Dataset Information

Patient relevant endpoints in oncology: current issues in the context of early benefit assessment in Germany.

ABSTRACT:

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The German AMNOG healthcare reform includes a mandatory early-benefit-assessment (EBA) at launch. As per German social code, EBA is based on registration trials and includes evaluation of the patient-relevant effect of the new medicines compared to an appropriate comparator as defined by the Federal Joint Committee (G-BA). Current EBA decisions released have unveiled issues regarding the acceptance of some patient-relevant endpoints as G-BA and IQWiG are grading the endpoints, focusing on overall survival as the preferred endpoint in oncology.A taskforce of experienced German outcomes research, medical, health-technology assessment and biostatistics researchers in industry was appointed. After agreement on core assumptions, a draft position was prepared. Input on iterative versions was solicited from a panel of reviewers from industry and external stakeholders.Distinctive features of registration trials in oncology need to be considered when these studies form basis for EBA, especially in cancer-indications with long post-progression survival; and with several consecutive therapeutic options available post-progression. Ethical committees, caregivers and patients often demand cross-over-designs diluting the treatment-effect on overall survival. Regulatory authorities require evaluation of morbidity-related study endpoints including survival of patients without their disease getting worse (i.e., progression-free survival). Also, progression requires treatment-changes, another strong indicator for its relevance to patients.Based on specific guidelines and clinical trial programs that were developed to be consistent with regulatory guidance, endpoints in oncology are thoroughly evaluated in terms of their patient-relevance. This extensive knowledge and experience should be fully acknowledged during EBA when assessing the patient-relevant benefit of innovative medicines in oncology.

Jel codes

D61; H51; I18.

SUBMITTER: Dabisch I

PROVIDER: S-EPMC3901346 | biostudies-literature | 2014 Jan

REPOSITORIES: biostudies-literature

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Patient relevant endpoints in oncology: current issues in the context of early benefit assessment in Germany.

Dabisch Inna I Dethling Jürgen J Dintsios Charalabos-Markos CM Drechsler Melanie M Kalanovic Daniel D Kaskel Peter P Langer Frank F Ruof Jörg J Ruppert Thorsten T Wirth Daniel D

Health economics review 20140124 1

<h4>Unlabelled</h4>The German AMNOG healthcare reform includes a mandatory early-benefit-assessment (EBA) at launch. As per German social code, EBA is based on registration trials and includes evaluation of the patient-relevant effect of the new medicines compared to an appropriate comparator as defined by the Federal Joint Committee (G-BA). Current EBA decisions released have unveiled issues regarding the acceptance of some patient-relevant endpoints as G-BA and IQWiG are grading the endpoints, ...[more]

PMID: 24460706

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Project description:BACKGROUND:Since the implementation of the Regulation on Patient Integration (2003), the Act on the Reorganization of the Pharmaceutical Market (2011), and the Patient Rights Law (2013), the inclusion of patient perspectives has been further anchored in the German early benefit assessment process. During the assessment of rare disease interventions, patient perspectives are particularly important, as clinical studies are often designed acknowledging small samples and patients suffering from severe symptoms and the chronic course of the disease. Therefore, our research question is whether patient perspectives are considered as part of early benefit assessments for rare diseases. We also strive to examine how patient perspectives are methodologically elicited and presented. METHODS:Our empirical evidence comes from a systematic review of orphan drug value dossiers submitted to the German Federal Joint Committee as well as the corresponding evaluations conducted between January 1, 2011 and March 1, 2019 (n?=?81). Data on patient perspective integration were extracted using the following patient-reported outcome subcategories: clinical patient-reported outcomes, health-related quality of life, patient preferences, and patient satisfaction. RESULTS:The analysis demonstrates the specific relevance of patient-reported outcomes raised as part of the medical data set and presented during the early benefit assessment process. They are predominantly presented in the form of health-related quality of life data (n?=?75%) and clinical outcomes (n?=?49%). Preferences (n?=?2%) and satisfaction (n?=?1%) are still rarely presented, although the heated methodological discussion in Germany would suggest otherwise. While various methodologies for the integration of clinical outcomes and quality of life data were found, presenting data on satisfaction and preferences still lacks methodological rigor. The German Federal Joint Committee has not yet integrated these data in their decision text. Clinical outcomes and quality of life have been included in 46% and 73% of the cases, respectively. CONCLUSIONS:The underlying analysis demonstrates that there is still a relative high potential for the regular and systematic inclusion of patient perspectives within the early benefit assessment process for rare diseases. In particular, patient preferences and patient satisfaction are still rarely included suggesting the need for a clear-cut methodological foundation and incentives.

