For certain medicinal products, such as those for orphan or ultra-orphan diseases, or severely debilitating or life-threatening conditions, normal marketing authorisation (MA) processes can be sub-optimal to accelerate patient access due to data limitations. In this guidance note, we cover two alternatives – conditional MA and approval under exceptional circumstances, and the impact of these licensing routes on national pricing and reimbursement. Conditional MA has not been found to be detrimental to a medicinal product’s chances of receiving positive health technology assessment (HTA) recommendations. MAP BioPharma’s research found insufficient data to judge the impact of approval under exceptional circumstances on HTA outcomes. The impact of conditional MA and approval under exceptional circumstances on national pricing and reimbursement is dependent on the certainty of the incremental cost-effective ratio (ICER) base case and range. Conditional MA or approval under exceptional circumstances remain attractive options to accelerate patient access to important medicinal products.

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