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  4. > Orphan Drug Market Dynamics and Patient Recruitment Best Practices

Orphan Drug Market Dynamics and Patient Recruitment Best Practices

  • February 2015
  • -
  • Industry Standard Research
  • -
  • 68 pages

Report Overview

The recruitment of patients for orphan drug trials adds levels of complexity to the already difficult process of patient recruitment. ISR sought the opinions of individuals with extensive experience with orphan drug trial recruitment in order to understand the most and least effective practices in orphan drug trial patient recruitment, how orphan drug trials are organized, the regulatory guidelines surrounding orphan drug trial recruitment, and ultimately what to expect for the future in terms of orphan drug trials.
ISR also analyzes key trial metrics, such as number and phase of orphan drug trials, outsourcing trends, study type, source of funding, projected vs. actual trial lengths, and enrollment figures for ongoing orphan drug/rare disease trials to provide a comprehensive look at the orphan drug market.

Report Scope

What you will learn in this report:

- The most and least effective practices in orphan drug trial patient recruitment
- How orphan drug trials are typically organized within a company’s structure
- The regulatory guidelines surrounding orphan drug trial recruitment
- What to expect for the future in terms of orphan drug trials
- How individual companies perceive and handle orphan drug trials (number of
orphan drug trials, outsourcing trends, deadlines met, etc.)
- Current orphan drug trial metrics, such as the phase, source of funding, study type, length of trial, projected vs. actual trial lengths, and enrollment figures
- Best practices for:
- Marketing CRO orphan drug services
- Direct recruiting practices
- Involving Key Opinion Leaders
- Patient advocacy groups and social media
- Sponsor methods for CRO selection

How you can use this report:

-Anticipate potential problems in orphan disease patient recruitment in order to
reduce trial delays
- Understand regulatory guidelines for orphan drug trials and the difficulties these
guidelines pose
- Benchmark your company’s orphan drug trial recruiting structure to those of other
companies outlined in the report
- Understand the bigger picture of the orphan drug recruitment process and what to
expect in the future




Table Of Contents

Orphan Drug Market Dynamics and Patient Recruitment Best Practices
Table of Contents

Copyright and Usage Guidelines 5
Methodology 6
Telephone Interviews 6
Interviewee Titles 6
Web-based Surveys 6
Secondary Sources 7
Introduction 8
Executive Summary 9
Sponsor Interest in Orphan Drug Compounds 11
Change in Orphan Drug Compound Interest Over Time 12
Future Interest in Orphan Drug Compounds 12
Organizational Structures 13
Therapeutic Focus 13
Trial-Specific Teams 14
Outsourcing Behavior 15
In-house vs In-licensed Compounds 17
Percent of Orphan Drug Trials Outsourced to CROs 18
Use of In-house Resources 19
Orphan Drug Sponsor Needs 20
Preference for Orphan Drug Experience vs Other Attributes 21
Preference for Site ID Capability vs Patient Recruitment Capability 22
Implications for Sponsors 23
Implications for Service Providers 23
Case Study: Patient Recruitment Specialists 24
Siren Interactive - Recruitment Practices 25
Siren Interactive Recruitment Work Flow 26
Regulatory Influence 28
Open Dialogue with Regulatory Bodies 29
Fair Recruitment Expectations 29
Protocol Development 29
Impact of Key Opinion Leaders 30
Value of KOLs 31
Difficulties with KOLs 31
How to Best Utilize KOLs 32
Alternative Tools for Patient Recruitment 33
Patient Advocacy Groups 34
Social Media 35
Difficulties Surrounding Social Media 35
How to Best Use Social Media 36
Enrollment Best Practices 37
Recruitment and Enrollment Methods 38
Best Practices and Recommendations 40
Marketing CRO Orphan Drug Services 41
Direct Recruiting Practices 41
Key Opinion Leaders 41
Patient Advocacy Groups and Social Media 41
Study Data 42
Success of Orphan Drug Patient Recruitment Company Type 43
Projected Use of Patient Recruitment by Company Type 45
Company Interest in Orphan Drug Compounds 46
Change in Orphan Drug Compound Interest 46
Projected Interest in Orphan Drug Compounds 47
In-house vs In-licensed Compounds 48
Percent of Orphan Drug Studies Outsourced to CROs 49
Preference for Orphan Drug Experience vs Other Attributes 50
Use of In-house Resources 51
Preference for Site ID Capability vs Patient Recruitment Capability 52
Average Percent of Trials Meeting Recruitment Timelines 53
Average Percent of Trials Meeting Original Recruitment Goals 53
Demographics 54
Type of Company 54
Job Title 54
Corporate Headquarters and Office Location 55
Therapeutic Expertise 56
Involvement in Orphan Drug Trials 57
Current Orphan Drug Clinical Trial Figures 58
Number of Trials by Phase 59
Study Type Distribution 59
Number of Interventional Trials by Phase 60
Number of Observational Trials by Phase 60
Number of Expanded Access Trials by Phase 61
Study Type Distribution Funded by Industry Only 61
Number of Industry Funded Interventional Studies by Phase 62
Number of Industry Funded Observational Trials by Phase 62
Number of Industry Funded Expanded Access Trials by Phase 63
Average Trial Length by Phase 63
Average Interventional Trial Length by Phase 64
Average Observational Trial Length by Phase 64
Average Targeted Enrollment by Phase 65
Average Targeted Interventional Enrollment by Phase 65
Average Targeted Observational Enrollment by Phase 66
Average Targeted Industry Funded Interventional Enrollment by Phase 66
Average Targeted Industry Funded Observational Enrollment by Phase 67
About Industry Standard Research 68

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