Patient Reported Outcomes: Strategies for gaining or expanding market access

May 2016

A FirstWord ExpertViews Dossier Report

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Patient Reported Outcomes: Strategies for gaining or expanding market access

Published May 2016© Copyright 2016 Doctor’s Guide Publishing Limited

All rights reserved. No part of this publication may be reproduced or used in any form or by any means graphic, electronic or mechanical, including photocopying, recording, taping or storage in information retrieval systems without the express permission of the publisher.

This report contains information from numerous sources that Doctor’s Guide Publishing Limited believes to be reliable but for which accuracy cannot be guaranteed. Doctor’s Guide Publishing Limited does not accept responsibility for any loss incurred by any person who acts or who fails to act as a result of information published in this document. Any views and opinions expressed by third parties and reproduced in this document are not necessarily the views and opinions of Doctor’s Guide Publishing Limited. Any views and opinions expressed by individuals and reproduced in this document are not necessarily the views and opinions of their employers.

Cover image: © Bacho Foto | AdobeStock

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Patient Reported Outcomes: Strategies for gaining or expanding

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ContentsExecutive summary ..............................................................................................................................1

Research objectives, methodology and definitions .....................................................4

Objectives ....................................................................................................................................................4

Methodology ................................................................................................................................................5

Definitions ....................................................................................................................................................6

Introduction ................................................................................................................................................7

Patient-reported outcomes (PROs): gauging the patient experience ............10

Defining a PRO ........................................................................................................................................12

Observer-reported outcomes (ObsRO) ........................................................................................13

A more holistic approach to health benefits .....................................................................................13

How PRO data differ from other clinical data ..................................................................................15

Growth in the significance of PROs to pharma ...............................................................................17

Focus on: Oncology and haematology .............................................................................................. 19

Growth in PRO measures and their use ..............................................................................20

Patient experience is recognised as more than symptoms alone .............................................20

The value of PROs pre- and post-marketing ...................................................................................22

Payers seek a different approach to understanding the impact of disease ...........................22

FDA guidance for industry on PRO measures ................................................................................23

Focus on: The PRO Consortium – liaison with the FDA ..............................................................25

European Medicines Agency (EMA) guidance on PROs .............................................................25

Patient centricity is a key driver of growth in PROs .......................................................................27

Case study: Jakafi, one of the first successful uses of PROs for product approval with the FDA ............................................................................................................28

Structure of the Jakafi PRO .............................................................................................................29

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Patient Reported Outcomes: Strategies for gaining or expanding

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PRO measures, instruments and classification ...........................................................31

Categories of PROs ................................................................................................................................31

Which PRO measure? Choosing the right one to suit your needs............................................34

Types of PRO measures and the tools used to obtain data ......................................................36

Quality of life ........................................................................................................................................36

Generic PRO e.g. SF-36 (Short Form-36) ..................................................................................37

Utility measure ....................................................................................................................................37

Disease/condition-specific PROs ...................................................................................................38

Patient-centric outcomes .......................................................................................................................40

Development of a PRO instrument to capture optimal PRO data ...................41

Determining and developing PRO instruments optimal to the data required ......................... 41

Guidance on development of PRO instruments ........................................................................43

Timing and development of PRO measures ....................................................................................47

Instruments to capture PRO measure data .....................................................................................48

Quantitative data, qualitative data and questionnaires............................................................49

PRO instruments obtain individual-level data .................................................................................49

Data capture with electronic PROs (ePROs) ..................................................................................50

Other methods of data capture ............................................................................................................53

Teasing out the value of PROs for payers, pharma and patients ....................56

What the experts say about the value of PROs ..............................................................................57

The added value of PRO measures for different stakeholders – benefits and limitations .....58

Benefits ..................................................................................................................................................58

Limitations .............................................................................................................................................60

PROs can help predict a product’s chance of success with reimbursement authorities/HTA ...........................................................................................................61

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Patient Reported Outcomes: Strategies for gaining or expanding

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What payer and reimbursement authorities expect from PRO measures ..............................61

How PRO measures can improve market access .........................................................................64

Employers as payers in the US ...........................................................................................................65

PROs: pharma’s opportunity to improve the product offering and achieve success ...........66

PROs can drive pharma innovation ...................................................................................................67

