ENGAGING THE CANADIAN PUBLIC ON REIMBURSEMENT DECISION-MAKING FOR DRUGS FOR RARE DISEASES: A NATIONAL ONLINE SURVEY

Purpose:

Funding of drugs for rare diseases (DRDs) require decisions that balance fairness for all individuals within the healthcare system with compassion for affected individuals. Our study objective was to conduct a national online survey to determine the respondents’ perspective, including regional variations, associated with decision-making for DRDs.

 Method:

The survey collected responses from 1,631 Canadians. Respondents were asked to rank at least three and up to five DRD decision-making priorities, out of a total of eight priorities presented in the survey. They were also asked to compare and rate their agreement with four funding scenarios described. The frequency of each priority was ranked independent of where it was ranked in relation to the other priorities. Regression analyses were conducted to measure the association between respondents’ demographics and selected priorities with their agreement level with each scenario.

Result:

Among the survey respondents, Improved Quality of Life and Effective Health Care were the most frequently selected as top priorities. Also, 79.2% of respondents agreed with equal access across Canada, 73.0% agreed with DRD funding if additional expenses are justified, and 50% agreed to pay for DRDs. The responses did not identify significant regional differences.

Our analyses did not reveal any significant associations between demographic characteristics and respondents’ agreement level with any specific funding scenario. Selecting Effective Health Care in the top priorities was positively associated with both prioritizing other programs over programs for rare diseases and funding for DRDs only if justified.  Respondents, who selected National Access as one of the top priorities, were less likely to agree to DRD funding only deemed as cost-effective and were more likely to agree with the scenario to provide equal access to DRDs across Canada.

 Conclusion:

The survey results suggest the level of public support for funding decisions and programs that incorporate assessment of the effectiveness of drugs for improving quality of life, and to promote similar access across Canada. There were no significant relationships between the rating of the scenarios and demographics, including geographic location, identified in our analyses.  The responses anticipate public responses to different policy scenarios and the priorities that underlie them. Decision-makers may find it useful to consider whether and how to incorporate these results into policy decisions and their justification to citizens and patients.