Patient-Reported Outcomes

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This report details PRO teams, budgets, contributions to clinical goals, PRO delivery mechanisms and the future of PROs. Use these findings to begin PRO planning early in the product lifecycle while focusing on data quality and labeling impact. Benchmarks show PRO team size, budget management and average cost per patient, among many other data points, and timelines outline planning, administration and data retrieval. Explore guidelines for optimizing PROs’ impact on regulatory discussions and data effectiveness, and read insights into electronic PROs as well as various PRO delivery mechanisms, from pen-and-paper to tablets and digital media.

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TABLE OF CONTENTS

Report Details

Publication Date: July 2014
Pages: 131
Chapters: 5
Metrics: 500+
Charts/Graphics: 75

Top Reasons to Buy This Patient-Reported Outcomes Report

Leverage a dedicated PRO team to ensure strategic focus and regulatory compliance: As patient-reported outcomes become more important to the clinical development process and market access initiatives, companies should establish dedicated PRO teams.  Though these teams are new to the industry, many pharmaceutical organizations recognize the potential benefits of these dedicated groups.  This report highlights PRO team structures and resources, as well as provides a look at their growing impact within the organization.

Focus on data quality for PRO endpoints to impact final product labels: Clinical teams have collected patient-reported data for some time.  However, this data was not used to its full potential until regulatory agencies in the US and EU released guidances on PRO usage in 2006.  Companies are now better able to harness clinical trial PRO data during regulatory and reimbursement conversations.  Ultimately, PRO teams hope to provide regulatory agencies with data that will enhance final product labels and better their own marketing strategies.  This report showcases how regulatory bodies perceive PROs as well as surveyed companies’ success rates in impacting final product labels via these findings.

Adopt ePRO technology to maximize PRO efficiency:Patient-reported outcomes delivery mechanisms are key to PRO strategy. Traditionally, PRO instruments have been pen-and-paper or telephone-based.  However, with growing digital technology, the use of electronic patient-reported outcomes (ePROs) is becoming a viable and efficient solution.  This report examines how innovative pharmaceutical companies utilize advanced ePRO instruments to collect patient-reported data more accurately and efficiently. Data also show the percentage of companies using specific PRO modalities, as well as how long companies have had ePRO technology in place.

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Excerpt from Patient-Reported Outcomes

Patient-reported outcomes (PROs) offer clinical teams and regulatory agencies a more complete look at a drug’s effects and future success. Physiological effects are of course essential — if the drug is not producing the desired medical effect, there is no use in continuing the clinical trial. But PROs offer an additional look at the drug’s impact. In an era of increasing competition and rising payer demands, capturing these results directly from the patient is crucial. Adding the “patient’s voice” to the value proposition positions the product well come approval and reimbursement discussions.

Companies generally discount the value of these self-reported health outcomes in favor of quantitative clinical results. But PROs serve as an important indicator of the drug’s potential for future success: however well the product may perform clinically, a patient will be reluctant to comply with a regimen that is difficult to follow or that negatively impacts his or her quality of life. Non-adherence is estimated to cost the US healthcare system up to $289 billion each year, so payers are looking for these patient data when comparing drugs for formulary position. Companies must thus incorporate these data points starting in early clinical development to best incorporate these endpoints.

