Varying results of early benefit assessment of newly approved pharmaceutical drugs in Germany from 2011 to 2017: A study based on federal joint committee data

Abstract

Background: Since January 2011, the Federal Joint Committee (FJC) conducts early benefit assessments (EBA) of newly approved pharmaceutical drugs compared to appropriate standard therapies. The FJC commissions the Institute for Quality and Efficiency in Healthcare (IQEH) to prepare preliminary reports. We aimed to evaluate the extent, impact, and reason for different judgments on added benefit of both institutions. Methods:We searched EBA data on the FJC website and included completed procedures from 2011 to 2017. We conducted a quantitative analysis of the difference between FJC and IQEH on divergent judgments, a quantitative analysis of the impact of EBA on market withdrawal, and a qualitative analysis to identify potential factors contributing to divergent judgments.Results: FJC rated an added benefit in 30% (139 of 457) and IQEH in 22% (101 of 457) matching research questions (P = 0.004). In the aftermath of EBA, 28 pharmaceutical drugs were withdrawn from the German market. We identified three potential factors that might have contributed to the divergent judgments. IQEH used a unique threshold concept to define the rating, FJC conducted additional public hearings, and FJC showed more flexibility with adherence to stringent criteria and interpretation of results.Conclusions: FJC and IQEH differed significantly in their early benefit assessment. In response to negative EBA decisions, pharmaceutical companies withdrew a considerable number of medicines from the German market. The present work uncovers the subjectivity and possible variance inherent in benefit assessment, as the two institutions observe the same rules of procedure.

The impact of additive or substitutive clinical study design on the negotiated reimbursement for oncology pharmaceuticals after early benefit assessment in Germany

“…Interestingly, Cabazitaxel shows a higher added premium than Abiraterone, even it was granted a lower added benefit (minor versus considerable) and both pharmaceuticals have exactly the same ACT, study design, target population and comparable pharmaceuticals in their indication (mCRPC). 17 Further influencing factors on the premium have to be looked at for this specific case. The low multiplicative and additive premium for Radium-223-dichloride can be explained by the fact that in this case the manufacturer's selling price (MSP) is taken into account (direct distribution to the nuclear medicine ambulatories) and thereby the reimbursement amount is lower.…”

“…For the observed variance in the added benefit reported by pharmaceutical companies, IQWiG, and the FJC see also Ruof et al[16] and Peinemann and Labeit[17] 3. Until end of June 2016, the prices had to be weighted by the countries' population 4.…”

Cost Effectiveness of Pharmacological Management for Osteoarthritis: A Systematic Review

“…An overview of existing studies analyzing the cost effectiveness of pharmacological interventions for OA would be useful for identifying the gaps in the current evidence, guiding researchers in designing and conducting high-quality economic evaluations, and helping administrators make decisions based on high-quality evidence. In the absence of a current review on this topic, and in light of previous reviews published in related areas [18][19][20][21][22][23][24][25], the purpose of this study is to systematically review economic evaluations for the pharmacological management of OA.…”