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Confirmatory versus explorative endpoint analysis: Decision-making on the basis of evidence available from market authorization and early benefit assessment for oncology drugs

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  • Niehaus, Ines
  • Dintsios, Charalabos-Markos

Abstract

The early benefit assessment of pharmaceuticals in Germany and their preceding market authorization pursue different objectives. This is reflected by the inclusion of varying confirmatory endpoints within the evaluation of oncology drugs in early benefit assessment versus market authorization, with both relying on the same evidence. Data from assessments up to July 2015 are used to estimate the impact of explorative in comparison to confirmatory endpoints on market authorization and early benefit assessment by contrasting the benefit-risk ratio of EMA and the benefit-harm balance of the HTA jurisdiction. Agreement between market authorization and early benefit assessment is examined by Cohen’s kappa (k). 21 of 41 assessments were considered in the analysis. Market authorization is more confirmatory than early benefit assessment because it includes a higher proportion of primary endpoints. The latter implies a primary endpoint to be relevant for the benefit-harm balance in only 67% of cases (0.078). Explorative mortality endpoints reached the highest agreement regarding the mutual consideration for the risk-benefit ratio and the benefit-harm balance (0.000). For explorative morbidity endpoints (−0.600), quality of life (−0.600) and side effects (−0.949) no agreement is ascertainable. To warrant a broader confirmatory basis for decisions supported by HTA, closer inter-institutional cooperation of approval authorities and HTA jurisdictions by means of reliable joint advice for manufacturers regarding endpoint definition would be favorable.

Suggested Citation

  • Niehaus, Ines & Dintsios, Charalabos-Markos, 2018. "Confirmatory versus explorative endpoint analysis: Decision-making on the basis of evidence available from market authorization and early benefit assessment for oncology drugs," Health Policy, Elsevier, vol. 122(6), pages 599-606.
    • Handle: RePEc:eee:hepoli:v:122:y:2018:i:6:p:599-606
    DOI: 10.1016/j.healthpol.2018.03.017
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    References listed on IDEAS

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    1. Fischer, Katharina Elisabeth & Heisser, Thomas & Stargardt, Tom, 2016. "Health benefit assessment of pharmaceuticals: An international comparison of decisions from Germany, England, Scotland and Australia," Health Policy, Elsevier, vol. 120(10), pages 1115-1122.
    2. Inna Dabisch & Jürgen Dethling & Charalabos-Markos Dintsios & Melanie Drechsler & Daniel Kalanovic & Peter Kaskel & Frank Langer & Jörg Ruof & Thorsten Ruppert & Daniel Wirth, 2014. "Patient relevant endpoints in oncology: current issues in the context of early benefit assessment in Germany," Health Economics Review, Springer, vol. 4(1), pages 1-8, December.
    3. Jörg Ruof & Friedrich Schwartz & J.-Matthias Schulenburg & Charalabos-Markos Dintsios, 2014. "Early benefit assessment (EBA) in Germany: analysing decisions 18 months after introducing the new AMNOG legislation," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 15(6), pages 577-589, July.
    4. Ruof, Jörg & Knoerzer, Dietrich & Dünne, Anja-Alexandra & Dintsios, Charalabos-Markos & Staab, Thomas & Schwartz, Friedrich Wilhelm, 2014. "Analysis of endpoints used in marketing authorisations versus value assessments of oncology medicines in Germany," Health Policy, Elsevier, vol. 118(2), pages 242-254.
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    1. Agnes Kisser & Joschua Knieriemen & Annette Fasan & Karolin Eberle & Sara Hogger & Sebastian Werner & Tina Taube & Andrej Rasch, 2022. "Towards compatibility of EUnetHTA JCA methodology and German HTA: a systematic comparison and recommendations from an industry perspective," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 23(5), pages 863-878, July.
    2. Matthias Herpers & Charalabos-Markos Dintsios, 2019. "Methodological problems in the method used by IQWiG within early benefit assessment of new pharmaceuticals in Germany," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(1), pages 45-57, February.
    3. C. M. Dintsios & F. Worm & J. Ruof & M. Herpers, 2019. "Different interpretation of additional evidence for HTA by the commissioned HTA body and the commissioning decision maker in Germany: whenever IQWiG and Federal Joint Committee disagree," Health Economics Review, Springer, vol. 9(1), pages 1-15, December.

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