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

From: 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

Patient # Qualitative assessment Quantitative assessment
Physical functioning Overall response by IERC per RECISTa Change in FACT-M score related to Physical functioning
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
  1. NC Not computed, due to missing baseline data, IERC Independent Endpoint Review Committee; RECIST: Response Evaluation Criteria In Solid Tumors version 1.1
  2. aOverall response by IERC per RECIST identical at both Week 13 and Week 25 for all patients