011. Accessible at: http://www.economist.com/node/18743951 ivBjorkholm M, Ohm L, Eloranta S, et al. Success story of targeted therapy in chronic myeloid leukemia: a population-based study of patients diagnosed in Sweden from 1973-2008. J Clin Oncol: 29: 2514-2520. 2011. Accessible at: http://jco.ascopubs.org/content/early/2011/05/16/JCO.2011.34.7146.full.pdf