Inside the World of New Pharma
(Simon & Schuster)
Mary Poppins made it sound simple: just a spoonful of sugar makes the medicine go down. But what if you can't afford the medicine? Or it's not covered by insurance? Or you don't own a spoon?
In his new book, The Antidote: Inside the World of New Pharma, Barry Werth touches briefly on such questions but mostly describes the challenges of a young pharmaceutical company as it tries to balance the conflicts between, "cutting edge science and profitable business."
This is not, then, as the subtitle implies, a hard-hitting, insider's account of the mammoth and obscenely profitable pharmaceutical industry in this country or why Americans have to go bankrupt to stay healthy. It is, instead, the rather narrow story of one company, Vertex Pharmaceuticals, Inc., and its founder Joshua Boger.
When Vertex was established in Cambridge, Massachusetts in 1989, the goal was to create a small company that would be light on its feet and yet stocked with brilliant scientists and engineers who would take the road less travelled to create exciting new "designer" pharmaceuticals; a company that could challenge industry giants such as Merck and Pfizer and go on to transform healthcare. In short, David v. Goliath ... with biologics.
Despite the fact that the pharmaceutical business is a $325 billion a year industry, it is still an uphill climb to surmount the multiple obstacles inherent in bringing a new product to market. Drugs need to be designed, tested in animals and tried out on humans. To get FDA approval, researchers are required to show that a drug is safe as well as effective --- but not necessarily better than other products already on the market. This is one reason why we have eight types of statins which are all about equally useful in treating elevated cholesterol.
Altogether, getting a drug from the laboratory to your medicine cabinet can take years and cost upwards of a billion dollars. For every thirty drugs out of the starting blocks, only one makes it to the finishing line. It is a depressing ratio until you consider the markup.
As Werth relates in detail worthy of a master class at the Harvard Business School, one of Vertex's biggest success stories is the drug ivacaftor or Kalydeco®. It is the first drug to reach pharmacist's shelves to treat one of the underlying genetic defects carried by persons with cystic fibrosis. By any measure, this was a remarkable achievement and was developed over the course of thirteen years with the support of the Cystic Fibrosis Foundation. There are approximately 30,000 people in United States who have cystic fibrosis but only 4% --- or 1,200 --- have the gene mutation targeted by Kalydeco®. While this breakthrough does not represent a cure, it can markedly improve a person's quality of life.
For patients and their families, it comes remarkably close to being a miracle. The only catch is the price. A one-year supply costs more than $300,000. So who exactly can afford this medicine? Where do you find enough sugar to help this go down?
In 2012, 24 US doctors and researchers involved in the development of the drug wrote to Vertex: "We have invested our lives and careers toward the success of these inspiring therapeutic agents. We also write with feelings of dismay and disappointment that the triumph and honor that should be yours is diminished by the unconscionable price assigned to Kalydeco®."
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Werth is an accomplished journalist who has written five other books. His first book, The Billion-Dollar Molecule, was also about Vertex and described the rollicking early days of the company which one reviewer described as a "razzle-dazzle new saga." Another proclaimed it to be "masterful." BusinessWeek referred to it as
a riveting tale that has more in common with a John Grisham thriller than with tomes on modern science.
With these kinds of comments, this reviewer was anticipating that The Antidote would be another page-turner and perhaps even an all nighter. Instead, it is an exhaustive account of the inner workings of a company trying to live up to its lofty ambitions while still staying afloat.
There is much to be learned here about how, for instance, a small start-up struggles day-by-day to compete against much bigger and better financed organizations in a highly competitive and sometimes onerously regulated market. The only downside to this molecular analysis is that there is frequently more detail than is needed to move the story forward and more than one cares to know. Do we really need to know the name and location of the hotel one of the Vertex executives stayed at or a description of what one of the researchers was wearing at a meeting including the color of the buttons on her suit and that she had on stiletto heels. At other times, the detail drifts into insider jargon, difficult for an outsider to parse. Consider,
Its salespeople had pressed physicians before the AdComm to begin the four-week lead-in with peg-riba early so that their patients could get on Victrelis at launch.
For someone looking for a comprehensive but insightful guide of the U. S. pharmaceutical industry and how it impacts our politics, our culture and individual patient lives, you'll probably need to look elsewhere. The Antidote, for all its many virtues, is not for the general reader or for someone who wants to know why their insurance company won't approve a generic medicine (or why Canadians pay far less for the same drug as we do in this country).
On the other hand, if you want to learn more about Vertex, this is the book for you.--- Larissa Belmondo