Study Questions Value of Costly Cancer-Drug Combinations

By Peter Loftus Features Dow Jones Newswires

A new study is stirring debate about whether the benefits of cancer drugs are worth their cost, particularly as drugmakers develop treatments that combine multiple pricey drugs.

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The study of about 4,800 women with an aggressive type of breast cancer found that adding Roche Holding AG's drug Perjeta to the company's older treatment Herceptin conferred a slight benefit versus Herceptin alone, after the women had undergone surgery to remove tumors. Some 94.1% of women receiving the combination in the study stayed free of invasive breast cancer three years after the start of treatment, versus 93.2% of those who received Herceptin alone -- a difference of less than a percentage point.

That modest benefit comes at a high price: more than doubling the one-year treatment cost to $158,000 a patient from $74,500. The combination also had higher rates of certain side effects than Herceptin alone, including severe diarrhea.

"The marginal improvement of adding Perjeta is tiny for the average person, but the incremental cost is going to be substantial," said Richard Schilsky, chief medical officer of the American Society of Clinical Oncology, which is hosting a conference where the study results were released Monday. He wasn't involved in the study.

Dr. Kathy D. Miller, an oncologist at Indiana University Melvin and Bren Simon Cancer Center in Indianapolis, wrote a critical editorial in the New England Journal of Medicine, published online along with the study results Monday. "The toxic effects (and cost) are too great for too many to benefit too few," she wrote.

Roche said the benefit of Perjeta and Herceptin in the new study could mean the difference between relapse and staying disease-free for many women. "We see this as a very meaningful benefit," Daniel O'Day, CEO of Roche's pharmaceutical unit, said in an interview. The company plans to file for regulatory approval to market the combination for patients after breast-cancer surgery, also known as adjuvant treatment.

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The finding comes as many drug companies including Roche, Merck & Co. and AstraZeneca PLC race to test whether new combinations of cancer drugs can improve outcomes versus single-drug therapy or older treatments.

Last month, the Food and Drug Administration approved adding Merck & Co.'s Keytruda, which costs $13,000 a month, to an older combination of lung-cancer treatments that includes Eli Lilly & Co.'s Alimta, which costs more than $5,700 for a 21-day treatment cycle.

In 2015, the FDA approved the use of a combination of Bristol-Myers Squibb Co.'s Yervoy and Opdivo to treat the deadly skin cancer melanoma, based on a study showing it slowed cancer progression better than either drug alone. The two drugs together cost more than $250,000 for the first full year of treatment.

For one class of cancer drugs, which harness the body's immune system to attack tumors, the number of clinical trials testing them in combination with other drugs more than tripled to 765 from 215 in late 2015, according to a new report from industry research firm Evaluate Ltd. Roche alone is running 45 clinical trials that test combinations of cancer immunotherapies, said Sandra Horning, chief medical officer of Roche's Genentech unit.

The trend concerns health insurers. UnitedHealth Group sometimes has to pay much more for the use of cancer combination therapies that contain pricey new brand-name drugs than it does for cheaper combinations of generic drugs that may be just as good, Lee Newcomer, senior vice president of oncology and genetics, said at a panel discussion about cost at the ASCO conference.

In the study, the Perjeta-Herceptin combination had a more pronounced benefit in a subset of women considered at higher risk of relapse, including those with cancer that had spread to lymph nodes, Gunter von Minckwitz, lead researcher for the study and head of the German Breast Group, an academic alliance that runs clinical trials, said in an interview. He said he expects health insurers and government health programs to be more open to paying for the combination's use in higher-risk patients.

Some doctors said they would probably avoid using the Perjeta-Herceptin combination in patients at lower risk of relapse. "Would I have preferred to see a higher benefit? Certainly yes," Eleni Andreopoulou, a breast-cancer specialist at Weill Cornell Medicine and NewYork-Presbyterian hospital, said in an interview. Dr. Andreopoulou, who enrolled patients in the study but wasn't a lead researcher, called it a positive result but said she would like to see more tests developed to predict which patients are more likely to benefit from the combination based on biological traits of tumors, and which aren't.

Herceptin, introduced in 1998, improved treatment of breast tumors with high levels of the protein HER2, particularly after surgery. Roche introduced Perjeta in 2012 to be used in combination with Herceptin to try for an even greater benefit.

Regulators previously approved the Herceptin-Perjeta combination to treat HER2-positive breast cancer at earlier and later stages of development than the one in the new study: cancers that have spread to other parts of the body, and cancers before women undergo surgery.

Roche reported about $7 billion in global Herceptin sales last year, and about $1.9 billion in Perjeta sales.

Write to Peter Loftus at peter.loftus@wsj.com

(END) Dow Jones Newswires

June 05, 2017 07:44 ET (11:44 GMT)