the drugs had serious side effects, were ineffective, or the trial sponsor ran out of money. Clinical trials cost huge sums of money, can take years, and depend in the end on the drug not only being effective, but in being able to actually prove this to the satisfaction of one or more regulatory agencies.
Drug sales? Let’s focus on the MBAs of the biotech world, the sales and marketing group. A drug that sells more than a billion dollars a year is usually classified as a blockbuster or, to continue the sports metaphors, a home run. These types of successes are hard to come by, because to meet this requirement a drug must treat a very large number of patients, be very expensive, or both. It costs just as much to get a drug that brings in $100M/year approved as one that brings in $1B. This is one reason Big Pharma and biotech companies like to swing for the fences with drugs tailored for large markets.
Number of patients taking the drug? This is the primary focus of the pharmaceutical development team. Working with the clinical group, they try to convince doctors (as well as potential patients) of the benefits of taking the new drug. They concentrate their efforts on patients who will most clearly benefit from the drug and overcome physician inertia. This is the natural (and often well-deserved) resistance of doctors to prescribe a new drug over one that has been on the market for years. Many doctors will wait until a new drug proves itself to be safe in the general population before prescribing it for their own patients.
So far I have covered six different definitions of success. Now imagine a biotech company that hit every one of these marks. (Genentech’s Avastin and Amgen’s Enbrel can claim to have reached all these measures of success.) Sounds like a pretty stellar outfit that should be on top of the pharma world. I wonder how many of you that noticed that one extremely important (and to my mind the most valuable) definition of success is missing: meeting unmet medical needs. Avastin and Enbrel have cleared this final hurdle as well. It might be hard to believe that a drug that satisfied all of the above criteria wouldn’t be the best thing since sliced bread, but to quote Gershwin “it ain’t necessarily so.” It is certainly possible to develop a drug that meets every definition of success that I’ve laid out above, but is simply not very effective or is no better than drugs currently on the market. New doesn’t mean better, it just means new. Tom Nesi, in his sloppily written but still interesting book Poison Pills: The Untold Story of the Vioxx Drug Scandal makes a compelling case for Vioxx being just such a drug.
So how can we as a society facilitate a process to generate more drugs that meet unmet medical needs? I would suggest that we create incentives for companies that develop drugs that meet this definition. Congress is currently debating (as part of health care reform legislation) the length of marketing exclusivity drug makers should get for follow-on biologics (this is distinct from patent considerations). Consumer groups are arguing for five years of exclusivity, while drug companies are asking for a twelve-year period. In a similar vein, I would use a period of market exclusivity to drive the marketplace towards the development of novel drugs. Companies that want to register “me too” drugs, agents that essentially act via the same mechanism as drugs already on the market should not be awarded market exclusivity (or get this for a very limited time). Firms that spend the extra effort (and likely money) to develop truly innovative drugs that hit new molecular targets should be rewarded for their investment by being rewarded with a 12-year period of exclusivity. Giving companies a greater reward for actual innovation should help spur the development of novel treatments for illnesses that still hunger for new medicines.
