Potentially curative gene/cell therapies face clinical uncertainties about long-term safety and duration of effect. This is compounded by the financial risks in the asymmetry between significant upfront costs and promised life-long benefits. While current tools, such as managed entry agreements (MEAs), may be suitable to address one uncertainty at a time, tackling clinical and financial uncertainties together will show the limitations of existing contracts and models.
With a MEA, manufacturers face the binary choice of either being denied access or having to reduce their initial target price. This price reduction often occurs through confidential means to address thefinancial uncertainty in terms of cost effectiveness and/or budget impact that payers deem unacceptable. These financial MEA contracts have various modalities on several levels: unit price, patient eligibility or population cap. Clinical performance-based agreements are linked to a clinical measurement that in turn triggers payment, additional discounts or no payment. Response, survival, prevention or other parameters are potentially used, but data collection and interpretation create another loop of complexity. Innovation is paramount for MEAs to work in the context of gene and cell therapies.
World Orphan Drug Congress Europe 2024 Review: 3 Themes for sustainable and equitable access to medicines for RD patients
This year’s European World Orphan Drug Congress, attended by CRA’s Bhavesh Patel, Michele Pistollato, Charlotte Poon, Owen Male and Clara Zacharko in...