Health Economics, University Duisburg-Essen, Essen, Germany.
Institute for Health Services Research and Health Economics, Medical Faculty, Heinrich-Heine-University Düsseldorf, Building: 12.49, Moorenstr. 5, 40225, Düsseldorf, Germany.
Pharmacoeconomics. 2020 Apr;38(4):397-411. doi: 10.1007/s40273-019-00872-8.
Legislation introduced in 2011 in Germany has instituted an early benefit assessment of newly licensed pharmaceuticals with a subsequent price negotiation. For orphan drugs (ODs) a special legal framework applies, which accounts for the fact that ODs do not have to prove an added benefit over an appropriate comparative therapy previously determined by the decision maker. As, in addition, the content of negotiations between pharmaceutical companies and the payer is confidential, the aim of this study was to identify factors influencing the negotiated prices of ODs.
Twelve hypotheses on factors influencing the negotiated OD price were derived based on the existing literature and framework agreement between payers and pharmaceutical unions according to German social legislation. Univariate analyses were applied to detect statistically significant correlations between annual therapeutic costs of ODs and the hypothesized factors. Bivariate analyses were used to determine confounding factors. In addition, a multiple ordinary least squares (OLS) regression with backward selection was conducted. Finally, sensitivity analyses assessed the robustness of the results.
Thirty-five ODs were included in the analysis. The univariate analyses and subsequent sensitivity analyses validated five of the 12 hypotheses formulated. Univariate analyses suggest a statistically significant association between the OD price and the (i) therapeutic area; (ii) approval for pediatric care; (iii) treatment population size; (iv) cost of comparative therapies; and (v) European prices. The OLS regression identified European prices as the variable with the strongest association with the negotiated prices.
We show that German OD pricing is a multivariate phenomenon. However, due to interdependencies, these results must be treated with caution.
2011 年德国出台的立法对新获得许可的药品进行早期效益评估，并进行后续的价格谈判。对于孤儿药（ODs），有一个特殊的法律框架适用，这是因为 OD 不必证明相对于决策者先前确定的适当比较疗法有额外的益处。此外，制药公司和支付方之间谈判的内容是保密的，因此本研究的目的是确定影响 OD 谈判价格的因素。
根据现有文献和德国社会立法中支付方和制药联盟之间的框架协议，我们提出了 12 个关于影响 OD 谈判价格的因素的假设。采用单变量分析来检测 OD 年治疗费用与假设因素之间是否存在统计学上显著的相关性。采用双变量分析来确定混杂因素。此外，还进行了多元最小二乘法（OLS）回归分析，并采用向后选择法进行回归。最后，敏感性分析评估了结果的稳健性。
共纳入 35 种 OD。单变量分析和随后的敏感性分析验证了 12 个假设中的 5 个。单变量分析表明，OD 价格与以下因素存在统计学上显著的关联：（i）治疗领域；（ii）儿科治疗批准；（iii）治疗人群规模；（iv）比较疗法的成本；以及（v）欧洲价格。OLS 回归确定了欧洲价格是与谈判价格关联最强的变量。
我们表明德国 OD 定价是一个多变量现象。然而，由于存在相互依存关系，这些结果必须谨慎对待。
