According to the FDA, a registry can be defined as “an organized system that collects clinical and other data in a standardized format for a population defined by a particular disease, condition, or exposure”1.

Disease registries can be useful ways to:

  • understand the natural history of a disease
  • inform the design of clinical trials or observational studies (e.g., inform sample size, eligibility criteria, and study endpoints)
  • facilitate recruitment into a clinical trial (it’s particularly important to consider early patient engagement if this is one of your goals)
  • investigate the safety and effectiveness of a drug received during routine medical practice (in an observational study), or where registry data is used as an external control arm in a trial.

The FDA issued draft guidance in November 20211 to help industry assess the value of using registries to support regulatory decision-making for drug and biological products. The guidance has yet to be finalized; however, it provides useful considerations for designing a new registry, or repurposing an existing registry to generate evidence for use in a regulatory context. It highlights the benefits of using registry data: collecting longitudinal, structured data from a defined population that can include the systematic collection of patient-reported data, which electronic medical records and medical claims databases may lack.

It may be that you have a registry already in mind. The FDA suggests checking for:

1. data relevance: the availability of key data elements, e.g., patient characteristics, outcomes

2. data reliability: whether data are accurate, complete, and traceable.

Whether creating a new registry or repurposing an existing one, it’s important to adhere to applicable jurisdictional human subject protection requirements, including the protection of participants’ health information. The FDA also recommends that an institutional review board (IRB) be consulted when designing the registry.

Of course, one of the distinguishing factors of disease registries is the ability to watch the data “roll in” (after designing it rigorously of course!). However, the FDA warns that, during longitudinal data collection, one must account for any changes in diagnostic criteria or clinical definitions. Furthermore, changes to software, hardware and operating systems could affect data integrity.

At Vitaccess, we take a whole-systems approach to generating real-world evidence in a patient-centric way, combining rigorous scientific methodology with our proprietary technology.

A recent example is the “MyRealWorld MG” study. This global, digital, disease registry is collecting patient-reported data on the impact of living with myasthenia gravis, a rare neuromuscular condition. Subscribers to study data are using the data from this registry to prepare for their upcoming product launches and to support reimbursement decisions.

To read the full success story, please click here.

To subscribe to the MG disease registry dataset, please contact us.

1  Food and Drug Administration (FDA). Real-world data: Assessing registries to support regulatory decision-making for drug and biological products. Guidance for industry. Draft guidance. 2021. Available at: Accessed: 24th August 2023.

By Emily Boxell

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