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

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.

AMNOG Arbitral Awards: Analysis of the Decisions of the Arbitration Board Ten Years after AMNOG Implementation

“…the Institute for Quality and Efficiency in Health Care (IQWiG) to carry out the assessment of the benefit dossier within three months [1,5,8]. The IQWiG's benefit assessment is a recommendation to the G-BA [1,2,9,10].…”

“…The G-BA finally decides on the pharmaceutical’s additional benefit in its resolution within three further months 1 , 2 , 3 . The G-BA’s resolution differentiates between six extents of the additional benefit: major, considerable, minor, non-quantifiable, no additional benefit and less benefit 1 , 2 , 3 , 5 , 8 , 11 .…”

“…The negotiating parties have six months to negotiate the reimbursement price becoming effective as of the thirteenth months after launch 1 , 3 , 11 , 12 . If the pharmaceutical was not assigned an additional benefit, the reimbursement price should not lead to higher annual therapy costs than the most economic ACT 5 , 9 . If an additional benefit was assigned by the G-BA, the reimbursement price of the assessed pharmaceutical could lead to a price premium in addition to the annual therapy costs of the ACT and considers the extent of the additional benefit, the actual selling prices in other European countries and the annual therapy costs of comparable pharmaceuticals 5 , 9 .…”

“…This changed when the Act to Reorganise the Pharmaceuticals’ Market in the Statutory Health Insurance System (AMNOG) became effective in Germany as of 1 January 2011. The AMNOG implemented a new two-step process to determine a pharmaceutical’s reimbursement price valid after an initial twelve months of free pricing 2 , 3 , 4 , 5 . The two-step process begins with an early benefit assessment initiated at the launch of the pharmaceutical.…”

“…The two-step process begins with an early benefit assessment initiated at the launch of the pharmaceutical. The objective of the early benefit assessment is to assess whether a pharmaceutical has an additional benefit in comparison to the appropriate comparator therapy (ACT) 5 , 6 , 7 .…”