The Pill That Could · Approval

Approval, part 3

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The press conference announcing the approval was held in Washington on May 10, 2001.

Tommy Thompson, the Secretary of Health and Human Services, announced the approval personally. This was, by the standards of cabinet-level public statements, unusual. Drug approvals are normally announced by the FDA, in a regulatory press release that is read carefully by industry analysts and reporters covering the relevant therapeutic area, and barely noticed elsewhere. Cabinet secretaries do not normally appear at podiums to talk about a single new oncology indication.

What Thompson said, in the prepared remarks of that morning, was that the FDA had approved a drug for a form of leukemia, that the drug had been developed in unusually short time, and that the approval represented — he used a phrase that the next several years of media coverage would repeat — a new era in cancer treatment. The phrase did some work. It was the kind of phrase that gets attached to product launches in industries with marketing budgets, and it was the kind of phrase that, when used about a real new drug for a real disease that really worked, deserved to be taken seriously. Both things were true at once.

Druker, Lydon, Sawyers, Talpaz, Vasella, and several others were present or spoke during the announcement and the surrounding events. The approval was, by any measure, a moment in their careers. It was also, more importantly, a moment in the lives of the patients who had been waiting.

On the day of approval, Gleevec was available by prescription in the United States. In the days after, Novartis began shipping. The phase 1 patients, who had been on the drug for almost three years on a research basis, transitioned to commercial supply. The patients who had been waiting through the petition campaigns of 1999 and 2000 began calling their doctors. Within the first week, several thousand prescriptions had been written. Within the first month, demand had exceeded what the company had projected.

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Two months after the approval, on May 28, 2001, Time magazine published an issue with Gleevec on the cover.

The cover image was a tall yellow capsule, dominating the frame, with a headline reading “There is new ammunition in the war against CANCER. These are the bullets.” Inside, a long feature by the magazine's senior science writer, Michael D. Lemonick, traced the history of the drug from the Philadelphia chromosome through the Ciba-Geigy program to the phase 1 trial to the New England Journal papers and the FDA approval. The piece was sympathetic, well-reported, and unusually accurate by the standards of mainstream cancer journalism. It also did something that had not been done before, at least in this volume: it told a general American audience, in cover-story language, that a fundamental shift in cancer treatment had occurred.

The framing was the magic-bullet framing — the dream of Paul Ehrlich, finally realized, in a single yellow capsule. The framing was, in some respects, a slight oversimplification of what Gleevec was and what it would turn out to be. The drug worked beautifully in CML, well in some related cancers, and not at all in others; it would not generalize as cleanly to all cancers as the cover suggested it might. CML was an unusually clean target. Most cancers are messier. We will come to this in chapter eleven.

But the cover was also, on its own terms, correct. Something had shifted. Cancer drug development, after Gleevec, was not the same enterprise it had been before Gleevec. The idea of identifying a specific molecular driver and building a specific molecule to inhibit it had moved, over the course of one drug's development, from a thing that ambitious laboratories did to a thing that the pharmaceutical industry took seriously enough to invest in at scale. The Time cover was, in a sense, the moment that change went public.

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Then there was the question of price.

Novartis launched Gleevec at a list price, in the United States, of approximately twenty-six thousand dollars per patient per year for the standard 400-milligram daily dose, with the price scaling roughly proportionally for higher doses (chronic-phase patients typically received 400 milligrams a day, accelerated-phase 600, blast-crisis 800). The figure that became attached to the drug in popular coverage was about thirty thousand dollars a year, the rough cost for a representative patient. Either number was, in 2001, considered a high price for a cancer drug. It was not unprecedented; some chemotherapy regimens of the era cost similar amounts on a course basis. But Gleevec was meant to be taken every day, indefinitely, for as long as the patient continued to respond. The lifetime cost, for a patient who responded well and stayed on the drug, would compound year after year.

The pricing decision was, internally at Novartis, the subject of considerable analysis and considerable disagreement. The arguments in favor of the price went something like this. The drug had cost more than a billion dollars to develop. (This is the kind of figure that gets quoted in pharmaceutical contexts; it is also the kind of figure that includes a great deal of allocation and amortization that economists argue about, but it was the figure the company used.) The market — perhaps thirty thousand CML patients in the United States, and similar fractions of similar populations in other developed countries — was small enough that the company would have to charge a meaningful per-patient price to recover the cost. Insurance, in most of the developed world, would cover most of the price for most patients, so the bill would not fall directly on patients in the way the headline number suggested. Comparable cancer therapies cost similar amounts. The company also pointed to its plan, announced at launch, to provide the drug free of charge to any United States patient whose income was below a threshold and who had no insurance coverage — a patient assistance program that would, in subsequent years, distribute Gleevec to many thousands of patients at no cost.

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