PublicationINTERDISCIPLINARY series PLATFORM ON BENEFIT ASSESSMENT VolumeVol. 2 10 INTERDISCIPLINARY PLATFORM ON BENEFIT ASSESSMENT AprilMarch 2020 2016 WhatClinical are Studies the (additional) – which benefits ofendpoints registry data? count? 2 INTERDISCIPLINARY PLATFORM ON BENEFIT ASSESSMENT PUBLICATION SERIES

PUBLICATION SERIES INTERDISCIPLINARY PLATFORM ON BENEFIT ASSESSMENT

VOLUME 1 Four years of AMNOG – Discourse and impulses VOLUME 2 Clinical studies – Which endpoints count? VOLUME 3 Adaptive Pathways – Opportunities and risks VOLUME 4 AMNOG 2.0 – Information problems VOLUME 5 Evidence gaps – What does registry data offer? VOLUME 6 Physician information via software – Ways and goals VOLUME 7 Physician information via software – Orientation or control? VOLUME 8 European Benefit Assessment – Opportunities and risks VOLUME 9 Contextual evidence – Strategies for targeted therapy VOLUME 10 What are the (additional) benefits of registry data?

ALL VOLUMES AVAILABLE ONLINE AT: HTTPS://WWW.AERZTEZEITUNG.DE/KOOPERATIONEN/PLATTFORM-ZUR-NUTZENBEWERTUNG INTERDISCIPLINARY PLATFORM ON BENEFIT ASSESSMENT CONTENTS 3

Volume 10 April 2020

Contents

EDITORIAL Confirmed benefit – but also an additional benefit? Registry data in the discourse 6

PETER G. M. MOL Good Registry Practice – setting the context 8

JÜRGEN STAUSBERG Overview of registries in Germany 12

STEFAN LANGE and registry data: The IQWiG’s perspective 24

ANTJE BEHRING Additional benefit of registry data for the early benef it assessment 30

BIRGIT BURKHARDT Registry data of the GPOH by the example of the NHL-BFM study group 42

STEFAN BRAUNE UND ARNFIN BERGMANN NeuroTransData Registry by the example of multiple sclerosis 50

JANBERND KIRSCHNER Registry data in neuromuscular diseases: SMArtCARE Registry 62

ANDREW JOHN ULLMANN Status and potential of registry research in Germany 68

FLORIAN STAECK Unclear regulatory requirements represent an obstacle for registries in Germany 74 4 INTERDISCIPLINARY PLATFORM ON BENEFIT ASSESSMENT INTERDISCIPLINARY PLATFORM ON BENEFIT ASSESSMENT GOALS 5

Goals of the plattform

ince the introduction of AMNOG in 2011, Ger- and which methodological problems occur during the many has a well-established and widely ac- process, cepted „adaptive system“ for the assessment • dentifying possible undesirable developments, in parti- of the patient-relevant additional benefit cular with regard to supplying patients with new active (Health Technology Assessment, HTA). The as- substances, Ssessment of the additional benefit by the Federal Joint • Enabling and holding a constructive dialogue with all Committee (G-BA) is the result of expert work based on a players involved in the benefit assessment procedure, law (AMNOG) and procedural and methodical regulations. e. g. on the further development of the legal framework The active players on the side of the G-BA and the health conditions of AMNOG. insurance funds are classified as scientists, hospital physici- Moreover, the European perspective in HTA of innovative ans and office-based statutory health insurance physicians, pharmaceuticals was reinforced by the European Commis- the Medical Service of the Health Funds and employees of sion’s proposal for a Regulation on HTA in 2018. Monito- the insurance fund administration, but also as patient re- ring the conflict between the well-established national as- presentatives, however, they act on the basis of their own sessment and the intended European HTA harmonisation interests. Value dossiers for new pharmaceuticals, likewise is also a central concern of the platform. The Interdiscipli- qualified and interest-based, are submitted to the G-BA by nary Platform would like to make a contribution to ensu- the pharmaceutical companies, which serve as the basis ring that new active substances are transparently and fairly for the assessment of the additional