Launching a pediatric rare disease drug in China

Child's ear
Kantar implemented a mixed-method research approach to maximise uptake and patient benefit for a critical metabolic disease medicine in China.


A world leader in biotech for rare disease launched a drug in China for the treatment for a rare genetic metabolic disease. While the company has been engaging key opinion leaders (KOLs) in Tier 1 cities, its understanding of physician diagnosis and treatment patterns in Tier 2 and 3 cities was limited. In order to maximise penetration across all regions, the company wanted to identify potential barriers across the patient journey and address any unmet needs, as well as better understand the market landscape in the regions where they had little or no presence.

Considering that many challenges exist for conducting research in pediatric rare diseases, greater efforts are needed to secure the caregiver’s perspective in research. Additionally, with the company not possessing a list of KOLs to be engaged in Tier 2 and 3 regions, a database needed to be generated for recruitment. Disease prevalence and reimbursement policy could potentially vary across provinces – further complicating matters.


Kantar implemented an approach that combined qualitative and quantitative research. Using a combination of desk research and in-depth interviews, we identified the KOLs. We also conducted interviews to identify the barriers across the patient journey and to gather insights around obtaining information in the regions with little or no coverage.

In the first phase of our work we employed a mix of online research – including hospital websites, forums and publications – telephone research, and snowball sampling to build out the recruitment database. A rigorous methodology was performed to ensure data quality.


In the second phase, which entailed in-depth interviews, we conducted face-to-face discussions with KOLs across 11 Chinese provinces to gain a holistic view of the diagnosis and treatment landscape for the rare disease. Additionally, we gathered caregivers’ testimonies that offered a perspective on the rare disease and the current treatment, as well as key insights on opportunities to improve care across the patient journey.


This client engagement was a complete success. We identified multiple opportunities across the patient journey for our client to pursue, including: a valuable summary of the current reimbursement status across provinces that will benefit the client’s discussion with payers and policy makers; shared examples of patient access programmes sponsored by government bodies and companies; and key insights on the roles of healthcare professionals in the treatment of this disease, which can be used to empower patients and achieve better health outcomes.

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