title: [Patient-relevant outcomes and surrogates in the early benefit assessment of drugs: first experiences].
abstract: The Act on the Reform of the Market for Medicinal Products (AMNOG) became effective in Germany on January 1, 2011. Since then, the assessment of the added benefit of new drugs versus a therapeutic standard on the basis of dossiers submitted by pharmaceutical companies has been required by law. The Federal Joint Committee (G-BA) generally commissions the Institute for Quality and Efficiency in Health Care (IQWiG) with this task. The added benefit is primarily to be demonstrated on the basis of patient-relevant outcomes. The aim of this paper is to describe the feasibility of the early benefit assessment on the basis of patient-relevant outcomes by systematically characterising the outcomes available in company dossiers and comparing the companies' and IQWiG's evaluations regarding patient relevance and surrogate validity. Dossier assessments published between October 2011 and June 2012 were used for this purpose. The outcomes available and the respective evaluations were extracted and compared. 12 out of 22 submitted dossiers contained sufficient data to assess outcomes; all 12 assessable dossiers provided data on patient-relevant outcomes. Data on mortality and adverse events were available in all dossiers, except that one dossier did not contain adverse event data on the relevant subpopulation. In contrast, data on morbidity and health-related quality of life were available in 8 and 7 dossiers, respectively. Of a total of 214 outcomes extracted by IQWiG, 124 patient-relevant and 3 surrogate outcomes were included in IQWiG's assessment (companies: a total of 183 outcomes included, of which 172 were patient-relevant and 11 were surrogates). The first experiences with AMNOG have shown that in principle an early benefit assessment of drugs based on patient-relevant outcomes is feasible. The companies' and IQWiG's evaluations regarding patient relevance and surrogate validity of outcomes partly deviated from each other. By increasingly considering patient-relevant outcomes in approval studies, pharmaceutical companies can create the necessary data basis for the early benefit assessment.
authors.affiliation: Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Köln, Deutschland. Electronic address: tatjana.kvitkina@iqwig.de.
authors.forename: Tatjana
authors.initials: T
authors.lastname: Kvitkina
authors.affiliation: Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Köln, Deutschland.
authors.forename: Anette
authors.initials: A
authors.lastname: ten Haaf
authors.affiliation: Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Köln, Deutschland.
authors.forename: Stefanie
authors.initials: S
authors.lastname: Reken
authors.affiliation: Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Köln, Deutschland.
authors.forename: Natalie
authors.initials: N
authors.lastname: McGauran
authors.affiliation: Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Köln, Deutschland.
authors.forename: Beate
authors.initials: B
authors.lastname: Wieseler
mesh_terms: D016907:Adverse Drug Reaction Reporting Systems / Q000331:legislation & jurisprudence; D015415:Biomarkers; D017277:Drug Approval / Q000331:legislation & jurisprudence*; D004345:Drug Industry; D023381:Endpoint Determination / Q000592:standards*; D005858:Germany; D018166:Health Care Reform / Q000331:legislation & jurisprudence*; D009313:National Health Programs / Q000331:legislation & jurisprudence*; D063868:Patient Outcome Assessment; D018570:Risk Assessment / Q000331:legislation & jurisprudence
chemical_list: D015415:Biomarkers
keywords: AMNOG; Early benefit assessment; Frühe Nutzenbewertung; Patientenrelevante Endpunkte; Surrogatendpunkte; patient-relevant outcomes; surrogate outcomes