The Importance of Real World Evidence in Orphan Drugs

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Publication Date:
November 2017
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Proof of value is an imperative for orphan drug manufacturers: it's the only route to improving patient access and overcoming unmet therapeutic needs for rare conditions. Progress has been made with real world data (RWD) and real world evidence (RWE), however there are still gaps that must be addressed. The Importance of Real World Evidence in Orphan Drugs explores the three-dimensional value story – clinical, patient and economic – and sheds light on how the core challenges facing orphan drug manufacturers can be overcome.

Discover on this page…

  • Key themes covered in the report
  • What you will learn from the report
  • Detailed table of contents
  • Research objectives and methodologies employed producing in the report
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Reasons to buy this report

There is still a lack of understanding in some quarters as to what real world evidence is or should be – which clearly demonstrates that greater clarity is needed, and that some areas are being missed. Getting to grips with the true patient experience can be harder than expected. Take 'quality of life' for example. How a doctor or a pharmaceutical researcher assesses this will differ from how the patient feels about their experience. The patient conversation is crucial – but with much smaller patient populations to work with, orphan drugs manufacturers must make the most of every opportunity to engage with patients and build the value story.

This report will enable you to:

  • Understand how RWE in orphan drugs differs to the mainstream and gain new insight on how to overcome specific challenges and achieve success.
  • Separate the constituent parts of the orphan drug value story and understand the benefits of each from a clinical, economic and patient perspective.
  • See where opportunities to leverage RWE occur throughout the orphan drug lifecycle and identify gaps in your current strategy.
  • Discover the latest views on the most effective RWE infrastructure and find out how to leverage technology to maximize advantage.
  • Gain insight into how to formulate and use partnerships productively across the spectrum of stakeholder groups.
  • Find out about the expected future developments likely to impact RWE in orphan drugs – including risk-sharing, wearables and patient centricity programs.
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Table of Contents

Executive summary

Research objectives and methodology

Experts interviewed

The distinct role for real-world evidence in orphan drugs

  • Incentives push orphan drug development
  • Hurdles to patient access to orphan drugs
  • Real-world data's role in the shift from volume to value
  • Embracing RWE in orphan drugs
  • Ensuring orphan drug success with RWE

RWE and the orphan drug value story

  • Key insights
  • Evidence of the three-dimensional value story
  • The clinical benefits
  • Patient benefits
  • Economic benefits
  • Outcomes selection and measurement in orphan drugs

Opportunities to leverage RWE across the orphan drug life cycle

  • Key insights
  • Preclinical stage: building an understanding of the disease and improving diagnosis
    • Improved diagnosis
    • Assessment of unmet needs
    • Setting the baseline for clinical improvements
  • Early developmental and pre-approval stages
  • Post-approval: ongoing studies on the value of the orphan drug

Overcoming challenges in leveraging RWE in rare diseases

  • Key insights
    • Collecting data on rare diseases is difficult and costly
    • The lengthy timeframe of clinical trials is not conducive to the life-limiting conditions of participants
    • There is a potential lack of an internal drive to utilise real-world data and a lack of action
  • SWOT analysis

Overcoming challenges in leveraging RWE in rare diseases

Key insights

  • Technology and the synergy of datasets
    • Data collection tools
    • Data analytics and data technologies
  • Institutional focus
  • A refinement framework

Partnerships with Patient Organisations, Experts and other Stakeholders

  • Key insights
  • Clinicians and researchers
    • Partnering with clinics and hospitals to conduct studies
    • Partnering with experts and academia to drive the quality of the science
  • Payers and HTA agencies
  • Patient advocacy groups and patient communities
    • To drive patient involvement and gather patient input
    • To locate specific patient populations
    • To drive awareness among patients
    • To establish give and take of support

Preparing for the future of RWE in orphan drugs

  • Key insights
  • Acknowledgement of the future trends
    • Risk-sharing schemes
    • Wearable technology
    • Patient-centricity
  • RWE's continued importance for pricing, reimbursement and market access
  • A constant need for a strategic approach to data collection and utilisation



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Research Methodology and Objectives

This report assesses how well orphan drug manufacturers are collating and using real world data and evidence (RWD and RWE) to boost market access and build the value story within the rare disease space.

The insights presented in this report are based on in-depth interviews conducted from August to October 2017 with 9 experts from leading orphan drug manufacturers. One interviewee chose to remain anonymous.

  • Professor Dorothee B. Bartels, Corporate Head of Global Epidemiology, Boehringer Ingelheim GmbH
  • Jay Chatfield, Senior Manager/Associate Director, US Market Access & Strategic Alliances, Orexigen
  • Usman Iqbal, Senior Medical Affairs Leader – Neuroscience, Global Medical Affairs, AstraZeneca US
  • Jean Malacan, Senior Health Economics Manager, Amgen
  • Claudio Marelli, MD, F.A.C.C., Global Medical Lead Director Internal Medicine, Shire International GmbH
  • Kay Parkinson, CEO, Cambridge Rare Disease Network UK
  • Aseel Bin Sawad, Senior Manager – Global Health Economics and Outcomes Research (HEOR), Baxter International, Inc.
  • Dr Christian Velten, Global Medical Project Manager, Actelion
  • Anonymous, Regional Market Access Senior Manager of a pioneering orphan drugs company

Key questions explored in this report include:

  • What new trends in RWE are impacting the orphan drug value story?
  • Which stakeholders in orphan drugs are most influenced by RWE?
  • What specific evidence are HTA agencies in the US and EU5 looking for?
  • What factors should be considered during RWE research?
  • What sources can be used to gather RWD?
  • How does RWE help in mapping and understanding the patient journey?
  • How can companies help payers to evaluate RWE?
  • What RWE are stakeholders asking for and what RWD are they interested in?
  • What is the future of RWE for attaining market access for orphan drugs?
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