Are companies fully grasping the opportunities and values associated with use of PRO measures? .................................................................................................................68

Down the road – opportunities and challenges for PROs over the next 5-10 years ..................................................................................................................69

Redefining ‘good outcomes’ ...........................................................................................................69

New PRO measures and PRO instruments will be developed ............................................69

Reimbursement authorities will place more emphasis on PROs ........................................69

Improvements in the interpretation of PROs .............................................................................69

PROs will be recognised for their value in understanding patient compliance ............... 70

PRO measures and PRO instruments are set to expand in number and become more refined, improving the patient experience ......................................................70

PROs need to be incorporated into treatment algorithms .....................................................70

Greater engagement with PROs by physician and patients associations ........................70

Technology will drive PRO measure development and use ................................................. 71

Increased emphasis on post-marketing use ..............................................................................71

PRO assessment in combination with performance measures ...........................................71

Making the case for the value of PRO assessment ................................................................72

Feedback from authorities on PRO measure development .................................................. 72

Conclusion ................................................................................................................................................73

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Research objectives, methodology and definitions

ObjectivesThis FirstWord report provides an analysis of where patient-reported outcomes (PROs) feed into drug development and market access strategies. It aims to highlight the benefits of PROs in complementing more conventional sources of data derived from clinical trials, and how the two sources of data can be used synergistically to build a clinical evidence base, sourced from patients as well as clinicians, to present to payers and regulatory agencies and to enhance the product offering more broadly. This report also shows how PROs provide a means of implementing the patient-centric approach that underpins much of the macro-level strategy present in the industry today.

Key questions that were asked during the course of the research included:

nTo what extent and how effectively is pharma currently using PRO measures generally, and is there room for improvement?

nWhat different types of PRO measures exist and how are they classified?

nWhat are the key benefits associated with obtaining PRO measures for pharma and for patients?

nWhat challenges are faced during PRO instrument development and implementation?

nWhat considerations are there in the design of a PRO study, for example, the patient types and processes involved?

nHow are findings related to PRO measures used to inform clinical trial programmes?

nIn what context do payers and/or reimbursement authorities require PRO data?

nHow are PRO data used to enhance market access?

nHow do you see the use of PROs by pharma developing over the next 5-10 years?

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MethodologyInformation in this report was gathered from interviews with experts in the pharmaceutical industry who work within medical affairs, health economics and outcomes research (HEOR), integrated healthcare informatics, heads of patient-reported outcomes and heads of patient-focused research. Some external specialists were also consulted. PROs fall under the responsibility of a diverse selection of in-house roles. Interviewees were sought from the US and Europe. Most interviewees are public speakers or authors on the topic, often presenting at specialist conferences.

Experts Interviewed

nDr Hendrik-Tobias Arkenau, MD, PhD, Founding Medical Director of the Sarah Cannon Research Institute UK, and Fellow of the Royal College of Physicians.

nAshok Bhaseen, Global President of Thyroid Federation International.

nClaire Burbridge, Senior Director of Clinical Outcomes Assessment at Clinical Outcomes Solutions Ltd, a company of specialists in the PRO field, providing strategic, qualitative and quantitative support to the pharmaceutical industry in the development, validation and analysis of PRO data.

nDr Diego Cadavid, Senior Medical Director, Biogen, US.

nDr Stephen Joel Coons, PhD, Executive Director, Patient-Reported Outcome Consortium, Critical Path Institute, US. His work involves the development and qualification of PRO measures for use as primary or secondary endpoints and endpoint measures in clinical trials.

nDr Gordon Crawford, MD, Director and co-founder of Patients Direct. He is also a senior partner in a GP practice.

nLinda Deal, Patient-Centered Outcomes Assessment (PCOA) Lead, Global Health & Value, Pfizer, US. Deal and the PCOA team work across the Pfizer Global Innovative Pharma portfolio. The views and opinions expressed in this report by Linda Deal are based upon her experience developing, validating and utilising PROs and Clinical Outcomes Assessments (COAs) and are not necessarily those of Pfizer.

