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 4 Progression trend of the ‘Physical functioning’ concept since starting study treatment up to Week 25 and its correspondence with quantitative assessments

Patient #	Qualitative assessment	Quantitative assessment
	Physical functioning	Overall response by IERC per RECIST^a	Change in FACT-M score related to Physical functioning
	Physical functioning	Overall response by IERC per RECIST^a	Physical Well-being	Functional Well-being
1	Improvement	Partial/complete responder	No change	Improvement
2	Improvement	Partial/complete responder	Worsening	Worsening
3	Improvement	Partial/complete responder	No change	Worsening
4	Improvement	Partial/complete responder	Improvement	Improvement
5	No change (no impact)	Partial/complete responder	No change	Improvement
6	No change (no impact)	Partial/complete responder	No change	Improvement
7	No change (no impact)	Partial/complete responder	Improvement	Improvement
8	Improvement	Partial/complete responder	NC	NC
9	No change (no impact)	Progressive disease	Worsening	Improvement

NC Not computed, due to missing baseline data, IERC Independent Endpoint Review Committee; RECIST: Response Evaluation Criteria In Solid Tumors version 1.1
^aOverall response by IERC per RECIST identical at both Week 13 and Week 25 for all patients

ISSN: 1750-1172