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Chron Respir Dis. 2019 Jan-Dec;16:1479973119881777. doi: 10.1177/1479973119881777.

The importance of effective registries in pulmonary diseases and how to optimize their output.

Author information

1
Department of Genetics and Clinical Immunology, National Institute of Tuberculosis and Lung Diseases, Warsaw, Poland.
2
Conresp, Lörzweiler, Germany.
3
Department of XIV, National Koranyi Institute for Pulmonology, Budapest, Hungary.
4
Department of Public Health, Semmelweis University, Budapest, Hungary.

Abstract

Randomized controlled trials (RCTs) are essential for the approval of new therapies; however, because of their design, they provide little insight concerning disease epidemiology/etiology and current clinical practice. Particularly, in lung disease, rigid inclusion/exclusion criteria can limit the generalizability of pivotal trial data. Noninterventional studies (NIS), conducted through the well-established mechanism of patient registries, are undervalued as a means to close data gaps left by RCTs by providing essential data that can guide patient care at different levels from clinical decision-making to health-care policy. While NIS contribute valuable data in all disease areas, their importance in rare diseases cannot be underestimated. In respiratory disease, registries have been essential in understanding the natural history and different phenotypes of rare conditions, such as alpha 1 antitrypsin deficiency, cystic fibrosis, and idiopathic pulmonary fibrosis. Importantly, additional therapeutic outcome data were generated. While measures for enhancing data quality in RCTs have evolved significantly, the approach and effectiveness of registries is variable. Within this article, we review the contribution of registries to pulmonary disease and make recommendations for their effective management. Additionally, we assess limitations of registry data as well as challenges to registry operation, including the impact of the European Union General Data Protection Regulation.

KEYWORDS:

Alpha 1 antitrypsin deficiency; lung diseases; quality improvement; rare diseases; registries

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