Using Health Economic Methods to Design Clinical Studies

Project Summary

Our project was trying to find out what are the most important factors to people with scleroderma who might be trying to decide whether to undergo stem-cell transplant, a treatment that offers exciting benefits but with considerable risks. By talking to people with scleroderma, we found that although risks and benefits are important, there are many other considerations in treatment decisions that should be considered.

These included:

  • Burden to patients of cost and distance to the treatment center
  • The way that treatment is delivered
  • The experience of physicians of treating people like them
  • The extent to which multidisciplinary, holistic care would be available to support medical and non-medical needs through the treatment

Project Findings

Our survey confirmed that these factors are important in the decisions people with scleroderma would make when deciding when to undergo stem cell treatment.

The two main trials of stem cell treatment for scleroderma have been hindered by a lack of people wanting to participate. Our results suggest that if trials of the same treatments were designed with patient priorities in mind, the amount of people willing to participate in trials—and the chance of results improving the care of scleroderma patients in the future—would be increased.

This approach of patient involvement in the design of research studies could be used in any disease area for any treatment.


Aguiar, M., Laba, TL., Munro, S. et al. Co-production of randomized clinical trials with patients: a case study in autologous hematopoietic stem cell transplant for patients with scleroderma. Trials 22, 611 (2021).

Aguiar M, Harrison M, Munro S, Burch T, Kaal KJ, Hudson M, Bansback N, LabaT. Designing discrete choice experiments using a patient-oriented approach. Patient 2020.

Burch T. Patient Commentary: Added value and validity to research outcomes through thoughtful multifaceted patient-oriented research. Patient 2020.

This project is part of the Health Economics and Simulation Modelling Cluster.


Mark Harrison, co-PI
Tracey-Lea Laba, co-PI
Tiasha Burch
Nick Bansback
Magda Aguiar
Julia Kaal
Sarah Munro
Jennifer Beckett

Methods Section Blog Post

“Most clinical researchers who come up with their ideas do ask others if it’s a good idea… but the trouble is they ask other clinicians, and not potential patients.”

So, we asked patients. What did they want from their treatments?

Nick Bansback & Mark Harrison on rethinking how we design clinical studies.


In February 2021, Dr Harrison and Dr Bansback presented for The BC AHSN Patient Engagement in Clinical Trials Community of Practice and Clinical Trials BC.

In March 2020, Dr Harrison discussed the research and an overview of findings to date with to the Arthrtitis through their #CRArthritis Interview at the Arthritis Broadcast Network Booth, at the Canadian Rheumatology Society conference.

In October 2019, Tiasha Burch and Mark Harrison presented at the Scleroderma Association of British Columbia’s (SABC) Annual General Meeting.