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

摘要(不超过500字)这项研究的目标有三个。首先，它试图经验性地评估哪些是孤儿药物与非孤儿药物覆盖率的预测因素。特别是，这项研究将侧重于了解监管因素以及临床和经济不确定性在多大程度上预测覆盖决定。其次，这项研究将试图分析哪些因素减少或加快了孤儿药物与非孤儿药物的覆盖时间，如从上市授权到HTA资金决定的时间。孤儿药物受益于专门的审批路径，通常针对可能加速进入市场的严重条件，但另一方面，由于与其相关的高度临床不确定性，它们在满足HTA要求方面也可能面临困难。进一步的分析将调查缺乏正式的孤儿政策是否会影响选定国家案例研究的复盖时间。最后，将探讨拥有多个孤立适应症是否提供了基于适应症的定价机会。有证据表明，制造商为他们的产品寻求和接受多个孤立的指示，并利用孤立的法规，如果这不被认为是确保产品生存所必需的，可能会由支付者承担费用。在支付者艰难应对医疗支出、OD成本效益门槛明显高于非孤儿药物的时代，区分低价值和高价值孤儿药物的价格可能会减轻国家预算的压力。

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.