Presentation (CTI)

OBJECTIVES: Health technology assessment and regulatory approval do not guarantee timely patient access. The EU’s access architecture—harmonized Joint Clinical Assessment plus launch incentives in pharmaceutical legislation—aims to broaden access, yet US MFN pricing creates a countervailing incentive to protect the reference price by restricting European access. We propose a framework quantifying health forgone, distinguishing the two margins through which restriction operates.
METHODS: The framework distinguishes an extensive margin (whether and when a drug is launched and reimbursed) from an intensive margin (which patients within the label are covered). Eligible populations are estimated from incidence, subtype, stage, and treatment-line data. Per-patient forgone health is computed as restricted mean survival time (RMST) differences versus next-best therapy—the full survival benefit forfeited under non-access, a partial benefit under delay—benchmarked against the earliest-access country. Outcomes are scenario-conditional ranges, equity-weighted across income tiers, without asserting any specific launch has been delayed.
RESULTS: We illustrate both margins with ribociclib. On the extensive margin, differential launch and reimbursement timing across income tiers (Patients W.A.I.T. data) forfeits, for an untreated patient in the slowest-access tier, the full MONALEESA-2 survival benefit (63.9 vs 51.4 months; HR 0.76)—approximately 0.3-0.4 gross life-years and 0.2-0.3 QALYs. On the intensive margin, national HTA bodies narrow reimbursed populations within a fixed EU label: NICE initially excluded roughly 47% of ribociclib’s label-eligible early-breast-cancer population before widening. The extensive margin forfeits more health per patient and concentrates in lower-income Member States—the margin omitted from current formal pricing models.
CONCLUSIONS: The framework provides a transparent evidence base before MFN’s effects become observable and identifies the two access margins most influencing the estimated burden. The estimates measure gross forgone health; net welfare subtracts the opportunity cost of associated spending, largest in high-price, accommodating markets, which MFN’s upward price pressure would further increase, motivating research on survival, affordability, and displacement effects.