Niche cancer drugs highlight the targeting trend in R&D

With Roche's Zelboraf likely headed for blockbuster status and Pfizer's ($PFE) newly approved Xalkori out to make its mark against non-small cell lung cancer, the trend spotters at the Wall Street Journal have declared the arrival of a new development paradigm. Rather than try to push for new mega-blockbusters to replace aging market giants like Lipitor, the new wave of development work calls for targeted treatments--niche drugs with diagnostics on hand to identify subgroups of patients who are likely to respond.  

"There's been a change of paradigm," Pfizer scientist James Christensen tells the WSJ. "The new school of thought is, 'If you find the patients that the drug will work in, and if you see enough benefit, we will find a way to get this to market."'

The new school also includes sticker shock for payers. Pfizer's Xalkori (crizotinib) will arrive at a cost of $115,000 a year, with some built-in protections for the uninsured and underinsured. The melanoma drug Zelboraf (vemurafenib) is priced at $56,400 for 6 months of treatment.

"Perhaps the pharmaceutical industry has come kicking and screaming, but it's now come to realize" that targeting subgroups can lead to new approvals from regulators as well as coverage by payers, said Daniel O'Day, who runs Roche's diagnostic division.

Of course, there's not a lot in this report that will be new to drug developers, many of whom began to grapple with diagnostics and targeted drugs years ago. It's also worth noting Pfizer--which had become the poster child for dysfunctional R&D empires--hasn't forgotten its dreams of mega-blockbusters to come. The new data on its anticoagulant makes the pharma giant the odds-on winner in an emerging $9 billion market. That can help make up for lots of lost revenue as generic competitors arrive.

- here's the article from the Wall Street Journal