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 3 Progression trend of the ‘Overall change in patients’ perception of their cancer’ concept since starting study treatment up to Week 25 and its correspondence with overall response by IERC per RECIST

Patient #	Overall response by IERC per RECIST^a	Overall change in patients’ perception of their cancer
1	Partial/complete responder	Improvement
2	Partial/complete responder	Improvement
3	Partial/complete responder	Improvement
4	Partial/complete responder	Improvement
5	Partial/complete responder	Improvement
6	Partial/complete responder	Improvement
7	Partial/complete responder	Improvement
8	Partial/complete responder	Improvement
9	Progressive disease	No change (still worsened)

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