Nuijten Mark, Van Wilder Philippe
A2M, Amsterdam, The Netherlands.
Ecole de Santé Publique, Université Libre de Bruxelles, Bruxelles, Belgium.
BMC Health Serv Res. 2021 Mar 12;21(1):222. doi: 10.1186/s12913-021-06208-7.
Innovative orphan drugs often have an incremental cost-effectiveness ratio (ICER) which is higher than the maximum threshold for reimbursement. Payers have limited budgets and often cannot pay the full price of a new product, but pharmaceutical and biotechnology companies require a minimum price to satisfy their investors. The objective of this study was to present a possible solution to bridge this pricing gap by having early phase price agreements, which reduce the risk for investors.
We used a Pricing Model, which determines the minimum (break-even) price of an innovative drug from an investor's perspective. This model is based on economic valuation theory, which uses the expected free cash flows and the required cost of capital. We selected two orphan drugs with a positive clinical assessment and an ICER higsher than the Dutch maximum threshold of €80,000 per QALY gained to use as examples in the model: Spinraza for spinal muscular atrophy and Orkambi for cystic fibrosis.
The results show that early pricing agreements before phase III trials can substantially lower the drug price resulting from a lower cost of capital. The minimum price for orphan drugs can be reduced by 27.4%, when cost of capital decreases from 12 to 9%. An additional adjustment of other critical parameters due to early pricing agreements (lower probabilities of phase III failure and lower research and development (R&D) costs) can further reduce the minimal price by 62.8%.
This study shows that earlier timing of price negotiations resulting in an agreement on drug price can substantially lower the minimal price of orphan drugs for the investor.
创新型孤儿药的增量成本效益比（ICER）往往高于报销的最高阈值。支付方预算有限，通常无法支付新产品的全部价格，但制药和生物技术公司需要最低价格来满足其投资者。本研究的目的是提出一个可能的解决方案，即通过达成早期阶段价格协议来弥合这一定价差距，从而降低投资者的风险。
我们使用了一种定价模型，该模型从投资者的角度确定创新药物的最低（盈亏平衡）价格。此模型基于经济估值理论，该理论使用预期自由现金流和所需资本成本。我们选择了两种临床评估为阳性且ICER高于荷兰每获得一个质量调整生命年（QALY）80,000欧元最高阈值的孤儿药作为模型示例：用于脊髓性肌萎缩症的Spinraza和用于囊性纤维化的Orkambi。
结果表明，在III期试验前达成早期定价协议可因资本成本降低而大幅降低药品价格。当资本成本从12%降至9%时，孤儿药的最低价格可降低27.4%。由于早期定价协议（III期失败概率降低和研发成本降低）对其他关键参数的额外调整可进一步将最低价格降低62.8%。
本研究表明，提前进行价格谈判并就药品价格达成协议可大幅降低投资者的孤儿药最低价格。
