Living with Chronic Disease: Measuring Important Patient-Reported Outcomes

  • Paul Kamudoni
  • Nutjaree Johns
  • Sam Salek

Table of contents

  1. Front Matter
    Pages i-xii
  2. A New Roadmap for Development of a PRO Measure

    1. Front Matter
      Pages 1-1
    2. Paul Kamudoni, Nutjaree Johns, Sam Salek
      Pages 3-13
    3. Paul Kamudoni, Nutjaree Johns, Sam Salek
      Pages 15-49
  3. An Illustration of the New PRO Measure Development Roadmap Using Research in Hyperhidrosis as A Practical Example

    1. Front Matter
      Pages 51-51
    2. Paul Kamudoni, Nutjaree Johns, Sam Salek
      Pages 53-73
    3. Paul Kamudoni, Nutjaree Johns, Sam Salek
      Pages 75-90
    4. Paul Kamudoni, Nutjaree Johns, Sam Salek
      Pages 91-113
  4. Practical Considerations When Applying PRO Measures

    1. Front Matter
      Pages 141-141

About this book


This book serves as a roadmap for the development and application of patient-reported outcome (PRO) measures, supporting beginners through to experts, as a practical guide. To elucidate on key concepts in the book, examples from clinical research in hyperhidrosis and health-related quality of life and medicines clinical development context, are used. Health-related quality of life represents one of the most commonly measured PROs in both routine clinical practice and research. The book demonstrates the importance of PROs to patients with chronic disease and how such outcomes can assist clinicians in managing patients and monitoring their response to treatment in terms of both symptoms and impacts.

This book will benefit readers as a single-source practical guide on the development of modern PRO measures and may also serve as a blueprint for the conceptualization and planning of evidence generation related to PROs in various settings.  Ideas and suggestions on how to navigate recent developments shaping the field of PRO measurement are also offered.

Dr Paul Kamudoni (PhD MSc.) is a Scientific Associate Director in the Global Evidence and Value Development – R&D department at Merck KgaA in Darmstadt (Germany) where he leads the Centre of Excellence for Patient Reported Outcomes. Dr Kamudoni trained as a Pharmacoeconomist and a Clinical Outcome Measurement Scientist at Cardiff University. In his day to day work, he is involved in the development and validation of PRO measures, and the design and integration of PRO endpoints in clinical trials and other types of studies. He maintains an active research interest in methodological issues in the integration of electronic health records with prospective PRO assessments, and the application of item response theory in PRO measurement among others.

Dr Nutjaree Pratheepawanit Johns (PhD PharmD) is an Associate Professor at the Faculty of Pharmaceutical Sciences, and melatonin research group, Khon Kaen University, Thailand. She has held a number of academic positions both in Thailand and abroad, including a fellowship at the Western Institutional Review Board (WIRB) (Olympia, WA, USA). Dr Johns has an expertise in cancer care and quality of life (QoL) research and collaborates with several cancer centres in Northeastern Thailand. She has developed and tested a number of PRO measures including cross-cultural adaptation into the Thai language. Dr Johns is also a principal investigator in many clinical trials investigating the effects of disease or interventions on QoL of cancer patients. She has been a recipient of several prestigious awards including Scholarship for Developing Country, International Society for Quality of Life Research and the Doyle Prize: Postgraduate section, Palliative Medicine Journal.

Professor Sam Salek (PhD RPh FFPM FRPS MCMS FESCP) is Professor of Pharmacoepidemiology at the University of Hertfordshire, Hatfield, visiting Professor at the Estate of Hessen, Germany, and Director of the Institute for Medicines Development, Cardiff, UK. Professor Salek’s research focuses mainly on four areas: development and application of PRO measures; drug safety evaluation and pharmacovigilance; pharmacoeconomics and economic evaluation of healthcare; and pharmaceutical regulation & medicines development in the mature and emerging markets. He has published 22 books and 600+ journal articles and abstracts. Professor Salek is a member of five Editorial Boards and an active member of several societies.


Clinical outcome assessment Quality of life Validation Construct validity Content validity Responsiveness Minimal clinically important differences Clinical significance Interpretability Patient engagement Item response theory EXACT-PRO Hyperhidrosis Quality of Life Index Qualification process

Authors and affiliations

  • Paul Kamudoni
    • 1
  • Nutjaree Johns
    • 2
  • Sam Salek
    • 3
  1. 1.Global Evidence & Value Development – R&D, Merck KgaADarmstadtGermany
  2. 2.Faculty of Pharmaceutical SciencesKhon Kaen UniversityKhon KaenThailand
  3. 3.School of Life & Medical SciencesUniversity of HertfordshireHatfieldUK

Bibliographic information

  • DOI
  • Copyright Information Springer Nature Singapore Pte Ltd. 2018
  • Publisher Name Adis, Singapore
  • eBook Packages Medicine Medicine (R0)
  • Print ISBN 978-981-10-8413-3
  • Online ISBN 978-981-10-8414-0
  • Buy this book on publisher's site