How to address the challenges of evaluating treatment benefits-risks in rare diseases? A convergent mixed methods approach applied within a Merkel cell carcinoma phase 2 clinical trial

Table 2 Patient characteristics and clinical outcomes

Variable	Population (N = 9)
Age (years)
Mean (Sd.)	70.8 (9.8)
Median	71.0
Min - Max	55.0–85.0
Gender (n)
Male	7
Female	2
Country (n)
Germany	1
USA	8
Overall response by IERC per RECIST at Week 13 / Week 25 (n)
Partial / Complete responder	8 / 8
Stable responder	0 / 0
Progressive disease	1 / 1

Sd standard deviation, USA United States of America, IERC Independent Endpoint Review Committee, RECIST Response Evaluation Criteria In Solid Tumors version 1.1

ISSN: 1750-1172