A press release issued today by Boston’s Vertex Pharmaceuticals led a doctor at Johns Hopkins to say that “he was extremely excited” about Vertex’s new combination therapy for cystic fibrosis–and it drove the company’s stock up 40.4% on the Nasdaq exchange.
Robert Weisman at The Boston Globe wrote that the combination of two drugs “helped people with the deadly disease breathe more easily, raising hopes the treatment could be on the market by late next year and sending the company’s stock soaring.”
Weisman was right to put both the potential value of the drugs and their effect on the company’s stock in the lede. Both developments are important for patients and for investors.
The treatment is designed only for the estimated 22,000 patients around the world with the most common form of cystic fibrosis, Weisman reported. Even with that limited market, however, the company could eventually reach annual sales of $5.5 billion, Mark Schoenebaum, a biotech analyst, told Weisman.
Weisman didn’t do the math, but I will. Sales of $5.5 billion from 22,000 patients works out to $250,000 per patient.
If Vertex is able to sell its drugs to only half of those patients, it will have to charge double that to reach $5.5 billion in sales.
“The data are good and will clearly support worldwide regulatory approval for this drug regimen,” Schoenebaum wrote in a note to investors, according to Weisman. “I can’t see any reason why the vast majority of patients with the appropriate mutation will not want to take this regimen.”
I can see a reason. The patients might want to take the regimen. But how many will be able to afford it? Matthew Herper at Forbes reported that the drug combination “could cost $160,000 per patient per year.” That’s less than what my arithmetic came up with, but still far more than most patients can afford.
Herper also notes that he agreed to an unfortunate restriction in reporting on the story. “The company shared the data with me ahead of its release this morning on the condition that I only talk to Vertex executives, Ramsey [one of the study’s authors], and a representative of the Cystic Fibrosis Foundation , which funded the drugs development. I think the data are strongly positive, but I haven’t had a chance to show the results to outside experts as I usually would.”
Engaging in a bit of instant peer review is what science reporters do to help verify the accuracy of what they are reporting–we run it by experts not involved in the study. Herper might now follow up by checking with outside experts, but that story tomorrow or the next day will likely get nowhere near the exposure that this one did. The company, in effect, released the data in a way that guaranteed good press. And even Herper, a smart and crafty reporter, apparently felt he had no choice but to agree. Forbes could hardly afford to be the only business publication without this big news from Wall Street.
Herper talked to the Cystic Fibrosis Foundation, which supported much of the early research on these drugs. The foundation’s executive vice president for medical affairs, Preston Campbell, told Herper that “it’s our impression that all of these drugs should be available to all cystic fibrosis patients.” That’s wishful thinking. “Our impression” that patients “should get” the drug?
Here’s what Campbell didn’t say: As a condition of our research funding, we agreed with the company that the price of the drugs should be kept affordable by even the neediest cystic fibrosis patients.
A further concern about this report is that the studies of the new drugs have not yet been published, so they have not had to undergo peer review. As far as I can tell, the research was reported only in a press release from the Cystic Fibrosis Foundation and one from Vertex.
Andrew Pollack of The New York Times noted that the improvement in lung function in the patients in the study was “2.6 to 4 percentage points after six months, compared with the lung function of patients who took the placebo.” That put it “at the low end of what some doctors would consider meaningful and what some investors had anticipated,” Pollack wrote.
Joseph Walker and Anna Prior at The Wall Street Journal wrote that the improvement in lung function was “perhaps not even perceptible to patients,” but “could have a large impact on patients’ quality of life.” Patients gained weight and showed a reduction in coughing episodes and shortness of breath, they wrote.
I didn’t see anyone else acknowledge, as Herper did, that he could get information on the new trials only by accepting unfortunate restrictions on how he could use it. If the others made the same agreement, they should have acknowledged it.
It’s clear that this research was a huge success for the company’s shareholders. What’s less clear, partly because of the restrictions on the reporting, is exactly how helpful it might one day be to the multimillionaire cystic fibrosis families who can afford it, or whose insurance companies pay for it.