ABSTRACT

Objective

To analyze how ophthalmic drugs fared in the early benefit assessment (EBA) after its introduction in Germany up to 2020 and to quantify its impact on their negotiated prices.

Methods

Relevant documents were screened and essential content on added benefit outcomes and the underlying evidence was extracted next to pricing information. In addition to descriptive statistics, cross-stakeholder analyses and agreement statistics were implemented.

Results

Thirteen completed EBA were identified involving eight drugs. Only four drugs (30.8%) received an added benefit. The OR for no added benefit of ophthalmic drugs versus all other drugs was 2.971 (0.902–9.781). The agreement between manufacturers’ claims and decision-maker appraisals is fair (kappa 0.435). In all cases, evidence was derived for RCTs, but for different reasons, not all of them allowed direct comparisons with the comparator as defined by the decision-maker. The negotiated rebates on manufacturer’s selling prices varied from 6.8% up to 47.4%. Nevertheless, the rebates for ophthalmic drugs (median 14.5%) were lower than those for all negotiated drugs (median 24%).

Conclusion

Over the past decade, the EBA of ophthalmic drugs was not necessarily a success story, but in most of the cases, the drugs were successful in the market.

中文翻译：

---

德国眼科药物早期效益评估十年：成功与否？

摘要

客观的

分析眼科药物在德国推出至 2020 年后在早期效益评估 (EBA) 中的表现，并量化其对其谈判价格的影响。

方法

筛选了相关文件，并在定价信息旁边提取了有关附加收益结果的基本内容和基础证据。除了描述性统计之外，还实施了跨利益相关者分析和协议统计。

结果

确定了 13 个已完成的 EBA，涉及 8 种药物。只有四种药物（30.8%）获得了额外的好处。眼科药物与所有其他药物相比无额外益处的 OR 为 2.971 (0.902–9.781)。制造商的索赔和决策者的评估之间的一致性是公平的（kappa 0.435）。在所有情况下，都为 RCT 提供了证据，但由于不同的原因，并非所有这些都允许与决策者定义的比较对象进行直接比较。制造商销售价格的谈判回扣从 6.8% 到 47.4% 不等。然而，眼科药物的回扣（中位数 14.5%）低于所有谈判药物的回扣（中位数 24%）。

结论

在过去十年中，眼科药物的 EBA 不一定是成功的故事，但在大多数情况下，这些药物在市场上是成功的。