Chronic myeloid leukemia (CML) 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. 

But that focus on better treatments was not done for free. The downside to a concentrated focus is that the increasing cost of treatments for chronic myeloid leukemiain the United States has reached unsustainably high levels and may be leaving many patients under- or untreated because they cannot afford care, according to an article in Blood.

In the article,
corresponding author Hagop Kantarjian, MD, chairman of the leukemia department at The University of Texas MD Anderson Cancer Center
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 year.

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. 

Treatments are better but increased liability and the regulatory climate have made costs accelerate as well.

"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 Kantarjian in their press release. "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. 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.  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. 

"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 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 Kantarjian.

Citation: Experts in chronic myeloid leukemia, 'Price of drugs for chronic myeloid leukemia (CML), reflection of the unsustainable cancer drug prices: perspective of CML Experts', Blood April 25, 2013 blood-2013-03-490003 doi: 10.1182/blood-2013-03-490003