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Eliciting Exploratory Patient Preference Data: A Case Study in a Rare Disease.

Kerrie-Anne Ho1, Anna Pierce2, Meredin Stoltenberg3

  • 1UCB Pharma, 216 Bath Rd, Slough, SL1 3WE, UK. kerrie-anne.ho@ucb.com.

Pharmaceutical Medicine
|December 20, 2023
PubMed
Summary

This study used a novel mixed-methods approach to efficiently gather patient preferences for generalized myasthenia gravis (gMG) treatments. The findings provide valuable insights for rare disease treatment decisions and future research.

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Area of Science:

  • Rare disease research
  • Patient-centered outcomes
  • Health economics and outcomes research

Background:

  • Qualitative and quantitative methods offer complementary insights into patient treatment preferences.
  • Generalized myasthenia gravis (gMG) is a rare disease with specific treatment considerations.

Purpose of the Study:

  • To employ a novel mixed-methods approach to explore patient preferences for generalized myasthenia gravis (gMG) treatments.
  • To generate preliminary insights into patient preferences within a condensed timeline.

Main Methods:

  • A mixed-methods study was conducted over approximately 4 months.
  • Participants completed an online quantitative survey with a risk threshold task.
  • Online focus groups were held to discuss qualitative questions and complete additional quantitative tasks.

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Main Results:

  • The study collected quantitative data on risk tolerance and qualitative data on treatment experiences from 18 participants.
  • Most participants (15/18) consistently chose the same hypothetical treatment across survey and focus group tasks.
  • Focus groups provided context for choices, confirmed attribute relevance, and clarified attribute importance.

Conclusions:

  • Patient preference data can be effectively collected using tailored qualitative and quantitative methods.
  • The novel mixed-methods approach efficiently captured informative patient preference data for exploratory research and decision-making.