For Jazz Pharmaceuticals, Failure Is The New Winning

Just 6 percent of people diagnosed with metastatic small cell lung cancer live five more years, according to the American Society of Clinical Oncology. In comparison, 13 percent of people diagnosed with metastatic pancreatic cancer reach that milestone.

Pharmaceutical companies such as AbbViePoniard Pharmaceuticals, and United Therapeutics have poured billions of dollars into the development of drugs for small cell lung cancer and clinical trials, but few life-extending options have materialized over the past three decades. All told, 40 clinical trials for small cell lung cancer treatments have failed.

Failing scientifically, yet scoring commercially

Traditionally, the FDA has viewed a drug’s inability to outperform current treatments in extending or improving the life of study participants as disqualifying — except for medications in its accelerated approval program, such as lurbinectedin. And lurbinectedin, like many cancer drugs, has documented side effects including fatigue, nausea, and declines in white blood cell counts.

Commercially, though, lurbinectedin has been far from a failure, and brokerage analysts have projected that Jazz could take in $200 million to $225 million in revenue this year from sales of the drug. One Wells Fargo analyst, in a June 2020 research note went so far as to suggest that Zepzelca’s annual sales might peak at $700 million. PharmaMar is benefitting handsomely, too: Jazz’s annual report noted the $300 million in payments it made to PharmaMar last year.

And the actions of Jazz stand in sharp contrast with those of larger rivals with oncology drugs. After Bristol Myers Squibb and Merck recently disclosed the failure of confirmatory trials for their own bestselling drugs under accelerated FDA approval for treatment of small cell lung cancer, both companies immediately stopped marketing them for that purpose and removed the indication from their labels. Bristol Myers Squibb’s Opdivo and Merck’s Keytruda are still indicated for the treatment of other cancers, including non-small cell lung cancer, though.

The FDA is rather vague about what is supposed to happen after a drug in the accelerated approval program fails its confirmatory trial. The FDA’s website simply states that the agency has “procedures in place that could lead to removing the drug from market.”

FDA press officer Chanapa Tantibanchachai wrote in an email reply to the Foundation for Financial Journalism’s questions, “The FDA is committed to ensuring the integrity of the accelerated approval program. The agency is currently in the process of evaluating oncology accelerated approvals.”

(On April 27, the FDA’s Oncologic Drugs Advisory Committee will hold a public hearing to examine accelerated approval drugs “with confirmatory trials that have not verified clinical benefits,” the agency has announced.)

Over the past two decades, the FDA appears to have shifted away from evaluating a drug solely on its trial data in favor of also including other data sets, according to Dr. Thomas Frieden, a former director of the Centers for Disease Control and Prevention. Thus, U.S. regulators might not remove medications like lurbinectedin from the marketplace in the near future.

At the center of this shifting regulatory landscape is the FDA’s acting leader, Dr. Janet Woodcock, who has served two long stints at the helm of the agency’s Center for Drug Evaluation and Research. Dr. Woodcock has been a vocal advocate of accommodating the pharmaceutical industry to speed the availability of drugs. She is well-known for regulatory leniency toward drugs whose clinical trials cast doubts on their efficacy.

What is less clear is how people with small cell lung cancer would benefit from access to expensive drugs that rarely work well, if at all.

Sacrificing clinical rigor, a casualty of war on cancer

The nature of cancer, and particularly small cell lung cancer, results in very complex and brutal tradeoffs as researchers and pharmaceutical companies hasten to bring new drugs to market.

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Disclosure: Neither SIRF nor its employees, contractors, board members or advisors has any economic stake in what we report on, before or after the release of the investigation. No one sees or is ...

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