Qualitative research can is valuable for its potential to provide in depth insights into complex issues and is particularly valuable for capturing patient perspectives. It can be used to inform healthcare decision making by assessing treatment acceptability and value, by supplementing quantitative data, and by providing insights into lived experiences.[1] The later is particularly important because small sample sizes, mean other types of data are often not available in rare disease.
Yet despite increasing recognition of its value,[2, 3] qualitative evidence is still not consistently included in rare-disease drug submissions. In Canada, sponsors of reimbursement submissions may submit qualitative evidence to the country’s two HTA agencies – Canada’s Drug Agency / L’Agence des médicaments du Canada (CDA-AMC) and Institut national d’excellence en santé et services sociaux (INESSS). Additionally, these HTAs collect patient group input, which often includes qualitative data.
At ISPOR 2025, Broadstreet team members Lauren Powell, Jessica Dunne, Evelyn Griffin, and Cassandra Debard presented a study that examined the use of patient- or caregiver-centred qualitative research by sponsors and patient groups in Canadian reimbursement reviews of pharmaceuticals for rare disease, with a focus on CDA-AMC, and how guidelines are evolving to amplify patient voices.

A review of 2024 CDA-AMC submissions

The team reviewed 10 CDA-AMC reimbursement reviews issued in 2024 for non-oncology pharmaceuticals for rare disease. We examined the public review documents to identify whether de novo patient- or caregiver-centred qualitative research was submitted by sponsors and/or patient groups, or whether any additional qualitative data were cited. We also reviewed the corresponding INESSS reimbursement reviews. Each qualitative study was categorised for its contribution using a published framework,[1] and the quality was assessed using the Critical Appraisal Skills Programme qualitative studies checklist.

Key findings

Few sponsors submitted qualitative evidence. Only 2 of the 10 included sponsor-led qualitative interviews with patients and caregivers.[4-6] In these cases, the sponsors used the qualitative research to: understand perspectives and provide context (n=2), inform subsequent quantitative exercises (n=2), assess treatment acceptability and subjective value (n=1), reach groups other methods cannot reach (n=1), and contribute to economic model development (n=1).
In all reviews, patient groups provided input, and 8 of the 10 included qualitative data from patients/caregivers such as interview quotes, open-text survey comments, or social-media posts. However, these patient-group submissions lacked methodological details to allow assessment of the quality of these data collection methods, and there was no indication that qualitative analysis methods were applied.
None of the 10 corresponding INESSS reimbursement reviews detailed qualitative research included by the sponsors, and only 4 of the 10 included qualitative data submitted by patient groups.
These results show that patients’ voices are often present but not always methodologically robust or consistently leveraged across the two HTA agencies in Canada.

A changing landscape

Available guidelines and information from CDA-AMC and INESSS on the use of qualitative research and patient involvement in reimbursement submissions were also reviewed. This review highlighted encouraging guideline developments:

In December 2024, CDA-AMC appointed the first-ever patient member to its board of directors.

This year, CDA-AMC:

• Issued a new methods guide that provides some guidance on the use of qualitative research – gathered through formal qualitative studies or systematic reviews – to support elements of value
• Announced their plans to evolve the patient group input processes
• Published a new deliberative framework and launched the inclusion of a person with lived experience presentation in the deliberation committee meeting

Also, enhancing involvement of patient organizations and people with lived experience; and evolving how patient voices are incorporated into drug reimbursement reviews is one of CDA-AMC’s goals for this year.

INESSS appointed a patient-consultation specialist in 2022 and updated its methodological guide to encourage use of qualitative data and patient input (via questionnaires and interviews) to inform evaluations.

These shifts suggest that qualitative research will play a growing role in rare-disease HTA decisions going forward.

Implications

For patient advocacy groups, the study underscores that their contributions are essential – but could have even greater impact with the inclusion of greater methodological detail. For sponsors, the findings highlight that qualitative evidence remains underused, even though it can strengthen submissions. Sponsors should work with patient groups to leverage the valuable qualitative data they collect. Clearer guidance from HTA bodies could help both sponsors and patient groups deliver high-quality qualitative research to capture patient and caregiver voices in rare diseases.

References

1. Szabo SM, Hawkins NS, Germeni E. The extent and quality of qualitative evidence included in health technology assessments: a review of submissions to NICE and CADTH. International Journal of Technology Assessment in Health Care. 2024;40(1):e6
2. Germeni E, Szabo S. Beyond clinical and cost-effectiveness: The contribution of qualitative research to health technology assessment. International Journal of Technology Assessment in Health Care. 2023;39(1):e23
3. Lewin S, Glenton C. Are we entering a new era for qualitative research? Using qualitative evidence to support guidance and guideline development by the World Health Organization. International journal for equity in health. 2018;17(1):126
4. Torfgard K, Gwaltney C, Paty J, Mattsson J, Soni P. Symptoms and daily impacts associated with progressive familial intrahepatic cholestasis and other pediatric cholestatic liver diseases: a qualitative study with patients and caregivers [abstract HP-088]. J Pediatr Gastroenterol Nutr. 2018;66(suppl 2):813-4
5. Marshall J, Skrobanski H, Moore-Ramdin L, Kornalska K, Swinburn P, Bowditch S. Caregivers’ perspectives on the impact of cannabidiol (CBD) treatment for Dravet and Lennox-Gastaut syndromes: a multinational qualitative study. Journal of Child Neurology. 2023;38(6-7):394-406
6. Gwaltney C, Bean S, Venerus M, et al. Development of the patient-and observer-reported PRUCISION instruments to assess pruritus and sleep disturbance in pediatric patients with cholestatic liver diseases. Advances in Therapy. 2022;39(11):5126-43