Medication Adherence and Non-adherence in Adults With Rare Disease

Withdrawn

• Conditions: Idiopathic Pulmonary Fibrosis; Hemophilia A; Hemophilia B; Myasthenia Gravis; Cystic Fibrosis; Sickle Cell Disease
• Interventions: Other: MAR-Scale

• Registration Number: NCT04541875
• Lead Sponsor: Xperiome

Brief Summary

The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease, and to identify the top patient-reported reasons for non-adherence. Internal reliability of the MAR-Scale will also be assessed in each condition.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-08-26
• Study First Submitted QC: 2020-09-01
• Study First Posted: 2020-09-09
• Study Start: 2021-01
• Status Verified: 2022-05
• Last Update Submitted: 2022-05-07
• Last Update Posted: 2022-05-12
• Primary Completion: 2022-09
• Study Completion: 2022-09

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Have a confirmed (self-reported) diagnosis by a doctor or other licensed healthcare professional of one of the following conditions:
• Cystic fibrosis
• Hemophilia (A or B)
• Idiopathic pulmonary fibrosis
• Myasthenia gravis
• Sickle cell disease
• Have access to the internet
• Are aged 18 years or above
• Are comfortable reading and answering questions in English
• Have an active prescription for at least one medication indicated to treat the relevant condition (self-reported)
• NB: Individuals will be eligible even if the prescription has not been dispensed (filled) yet, and also if they have the medication(s), but are not actually taking it/them Are able and willing to provide consent electronically through the my.raremark.com platform

Exclusion Criteria

• There are no exclusion criteria. Any member of a Raremark community will be able to take part in this study if they meet the inclusion criteria and can provide their informed consent.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cystic fibrosis	MAR-Scale	Patients aged 18+ and diagnosed with cystic fibrosis. Patients will answer the MAR-Scale once every three months for a year.
Myasthenia gravis	MAR-Scale	Patients aged 18+ and diagnosed with myasthenia gravis. Patients will answer the MAR-Scale once every three months for a year.
Hemophilia A or B	MAR-Scale	Patients aged 18+ and diagnosed with hemophilia A or B. Patients will answer the MAR-Scale once every three months for a year.
Idiopathic pulmonary fibrosis	MAR-Scale	Patients aged 18+ and diagnosed with idiopathic pulmonary fibrosis. Patients will answer the MAR-Scale once every three months for a year.
Sickle cell disease	MAR-Scale	Patients aged 18+ and diagnosed with sickle cell disease. Patients will answer the MAR-Scale once every three months for a year.

Primary Outcome Measures

Name	Time	Method
To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.	1 year	Tally of reasons for non-adherence.
To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.	1 year	Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.

Secondary Outcome Measures

Name	Time	Method
To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients.	Baseline	Use the 200 responses collected from each condition to determine the Cronbach's alpha of the scale for each condition.