Rieder Veronika, Hammerschmidt Thomas
Hochschule Rosenheim, Fakultät für Angewandte Gesundheits- und Sozialwissenschaften, Rosenheim.
Gesundheitswesen. 2018 Aug;80(8-09):e44-e53. doi: 10.1055/a-0592-6725. Epub 2018 Apr 26.
Since 2011, new drugs are assessed at the time of launch in Germany (AMNOG). Based on this early benefit assessment (EBA), drug prices are negotiated. At this time, the evidence base might be weak. A later benefit assessment (LBA) is not done on a regular basis except for selected drugs. Our objective was to analyze the impact of LBAs of drugs for the same indication.
Analysis of all completed LBAs between 2011 and 2016.
228 benefit assessments have been performed since 2011. 26 drugs were assessed twice for the same indication. Oncology and diabetes were the most common therapeutic areas in LBA and more pronounced than in EBA. 15 LBAs were due to the EBAs having a time limitation because of insufficient evidence base partially based on conditional approval. Time between EBA and LBA was 2.6 years. All 15 drugs had added benefit in the EBA, 4 got a better, 5 a worse assessment in the LBA. Seven drugs without added benefit in the EBA were assessed at the request of the manufacturer because of new data after 1.7 years. Three drugs could show added benefit in the LBA. Finally, 4 orphan drugs were reassessed according to the AMNOG regulation after achieving annual sales of 50 million euros. One got a better, 2 got a worse benefit assessment. Average improvement of benefit was +1.5 on a scale between - 3 (worst negative benefit) and +9 (highest positive benefit). Average deterioration of added benefit was - 1.4. Negotiated prices were significantly correlated with the change in the benefit assessment.
LBA on a broader evidence base did not result in a significantly changed outcome. A general LBA for all drugs does not appear to be necessary because of the limited effect on the benefit assessment and the price when considering cost and administrative burden of the AMNOG benefit assessment. The selective approach of LBA for specific drugs is sufficient in cases in which the evidence base was limited at launch.
自2011年起，德国在新药上市时进行评估（德国药品市场改革法案）。基于这一早期效益评估（EBA）对药品价格进行谈判。此时，证据基础可能较为薄弱。除部分选定药物外，后期效益评估（LBA）并非定期进行。我们的目的是分析针对同一适应症的药物进行后期效益评估的影响。
分析2011年至2016年间所有已完成的后期效益评估。
自2011年以来共进行了228次效益评估。26种药物针对同一适应症进行了两次评估。肿瘤学和糖尿病是后期效益评估中最常见的治疗领域，且比早期效益评估中更为显著。15次后期效益评估是由于早期效益评估因证据基础不足（部分基于有条件批准）而有时间限制。早期效益评估和后期效益评估之间的时间间隔为2.6年。所有15种药物在早期效益评估中均有额外效益，4种在后期效益评估中得到更好的评估，5种得到更差的评估。7种在早期效益评估中无额外效益的药物，因1.7年后有新数据应制造商要求进行了评估。3种药物在后期效益评估中显示有额外效益。最后，4种孤儿药在年销售额达到5000万欧元后，根据德国药品市场改革法案规定进行了重新评估。1种得到更好的效益评估，2种得到更差的效益评估。效益平均改善值在-3（最差负效益）至+9（最高正效益）的量表上为+1.5。额外效益的平均恶化值为-1.4。谈判价格与效益评估的变化显著相关。
基于更广泛证据基础的后期效益评估并未导致结果有显著变化。考虑到德国药品市场改革法案效益评估的成本和行政负担，由于对效益评估和价格的影响有限，似乎没有必要对所有药物进行常规后期效益评估。在上市时证据基础有限的情况下，针对特定药物的选择性后期效益评估方法就足够了。
