Medication Adherence and Non-adherence in Adults With Rare Disease

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

Study Overview

• Status: Withdrawn
• Conditions: Hemophilia A; Hemophilia B; Idiopathic Pulmonary Fibrosis; Myasthenia Gravis; Cystic Fibrosis; Sickle Cell Disease
• Intervention / Treatment: Other: MAR-Scale

• Study Type: Observational

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Anyone who fits the eligibility criteria and gives their consent to take part in this study.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

5

Cohorts and Interventions

Group / Cohort	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Xperiome
• Collaborators: The Touro College and University System
• Investigators: Principal Investigator: Elizabeth J Unni, BpharmMBAPhD, Touro College of Pharmacy

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): January 1, 2021
• Primary Completion (ANTICIPATED): September 1, 2022
• Study Completion (ANTICIPATED): September 1, 2022

Study Registration Dates

• First Submitted: August 26, 2020
• First Submitted That Met QC Criteria: September 1, 2020
• First Posted (ACTUAL): September 9, 2020

Study Record Updates

• Last Update Posted (ACTUAL): May 12, 2022
• Last Update Submitted That Met QC Criteria: May 7, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Keywords: Adherence; Observational; Rare disease; MAR-Scale
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Nervous System Diseases; Respiratory Tract Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Lung Diseases; Neoplasms by Site; Disease Attributes; Hematologic Diseases; Infant, Newborn, Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Neuromuscular Diseases; Neurodegenerative Diseases; Anemia; Nervous System Neoplasms; Blood Coagulation Disorders; Pancreatic Diseases; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Fibrosis; Hemophilia A; Hemophilia B; Pulmonary Fibrosis; Idiopathic Pulmonary Fibrosis; Myasthenia Gravis; Cystic Fibrosis; Anemia, Sickle Cell; Rare Diseases
• Other Study ID Numbers: RM-RP005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No