Drug development is a tough business. Sometimes, just when you think you're getting to the pot at the end of the rainbow, you find the trap door one step away.
It's not supposed to work that way. The further you get down the pipeline, the better your shot at success. By the end of Phase II, investigators are supposed to have a solid set of significant safety and efficacy data on humans. Phase III is where the big bets are made, based on good science and some unprecedented demands for new products.
Sometimes, though, it's hard to tell where the science leaves off and the magical thinking begins. The desire to score a modest clinical success for Alzheimer's, an automatic gold ticket to a megablockbuster market entré, convinced some experts to push ahead with some incredibly risky bets--which flopped. The same goes for hepatitis C, where there's gold at the end of a rainbow for the first interferon-free drug to hit the marketplace.
About a third of all Phase III studies end in disaster. The analyst Tim Anderson recently produced a report pointing to a 65% success rate, which is down from the 70% success rate tracked just a few years ago.
Of course, it's also worth noting that not all the big blockbusters we hear about in the clinic actually live up to analysts' expectations. Making a peak sales projection is more art than science, and the art often looks rather comical in retrospect.
Dendreon's ($DNDN) big blockbuster Provenge? The prostate cancer therapy earned $80 million in the second quarter, down a bit from Q1 with new competition piling up. The company's been forced to cut costs, changed CEOs and talks tough, but their drug is flailing.
Human Genome Sciences' big lupus breakthrough Benlysta? Disappointing sales drove down HGS shares, leaving the biotech virtually defenseless when its longtime partner GlaxoSmithKline ($GSK) scooped it up for the bargain basement price of $3 billion. At one point, analysts projected that Benlysta could make more than that in a year.
Every year we see a good number of potential blockbusters come down to the pivotal wire. And in Part II of this feature I'll highlight the biggest potential blockbusters in the clinic stirring a buzz. But no review of the latest crop of blockbusters would be complete without an assessment of the biggest failures. And 2012 has seen some of the biggest failures in the history of drug R&D. Click here to see my top 5 flops of the year >> -- John Carroll, Editor-in-Chief. Follow me on Twitter and LinkedIn.