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Table 6 Excerpts of patient quotes illustrating the status of the selected concepts

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

Clinical statusa Progression of concepts at Week 13 or Week 25 interviewsb Patients’ quotes extracted from Week 13 or Week 25 interviews
Perceived change in cancerc Physical functioning Fatigue/Energy Pain
Responders (n = 8 patients) Improved “It’s like much, much better. I’m doing very well. I’m doing especially well. Well, it happens to be that one of my tumours is something that I can touch, and I can tell it’s getting smaller, and the CAT scans I’m getting every six weeks are indicating the same thing.” (Week 25) “I’m doing things that I haven’t done in a long time just like being able to go out and walk” (Week 25) “I have more energy... I mean, I had energy when I started, but it was more of a forced energy, now it’s not a... It’s like I forced myself to do things, so that I would keep going. Now I don’t need to force myself […] Sometimes I have some fatigue, but it’s not bad, I mean, it’s just a mild fatigue” (Week 25) “well, I have, I’m still having some pain in my back, but it has lessened since I started the infusions” (Week 13)
Unchanged “not to my knowledge. I’m due for another set of scans next Monday, so…” “I feel fine and continue to do what I can. I mean, I am 74 so, you know, I don’t do, unlike former President Bush, I don’t parachute jumping and stuff like that on my birthday, but, you know, I do not feel limited due to the cancer and what I do in terms of be daily activities” (Week 25) “I’m not that strong, I mean I’m up and about all day and I work for a couple of hours then I come in and lay down and rest for about 30 min and then go out and work some more […] I’m draggy, I wear out quick” (Week 13) “No, no pain whatsoever” (Week 25)
Worsened No patients “I wanted to dance with my wife, and I could get maybe one dance in and then I might have to rest for 45 min before I can dance again, […] I’m down, I can’t probably walk much less, getup and dance” (Week 13)   No patients
Progressive disease (n = 1 patient) Improved No patients No patients N/A No patients
Unchanged No patients “Physically I’m still fairly strong in what I’m doing, and active as much as I can be […] at this point in time noticed any drop off in my physical capabilities.” (Week 13) N/A No patients
Worsened “To the best of my knowledge it has metastasized just slightly, based on the last scan I had. Still located in the abdominal area and esophagus area, pancreas area, all that just generally in that area. I’m seeing a slow worsening at this point in time.” (Week 13) No patients “after I received my infusion I think it was a day or so after, I’m a little fatigued […] not very bad, not that I can’t’ do everything but I’d like to kind of back off that day and take it easy for that day” (Week 25) “I’m seeing a slow worsening at this point in time. A little bit, like I say, a little more abdominal pain […] which I would grade now on a level of one to ten, probably in the 3 range, 4 range, when I have it, it’s not a continuous pain, it’s when I’m in certain positions like laying down in bed on my back, it will bother me.” (Week 13)
  1. N/A not applicable as data is missing for the patient with progressive disease
  2. aClinical status, as defined based on the overall response by Independent Endpoint Review Committee per Response Evaluation Criteria In Solid Tumors version 1.1
  3. bAs categorized during qualitative analysis [35]
  4. Cpatients answered to the question: “Has your cancer changed at all, since you started the study and received the study treatment?” during the follow-up interview