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The tribulations and triumph of drug development
on February 5, 2014
As a biomedical scientist I really enjoyed this book. It is the sequel to the author's previous volume "The Billion Dollar Molecule". Both books provide a fly-on-the-wall account of Vertex Pharmaceuticals, an innovative biopharmaceutical company that brought two breakthrough medicines - one for hepatitis C and the other for cystic fibrosis - to market two years ago. Both these drugs were the first of their kind and have given thousands of patients a new lease on life. From a broader perspective however, the author's goal is to shed light both on the immensely challenging process of bringing a new drug to market and the brilliant scientists and driven personalities that make it possible.
Werth's account of Vertex focuses mainly on the hepatitis C drug, with the cystic fibrosis drug playing a smaller but still important role. The previous book was much more science-heavy than this one, emphasizing the chemistry, biology and computer science that goes into the early stages of drug discovery. In spite of the intense scientific competition and research depicted in that volume (much of it spanning the late 80s and early 90s), Vertex did not bring a successful drug to market until 2010, underscoring the challenge of drug discovery in which you have to furiously paddle simply to stay afloat. The two books thus mirror two different phases of the company: the first dealing mainly with the science and the birthing pains of a new startup, and the second dealing with the transformation of the startup into a commercial enterprise. You will thus find much more of the business, legal and commercial aspects of drug development in this volume. Boardroom deliberations and the subtleties of drug pricing litter the narrative. Both books, however, provide an excellent overview of the multiple challenging stages of drug discovery, from discovering an initial "hit", to optimizing its properties to formulating it and finally selling it in the form a once-a-day pill. The account of formulation difficulties was especially revealing to me. As the founder of Vertex put it, taking a drug all the way from initial discovery to the market is harder than putting a man on the moon, partly because the biology is so complex and incompletely understood.
Werth has always been a skilled purveyor of the personalities and egos that populate the highest echelons of the science and business and he continues this tradition in this book. Along with highly driven scientists we meet egotistic but talented lawyers, executives, government officials, Wall Street analysts and venture capitalists. The founder of Vertex is described as having a "reality distortion field" akin to Steve Jobs. The character descriptions are far more colorful in the previous book but this volume does have its share of sharp profiles: for instance one management consultant "dressed like George Will but sounded like Don Draper channeling Alan Ginsburg". One of the strengths of the previous volume was that many of the personalities portrayed in it - including academic giants like Robert Burns Woodward and industrial giants like George Merck - were fundamentally more interesting but the present account on the other hand does a better job of describing the vast and sundry set of individuals required to sustain the diverse drug enterprise. We also see how cruel the business can be, bringing failure to years of intense effort and laying off dedicated scientists who have given their heart and soul to the process.
Werth's discussion of the two Vertex drugs also raises important questions about the future of the industry. The hepatitis drug is targeted toward a vast patient population, much of which is located in poor countries. The cystic fibrosis drug is targeted toward a small patient population, much of which is located in rich countries. The stories of patients in both groups - one of which Werth documents in detail - are heartbreaking, but in both cases the challenge is to balance profits with cures. In one case the company was compelled to charge exorbitant amounts of money because of the small patient population while in the other case the lack of wealthy patients led to lower revenues. Whether you are developing a drug for millions or for thousands, the R&D costs are roughly the same - and enormous. This is the moral and financial challenge the industry faces right now; how, when it is trying to develop highly personalized therapies for smaller and smaller patient groups, can it get away with both charging reasonable prices and recovering the expenditure on R&D that it can plug into developing the next drug. Werth deftly discusses these issues along with the underlying ones of FDA approvals and Medicare and government controls.
Ironically, the successful Vertex story - which culminated last year in a move to a shining, sprawling new building near Boston Harbor - ends in a somewhat somber note. Many of the most important scientists - including the founder - who discovered the two medicines are no longer with the company and the highly-science driven approach that was described in the first book seems to have taken a backseat to commercial pursuits. The future of the company is thus not completely certain, and Werth ends with an afterword in which he discusses the fate of big pharmaceutical companies which seem to be laying off thousands, making incremental advances and cutting each others' throats to bring the next "me-too" pill to market. Unlike Vertex, many of these organizations spend much more on marketing than on R&D and are busy pleasing Wall Street and shareholders rather than patients. As Werth reminds us, if we want to advance the next groundbreaking medicines we need to remember George Merck's advice: focus on patients, the profits will follow. The story of Vertex provides both a shining example and a cautionary note.