Presentation
Navigating Recruitment Challenges for Rare Patient Populations: Strategic Characterization to Optimize Study Pathways
SessionPoster Session 2
DescriptionRare diseases affect a small segment of the population, making participant recruitment for Human Factors research inherently difficult. The limited number of eligible individuals combined with geographic dispersion, significantly restricts access to suitable participants. Low awareness among healthcare professionals and the public further reduces referrals, while multiple concurrent studies often compete for the same small pool of candidates.
Even when patients are identified, their condition may be poorly managed or too severe to allow participation. These factors make it challenging to meet regulatory expectations, for example how to meet the FDA’s requirement for 15 representative participants per distinct user group, which pose a major barrier to conducting robust Human Factors research in rare disease contexts.
The FDA may allow surrogate participants in validation studies when recruitment of the intended user population is exceptionally difficult or impossible and provided that clinical similarity is clearly demonstrated. Before proposing surrogate users, it is essential to fully understand the intended user population. This includes:
• Identifying how the disease’s clinical manifestations affect usability.
• Recognizing unique traits that influence device interaction.
Alexion and Team Consulting collaborated to conduct user research aimed at understanding the lived experiences of individuals with a rare disease. The goal was to characterize the patient population, explore how the condition affects daily life, and inform future device development. This presentation will share strategies for navigating recruitment challenges in rare disease populations, along with different ways you may want to use the collected data to advance program objectives.
Key discussion points include:
• The value of incorporating an exploratory recruitment phase.
Allocating time upfront for recruiters to build relationships, identify key markets, and determine optimal travel locations proved to be a cost-effective way to mitigate recruitment risks. This phase enabled the team to refine the recruitment strategy and select the most appropriate methodology for engaging participants.
• The practicality of meeting participants where they are.
Although most research activities could have been conducted remotely, doing so would have introduced delays and compromised data quality, particularly for capability assessments. To reduce participant burden and ensure valid results, a flexible, participant-centric approach was adopted: if it was easier for the participant, the team travelled to them.
• The importance of creating a positive participant experience.
Home visits required careful planning to allow time for arrival, introductions, and rapport-building. Many participants were unfamiliar with research settings, and entering their homes with technical equipment could feel intrusive. A respectful and warm approach helped ensure a positive experience, increasing the likelihood of future engagement.
• Respecting participant effort and emotional investment.
Individuals with rare diseases often provided profound insights, but participation could be physically and emotionally demanding. Interviews were sometimes the only activity participants could manage that day. To reduce burden, questionnaires were sent in advance for private completion, sessions were held in participants’ homes, and their duration was kept manageable.
• How to build a strong justification for the use of surrogate patient populations.
To gain FDA endorsement, a surrogate strategy must be supported by a strong justification built around three key elements:
1. Intentionality
Defining why the chosen surrogate population is appropriate. Explaining the selection criteria, the rationale behind the choice, and how the surrogate group reflects the intended users.
2. Similarity
Demonstrating clinical and functional similarities between the surrogate and intended users. Using data from evaluations to compare usability-relevant traits and show that the surrogate group interacts with the device in a comparable way.
3. Recruitment Difficulty
Providing clear evidence of the challenges in recruiting the intended users. Documenting outreach efforts, barriers encountered, and steps taken to engage the target population.
Ultimately, the goal is to show that surrogate use is a last resort, backed by thoughtful planning, clinical justification, and genuine effort to recruit the intended users.
• Fully understanding and characterizing intended patient populations can open the door to more possibilities and submission pathways.
Even when patients are identified, their condition may be poorly managed or too severe to allow participation. These factors make it challenging to meet regulatory expectations, for example how to meet the FDA’s requirement for 15 representative participants per distinct user group, which pose a major barrier to conducting robust Human Factors research in rare disease contexts.
The FDA may allow surrogate participants in validation studies when recruitment of the intended user population is exceptionally difficult or impossible and provided that clinical similarity is clearly demonstrated. Before proposing surrogate users, it is essential to fully understand the intended user population. This includes:
• Identifying how the disease’s clinical manifestations affect usability.
• Recognizing unique traits that influence device interaction.
Alexion and Team Consulting collaborated to conduct user research aimed at understanding the lived experiences of individuals with a rare disease. The goal was to characterize the patient population, explore how the condition affects daily life, and inform future device development. This presentation will share strategies for navigating recruitment challenges in rare disease populations, along with different ways you may want to use the collected data to advance program objectives.
Key discussion points include:
• The value of incorporating an exploratory recruitment phase.
Allocating time upfront for recruiters to build relationships, identify key markets, and determine optimal travel locations proved to be a cost-effective way to mitigate recruitment risks. This phase enabled the team to refine the recruitment strategy and select the most appropriate methodology for engaging participants.
• The practicality of meeting participants where they are.
Although most research activities could have been conducted remotely, doing so would have introduced delays and compromised data quality, particularly for capability assessments. To reduce participant burden and ensure valid results, a flexible, participant-centric approach was adopted: if it was easier for the participant, the team travelled to them.
• The importance of creating a positive participant experience.
Home visits required careful planning to allow time for arrival, introductions, and rapport-building. Many participants were unfamiliar with research settings, and entering their homes with technical equipment could feel intrusive. A respectful and warm approach helped ensure a positive experience, increasing the likelihood of future engagement.
• Respecting participant effort and emotional investment.
Individuals with rare diseases often provided profound insights, but participation could be physically and emotionally demanding. Interviews were sometimes the only activity participants could manage that day. To reduce burden, questionnaires were sent in advance for private completion, sessions were held in participants’ homes, and their duration was kept manageable.
• How to build a strong justification for the use of surrogate patient populations.
To gain FDA endorsement, a surrogate strategy must be supported by a strong justification built around three key elements:
1. Intentionality
Defining why the chosen surrogate population is appropriate. Explaining the selection criteria, the rationale behind the choice, and how the surrogate group reflects the intended users.
2. Similarity
Demonstrating clinical and functional similarities between the surrogate and intended users. Using data from evaluations to compare usability-relevant traits and show that the surrogate group interacts with the device in a comparable way.
3. Recruitment Difficulty
Providing clear evidence of the challenges in recruiting the intended users. Documenting outreach efforts, barriers encountered, and steps taken to engage the target population.
Ultimately, the goal is to show that surrogate use is a last resort, backed by thoughtful planning, clinical justification, and genuine effort to recruit the intended users.
• Fully understanding and characterizing intended patient populations can open the door to more possibilities and submission pathways.
Event Type
Poster Presentation
TimeTuesday, March 244:45pm - 6:15pm EDT
LocationRhinelander Gallery
Medical and Drug Delivery Devices