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Health Economics Review
Published in: Health Economics Review 1/2020

01-12-2020 | Research

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

Authors: C. M. Dintsios, I. Beinhauer

Published in: Health Economics Review | Issue 1/2020

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Abstract

Background

We analysed the impact of clinical study design for oncological pharmaceuticals on the subsequent price negotiations after early benefit assessment between pharmaceutical companies and the German National Association of Statutory Health Insurance Funds. The analysis was conducted for all oncology pharmaceuticals that underwent the early benefit assessment in Germany since its introduction in 2011 up to September 2016.

Methods

It was differentiated between additive (new therapy in addition to baseline therapy) and substitutive study designs (baseline therapy to be replaced). The study design was derived from the dossiers of the pharmaceutical companies submitted to the Federal Joint Committee. Subgroup specific costs in case of granted added benefit were calculated as annual therapy costs and compared with the costs of the appropriate comparators to quantify price premiums. Further price influencing factors were analysed in univariate and multivariate regression analysis considering the budget impact for the statutory health insurance as well.

Results

The mean and the median of the additive premiums for substitutive designs (€50,477.68 and €49,841.24) were higher than for additive designs, if the comparator was different to best supportive care (€48,750.00 and €42,820.44). The mean multiplicative premium for the substitutive designs was 15.07 versus 2.29 for the additive designs. EU-Prices and target population size had a significant effect on the reimbursement. The adjusted R-square in the log Premium OLS-regressions reached 0.708 when including all explanatory variables and considering interaction between target population and annual costs of the comparator.

Conclusions

Study design as an additional important influencing factor of the negotiations next to those stated in the framework agreement was identified and verified. Therefore, study design should be considered by pharmaceutical companies and by decision makers and payers within strategic price planning as a potential predictor. For some specific categories the number of cases was small. Further analyses should be performed when more oncology pharmaceuticals have passed the early benefit assessment.
Appendix
Available only for authorised users
Footnotes
1
Points of criticism on IQWiG’s methods can be found in Röhmel [8], Witte and Greiner [9], Vach [10], Herpers and Dintsios [11].
 
2
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
The pharmaceutical companies are obliged by law to report the actual sales price, which is the retail price not including value-added tax minus the discounts that have been granted.
 
5
Mandatory manufacturer, wholesale and pharmacy rebates and their changes over time as well and new indications of the included pharmaceuticals were incorporated in the calculations, respectively. Up to the end of 2013, the mandatory rebate for manufacturers was 16%. On 1st January 2014 it was reduced to 6% and after 1st April 2014 it increased to 7%.
 
6
On a pharmacy sales price (PSP) basis.
 
7
The only exception was the price calculation for Radium-223-dichloride which is distributed due to its technology (alpha radiation emitter) direct to consumer (nuclear medicine ambulatory) and, therefore, the manufacturer sales price (MSP) was used for the calculation.
 
8
Par. 130b German Social Code V
 
9
Abiraterone, Eribulin, Regorafenib, Enzalutamide, Pertuzumab and Dabrafenib were approved for two indications, which were assessed in two different points of time.
 
10
Some data on market penetration of assessed pharmaceuticals is presented in de Millas et al. [24].
 
11
Austria, Denmark, Finland, the Netherlands, Sweden, and the UK.
 
12
For influencing factors on Orphan drug pricing in Germany see Schlander et al. [32].
 
13
One-armed studies (cohorts), not adjusted indirect comparisons or historical controls.
 
14
Cabazitaxel 2. SG, Eribulin 2. SG, Abiraterone 2. SG, Tegafur, Axitinib 1. SG, Crizotinib 2. SG, Pixantron, Pertuzumab 3. SG, Vismodegib 1. & 2. SG, Afatinib 2. & 3. SG, Ipilimumab new ind., Trastuzumab 1. SG & 3. SG, Eribulin 2. Ind. of 2. SG, Radium 223-dichloride 1. SG, Regorafenib new ind., Afatinib new ass. 2. & 3. SG, Nivolumab 1. SG & 3. SG, Pembrolizumab 1. SG, Trametinib 1. SG
 
15
Pertuzumab (first indication SG and second indication), and the re-assessment of Regorafenib after the termination of the time limitation of the first FJC.
 
16
Par. 130b sec. 3 German social code V
 
17
mCRPC: Metastatic castration-resistant prostate cancer.
 
18
The extreme value for Axitinib can be explained by the fact that the added benefit was granted only for one subgroup which refers to 1% of the labelled target population and which generates the respective premium.
 
19
One possible explanation for the price policy of the pharmaceutical company might be a very close launch price with regard to the comparable pharmaceuticals Gefitinib and Erlotinib for the indication of interest.
 
20
This was next to Nintedanib and Aflibercept one of the three presented cases with an add on design to a curative ACT.
 
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Metadata
Title
The impact of additive or substitutive clinical study design on the negotiated reimbursement for oncology pharmaceuticals after early benefit assessment in Germany
Authors
C. M. Dintsios
I. Beinhauer
Publication date
01-12-2020
Publisher
Springer Berlin Heidelberg
Published in
Health Economics Review / Issue 1/2020
Electronic ISSN: 2191-1991
DOI
https://doi.org/10.1186/s13561-020-00263-2

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