Appendix B

INTERVIEW GUIDE: ECULIZUMAB

Characteristics of the interview

Face-to-face and individual interview

Location convenient for participant (e.g. home, hospital, public area, cafe)

Direct approach: participant informed about the purpose of the interview

Semi-structured interviews: main questions with potential sub-questions

Duration of 1 hour (maximum)

Digitally recorded

Aims of the interview

To inform the development of the PESaM questionnaire, with a focus on disease-specific items, by gaining knowledge and information about:

Patient experiences and perceptions about the efficacy of eculizumab

Patient experiences of side-effects of the medication

Patient experiences with the administration of the medication

The bothersomeness of the side-effects and administration

The impact of the efficacy, side-effects and administration mode on daily life

Satisfaction with the medication

Main questions and potential sub-questions

When where you diagnosed with aHUS? Can you tell me a little bit more about this?

  • Disease symptoms? Disease trajectory?

When did you start witheculizumab?

  • Continuous or with breaks?

Where you informed by your health care professional (specialist nurse or medical specialist) about the expected efficacy of the medication and potential side-effects?

  • What were your expectations after this conversation?

Do you have the feeling/are you experiencing that the medication is effective?

  • How have you noticed?
  • Has your general quality of life improved? How do you notice? Examples?

Many medications will have unwanted side-effects, which may (or may not) be very unpleasant. Have you experienced side-effects of eculizumab?

  • Do these side-effects bother you?
  • How do they impact on your daily life?
  • Do you feel hindered in daily activities by the side-effects?
  • Are you able to cope with the side-effects? How do you do that?
  • Do you balance the side-effects with the effectiveness of the medication?
  • Do you ever skip the medication due to its side-effects? Do you consider to stop with the medication?

Are there side-effects, or aspects of side-effects, we haven’t discussed yet?

Different aspects of the medication can lead to satisfaction or dissatisfaction. These aspects differ for each individual. To what extent are you happy with and how important are the following aspects of eculizumab?

  • Doses, administration mode, how long (perhaps lifelong) you need to take the medication

Looking back, what is the most difficult about using eculizumab?

What are you most satisfied about?

Does eculizumab live up to your expectations?

To what extent do you balance the effectiveness with the side-effects and ease of use?

Are there any issues that we haven’t discussed that you would like to tell me about?

How satisfied, on a scale from 1 to 10, are you with eculizumab? Please take all advantages and disadvantages into account.

Do you have any final questions?