Patient Reported Outcomes: Strategies for market access
Harness the power of Patient-reported outcomes
Patient-reported outcomes (PRO) can refine research design, support new product applications and build powerful evidence of your brand’s value with payers. But how do you create effective PRO instruments, in what settings do they bring greatest value and what developments are on the horizon?
Informed by expert opinion, this report examines the emerging science of PROs and identifies how you can practically build the tools, and apply the techniques, that will harness the power of the patients’ experience for your brand.
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Answering key questions:
Key Topics Explored
- Could do better: Pharma is not fully benefitting from PROs – in what areas could they do better?
- Expanding use: In what conditions and therapy areas are PROs playing a key role?
- Market Access: How can PRO data be used to enhance market access?
- Key PRO ingredients: How can you define a PRO measure and what patient and operational issues do you need to consider when designing a study?
- Measuring up: What different types of PRO measures currently available and how might they develop in the future?
- Improving research: In what ways can PRO measures be added into clinical trial design to deliver more valuable outcomes?
- Payer influence: What PRO data can be persuasive to payers and support pricing?
- Guidance: What guidance is available from the FDA, EMA and PRO Consortium on the selection and use of PRO instruments?
- PRO measures articulate the patients’ experiences in a structured manner for application across a range of pharma’s activities, but it is essential to design studies that deliver insights not just data.
- PRO data is no longer just a “nice to have”. Regulators and payers are increasingly demanding PRO data to support their evaluations.
- While traditionally used in pre-marketing settings, PROs are now positively impacting drug applications and post-marketing assessments
- Incyte’s Jakafi (ruxolitinib) is seen as a model application of a PRO strategy – what benefits did the company accrue and what lessons can be learnt?
- Dr Hendrik-Tobias Arkenau, MD, PhD, Founding Medical Director of the Sarah Cannon Research Institute UK, and Fellow of the Royal College of Physicians.
- Ashok Bhaseen, Global President of Thyroid Federation International.
- Claire Burbridge. Senior Director of Clinical Outcomes Assessment at Clinical Outcomes Solutions Ltd.
- Dr Diego Cadavid, Senior Medical Director, Biogen.
- Dr Stephen Joel Coons, PhD, Executive Director, Patient-Reported Outcome Consortium, Critical Path Institute, US.
- Dr Gordon Crawford, MD, Director and co-founder of Patients Direct.
- Linda Deal, Patient-Centered Outcomes Assessment (PCOA) Lead, Global Health & Value, Pfizer.
- Matt Reaney, Head of Patient-Reported Outcomes, Global Health Economics and Outcomes Research, Sanofi.
- Sue Vallow, Senior Director and Head of Patient Focused Outcomes at GlaxoSmithKline.
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- 1 Executive summary
- 2 Research objectives, methodology and definitions
- 2.1 Objectives
- 2.2 Methodology
- 2.3 Definitions
- 3 Introduction
- 4 Patient-reported outcomes (PROs): gauging the patient experience
- 4.1 Defining a PRO
- 4.1.1 Observer-reported outcomes (ObsRO)
- 4.2 A more holistic approach to health benefits
- 4.3 How PRO data differ from other clinical data .
- 4.4 Growth in the significance of PROs to pharma
- 4.5 Focus on: Oncology and haematology
- 5 Growth in PRO measures and their use
- 5.1 Patient experience is recognised as more than symptoms alone
- 5.2 The value of PROs pre- and post-marketing
- 5.3 Payers seek a different approach to understanding the impact of disease
- 5.4 FDA guidance for industry on PRO measures
- 5.5 Focus on: The PRO Consortium – liaison with the FDA
- 5.6 European Medicines Agency (EMA) guidance on PROs
- 5.7 Patient centricity is a key driver of growth in PROs
- 5.8 Case study: Jakafi, one of the first successful uses of PROs for product approval with the FDA .
- 5.8.1 Structure of the Jakafi PRO
- 6 PRO measures, instruments and classification
- 6.1 Categories of PROs
- 6.2 Which PRO measure? Choosing the right one to suit your needs
- 6.3 Types of PRO measures and the tools used to obtain data
- 6.3.1 Quality of life
- 6.3.2 Generic PRO e.g. SF-36 (Short Form-36)
- 6.3.3 Utility measure
- 6.3.4 Disease/condition-specific PROs.
- 6.4 Patient-centric outcomes
- 7 Development of a PRO instrument to capture optimal PRO data
- 7.1 Determining and developing PRO instruments optimal to the data required
- 7.1.1 Guidance on development of PRO instruments
- 7.2 Timing and development of PRO measures
- 7.3 Instruments to capture PRO measure data
- 7.3.1 Quantitative data, qualitative data and questionnaires
- 7.4 PRO instruments obtain individual-level data
- 7.5 Data capture with electronic PROs (ePROs)
- 7.6 Other methods of data capture
- 8 Teasing out the value of PROs for payers, pharma and patients
- 8.1 What the experts say about the value of PROs
- 8.2 The added value of PRO measures for different stakeholders – benefits and limitations
- 8.2.1 Benefits
- 8.2.2 Limitations
- 8.3 PROs can help predict a product’s chance of success with reimbursement authorities/HTA
- 8.4 What payer and reimbursement authorities expect from PRO measures
- 8.5 How PRO measures can improve market access
- 8.6 Employers as payers in the US
- 8.7 PROs: pharma’s opportunity to improve the product offering and achieve success
- 8.8 PROs can drive pharma innovation
- 8.9 Are companies fully grasping the opportunities and values associated with use of PRO measures?
- 9 Down the road – opportunities and challenges for PROs over the next 5-10 years
- 9.1 Redefining ‘good outcomes’
- 9.2 New PRO measures and PRO instruments will be developed
- 9.3 Reimbursement authorities will place more emphasis on PROs
- 9.4 Improvements in the interpretation of PROs
- 9.5 PROs will be recognised for their value in understanding patient compliance
- 9.6 PRO measures and PRO instruments are set to expand in number and become more refined, improving the patient experience
- 9.7 PROs need to be incorporated into treatment algorithms
- 9.8 Greater engagement with PROs by physician and patients associations
- 9.9 Technology will drive PRO measure development and use
- 9.10 Increased emphasis on post-marketing use
- 9.11 PRO assessment in combination with performance measures
- 9.12 Making the case for the value of PRO assessment
- 9.13 Feedback from authorities on PRO measure development
- 10 Conclusion