The 'Wonder Drugs' That Cost Thousands of Dollars and Do Almost Nothing

A new rating system developed by oncologists aims to fight what some of them call ‘the hype over 'miracle' cancer drugs.' The idea for it first arose in Israel.

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An employee in the chemistry research laboratory at the Santaris Pharma A/S biopharmaceutical company, acquired by Roche Holding AG, in Horsholm, Denmark, Sept. 16, 2014.
An employee in the chemistry research laboratory at the Santaris Pharma A/S biopharmaceutical company, acquired by Roche Holding AG, in Horsholm, Denmark, Sept. 16, 2014.Credit: AP

The often tenuous relationship between the clinical effectiveness of new cancer drugs and their exorbitant cost is a worldwide problem. Over the past decade, more and more such drugs have come onto the market and into national health systems.

“It happens in waves,” said Prof. Raphael Katan, a senior oncologist at both Sheba Medical Center and Shaare Zedek Medical Center and the former head of oncology at Sheba. “Every time there’s another drug that’s considered good and desirable, it creates hope. Then it turns out that it’s not so good, or that it’s only good in very specific situations.”

Impressive presentations about the latest clinical trials aren’t confined to the annual meeting of the American Association of Clinical Oncology, attended by some 40,000 oncologists from all over the world (the most recent was in Chicago a few weeks ago). They quickly find their way to the international media, and from there to patients and national health systems.

“The drug companies are commercial enterprises and they’re trying to get the best price,” Katan said. “Over the last few years, the public has evidently been willing to pay higher amounts for drugs, even if the benefit is small.

“Even wealthy countries like the United States and some European countries have begun to be aware of this and are trying to limit it,” he added. “But it’s not simple. When there’s a new drug, even if it’s not so effective, but just a little better than another drug, we feel obligated to recommend it.”

And when oncologists make recommendations, the decision winds up on the health system’s doorstep. The decision makers will tell you there’s nothing tougher than standing before a cancer patient in the guise of the person who’s blocking his last hope for a cure or for some improvement in his condition. And the drug companies are well aware of this.

The search for a cure for cancer, the fondest wish of scientists and physicians, is also part of a vast commercial industry. Every report of a new drug or clinical trial reverberates commercially, with analysts vying to spot the new blockbuster drug that will sell for hundreds of millions of dollars; such reports affect profit forecasts and stock value. And the key to all of this is media and medical buzz.

Dr. Karol Sikora, an oncologist and dean of the University of Buckingham medical school, warned in an op-ed in The Telegraph following the recent ASCO meeting of what he called “the hype over ‘miracle’ cancer drugs.” Sikora charged that there is “a sophisticated conspiracy to hype products being sold to bigger companies by small start-ups, to get more investment for the industry from the City or Wall Street, or simply to ramp up share prices and make short-term gains.”

A new applied research project by a group of oncologists, members of the European Society of Medical Oncology, aims to put an end to this phenomenon. The project was reported in late May in the journal Annals of Oncology and also presented at the recent ASCO meeting.

The oncologists developed a model for determining and rating the clinical effectiveness of cancer drugs. The rating scale takes numerous factors into account, including the drug’s impact on the patient’s overall survival, the amount of time it caused the cancer to go into remission or remain static, side effects, impact on quality of life, and how its clinical trials were conducted.

Once all the parameters have been statistically weighted, the drugs are ranked in two categories: Drugs to treat incurable cancers (on a scale of 1 to 5, with 5 standing for the most effective), and drugs to treat curable cancers (rated on a scale of A, B, C, with A being the most effective and C the least effective).

“The idea for the scale came about six years ago, following a discussion that actually arose in the Israeli Society for Clinical Oncology, concerning recommendations for the ‘health basket,’” said Prof. Nathan Cherny, a senior oncologist at Shaare Zedek and an ESMO member, referring to the list of medicines subsidized by the national health insurance plan.

“I saw recommendations that were very hard for me to justify based on the statistics,” added Cherny, who was one of the people involved in developing the new model.

After developing the model, Cherny and his colleagues used it to rank 77 cancer drugs. While some ranked high for clinical effectiveness, 21 expensive drugs received a ranking of only 1 or 2, having been found to be of low or negligible effectiveness.

One example is Tarceva, used to treat metastatic pancreatic cancer. According to the study, it extends patients’ lives by just nine days, and its impact on their quality of life has not been assessed. Yet it costs about 25,000 shekels per month ($6,600).

Then there’s Afinitor, “a third-line treatment for metastatic breast cancer,” which is currently in the health basket. It was found not to have contributed at all to patients’ overall survival or quality of life, but it costs 20,000 shekels per month.

“The chutzpah of the drug companies is mind-boggling,” Cherny said. “When a company is charging 25,000 shekels a month for a drug that extends a patient’s life by nine days, clearly there’s no connection between the price and the clinical benefit. If we know these drugs are so ineffective, why not take them out of the health basket and make room for other, more effective drugs?”

The hype surrounding new cancer drugs is not always completely deliberate. “There are many reasons why people interpret data in a more optimistic light than is justified, and I’m not just talking about people who have a clear interest here. We all have a certain tendency to optimism,” Cherny said. “In most cases, it’s motivated by goodwill and the need to offer encouragement, but there’s a marketing aspect here too, and some people exploit this optimism for sales purposes.”

It’s very easy to be persuaded when doctors say they’ve seen “miracle” results. “When you participate in a clinical trial and the results are positive, you want to believe that you’re changing the world,” Cherny said. “It’s important to always put things in the right proportion and to continue monitoring the findings and the latest data. I think our model will help with that.”

Last week, ASCO announced that it is also adopting a new model that will enable oncologists to compare drug treatments for four common types of cancer based on survival rates, side effects and cost.

Novartis, which manufactures Afinitor, responded that the drug in conjunction with hormonal therapy extends patients’ life expectancy to nearly three times what it was previously. With the treatment, their average survival time was 31 months, and the chance of the disease progressing was cut by 62 percent, it said.

Cherny blasted Novartis’ response, noting, “The addition of Afinitor did increase the time until the cancer started to progress by three times what it was previously. But (and importantly) this did not result in any improvement in the quality of life of patients taking the medication, nor did it statistically improve the amount of time they lived overall, which was [still] about 31 months.”

Roche, which makes Tarceva, did not respond to a request for comment.

Prof. Jonathan Halevy, director of Shaare Zedek and chairman of the health basket committee, said, “The committee’s discussions are based upon the very latest research. All our questions begin with the subject of the drug’s effectiveness. During the clinical stages of the discussions, there is no discussion of the financial aspect.

“I have great admiration for the new model, but it’s brand new,” he added. “It passes the test of logic, but it will also have to pass the test of reality. If it eventually becomes the ‘gold standard,’ we’ll definitely have to take it into account.”

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