Introduction: Generic statins are the standard of care for 3 million Americans affected annually by dyslipidemia. Approximately 20% of these individuals are intolerant, and PCSK9 inhibitors provide an effective alternative. Recent drug launches (such as sofosbuvirir) have raised Payer scrutiny regarding drug pricing, triggering proactive review of therapies, such as PCSK9 inhibitors, which could pose considerable budget impact. FDA approval of a broader than expected label for PCSK9 inhibitors has further exacerbated concerns. Analysts still predict combined 2016 US sales of $1.2 billion. Understanding drivers contributing to PCSK9 inhibitors market access can provide manufacturers facing similar business challenges critical insights for optimizing product value. Objective(s): Evaluate drivers for the value story and market access of PCSK9 inhibitors. Method(s): Conducted secondary research of publicly available information in PubMed and grey literature. Result(s): Clinical drivers:PCSK9 inhibitors target a segment of the dyslipidemia population. They have demonstrated considerable efficacy vs. placebo in Phase-II trials (40-72% reduction in LDL-C). Coincidentally, the IMPROVE-IT trial provided the first substantive evidence that lowering LDL-C reduces risk of CV events. Preliminary analysis of the ODYSSEY LONG TERM trial has demonstrated a lower rate of adjudicated MACE for the alirocumab vs. placebo group. Survey data indicated considerable increase in physician willingness to prescribe LDL-C lowering treatment after the availability of long term outcomes data. Marketing/ regulatory drivers: Sanofi/Regeneron made distinct efforts to ensure alirocumab was first to market. They also launched a national campaign, "Cholesterol Counts" to improve awareness of cholesterol monitoring. Regeneron engaged in Payer education programs for screening eligible patients, while Amgen engaged in pay-for-performance for evolocumab with Harvard Pilgrim. A considerable increase in published literature (PubMed) was also observed prior to launch. Conclusion(s): While defining and communicating value is necessary for product success, partnering with, and educating Payers, is critical to optimize market access.
Ramesh, Kochel, S., & Mwamburi, M. (2016). PCSK9 Inhibitors - Alirocumab and Evolucumab: Key Insights on Differentiation in a Generic Market. Value in Health, 19(3), A57. https://doi.org/10.1016/j.jval.2016.03.152