Searching for The Elusive Billion Dollar Molecule -- A 23 Year Old Book, Highly Relevant Today

When you contemplate how hard it is to bring a drug from a hypothetical construct in a scientist's mind to something you can pick at a drugstore it is really unfathomable how many false starts, blind alleys, toxicity concerns, capital expenses, and regulatory considerations will occur over the years of development. In all, it is often estimated that it takes one billion dollars to bring a drug to market. 

This level of capital expenditure may explain why large-sized pharmaceuticals companies are market leaders because past historical successes have provided them the resources to engage in this field. I recently came across an important book, published in 1994, that provides an in-depth biographical view of a company being born. The Billion Dollar Molecule by Barry Werth is an incredible book that really, for the first time, concretized to me how hard pharmaceutical research is and provided a full context for the heroic achievements of those who engage in this extremely challenging -- yet absolutely vital field. 

The company profiled in this book (and also the subject of a sequel by Werth) is Vertex Pharmaceuticals. Vertex, founded by visionary former Merck star scientist Joshua Boger, was premised on what was then a novel concept: structural and rational drug design in which the structure of a target and a candidate molecule were given paramount importance in an industry which largely relied on mass compound screening. Werth details the company's dual efforts in immunosuppression and HIV -- a disease Vertex deemed of "planetary significance" -- protease inhibitors.

My current work often has me discussing and contemplating emerging infectious disease and the relative lack of treatments against them and The Billion Dollar Molecule provides much needed explanations for this insoluble dilemma. Several important takeaways with respect to emerging infectious disease:

  • "Diseases are not treated first as diseases, but as markets"
  • "You don’t see blockbuster drugs for acute situations. You see them for chronic cases."

  • "A molecule, he knew, was worthless, a laboratory curiosity, if it couldn’t be made cheaply enough to sell at a profit."

  • "Vertex wouldn’t pick the project that would benefit the most people or target the most pressing need; no company would. It would pick the project with the best overall chance of success."

The goal of any pharmaceutical company is to sell product in order to earn revenue and the bigger the market, the bigger lure. An emerging infectious disease, even in the most dire zombie apocalypse scenario, is dwarfed by hypertension, diabetes, heart disease, depression, and the like. If the market does not exist or is minuscule, it follow that market entrants will not be abundant. This is often termed a "market failure" when, in reality, it truly is very obviously just normal market functioning. Realizing these objective facts, without damning the industry like many are wont to do, leads to a whole different type of strategy to make these smaller markets attractive -- a project I am currently engaged in (more to come).

The book also contains great historical information about Merck -- an incredibly successful company with an almost unrivalled historical interest in infectious disease amongst giant pharmaceutical companies -- as well as important insights into the personality traits of the highly motivated scientists who were at the forefront of drug development.

One of the other absolute treasures of the book, to someone from Pittsburgh, is the time devoted to Dr. Thomas Starzl, one of the fathers of organ transplantation and a heroic pioneering visionary figure of unrivalled stature, who was absolutely instrumental to the success of the immunosuppressant tacrolimus.

Today, Vertex has revenues over $1 billion (and was responsible for the hepatitis C protease inhibitor telaprevir as well as an innovative treatment for cystic fibrosis) and The Billion Dollar Molecule, published 23 years ago, contains important lessons for those trying to understand the mechanics of this crucial industry as well, like me, trying to understand the considerations as the apply to emerging infectious diseases. In an era in which we have a president-elect who openly derides this life-saving industry, understanding -- and defending -- the pathbreaking efforts of this industry is more crucial than ever.