The number of patient registries is increasing, generating valuable sources of data for holistic disease insights and well-informed therapeutic advances. This article describes findings from a global registry review in sickle-cell disease, assessing their current status as well as future directions that could benefit all stakeholders, including patients.

The value of patient registries in rare diseases

Patient registries are important tools in the healthcare system for systematically collecting data over time from a population living with a certain disease, condition, or exposure [1]. Well-designed and well-executed patient registries hold great potential in providing important disease insights into, for example, patient characteristics, patterns of treatment and care, clinical outcomes and comparative effectiveness, as well as disparities [2, 3].

When it comes to rare diseases, the value of patient registries may be even greater. A number of obstacles can emerge when conducting clinical trials in rare diseases, which may impact the completeness of the data collected. For instance, research may be hindered by an incomplete knowledge of the natural history of the condition, geographical dispersion of small groups of patients, or difficulty in capturing clinically significant changes over time [4]. By facilitating the collection of real-world data (RWD) on rare disease populations, patient registries can combat these obstacles. Registries can generate an important resource to improve understanding of the disease pathology and its epidemiology, alongside supporting the development of more targeted regulatory approvals for new therapies and reimbursement strategies