Designing for Recruitment and Retention in Rare Disease Studies

Description

An area of clinical study that is gaining momentum is rare disease. ISR acknowledges pharma’s growing interest in this space. While this means opportunity for the drug development industry, the unique nature of rare disease studies is burdensome. Greater challenges are baked into rare disease studies compared to typical studies: very small patient pools, specialized protocols, difficult-to-define endpoints, often very sick patients with limited ability or time, and the predicament of high cost versus low volume. ISR’s Designing for Recruitment and Retention in Rare Disease Studies report takes a deep dive into two areas impacting the success of these studies: recruitment and retention. Given the importance of each rare disease patient, these activities are time-intensive and resource-heavy. We asked 125 respondents involved in these activities about the challenges they face, methods and support services used, and their perspectives and practices regarding patient-friendly alternatives to the clinical setting.

Designing for Recruitment and Retention in Rare Disease Studies

Sponsors:

  • Understand the recruitment and retention challenges facing rare disease studies and how to mitigate them
  • Learn the practices and support services being utilized for rare disease studies, plus perspectives on unconventional methods
  • Explore various patient-friendly alternatives to the clinical setting and their impact on patient retention

Service Providers:

  • Understand the pain points associated with recruitment and retention activities in rare disease studies
  • Gain insight into rare disease study preferences and practices, and use this information to evaluate/market your offerings
  • Learn the specific support services needed by sponsors to best accommodate patients in rare disease studies
  • Baseline Challenges & Perspectives
  • Recruitment Methods
  • Retention Practices
  • Study Data

ISR understands that you’re looking for confidence in your market research. With ISR, you’ll consistently receive

  • Focused Domain Expertise — We’ve operated in pharmaceuticals for over 15 years and because it’s our sole focus, our domain expertise brings value to the work that “generalist” researchers can’t deliver.
  • Genuine Research Expertise — Our market research experience has developed over 20 years in many dynamic industries.  We capture appropriate sample sizes, given the research objectives, and we use appropriately sophisticated statistics to uncover everything that’s real and to give you confidence in your decisions. Read our Six Questions to Ask About Your Market Research to learn more about why our industry expertise sets us apart.
  • Transparency — If you’re like many, you’ve been disappointed more than once by research providers who fail to live up to their promises, providing you with their “professional judgment” in place of sound data; and suspect contacts instead of real decision-makers. We deliver the beliefs, attitudes, and intentions of people who matter – and we’ll prove it by showing you the titles of your respondents.

For additional questions about any of ISR’s reports or custom research services, please contact us at info@ISRreports.com.

For Pharma Project Managers

Baseline Challenges & Perspectives

Primary Section Takeaways
Challenges in Finding Patients
Finding Patients: Reason for Most Challenging
Challenges in Retaining Patients
Retaining Patients: Reason for Most Challenging
Beliefs about Non-Traditional Data Collection

Recruitment Methods

Primary Section Takeaways
Use of Traditional vs Non-traditional
Recruitment Methods
Traditional Recruitment Channels
Non-Traditional Recruitment Channels
Identification of Patients
Use of Patient Advocacy Group
Study Assistance Provided by Patient Advocacy Group

Retention Practices

Primary Section Takeaways
Use of Remote Screening
Remote Screening Methods
Use of Remote Patient Visits
Remote Patient Visits & Retention
Prevalence of Flexible Protocol
Data Collection Location
Impact of Flexible Protocol
Flexible Protocol & Retention
Surrogate Markers
Surrogate Markers & Retention
Fight for Clinical Endpoints versus Settle for Surrogate Measures
Use of In-Home Trial Support
Impact of In-Home Trial Support
In-Home Support & Retention
Helpful In-Home Support Service
In-Home Support & Dropout Rate
Use of Support Services
Source of Support Services
CRO/3rd Party Support Services: Performance Expectations
Respondent Comments on Performance

Study Data

Time Dedicated to Recruitment & Retention Activities
Activities Requiring Most Time
Studies in Therapeutic Areas
Pediatric Patients
Adult Patients
Use of Traditional Recruitment Methods
Use of Non-Traditional Recruitment Methods
Challenges in Finding Patients
Biggest Challenge in Finding Patients
Reason for Most Challenging (Finding Patients)
Challenges in Retaining Patients
Biggest Challenge in Retaining Patients
Reason for Most Challenging (Retaining Patients)
Traditional Recruitment Channels
Most Effective Recruitment Channel – Traditional
Non-Traditional Recruitment Channels
Most Effective Recruitment Channel – Non-Traditional
Remote Patient Visits & Retention
Flexible Protocol & Retention
Study Assistance Provided by Patient Advocacy Group
Advisement by Patient Advocacy Group
Unreasonable Clinical Endpoints
Endpoint Data Submission
Surrogate Markers & Retention
Fight for Clinical Endpoints versus Settle for Surrogate Measures
In-Home Support & Retention
Indication for Most Recent Study
Patient Type, Study Location, and CRO Type

Respondent Demographics

Company Type
R&D Spend
CRO Size
Respondent Location
Job Level
Job Title
Primary Area of Responsibility
Involvement in Recruitment & Retention
Studies in Development Phases

Additional information

License

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