Medication Adherence And Non-adherence In Adults With Rare Disease

Study Overview

Medication Adherence and Non-adherence in Adults With Rare Disease

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.

N/A

Cystic Fibrosis
Hemophilia A
Hemophilia B
Idiopathic Pulmonary Fibrosis
Myasthenia Gravis
Sickle Cell Disease

OTHER: MAR-Scale

RM-RP005

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates	Results Reporting Dates	Study Record Updates
First Submitted 2020-08-26	Results First Submitted N/A	Last Update Submitted that met QC Criteria 2022-05-07
First Submitted that Met QC Criteria 2020-09-01	Results First Submitted that Met QC Criteria N/A	Last Update Posted (ACTUAL) 2022-05-12
First Posted (ACTUAL) 2020-09-09	Results First Posted N/A	Last Verified 2022-05

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

Primary Purpose:
N/A

Allocation:
N/A

Interventional Model:
N/A

Masking:
N/A

Arms and Interventions

Participant Group/Arm	Intervention/Treatment
: Cystic fibrosis Patients aged 18+ and diagnosed with cystic fibrosis. Patients will answer the MAR-Scale once every three months for a year.	OTHER: MAR-Scale The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.
: Hemophilia A or B Patients aged 18+ and diagnosed with hemophilia A or B. Patients will answer the MAR-Scale once every three months for a year.	OTHER: MAR-Scale The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.
: Idiopathic pulmonary fibrosis Patients aged 18+ and diagnosed with idiopathic pulmonary fibrosis. Patients will answer the MAR-Scale once every three months for a year.	OTHER: MAR-Scale The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.
: Myasthenia gravis Patients aged 18+ and diagnosed with myasthenia gravis. Patients will answer the MAR-Scale once every three months for a year.	OTHER: MAR-Scale The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.
: Sickle cell disease Patients aged 18+ and diagnosed with sickle cell disease. Patients will answer the MAR-Scale once every three months for a year.	OTHER: MAR-Scale The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.

Primary Outcome Measures	Measure Description	Time Frame
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
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
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
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
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
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
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
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
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
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 Outcome Measures	Measure Description	Time Frame
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

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person’s general health condition or prior treatments.

Ages Eligible for Study:
ALL

Sexes Eligible for Study:
18 Years

Accepts Healthy Volunteers:
1

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

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.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

The Touro College and University System

Investigators

PRINCIPAL_INVESTIGATOR: Elizabeth J Unni, BpharmMBAPhD, Touro College of Pharmacy

Publications

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

No publications available

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Resources

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Clinical Trial Record