New Cancer Drugs: Very Costly but They Increase Survival

September 07, 2016

The high cost of cancer drugs is a huge concern within the oncology community, with some patients even facing bankruptcy while keeping up with payments.

But there may a silver lining ― along with the burgeoning price tags, the use of the newer expensive cancer drugs has led to large gains in life expectancy, concludes a new analysis.

The new findings are published in the September issue of Health Affairs.

The cost of treating patients with metastatic breast, lung, or kidney tumors or chronic myeloid leukemia (CML) has definitely spiked in recent years, but this has been accompanied by "meaningful improvements in survival," note the authors.

They give the example of metastatic breast cancer, for which 11 new drugs were approved during the period 1996-2011. During that time, the lifetime costs, including costs for outpatient and inpatient care, increased by $72,000, but life expectancy increased by 13 months, they point out.

In contrast, for patients who were not treated with the newer drugs, life expectancy increased by 2.0 months (possibly because of improvements in supportive care or lead-time bias), and costs increased by $8900.

Survival Gains With New Drugs

Led by David H. Howard, PhD, an associate professor in the Department of Health Policy and Management at Emory University, in Atlanta, Georgia, the authors used the Surveillance, Epidemiology, and End Results (SEER)–Medicare database to assess the value of new cancer treatments in routine clinical practice.

They limited their assessment to metastatic breast, lung, or kidney cancer or CML in the periods 1996-2000 and 2007-2011.

During the study periods, the US Food and Drug Administration approved more than 25 new drugs for these cancers.

Although it is possible that other therapies could figure into the survival gains, Dr Howard pointed out that these patients were generally not candidates for curative surgery or radiotherapy.

"We limited the sample to patients with late-stage metastatic disease," he told Medscape Medical News. "In some cases, they received radiation therapy, but treatment was really limited to drugs."

That said, improvements in imaging could have played a role. "As a result of better imaging, doctors are better able to identify the stage of cancer," Dr Howard said. "It is possible that, as a result of better imaging, patients who were categorized with metastatic disease towards the end of the study period would not have been categorized with metastatic disease at the beginning."

If that was the case, it could impart a bias to the study results, although that is unclear. "I don't think it is a major concern ― we show that survival hasn't improved much among patients who don't receive drugs ― but it can't be dismissed entirely," he added.

Value of Care

The cohort included 73,024 cancer patients across the four tumor types that were included. The great majority (n = 62,865) had lung cancer.

There were 25,174 patients diagnosed in 1996-2000; 47,850 who were diagnosed in 2007-2011.

Life expectancy increased by 3.9 months. Costs increased by $23,000 for lung cancer patients who received drug therapy; it was basically unchanged for those not receiving treatment.

Similarly, for patients with kidney cancer, life expectancy increased by 7.9 months, with accompanying lifetime costs increasing by $44,700. A number of oral drugs received approval for kidney cancer beginning in 2007, and the authors point out that survival and cost estimates do not fully reflect the impact of these drugs because they were not widely used in the latter study period.

Patients with CML experienced the largest gain in life expectancy ― 22.1 months ― which can probably be attributed to the introduction of imatinib (Gleevec, Novartis) in 2001. Lifetime medical costs for CML patients increased by $142,200, of which $126,300 was attributable to Medicare Part D spending.

"The changes in life expectancy relative to costs were much larger for those treated with physician-administered anticancer drugs than for those who were not," the authors comment. "The fact that survival gains were concentrated among patients who received drugs suggests that changes in life expectancy and costs were mainly attributable to changes in drug therapy — and were not attributable to changes in the timing of diagnosis or other factors that would have affected all patients diagnosed with metastatic disease.

"Even though life expectancy for patients diagnosed with metastatic breast, kidney, or lung cancer has improved, it remains low — especially for lung cancer," the authors comment. "Thus, there is the potential for future research and development on new drugs to produce substantial benefits."

Quality of Life?

One factor that the authors did not look at was quality of life and how that fits into the total picture regarding value.

"Quality of life is an important outcome for patients with early-stage cancer, given that 5-year survival rates often exceed 90%," Dr Howard said. For patients with early-stage disease, "we have that luxury," he commented. "But for late-stage disease, survival time is still the focus.

"I've heard people make the argument that in addition to prolonging survival, newer drugs are associated with fewer side effects," he continued. "However, studies that have examined this issue have not found that to be the case. Newer drugs may be associated with a different side-effect profile, on average, but overall quality of life is not better."

A key policy question is whether new and expensive cancer drugs offer sufficient value to justify the high cost. It is an issue that the American Society of Clinical Oncology (ASCO) has been working on and recently developed a strategic initiative to define value in cancer care.

In the ASCO framework, value is defined by clinical benefit, toxicity, and cost. Thus, theoretically, higher cost would be justified for drugs that provide long and high-quality survival, whereas drugs with minimal survival effect and/or high toxicity should cost less.

"That already goes on to some degree," said Dr Howard. "Drugs associated with longer gains in life expectancy have higher prices if you measure prices as the amount paid over the entire course of treatment.

"I don't favor price controls, but I think it would be fine if a private insurer threatened to withhold coverage from a low-value drug as a negotiating tactic," he added.

"Our results highlight the importance of considering outcomes and overall costs in routine practice when assessing the value of anticancer drugs as a group," the authors conclude.

"Our results also raise the question of whether back-of-the-envelope calculations based on drugs' prices and the survival benefits reported in clinical trials provide an accurate measure of value," they caution.

The study was funded by Pfizer, Inc. The authors have disclosed no relevant financial relationships.

Health Aff. 2016;35:581-1587. Abstract

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