|- candidate number||24358|
|- NTR Number||NTR5860|
|- ISRCTN||ISRCTN no longer applicable|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||17-mei-2016|
|- Secondary IDs||MEC-2015-265 |
|- Public Title||The development of a medication-related patient-reported experience measure to better inform reimbursement decision making and clinical practice|
|- Scientific Title||The development of a medication-related patient-reported experience measure to better inform reimbursement decision making and clinical practice|
|- ACRONYM||M-PREM study|
|- Healt Condition(s) or Problem(s) studied||Drug trials, Patient education|
|- 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|
|- mec approval received||no|
|- multicenter trial||no|
|- planned startdate ||1-nov-2014|
|- planned closingdate||1-nov-2016|
|- Target number of participants||150|
|- Primary outcome||Perceived effectiveness|
Ease of use
|- Secondary outcome||n/a|
|- Timepoints||New cases: Baseline (start of treatment), 3 and Prevalent cases: 3-monthly
|- Trial web site|
|- CONTACT FOR PUBLIC QUERIES|| Merel Kimman|
|- CONTACT for SCIENTIFIC QUERIES|| Merel Kimman|
|- Sponsor/Initiator ||Maastricht University Medical Center (MUMC+)|
(Source(s) of Monetary or Material Support)
|Dutch Lung Foundation, ZON-MW, The Netherlands Organization for Health Research and Development|
|- Brief 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 patientís 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.
|- Main changes (audit trail)|
|- RECORD||17-mei-2016 - 2-jul-2016|