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Mind the gapBridging the difference between efficacy and effectiveness of orphan drugsSchuller, Y.

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Citation for published version (APA):Schuller, Y. (2018). Mind the gap: Bridging the difference between efficacy and effectiveness of orphan drugs.

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Download date: 29 Jun 2020

Mind the gap Bridging the diff

erence between effi

cacy and effectiveness of orphan drugs Yvonne Schuller

Bridging the difference between efficacy and effectiveness

of orphan drugs

Yvonne Schuller

Mind the gap

Bridging the difference between efficacy and effectiveness of orphan drugs

Y. Schuller

Colofon

© 2018, Yvonne Schuller, Amsterdam, The Netherlands

All rights reserved. No part of this thesis may be reproduced or transmitted in any form or by

any means, without permission of the author.

Work in this thesis was supported by the Dutch Medicines Evaluation Board (College ter

Beoordeling van Geneesmiddelen).

Financial support for the printing of this thesis was kindly provided by AMC Medical Research,

de Gaucher Stichting, Dutch Medicines Evaluation Board and ChipSoft.

ISBN 978-94-028-1054-7

Author Yvonne Schuller

Lay-out Ilse Stronks, persoonlijkproefschrift.nl

Cover Evelien Jagtman, www.evelienjagtman.com

Printing Ipskamp Printing

Mind the gap

Bridging the difference between efficacy and effectiveness of orphan drugs

ACADEMISCH PROEFSCHRIFT

ter verkrijging van de graad van doctor

aan de Universiteit van Amsterdam

op gezag van de Rector Magnificus

prof. dr. ir. K.I.J. Maex

ten overstaan van een door het College voor Promoties ingestelde commissie,

in het openbaar te verdedigen in de Agnietenkapel

op woensdag 4 juli 2018, te 10.00 uur

door Yvonne Schuller

geboren te Beverwijk

Promotiecommissie Promotor

Prof. dr. C.E.M. Hollak AMC - UvA

Copromotores

Dr. M. Biegstraaten AMC - UvA

Dr. V.V. Stoyanova-Beninska CBG-MEB

Overige leden

Prof. dr. B.M. Nogueira Sepodes University of Lisbon

Prof. dr. D. Cassiman UZ Leuven

Prof. dr. J.H.M. Schellens Antoni van Leeuwenhoek ziekenhuis

Dr. R.P. Hagendijk Universiteit van Amsterdam

Prof. dr. M.G.W. Dijkgraaf AMC - UvA

Prof. dr. M.J. Kersten AMC - UvA

Faculteit der Geneeskunde

Contents

Chapter 1 General introduction 7

Chapter 2 The quality of economic evaluations of ultra-orphan drugs in Europe –

a systematic review.

17

Chapter 3 Factors contributing to the efficacy-effectiveness gap in the case of

orphan drugs for metabolic diseases.

39

Chapter 4 Dose-finding studies among orphan drugs approved in the EU – a

retrospective analysis.

81

Chapter 5 Hurdles in access to high-priced orphan drugs. 113

Chapter 6 Adaptive pathway development for Fabry disease: a clinical approach. 143

Chapter 7 Oncologic orphan drugs approved in the EU – Do clinical trial data

correspond with real-world effectiveness?

159

Chapter 8 Clinical benefit of systemic treatment in patients with advanced

pancreatic and gastro-intestinal neuroendocrine tumors according to

ESMO-MCBS and ASCO framework.

193

Chapter 9 General discussion and future perspectives 217

Chapter 10 Addendum

Summary

Nederlandse Samenvatting

List of co-authors and affiliations

List of publications

List of acronyms and abbreviations

PhD Portfolio

About the author

Acknowledgements/Dankwoord

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