OBJECTIVES: Assess the impact of price on the sustainability and long term viability of the biopharmaceutical business behind the emerging innovative potentially curative interventions

METHODS: Confidential poll using an instant electronic voting system with self-selected participants made of a mix of industry executives, patient association representatives, R&D researchers, strategy consultants and investors attending the World Orphan Drug Conference Washington DC (US) in 2018

RESULTS: CONCLUSIONS: Accepting a low price with the purpose of gaining market access could be the simplest solution to insure access of innovative treatment to all patients and health systems. Low price could in theory avoid the possible restrictions and or delays that a higher price would otherwise trigger. Although opinion is divided, the vast majority, perceive a potential long-term viability risk for gene/cell therapy if price negotiators prioritise access at the expense of price. Low epidemiology and incidence only conditions which can be potentially cured by gene/cell therapies will have to find the right price to be at the same time affordable and sustainable. All stakeholders involved or influencing pricing and access decisions should therefore continue to seek new, likely complex and mutually beneficial long-term solutions.