Orphan drugs: predictors of coverage, time-to-coverage and indication-based pricing

Summary (no more than 500 words) The objectives of this research are threefold.
First, it seeks to empirically assess which are the predictors of coverage for orphan versus non-orphan drugs. In particular, this research will focus on understanding to what extent coverage decisions are predicted by regulatory factors and clinical and economic uncertainties.
Secondly, this research will attempt to analyse which factors reduce or accelerate the time-to-coverage, indicated as the time from marketing authorisation to HTA funding decision, for orphan versus non-orphan drugs. Orphan drugs benefit from dedicated approval pathways and usually target severe conditions which might accelerate access to market but, on the other hand, they are also likely to face difficulties in meeting the HTA requirements due to the high clinical uncertainty associated with them. Further analyses will investigate whether the lack of a formal orphan policy impacts the time-to-coverage in selected country case-studies.
Finally, it will be explored whether having multiple orphan indications provides opportunities for indication-based pricing. Evidence suggests that manufacturers seek and receive multiple orphan indications for their products and take advantage of orphan regulations, also if this is not deemed as necessary for ensuring product viability, potentially at the expenses of payers. In a time when payers struggle with healthcare expenditure, with ODs cost-effectiveness threshold being sensibly higher than non-orphan drugs, differentiating prices among low and high-value orphan drugs could reduce the pressure on national budgets.