Case Study
The patient journey in rare disease, today and tomorrow
- Conditions: Rare Diseases • Myopathy
- Countries: France • Canada
- Timeline: 3 to 4 weeks
Key Deliverables
Client
A pharma company with an early asset on gene therapy for rare disease in pediatrics
Key Question
What is the current patient journey, and how should it look like in the future with the introduction of gene therapy?
Our approach
We supported the client and its insight agency with the qualitative research with parents of children affected, to understand potential barriers to future treatment in two countries. We mapped out the patient journey from very early symptoms to treatment, to identify root causes of delay in diagnosis. We explored the parents’ concerns and expectations regarding gene therapy, to understand what would drive their decision-making for their child’s treatment.
Results
We mapped the current patient journey and its gaps across several markets to understand local challenges and identify key points of change in the future.
