Innovations in genetics, molecular and computational biology and basic research are rapidly evolving. However, translating this progress into clinic research, clinical trials or commissioned therapies in clinical practice remains challenging.

Some of these challenges relate to difficulties in pinpointing the greatest unmet clinical needs, and may be addressed through a systematic collection of data in the form of longitudinal patient registries. Patient registries can include data on patient demographics, diagnosis, treatment and outcomes. Prospective, well-co-ordinated, long term patient registries are an often under-recognised tool for building a comprehensive knowledge base for heterogeneous diseases. 

The Society for Endocrinology has committed to promoting and supporting the development of national endocrine real-world data registries. An application for ethics approval for a UK Adrenal Tumour Registry has recently been submitted, with the Society’s support, paving the way for this initiative. See page 27 for more details of your Society’s work on registries.

MAKING USE OF REGISTRIES

Disease registries can serve a number of objectives and, as such, can benefit a variety of stakeholders. Such registries can provide data on the natural history and outcomes for a specific condition and inform disease guidelines, governmental policies and clinical service development. Registries can be utilised to innovate clinical trial design, particularly for rare disease, and can support the development of therapies by providing real-world evidence, which can be leveraged as an observational arm in a clinical trial setting. Furthermore, national registries can help identify patient populations suitable for recruitment to clinical trials, and can provide valuable clinical and molecular data for basic and clinical researchers.

Large scale data registries, such as the Surveillance, Epidemiology and End Results Program (SEER; www.seer.cancer.gov), have demonstrated the power of well-co-ordinated long term data collection. The SEER data registry has provided data on long term outcomes for cancer survivors,¹ temporal changes in cancer incidence and mortality,² and data supporting the risk of iatrogenic cancers,³ and continues to play an advisory role for new cancer registry development.

REGISTRIES FOR THE UK

The establishment of disease-specific national registries can facilitate annual auditing of standards and help identify discrepancies in resources and funding. Within the UK, it can contribute invaluable information for the commissioning of new therapies, biomarkers and technologies, through collaboration with organisations such as the National Institute for Health and Care Excellence (NICE) and NHS England. Furthermore, NHS Digital has set out strategic priorities for 2023–2034, which include a commitment to improving digital infrastructures and digital linkage within the UK, which will serve to improve the efficiency and accuracy of national data collection. 

There are a number of exemplar UK disease registries, such as RaDaR (National Registry of Rare Kidney Diseases), which has recruited over 29,500 patients from 107 UK sites. It has contributed to over 30 peer research manuscripts between 2018 and 2023, and currently supports several national clinical research projects (www.ukkidney.org/rare-renal/radar).

The British Association of Endocrine and Thyroid Surgeons (BAETS; www.baets.org.uk) performs an annual audit of data returned to the UK Registry of Endocrine and Thyroid Surgery. This provides real-life data on quality performance indicators, which has prompted changes and innovations in thyroid surgical practices in the UK. Collection and submission of thyroid surgical data are mandatory requirements for BAETS members and contribute to the NHS Consultant Outcome Publication initiative, promoted initially by the Medical Director of NHS England.

PATIENT BENEFITS

Arguably the most important stakeholder for any disease registry is the patient. Enabling patient interaction in data registries can provide unique opportunities to study quality of life and patient-reported outcome measures. 

The opportunity to involve patients in digital health registries has been pioneered by the patient-focused digital health platform PeopleWith (www.peoplewith.com). PeopleWith provides a secure digital platform to host anonymised data, which also enables patient interaction via an app. PeopleWith was developed to support better ‘user’ engagement with healthcare systems and to optimise the voice of the patient in the future of health.

A CO-ORDINATED EFFORT

Finally, national registries should be curated to ensure that data transfer can be easily facilitated to established international data registries or online disease catalogues, such as Online Mendelian Inheritance in Man (OMIM), and the Cancer Genome Atlas Program (TCGA), to name a couple. 

Endocrine conditions are frequently heterogeneous and many have a strong hereditary basis, so co-ordinated efforts for national data collection in addition to international collaboration and data sharing will be crucial in ensuring the continued advancement of the specialty and improved patient outcomes.

RUTH T CASEY
Wellcome–MRC Institute of Metabolic Science and Department of Medical Genetics, University of Cambridge, and NIHR Cambridge Biomedical Research Centre, Addenbrooke’s Hospital, Cambridge

JESSICA DAVIS
Society for Endocrinology

KRISTIEN BOELAERT 
Institute of Applied Health Research, University of Birmingham

REFERENCES

1. Dixon SB et al. 2023 Lancet 401 1447–1457 https://doi.org/10.1016/S0140-6736(22)02471-0.
2. Siegel RL et al. 2023 CA: A Cancer Journal for Clinicians 73 233–254 https://doi.org/10.3322/caac.21772. 
3. Pasqual E et al. 2022 Journal of Clinical Oncology 40 1439–1449 https://doi.org/10.1200/JCO.21.01841 [as corrected].

In this issue: More on the Society's work with PeopleWith