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 1 Sample of FACT-M items/scores corresponding to the selected qualitative concepts

Qualitative concept	Corresponding FACT-M item/score
Physical functioning	Physical Well-being score
Physical functioning	Functional Well-being score
Pain	GP4. I have pain
	M1. I have pain at my melanoma site or surgical site
	M5. I have aches and pain in my bones
	M13. Movement of my swollen area is painful
Fatigue	HI7. I feel fatigued

ISSN: 1750-1172