Using Performance-Based Risk-Sharing Arrangements to Address Uncertainty in Indication-Based Pricing.

Background: The rise in pharmaceutical expenditures in recent years has increased health care payer interest in ensuring good value for the money. Indication-based pricing (IBP) sets separate, indication-specific prices paid to the manufacturer according to the expected efficacy of a drug in each of its indications. IBP allows payers to consistently pay for value across indications. While promising, a limitation of IBP as originally conceived is that efficacy estimates are typically based on clinical trial data, which may differ from real-world effectiveness. An outcomes guarantee is a type of performance-based risk-sharing arrangement that adjusts payments according to prospectively tracked outcomes. We suggest that an outcomes guarantee contract, which has been used by some payers, may be adapted to achieve indication-based prices supported by real-world effectiveness.

Objective: To illustrate the potential of an outcomes guarantee to achieve indication-based prices aligned with real-world value, using a case study of trastuzumab for the treatment of metastatic breast and advanced gastric cancers.

Methods: We estimated costs and outcomes under traditional IBP (i.e., expected value IBP) and outcomes guarantee frameworks and calculated incremental cost-effectiveness ratios (ICERs) comparing treatment with and without trastuzumab. Efficacy data came from pivotal trials, whereas effectiveness data came from observational studies. We adjusted trastuzumab prices in order to achieve target ICERs of $150,000 per quality-adjusted life-year under each framework and for each indication.

Results: To achieve the ICER target under traditional IBP, the unit price of trastuzumab using efficacy evidence was adjusted for metastatic breast and advanced gastric cancers from an average sales price of $9.17 per mg to $3.50 per mg and $0.93 per mg, respectively. Under an outcomes guarantee, the unit price of trastuzumab using effectiveness evidence was adjusted for metastatic breast cancer and advanced gastric cancer to $8.66 per mg and $0.20 per mg, respectively.

Conclusions: Like expected value IBP, outcomes guarantee contracts can also vary payment based on indication. In addition, an outcomes guarantee can also reduce uncertainty regarding effectiveness and better align payment with the actual value of a treatment.

Disclosures: No funding supported this study. Carlson reports consulting fees from Genentech, Pfizer, and Seattle Genetics. The other authors have no conflicts of interest to disclose. Study concept and design were contributed by Carlson, Yeung, and Li. Yeung, Carlson, and Li collected and analyzed the data. The manuscript was written primarily by Yeung, along with Carlson and Li, and revised by all the authors.