Project description:BackgroundThe AMNOG regulation, introduced in 2011 in Germany, changed the game for new drugs. Now, the industry is required to submit a dossier to the GBA (the central decision body in the German sickness fund system) to show additional benefit. After granting the magnitude of the additional benefit by the GBA, the manufacturer is entitled to negotiate the reimbursement price with the GKV-SV (National Association of Statutory Health Insurance Funds). The reimbursement price is defined as a discount on the drug price at launch. As the price or discount negotiations between the manufacturers and the GKV-SV takes place behind closed doors, the factors influencing the results of the negotiation are not known.ObjectivesThe aim of this evaluation is to identify factors influencing the results of the AMNOG price negotiation process.MethodsThe analysis was based on a dataset containing detailed information on all assessments until the end of 2015. A descriptive analysis was followed by an econometric analysis of various potential factors (benefit rating, size of target population, deviating from appropriate comparative therapy and incorporation of HRQoL-data).ResultsUntil December 2015, manufacturers and the GKV-SV finalized 96 negotiations in 193 therapeutic areas, based on assessment conducted by the GBA. The GBA has granted an additional benefit to 100/193 drug innovations. Negotiated discount was significantly higher for those drugs without additional benefit (p = 0.030) and non-orphan drugs (p = 0.015). Smaller population size, no deviation from recommended appropriate comparative therapy and the incorporation of HRQoL-data were associated with a lower discount on the price at launch. However, neither a uni- nor the multivariate linear regression showed enough power to predict the final discount.ConclusionsAlthough the AMNOG regulation implemented binding and strict rules for the benefit assessment itself, the outcome of the discount negotiations are still unpredictable. Obviously, negotiation tactics, the current political situation and soft factors seem to play a more influential role for the outcome of the negotiations than the five hard and known factors analyzed in this study. Further research is needed to evaluate additional factors.

Project description: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.

Project description:ObjectivesTo support the shift from disease-oriented towards goal-oriented care, we aimed to develop a tool which is capable both to identify priorities of an individual older hospitalised patient and to measure the outcomes relevant to him.DesignMixed-methods design with open interviews, three step test interviews (TSTIs) and a quantitative field test.SettingUniversity teaching hospital and a regional teaching hospital.ParticipantsHospitalised patients ages 70 years and older.ResultsThe Patient Benefit Assessment Scale for Hospitalised Older Patients (P-BAS HOP) consists of a baseline questionnaire and an evaluation questionnaire. Items were based on 15 qualitative interviews with hospitalised older patients. Feedback from a panel of four community-dwelling older persons resulted in some adaptations to wording and one additional item. Twenty-six hospitalised older patients participated in TSTIs with Version 1 of the baseline questionnaire, revealing indications for a good content validity and barriers in completion behaviour, global understanding and understanding of individual items, which were solved with several adaptations. Four additions were made by participants. After TSTIs with ten patients with the evaluation questionnaire, one adaptation was made. A field test with 91 hospitalised older patients revealed a small number of missing values.To enhance the feasibility, the number of items was reduced from 32 to 22, based on correlations and mean impact score. The field test was repeated with 104 other patients in a regional teaching hospital. To enhance the understanding, the tool was split into two phases. This version was tested with TSTIs with eight patients and appeared to be understandable. The final version was an interview-based tool and took about 11 min to complete.ConclusionsThe P-BAS HOP is a potentially suitable tool to identify priorities and relevant outcomes of the individual patient. Further research is needed to investigate its validity, reliability and responsiveness.

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Patient relevant endpoints in oncology: current issues in the context of early benefit assessment in Germany.

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Jel codes

Publications

Patient relevant endpoints in oncology: current issues in the context of early benefit assessment in Germany.

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