Table of Contents

8             Executive Summary

12           Study Methodology

13           Study Definitions

14           Patient-Reported Outcomes: Five Principles for Success

25           Allotting Resources to Fuel a Successful PRO Strategy

26           Dedicate Staff to Creating a Robust PRO Strategy

38           Managing PRO Budgets via Clinical Resources

44           Implementing and Supporting PRO Initiatives During Clinical Trials

45           Incorporating Patient-Reported Endpoints into Clinical Trials

63           Outsourcing Patient-Reported Outcomes Initiatives for Innovation

76           Selecting a PRO Instrument to Achieve PRO-Driven Goals

77           Using Standard PRO Instruments to Drive Patient-Centered Outcomes Initiatives

99           Choose Innovative PRO Modalities to Improve Data Quality

100        PRO Delivery Mechanisms

104        Driving PRO Efficiency via Digital Technology

111        Leveraging the Patient Voice to Drive Regulatory and Reimbursement Decisions

112         Harnessing PRO Evidence to Influence Reimbursement Discussions with Regulators

116        Gaining Approval for PRO-Based Claims in Final Product Marketing

124        The Future of Patient-Reported Outcomes: Evolving Consortia and Regulatory Guidelines

CHARTS AND GRAPHICS

8             Executive Summary

9             Figure E.1: FDA Guidance Endpoint Model in Disease Treatment

15           Figure E.2: Benefits of Adopting ePRO Technology

16           Figure E.3: Average Percentage of PRO Endpoints on Final Product Label, by Company

17           Figure E.4: Average Percentage of PRO Endpoints on Final Product Label, by Therapeutic Area

19           Figure E.5: Percentage of Companies with a Dedicated PRO Team

22           Figure E.6: Phase at Which PRO Planning Begins

23           Figure E.7: Phase at Which a PRO Is First Administered

25           Allotting Resources to Fuel a Successful PRO Strategy

26           Dedicate Staff to Creating a Robust PRO Strategy

27           Figure 1.1: Regions Involved in PRO Strategy Involvement, by Company Size

28           Figure 1.2: Regional PRO Strategy Involvement, by Company Size

29           Figure 1.3: Percentage of Companies with a Dedicated PRO Team

30           Figure 1.4: Function Providing Oversight of PRO Teams, by Company

31           Figure 1.5: PRO Team Alignment, by Company Size

32           Figure 1.6: PRO Team Size

33           Figure 1.7: Number of PRO Team FTEs, by Level of Executive

36           Figure 1.8: Function Responsible for PROs in Absence of Dedicated PRO Team, by Function

37           Figure 1.9: Function Responsible for PROs in Absence of Dedicated PRO Team, by Company Size

38           Managing PRO Budgets via Clinical Resources

40           Figure 1.10: Function Managing PRO Team Budget, by Company

42           Figure 1.11: Average Cost per Patient for PROs, by Company and Therapeutic Area

44           Implementing and Supporting PRO Initiatives During Clinical Trials

45           Incorporating Patient-Reported Endpoints into Clinical Trials

46          Figure 2.1: Average Percentage of Company Trials Implementing PRO Measurements, by Company Size

47          Figure 2.2: Percentage of Companies Measuring Specific PRO Metrics: All Companies

49          Figure 2.3: Percentage of Companies Measuring Specific PRO Metrics: Top 10 Pharma

49          Figure 2.4: Percentage of Companies Measuring Specific PRO Metrics: Top 50 Pharma

50          Figure 2.5: Percentage of Companies Measuring Specific PRO Metrics: Small Pharma

51           Figure 2.6: Of Trials with PROs, Percentage of Endpoints that Are PROs, by Therapeutic Area

52          Figure 2.7: Of Trials with PROs, Percentage of Endpoints that Are PROs, by Therapeutic Area      (Including Minimum and Maximum Values)

