Faster, pricier and often worthless: Study slams regulators for wave of questionable cancer drug OKs

The explosion of new cancer drugs that’s occurred over the past decade includes some major standouts that include therapies that have registered some jaw-dropping results in clinical trials. But when researchers at King’s College London and the London School of Economics took a step back and examined 68 cancer indications approved in Europe over a 5-year period through 2013, they found that many were OK’d on only flimsy data derived from unreliable trial designs and at least 10 of these OKs have never proved to have any real value for patients.

Kicking back at industry’s criticism of groups like NICE, which often balk at covering high-priced cancer drugs, the biopharma critics say that the vogue of fast-tracking oncology therapies has put a long lineup of drugs into regular use, even though the manufacturers haven’t produced solid evidence of efficacy four or more years later.

Their study was published in the BMJ.

Courtney Davis

The two authors — Courtney Davis at King’s College in London, and Huseyin Naci of LSE Health — say 39 indications (57%) were sanctioned “without evidence of a survival or quality of life benefit.” And they excoriated the use of surrogate endpoints, saying they are unreliable at best.

While 24 of these 68 indications offered evidence for increased survival, the average was less than 3 months. And only 7 of 68 provided data demonstrating that they improved quality of life.

“When expensive drugs that lack clinically meaningful benefits are approved and reimbursed within publicly funded healthcare systems, individual patients may be harmed, important resources wasted, and the delivery of equitable and affordable care is undermined,” the researchers say. They added that while regulators have the authority to pull a drug back from the market if it doesn’t demonstrate efficacy, they don’t do it.

Cancer Research UK cautioned, though, that the system on approving and paying for cancer drugs has undergone some change in recent years, with a greater emphasis on some of the weak spots highlighted in the study.

Of course, many of these same drugs are also approved in the US. And the authors’ list of questionable drugs covers some blockbusters. It includes:

→ Avastin, OK’d for ovarian cancer by the EU in 2011, they say, slapping researchers for switching from overall survival to progression-free survival.

Vinay Prasad Washington Post

→ Herceptin also comes in for criticism, with the researchers flagging an approval for breast cancer long before it failed to demonstrate a survival benefit.

→ Celgene’s Pomalyst was OK’d in Europe after regulators criticized their comparison of the drug combined with low dose dexamethasone against high dose dexamethasone.

“The expense and toxicity of cancer drugs means we have an obligation to expose patients to treatment only when they can reasonably expect an improvement in survival or quality of life,” industry critic Vinay Prasad told CRUK. The findings suggest “we may be falling far short of this important benchmark”

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