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ID
Source
Brief title
Health condition
To develop and test a measure aimed to better understand patient experiences with (expensive) medications.
Sponsors and support
ZonMW
Intervention
Outcome measures
Primary outcome
Perceived effectiveness
Perceived side-effects
Ease of use
Satisfaction
Secondary outcome
n/a
Background summary
Background: Medications for rare diseases are often expensive and effectiveness can be uncertain due to small patient populations in clinical trials. This undermines value for money and increases the uncertainty facing the policy maker when considering such medications for reimbursement. The patient’s evaluation of the effectiveness and acceptability of a therapy is not explicitly incorporated into the reimbursement decision making process. Yet, this evaluation may influence a range of outcomes such as uptake of, compliance with, and ultimately effectiveness of the medication. Hence, evaluation of patient experiences can promote efficient use of medications.
Aim: To develop a patient-reported outcome measure to better understand experiences and satisfaction with medications.
Methods: Two expensive medications that are under consideration for reimbursement in the Netherlands were used as case studies; 1) pirfenidone (esbriet) for patients with idiopathic pulmonary fibrosis, and 2) eculizumab (soliris) for patients with atypical haemolytic uraemic syndrome.
Recognised methodology was used to develop the PESaM-questionnaire involving literature review, individual patient interviews (n=5), two focus groups (n=13), expert opinion (n=4), pre-testing of face and content validity using retrospective verbal probing (n=13), and evaluation of psychometric properties (n=175).
Results: Three measures were developed; a generic measure that can be used for any medication (16 items), a measure for pirfenidone (10 items) and one for eculizumab (12 items). The measures cover the following domains: perceived effectiveness, perceived side-effect, ease of use and satisfaction. Face and content validity of the three measures were considered good. Psychometric properties are under evaluation.
Conclusion: We developed three measures to assess patient experiences with medication in general and with pirfenidone and eculizumab specifically. In future these tools could be used to 1) inform reimbursement decision making, and 2) assist clinician and patient when making treatment choices.
Study design
New cases: Baseline (start of treatment), 3 and Prevalent cases: 3-monthly
Intervention
N/A
Inclusion criteria
IPF patients who 1) are prescribed Pirfenidone (current and new users), 2) can read and speak Dutch, and 3) are willing to participate.
aHUS patients who 1) are prescribed Eculizumab (current and new users), 2) can read and speak Dutch, and 3) are willing to participate.
Exclusion criteria
N/a
Design
Recruitment
Followed up by the following (possibly more current) registration
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Other (possibly less up-to-date) registrations in this register
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In other registers
| Register | ID |
|---|---|
| NTR-new | NL5707 |
| NTR-old | NTR5860 |
| Other | : MEC-2015-265 |