Patient Journey Study in Rare Neuromuscular Disease
A global pharmaceutical company developing a new treatment for a rare, intractable neuromuscular condition sought to understand current challenges and unmet needs from physician, patient, and caregiver perspectives. A key goal was to explore conditions for an ‘acceptable treatment’ and identify barriers to treatment adoption.
Our Work
- Online IDI (60 min) with N=15 physicians (neurologists and pediatric neurologists) — covering diagnosis pathways, treatment decision factors, multidisciplinary collaboration challenges, and Target Product Profile (TPP) evaluation
- Online IDI (60 min) with N=15 patients and caregivers — mapping the patient journey from onset to treatment, exploring daily physical/psychological burdens, and extracting emotional insights around ‘ideal treatment’ and TPP expectations
The Outcome
- Mapped the diagnostic pathway and barriers to specialist referral, revealing why early symptoms often go unrecognized and observation periods become prolonged
- Identified how information timing and explanation at diagnosis influence patient understanding and psychological readiness for treatment
- Clarified treatment selection criteria balancing specialist expertise with hospital accessibility
- Surfaced psychological and daily life challenges including strong preference for oral medication, unmet needs in rehabilitation, and gaps in daily support information
“Thank you so much for your help with this project. It was a very interesting study, and we really enjoyed working together. Should any other research opportunities arise in the future, we look forward to working with you again.”
– Client Voice