Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2014 Jan 24;4(1):2.
doi: 10.1186/2191-1991-4-2.

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

Inna Dabisch, Jürgen Dethling, Charalabos-Markos Dintsios, Melanie Drechsler, Daniel Kalanovic, Peter Kaskel  1 , Frank Langer, Jörg Ruof, Thorsten Ruppert, Daniel Wirth
Affiliations

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

Inna Dabisch et al. Health Econ Rev. .

Abstract

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.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Key AMNOG terms, as per German Social Code (Book V). *or indirect comparison; **or validated surrogate. AMNOG=Arzneimittelmarktneuordnungsgesetz (National health care law). HRQoL=Health related quality of life. G-BA=German Joint Federal Committee.
Figure 2
Figure 2
Task force position flow chart.
See this image and copyright information in PMC

References

    1. Soria JC, Massard C, Le Chevalier T. Should progression-free survival be the primary measure of efficacy for advanced NSCLC therapy? Ann Oncol. 2010;4:2324–2332. doi: 10.1093/annonc/mdq204. - DOI - PubMed
    1. Oncology Working Party. Guideline on the evaluation of anticancer medicinal products in man. London, UK: European Medical Agency; (EMA/CHMP/205/95/Rev.4). http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guidelin... 2012. Accessed 28 March 2013.
    1. Bundesinstitut für Arzneimittel und Medizinprodukte. German Medicinal Products Act: Non-official translation. Berlin, Germany: Bundesinstitut für Arzneimittel und Medizinprodukte; http://hh.juris.de/englisch_amg/index.html; 2011. Accessed 28 March 2013.
    1. Marckmann G, Meran JG, Year books of the Working Group of Medical Ehics Committees in the Federal Republic of Germany Issue 13. Ethical aspects of research in oncology. Cologne, Germany: Deutscher Ärzte-Verlag; 2007.
    1. Yothers G. Toward progression-free survival as a primary end point in advanced colorectal cancer. J Clin Oncol. 2007;4:5153–5154. doi: 10.1200/JCO.2007.13.6796. - DOI - PubMed

LinkOut - more resources