Medication Adherence and Non-adherence in Adults With Rare Disease

Cystic Fibrosis Clinical Trial

Official title:

A Longitudinal, Digital Study Using the Medication Adherence Reasons Scale (MAR-Scale) to Identify the Reasons for Non-adherence to Medications in Rare Disease

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04541875
• Other study ID #: RM-RP005
• Status: Withdrawn
• Start date: January 2021
• Est. completion date: September 2022

Study information

• Verified date: May 2022
• Source: Xperiome
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: September 2022
• Est. primary completion date: September 2022
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Cystic Fibrosis
• Fibrosis
• Hemophilia A
• Hemophilia B
• Idiopathic Pulmonary Fibrosis
• Myasthenia Gravis
• Pulmonary Fibrosis
• Rare Diseases
• Sickle Cell Disease

Intervention

Other:
• MAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Xperiome	The Touro College and University System

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.	Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.	Baseline
Primary	To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.	Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.	3 months
Primary	To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.	Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.	6 months
Primary	To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.	Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.	9 months
Primary	To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.	Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.	1 year
Primary	To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.	Tally of reasons for non-adherence.	Baseline
Primary	To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.	Tally of reasons for non-adherence.	3 months
Primary	To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.	Tally of reasons for non-adherence.	6 months
Primary	To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.	Tally of reasons for non-adherence.	9 months
Primary	To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.	Tally of reasons for non-adherence.	1 year
Secondary	To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients.	Use the 200 responses collected from each condition to determine the Cronbach's alpha of the scale for each condition.	Baseline