ISSUE

: Gene therapies are the new kids on the block, with potential for substantial patient/caregiver benefit, disease modification or cure, often in rare severely disabling and life-threatening diseases. However, they have a high upfront price tag meaning demonstrating cost-effectiveness is difficult and highly uncertain to the point that conventional HTA, whilst feasible to perform, is itself cumbersome and arguably of limited value for reliable decision-making. An alternative proposal is to bring key stakeholders together (payers, clinicians, patients, manufacturers) in order to negotiate a fair, affordable and sustainable price, and a payment plan that respects the constraints of the healthcare system. Alternative approaches need urgent discussion before more gene therapies hit the market.

OVERVIEW

: The panel will debate the merits and drawbacks of performing HTA on the relative clinical and cost-effectiveness of innovative gene therapies. The moderator will set up the debate on whether gene therapies represent a special case for exemption/modification from conventional HTA. Maarten Posta will present a case that all gene therapies should undergo rigorous HTA to assess value and quantify key areas of uncertainty for informing meaningful realworld outcomes data collection. Nevertheless, it maybe argued that the challenges and data uncertainties with gene therapies are clear without need for full HTA. Josie Godfrey will present the case for the patient organisation to be a key stakeholder in these negotiations in order to agree an equitable access plan that puts the needs of the patients and their carers at the centre of decision making.

The panelists will have 10 minutes each to present their case followed by audience Q&A/discussion. This panel will be of interest to all HTA stakeholders and decision-makers, including healthcare payers.