54           Figure 2.8: Phase at Which PRO Planning Begins

55           Figure 2.9: Phase at Which PRO Planning Begins, by Company Size

57           Figure 2.10: Phase at Which a PRO Is First Administered

57           Figure 2.11: Phase at Which a PRO Is First Administered, by Company Size

58           Figure 2.12: Frequency that PRO Is Administered to Patient

59           Figure 2.13: Frequency that PRO Is Administered to Patient, by Company Size

60           Figure 2.14: Frequency of PRO Data Retrieval

61           Figure 2.15: Frequency of PRO Data Retrieval, by Company Size

63           Outsourcing Patient-Reported Outcomes Initiatives for Innovation

65           Figure 2.16: Average Percentage of PRO Initiative Budget Outsourced, by Company

66           Figure 2.17: Average Percentage of PRO Initiative Budget Outsourced, by Therapeutic Area

67           Figure 2.18: Average Percentage of PRO Initiative Budget Outsourced, by Company: CNS

68           Figure 2.19: Average Percentage of PRO Initiative Budget Outsourced, by Company: Oncology

68           Figure 2.20: Average Percentage of PRO Initiative Budget Outsourced, by Company: Respiratory

69           Figure 2.21: Average Percentage of PRO Initiative Budget Outsourced, by Company: Immunology

71           Figure 2.22: Percentage of PRO Initiative Budget Outsourced in Each Stage, by Company Size

71           Figure 2.23: Percentage of PRO Initiative Budget Outsourced in Each Stage, by Company

73           Figure 2.24: Vendor Selection by PRO Initiative Stage: All Companies

74           Figure 2.25: Vendor Selection by PRO Initiative Stage: Top 10 Pharma

75           Figure 2.26: Vendor Selection by PRO Initiative Stage: Top 50 Pharma

75           Figure 2.27: Vendor Selection by PRO Initiative Stage: Small Pharma

76           Selecting a PRO Instrument to Achieve PRO-Driven Goals

77           Using Standard PRO Instruments to Drive Patient-Centered Outcomes Initiatives

79           Figure 3.1: Short Form 36 Version 2 Measurement Model

82           Figure 3.2: Ratings of PRO Effectiveness in Supporting Quality of Life Endpoints: All Companies

83           Figure 3.3: Ratings of PRO Effectiveness in Supporting Quality of Life Endpoints: Top 10 Pharma

84           Figure 3.4: Ratings of PRO Effectiveness in Supporting Quality of Life Endpoints: Top 50 Pharma

84           Figure 3.5: Ratings of PRO Effectiveness in Supporting Quality of Life Endpoints: Small Pharma

86           Figure 3.6: Ratings of PRO Effectiveness in Supporting Cost-Effectiveness Endpoints: All Companies

86           Figure 3.7: Ratings of PRO Effectiveness in Supporting Cost-Effectiveness Endpoints: Top 10 Pharma

87           Figure 3.8: Ratings of PRO Effectiveness in Supporting Cost-Effectiveness Endpoints: Top 50 Pharma

87           Figure 3.9: Ratings of PRO Effectiveness in Supporting Cost-Effectiveness Endpoints: Small Pharma

88           Figure 3.10: Percentage of Companies Implementing PROs, by Therapeutic Area

90           Figure 3.11: Ratings of PRO Effectiveness in Achieving Study Goals: All Therapeutic Areas

91            Figure 3.12: Ratings of PRO Effectiveness in Achieving Study Goals: CNS

92           Figure 3.13: Percentage of Trials Using Specific PRO Measurements: CNS

93           Figure 3.14: Ratings of PRO Effectiveness in Achieving Study Goals: Respiratory

94           Figure 3.15: Percentage of Trials Using Specific PRO Measurements: Respiratory

95           Figure 3.16: Ratings of PRO Effectiveness in Achieving Study Goals: Immunology

96           Figure 3.17: Percentage of Trials Using Specific PRO Measurements: Immunology

97           Figure 3.18: Ratings of PRO Effectiveness in Achieving Study Goals: Oncology

98           Figure 3.19: Percentage of Trials Using Specific PRO Measurements: Oncology

99           Choose Innovative PRO Modalities to Improve Data Quality

100        PRO Delivery Mechanisms

102        Figure 4.1: Percentage of Companies Using PRO Modalities, by Modality Type and Company Size

103        Figure 4.2: Percentage of Companies Using Specific Additional PRO Delivery Mechanisms

104        Figure 4.3: Companies Using ePRO Technology

104        Driving PRO Efficiency via Digital Technology

106        Figure 4.4: Benefits of Adopting ePRO Technology

108        Figure 4.5: Number of Years Since ePRO Implementation

109        Figure 4.6: Timeline of Planned ePRO Implementation

110        Figure 4.7: Breakdown of ePRO Initiative Costs

111        Leveraging the Patient Voice to Drive Regulatory and Reimbursement Decisions

112        Harnessing PRO Evidence to Influence Reimbursement Discussions with Regulators

113        Figure 5.1: Ratings of PRO Effectiveness in Discussions with Regulatory Bodies, by Region

116        Figure 5.2: Average Percentage of PRO Endpoints on Final Product Labels

116        Gaining Approval for PRO-Based Claims in Final Product Marketing

118        Figure 5.3: Average Percentage of PRO Endpoints on Final Product Label, by Therapeutic Area

120        Figure 5.4: Average Percentage of PRO Endpoints on Final Product Label, by Company: Pain

120        Figure 5.5: Average Percentage of PRO Endpoints on Final Product Label, by Company: Oncology

121        Figure 5.6: Average Percentage of PRO Endpoints on Final Product Label, by Company: Respiratory

121        Figure 5.7: Average Percentage of PRO Endpoints on Final Product Label, by Company: Immunology

122        Figure 5.8: Average Percentage of PRO Endpoints on Final Product Label, by Company: Infectious Diseases

122        Figure 5.9: Average Percentage of PRO Endpoints on Final Product Label, by Company: Pediatrics

123        Figure 5.10: Average Percentage of PRO Endpoints on Final Product Label, by Company: CNS

124        The Future of Patient-Reported Outcomes: Evolving Consortia and Regulatory Guidelines