OBJECTIVES: The conventional cost-effectiveness methodologies use quality-adjusted-life-years (QALYs) with a predetermined threshold in health technology assessments. While this approach was well established for decades, it also received criticisms for its limitations. The purpose of our study is to review the alternative value-based pricing models and assess their usefulness in complementing QALYs to advance access to high-cost innovations.

METHODS: We conducted a comprehensive search on PubMed using keywords “cost-effectiveness,” “innovation,” and “value-based pricing” to identify pricing models published between January 1, 2010 and December 31, 2019. We also retrieved expert opinions from MassBio Value of Health Series 2019, ISPOR 2019, ISPOR Europe 2019, and the National Health Council Value Assessment conference 2019. We used the following checklist to appraise each model: 1) Is the model quantitative or qualitative? 2) Is it about adding new value elements, employing new decision rules, or developing novel payment models? 3) Is it being used in practice, or is it a theory? 4) Can it differentiate pricing of real innovation from the “me too” products?

RESULTS: Among the thirteen models we have analyzed, the ISPOR Task Force value flower, “multi-criteria decision making,” and “discrete experiment by patient experiences” are new frameworks with novel value elements added to the QALY matrix. Regarding employing new decision rules, payers use higher willing-to-pay thresholds for curative therapies and consider the uncertainties of effectiveness in the equation. Additionally, novel pricing models such as “pay-for-performance,” “bundled payment,” “three-part pricing,” “indication-based pricing,” “pay-over-time,” “ risk-sharing,” and the “ subscription-based” were developed to address challenges in long-term outcome uncertainty paired with high upfront costs, time divergence between costs and benefits, and concerns about affordability and fair sharing of the economic surplus of transformative therapies.

CONCLUSIONS: The value-based models in our review could help to formulate new pricing strategies in advancing transformative innovations market access.