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nMatt Reaney, Head of Patient-Reported Outcomes, Global Health Economics and Outcomes Research (HEOR), Sanofi.

nDr Robin Turpin, PhD, Director, US Health Delivery Systems and Head, Field HEOR Team, at Takeda Pharmaceuticals. She focuses on PROs in the context of real-world evidence. Much of her work is in partnership with payer organisations.

nSue Vallow, Senior Director and Head of Patient-Focused Outcomes at GlaxoSmithKline. Areas she is involved in include rare diseases and gene therapies.

DefinitionsPatient-reported outcomes (PROs): according to the Food and Drug Administration (FDA), a PRO is any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.2

PROs and PRO measures: these terms are used interchangeably.

PRO instruments: these are the tools used to obtain the PRO data e.g. a questionnaire.

2 US Department of Health and Human Services, FDA, CDER, CBER, CDRH. (December, 2009). Guidance for Industry – Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Retrieved from http://www.fda.gov/downloads/Drugs/.../Guidances/UCM193282.pdf

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Patient Reported Outcomes: Strategies for gaining or expanding

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Which PRO measure? Choosing the right one to suit your needsCadavid explains that when deciding on the type of PRO needed, he initially seeks to identify the nature of the construct or characteristic being investigated. “For example, take the construct of walking ability. If that is all you are investigating then you have what we call an unidimensional PRO,” he explains. Other PROs focus on multiple dimensions and they are integrated together. “In this case, you may have seven or 10 constructs that are assessed individually, and then they are all pulled together into a common score. This is often done when we study health status because there are many different dimensions to health.”

Cadavid says that health status, a generic PRO, is an important tool that might be used in a comparison of the impact of different diseases on patients. “This measures deviation from health. You might want to compare diabetes to Alzheimer’s to schizophrenia if you use a PRO that’s measuring health. This is often used in public health and even in management of diseases and considerations of research.”

Claire Burbridge from Clinical Outcomes Solutions points out that people often understand PRO data as quality of life (QOL). “Traditionally, people equated PROs and quality of life with each other, but PROs are also used to capture things like symptoms. That’s a key thing, and that’s where PROs are coming up more and more now. You’ve got symptom diaries, often in pain for example, because there’s no other way of assessing pain,” she says.

Burbridge also notes that in clinical practice in oncology, PROs can be very useful in an effort to weigh-up the impact of side effects against benefits of the therapies. “That’s an area where it is very important to get that broader perspective,” she says.

Dr Hendrik-Tobias Arkenau leads research into PRO measures in oncology patients. He says certain cancer types are more likely to use PRO measures. “We often use patient-reported outcomes in, for example, lung cancer, to test the breathing – if the patient is short of breath. In breast cancer we would more commonly ask about general pain. In specific tumour types, PRO measures are well-tested, established and validated.”

Arkenau points out that with the arrival of new cancer therapies, for example the immunotherapies, patients stay in early clinical trials for a very long time, and it is important to have PROs as early indicators of toxicity and side effects.

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The added value of PRO measures for different stakeholders – benefits and limitationsBenefits

The patient voice is heardEssentially, PROs provide structure to the patient’s voice and perspectives on their disease, their therapy and any other issue that relates to their medical situation. A condition related to these measures is that the data are essentially raw in the sense that they are free from interpretation by clinicians or other HCPs.

PROs provide a pharma company with added value that can guide early development stages through to post-approval when efforts are made to liaise with stakeholders, including patients, prescribers and payers. In fact, referring to the latter, many experts assert that PRO measures are no longer optional, but critical for meeting the growing requirements of reimbursement (and regulatory) authorities that grant patient experience a heightened status in product evaluations.

Sue Vallow from GSK emphasises that ideally, if a drug is both efficacious and acceptable to the patient, then outcomes improve in all respects. “With a minimally efficacious product you can get drug approval, but you also want to show that it’s good for the patient at the end of the day and that can improve the overall outcomes,” she says.

Benefits commercial strategyVallow also highlights that monitoring PROs can deliver results that are advantageous for both patients and a company’s commercial strategy. By way of example, she highlights Zytiga (abiraterone acetate), a drug that was being investigated for prostate cancer, acquired by Johnson & Johnson several years ago. “In the course of drug development they noticed that there was some improvement in pain relief,” says Vallow. “Pain was then investigated as an exploratory endpoint. Although it was exploratory, the data showed clinically significant improvement. The EMA accepted it into the label because of that improvement. It then gained a line extension around pain.”

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