+ Site Statistics
+ Search Articles
+ PDF Full Text Service
How our service works
Request PDF Full Text
+ Follow Us
Follow on Facebook
Follow on Twitter
Follow on LinkedIn
+ Subscribe to Site Feeds
Most Shared
PDF Full Text
+ Translate
+ Recently Requested

A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers for improving the evidence and how can they be overcome?



A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers for improving the evidence and how can they be overcome?



Trials 18(1): 556



Evidence-based clinical practice is challenging in all fields, but poses special barriers in the field of rare diseases. The present paper summarises the main barriers faced by clinical research in rare diseases, and highlights opportunities for improvement. Systematic literature searches without meta-analyses and internal European Clinical Research Infrastructure Network (ECRIN) communications during face-to-face meetings and telephone conferences from 2013 to 2017 within the context of the ECRIN Integrating Activity (ECRIN-IA) project. Barriers specific to rare diseases comprise the difficulty to recruit participants because of rarity, scattering of patients, limited knowledge on natural history of diseases, difficulties to achieve accurate diagnosis and identify patients in health information systems, and difficulties choosing clinically relevant outcomes. Evidence-based clinical practice for rare diseases should start by collecting clinical data in databases and registries; defining measurable patient-centred outcomes; and selecting appropriate study designs adapted to small study populations. Rare diseases constitute one of the most paradigmatic fields in which multi-stakeholder engagement, especially from patients, is needed for success. Clinical research infrastructures and expertise networks offer opportunities for establishing evidence-based clinical practice within rare diseases.

Please choose payment method:






(PDF emailed within 0-6 h: $19.90)

Accession: 059420978

Download citation: RISBibTeXText

PMID: 29166947

DOI: 10.1186/s13063-017-2287-7


Related references

Improving evidence based practice in postgraduate nursing programs: A systematic review: Bridging the evidence practice gap (BRIDGE project). Nurse Education Today 63: 69-75, 2018

Rapid Evidence Assessment of the Literature (REAL(©)): streamlining the systematic review process and creating utility for evidence-based health care. Bmc Research Notes 8: 631, 2016

Current evidence on evidence-based practice training in allied health: a systematic review of the literature. International Journal of Evidence-Based Healthcare 10(4): 347-360, 2013

Implementing the new essential evidence-based practice competencies in real-world clinical and academic settings: moving from evidence to action in improving healthcare quality and patient outcomes. Worldviews on Evidence-Based Nursing 12(2): 67-69, 2016

The effect of evidence-based, treatment-oriented, clinical practice guidelines on improving patient care outcomes: a systematic review protocol. Jbi Database of Systematic Reviews and Implementation Reports 14(6): 42-51, 2016

Physician-reported barriers to using evidence-based recommendations for low back pain in clinical practice: a systematic review and synthesis of qualitative studies using the Theoretical Domains Framework. Implementation Science 14(1): 49, 2019

Barriers to evidence based practice in primary care: a review of the literature. International Journal of Nursing Studies 41(4): 369-378, 2004

Evidence-based practice within nutrition: what are the barriers for improving the evidence and how can they be dealt with?. Trials 18(1): 425, 2018

Evidence-based practice in physiotherapy: a systematic review of barriers, enablers and interventions. PhysioTherapy 100(3): 208-219, 2015

Barriers to using electronic evidence based literature in nursing practice: a systematised review. Health Information and Libraries Journal 2017, 2017

Barriers Associated With Evidence-Based Practice Among Nurses in Low- and Middle-Income Countries: A Systematic Review. Worldviews on Evidence-Based Nursing 2019, 2019

Improving evidence based practice in postgraduate nursing programs: A systematic review. Nurse Education Today 63: 69-75, 2018

Interventions to Reduce Perceived Stress Among Graduate Students: A Systematic Review With Implications for Evidence-Based Practice. Worldviews on Evidence-Based Nursing 14(6): 507-513, 2017

We should overcome the barriers to evidence-based clinical diagnosis!. Journal of Clinical Epidemiology 60(3): 217-227, 2007

Evidence-based practice in chiropractic practice: A survey of chiropractors' knowledge, skills, use of research literature and barriers to the use of research evidence. Complementary Therapies in Medicine 22(2): 286-295, 2015