Mitochondrial disease patient motivations and barriers to participate in clinical trials

Autor: Marni J. Falk, Xiaoyan Wang, Elizabeth M. McCormick, Amy Holberts, Zarazuela Zolkipli-Cunningham, Rui Xiao, Lauren Williams, John L.P. Thompson, Shana E. McCormack, Amy Stoddart, Natalie Burrill
Jazyk: angličtina
Rok vydání: 2018
Předmět:
Male
0301 basic medicine
Research design
Mitochondrial Diseases
Developmental Disabilities
lcsh:Medicine
Disease
Surveys
Severity of Illness Index
Cohort Studies
Families
0302 clinical medicine
Phlebotomy
Informed consent
Surveys and Questionnaires
Medicine and Health Sciences
Medicine
Child
lcsh:Science
Children
Clinical Trials as Topic
education.field_of_study
Informed Consent
Muscle Weakness
Multidisciplinary
Middle Aged
3. Good health
Research Design
Child
Preschool
Cohort
Female
Symptom Assessment
Attitude to Health
Research Article
Cohort study
Adult
medicine.medical_specialty
Drug Research and Development
Adolescent
Clinical Research Design
Population
Patient Advocacy
Research and Analysis Methods
Young Adult
03 medical and health sciences
Adults
Humans
Clinical Trials
education
Aged
Pharmacology
Clinical Genetics
Motivation
Survey Research
Epilepsy
business.industry
lcsh:R
Infant
Patient Acceptance of Health Care
Health Care
Clinical trial
030104 developmental biology
Clinical research
Age Groups
People and Places
Physical therapy
Population Groupings
lcsh:Q
Clinical Medicine
Health Expenditures
business
030217 neurology & neurosurgery
Zdroj: PLoS ONE, Vol 13, Iss 5, p e0197513 (2018)
PLoS ONE
ISSN: 1932-6203
Popis: Background Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD. Methods A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design. Results PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity. Conclusions These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions.
Databáze: OpenAIRE
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