Innovation is the lifeblood of the biopharmaceutical industry. New medicines pharma brings to market save lives, reduce suffering, and, not incidentally, generate new revenue streams. Today's increased emphasis on innovation—discovering breakthrough medicines that provide significant therapeutic advantages—is driven in part by the growing resistance of both public and private payers to pay premium prices for medicines that are not demonstrably superior to those already available.
In the United States, pharmacy benefits managers and managed care organizations have adopted tiered formularies and other cost-containment measures that relegate new drugs to a lower reimbursement status unless a significant advantage over existing medicines can be demonstrated. In Europe, a new drug may only receive reimbursement at the level of the lowest-cost alternative—perhaps a generic—if superiority cannot be demonstrated.
First, it is important to recognize that not all innovation is purely scientific. Innovation is in the eye of the beholder, and in the medicine, there are at least four beholders: patients, physicians, regulators, and payers. These stakeholders do not always focus on first-in-class medicines. They value innovations in safety, reduced side effects, convenience, and dosing schedule. Such improvements can also speed drug approval, promote physician prescribing, and enhance patient compliance.
The other axis shows whether the medicine has a , new target versus existing target, shows the extent to which a new drug is acting on a known target, for which medicines are already available, or has a novel mechanism of action.
The examples given, and there are many others, show that important medicines—those that add value to the company—arise from all four quadrants.
At one extreme, there is Enbrel, a drug that combined a novel mechanism of action with a reshaping of the therapeutic landscape. It has become a blockbuster, with 2004 sales of nearly $2 billion, according to the manufacturer's Web site. For this type of molecule, there is a high degree of technical risk, because it relies on a novel mechanism of action. The commercial risk tends to be lower, because there is no demonstrable therapeutic alternative.
Lipitor lies at the other end of the spectrum. The fifth statin entered a market that was well defined, with a biochemical target similar to its competitors: HMG-CoA reductase. Yet it has become the best-selling drug on the planet. For a molecule such as this, the technical risk was significantly lower. But it carried a higher level of commercial risk as a late entry into a market whose existing products already performed well.