Pfizer aims ambitious PCSK9 program at payers, skeptical regulators

Tantalized by various megablockbuster projections for the PCSK9 field of cholesterol drugs, Pfizer ($PFE) has mapped out one of the most ambitious late-stage programs in the industry for RN-316. This week, the pharma giant spelled out plans for a massive cardiovascular outcomes trial to prove to regulators and payers alike that the drug not only works as advertised, but delivers real value to patients. And there are already plans to follow up on its first approval with a more convenient dosing regimen, outlining the company's lifecycle management expectations.

With the PCSK9 development program, Pfizer is trying to cover a lot of bases at once. The combination of trials testing RN-316's ability to lower "bad" cholesterol with cardiovascular outcomes trials will yield a mix of data Pfizer hopes to use to win over not only regulators, but payers. "The issue is, in this marketplace, we need trials constructed to demonstrate value to society and payers," Pfizer's John Young, president of the primary care business unit, said during the third-quarter earnings call on Tuesday.

One of the outcomes trials looks specifically at some very high-risk patients: those who've been unable to get their LDL cholesterol levels below 100, even with statin therapy. If RN-316--now dubbed bococizumab--can show itself effective at improving outcomes for that group, payers could see it as a money-saving bet. "Those patients really do represent a tremendous cost to the healthcare system," Young said during the call. "A positive outcome in that group would be very significant." For more, check out FierceBiotech's story. -- Tracy Staton (email | Twitter)