OBJECTIVES The pharmaceutical R&D model currently rests upon an extensive period of clinical trials increasing in length, but also in complexity, threatening financial returns. While pharmaceutical companies could pull the pricing lever to increase value, increased pressure to control healthcare budgets can lead to struggles between pharmas and payers during HTA and pricing, impacting patients with treatments being unequally delayed throughout Europe, if not introduced at all. We aim to demonstrate the viability of an alternative pricing framework to foster value co-creation and overcome market access barriers.

METHODS : Simulations of Net Present Value were undertaken in oncology and rare diseases. NPV was derived using official data from pharmaceutical companies and sales forecast until LOE. We compare two scenarios: a traditional phase III approach and a model where products would be commercialized following phase II. Such products would not have proven their value on hard endpoints yet and hence would be initially negotiated at a discounted price. This system uses real-world clinical milestones: every time such milestone has been reached, the price of the treatment increases. Comparisons of NPV versus initial levels were used to test the framework, complemented with stakeholder interviews.

RESULTS : Our analysis has demonstrated a potential increase of 30% in NPV, led by an early access to market despite a lower initial price. Gains have been demonstrated for all stakeholders as treatment access is improved at an affordable cost, with final prices up to 20% lower while maintaining similar NPV levels, allowing uniformization of market access in Europe.

CONCLUSIONS : This framework has demonstrated financial viability for pharmaceutical companies as well as a potential to improve equity in treatment access combined with lower annual costs for payers. However, it will require a profound change in the HTA mindset and a radical improvement of real-world evidence platforms.