Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2021 Mar;71(3):561-568.
doi: 10.1007/s12020-021-02618-z. Epub 2021 Feb 3.

Patients with rare endocrine conditions have corresponding views on unmet needs in clinical research

Johan P de Graaf #  1 , Friso de Vries #  2 , Anne Dirkson  3 , Olaf Hiort  4 , Alberto M Pereira  5 , Márta Korbonits  6 , Martine Cools  7 ; Research and Science Work Package of Endo-ERN
Affiliations

Patients with rare endocrine conditions have corresponding views on unmet needs in clinical research

Johan P de Graaf et al. Endocrine. 2021 Mar.

Abstract

Purpose: European Reference Network on Rare Endocrine Conditions' (Endo-ERN) mission is to reduce and ultimately abolish inequalities in care for patients with rare endocrine conditions in Europe. This study assesses which themes related to rare endocrine conditions are prioritized by patients for clinical research.

Methods: A survey was developed, translated into 22 different European languages, and distributed to patients with rare endocrine conditions. Patients were asked to give priority scores to listed prespecified topics: fertility, heritability, tiredness, daily medicine intake, sleep quality, physical discomfort, and ability to work, partake in social life, and sports. They were also asked to suggest further important areas for research in open fields.

Results: After data cleaning, 1378 survey responses were analyzed. Most responses were received from Northern (47%) and Western Europeans (39%), while Southern (11%) and Eastern Europe (2%) were underrepresented. Respondents were most interested in research concerning ability to participate in social life and work. Patients suggested key areas to work: long-term side effects of medical treatments and quality of life. Some priorities differed between disease groups, both for prespecified and open topics and reflected aspects of patients' individual conditions.

Conclusions: With this large survey, Endo-ERN gained insight into patients' unmet needs in scientific research. Patients prioritized research on ability to work and participation in social activities, though needs differ between the disease groups. Clinical experts should incorporate the results of this survey into the design of future studies on rare endocrine conditions. We aim to utilize these results in designing patient-reported outcome measures for the disease areas covered by Endo-ERN.

Keywords: Endo-ERN; Endocrinology; Patient perspective; Rare disease; Research.

PubMed Disclaimer

Conflict of interest statement

The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1
Absolute number of responses per country
Fig. 2
Fig. 2
Mean priorities with standard deviations as rated by respondents from 1 to 3 in order of prioritization, 1 being the most important priority
Fig. 3
Fig. 3
Percentage of all respondents that listed source as a used source for scientific information on their disease. For Paediatrician, only patients younger than 18 years were analyzed. Paediatrician included paediatric endocrinologist
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Azzopardi-Muscat N, Brand H. Will European Reference Networks herald a new era of care for patients with rare and complex diseases? Eur. J. Public Health. 2015;25(3):362–363. doi: 10.1093/eurpub/cku144. - DOI - PubMed
    1. F. de Vries, M. Bruin, A. Cersosimo, C. N. van Beuzekom, S. F. Ahmed, R. P. Peeters, N. R. Biermasz, O. Hiort, A. M. Pereira. An overview of clinical activities in Endo-ERN: the need for alignment of future network criteria. Eur J Endocrinol. 183(2), 141–148 (2020) - PubMed
    1. Endo-ERN. Overview of specific expertise (2020), https://endo-ern.eu/activities/research-activities/
    1. Endo-ERN. Research activities (2020), https://endo-ern.eu/activities/research-activities/
    1. UNSD. UNSD methodology—geographic regions (2020), https://unstats.un.org/unsd/methodology/m49/

Publication types

LinkOut - more resources