A framework for applying unfamiliar trial designs in studies of rare diseases.
Explore this paper's citation graph
Summary
These techniques may facilitate research in rare diseases through explaining and providing a framework to assist researchers in identifying the most appropriate design for a given research question.
- Type
- review
- Published
- 2011-10-01
- Cited by
- 103
- References
- 54
- OpenAlex
- https://openalex.org/W1988778209
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:23753174
Keywords
MEDLINE, Medicine, Computer science, Psychology, Biology
References
- Internal pilot studies I: type I error rate of the naive t-test.
- Adaptive design methods in clinical trials – a review
- Clinical Epidemiology: The Essentials
- Designing Clinical Research: An Epidemiologic Approach
- Clinical Epidemiology: How to Do Clinical Practice Research
- Extracorporeal circulation in neonatal respiratory failure: a prospective randomized study.
- Conducting research in individual patients: lessons learnt from two series of N-of-1 trials
- Recombinant DNase in cystic fibrosis: a protocol for targeted introduction through n-of-1 trials. Scottish Cystic Fibrosis Group.
- The importance of pilot studies.
- Amitriptyline to relieve pain in juvenile idiopathic arthritis: a pilot study using Bayesian metaanalysis of multiple N-of-1 clinical trials.
- Users' Guides to the Medical Literature: XXV. Evidence-based medicine: principles for applying the Users' Guides to patient care. Evidence-Based Medicine Working Group.
- Why rare diseases are an important medical and social issue.
- Itraconazole to prevent fungal infections in chronic granulomatous disease.
- Clinical trials and rare diseases.
- Strategy for randomised clinical trials in rare cancers
- Adaptive Designs in Clinical Drug Development—An Executive Summary of the PhRMA Working Group
- Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment.
- Sequential design with boundaries approach in pediatric intervention research reduces sample size.
- Adopting orphan drugs--two dozen years of treating rare diseases.
- Case studies, single-subject research, and N of 1 randomized trials: comparisons and contrasts.
Cited by
- Translating rare-disease therapies into improved care for patients and families: what are the right outcomes, designs, and engagement approaches in health-systems research?
- Bayesian Hypothesis Testing in Two‐Arm Trials with Dichotomous Outcomes
- Tipifarnib and farnesyltransferase inhibitors in the treatment of inflammatory breast cancer: is the story over? A review
- Small sample sizes in clinical trials: a statistician's perspective
- Development and Use of New Therapeutics for Rare Diseases: Views from Patients, Caregivers, and Advocates
- Cognitive-Behavioural Therapy Targeting Fear of Progression in an Interdisciplinary Care Program: A Case Study in Systemic Sclerosis
- Prolonged rupture of membranes and pulmonary hypoplasia in very preterm infants: pathophysiology and guided treatment.
- Methodology of clinical trials for rare diseases.
- Achieving the “triple aim” for inborn errors of metabolism: a review of challenges to outcomes research and presentation of a new practice-based evidence framework
- Patient-centeredness in the design of clinical trials
- Strategies for Postmarketing Surveillance of Drugs for Rare Diseases
- Supraventricular tachycardia treatment efficacy in infants: on further review.
- Innovative research methods for studying treatments for rare diseases: methodological review
- A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov
- A methodological framework for drug development in rare diseases
- Experimental designs for small randomised clinical trials: an algorithm for choice
- Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach; study rationale and protocol
- Psychological well-being in systemic sclerosis: Moving forward in assessment and treatment
- Thymectomy for non-thymomatous myasthenia gravis: a propensity score matched study
- Developing methodology for the creation of clinical practice guidelines for rare diseases: A report from RARE-Bestpractices
Related papers
- ИСПОЛЬЗОВAНИЕ ПОТЕНЦИAЛA СОЦИAЛЬНЫХ ПAРТНЕРОВ В ПОДГОТОВКЕ БУДУЩИХ ПЕДAГОГОВ
- DETERMINING QUALITY REQUIREMENTS AT THE UNIVERSITIES TO IMPROVE THE QUALITY OF EDUCATION
- Genentech taps Affimed’s antibodies
- KOMPLIKASI REGIONAL ANESTESI PADA PEDIATRIK
- Enxerto livre de arteria epigastrica inferior: avaliacao hemodinamica e angiografica pre e pos-operatoria
- Cardiac retransplantation in children.
- Avaliacao hemodinamica sequencial em transplante de coracao
- Thoracoscopic discectomy and fusion in the treatment of spinal deformities