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Background <p>Countries have implemented different assessment pathways for public funding of drugs for rare diseases, resulting in inequities in access. Understanding public preferences for these processes could enhance decision-making legitimacy and acceptance.</p> Objective <p>We aimed to elicit societal preferences for different assessment pathways that lead to public reimbursement to drugs for rare diseases.</p> Methods <p>A discrete choice experiment was conducted among adults from the general population in Australia who were asked to assume the role of a government advisor and chose between two different assessment pathways. Attributes describing the assessment pathways were identified through a literature review and expert focus groups. Each respondent completed eight choice tasks. Data were analysed using conditional logit, mixed logit and latent class models. Willingness to wait was calculated as the ratio of attribute coefficients to access time.</p> Results <p>There were 1099 respondents who completed the survey. Overall, respondents preferred assessment pathways that incorporated comprehensive evidence and stakeholder input. The mixed logit model revealed preference heterogeneity across four of the five attributes, with the latent class analysis identifying three groups: Class 1 (28.1%) preferred use of all available evidence, Class 3 (20.2%) opposed long wait times, and Class 2 (52%) showed no strong preferences. On average, respondents were willing to wait 32 additional months to incorporate all types of evidence into the decision-making process, and an additional 14 months for broad stakeholder involvement. Acceptable delays indicate the value placed on greater certainty and broader consultation rather than recommended policy waiting times.</p> Conclusions <p>This study examined societal preferences for assessment pathways for rare disease medicines. Robust evidence, covering safety, clinical effectiveness and cost effectiveness, and enhanced stakeholder involvement were the most influential factors. Respondents were also willing to delay patient access to ensure decisions were supported by strong evidence.</p>

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Societal Preferences for Assessment Pathways of Rare Disease Drugs: A Discrete Choice Experiment

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月27日

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  • Constanza Vargas,
  • Richard De Abreu Lourenco,
  • Deborah J. Street,
  • Manuel Espinoza,
  • Stephen Goodall

摘要

Background

Countries have implemented different assessment pathways for public funding of drugs for rare diseases, resulting in inequities in access. Understanding public preferences for these processes could enhance decision-making legitimacy and acceptance.

Objective

We aimed to elicit societal preferences for different assessment pathways that lead to public reimbursement to drugs for rare diseases.

Methods

A discrete choice experiment was conducted among adults from the general population in Australia who were asked to assume the role of a government advisor and chose between two different assessment pathways. Attributes describing the assessment pathways were identified through a literature review and expert focus groups. Each respondent completed eight choice tasks. Data were analysed using conditional logit, mixed logit and latent class models. Willingness to wait was calculated as the ratio of attribute coefficients to access time.

Results

There were 1099 respondents who completed the survey. Overall, respondents preferred assessment pathways that incorporated comprehensive evidence and stakeholder input. The mixed logit model revealed preference heterogeneity across four of the five attributes, with the latent class analysis identifying three groups: Class 1 (28.1%) preferred use of all available evidence, Class 3 (20.2%) opposed long wait times, and Class 2 (52%) showed no strong preferences. On average, respondents were willing to wait 32 additional months to incorporate all types of evidence into the decision-making process, and an additional 14 months for broad stakeholder involvement. Acceptable delays indicate the value placed on greater certainty and broader consultation rather than recommended policy waiting times.

Conclusions

This study examined societal preferences for assessment pathways for rare disease medicines. Robust evidence, covering safety, clinical effectiveness and cost effectiveness, and enhanced stakeholder involvement were the most influential factors. Respondents were also willing to delay patient access to ensure decisions were supported by strong evidence.