What is a registry?

This morning we posted a video of Dr. Heather Van Mater on our social media, where she talks about a registry as an area of opportunity to advance AE Research. But what is a registry?

Rare diseases present unique challenges for researchers and companies working towards treatments and cures; small and scattered patient populations, difficulty to diagnose, no specific treatment protocols available, and a poor understanding of the natural history of the disease and its progression with and without treatment.

We can address a lot of these challenges through a systematic collection of clinical, genetic, and biologic data in the form of long-term registries. Registries come in different shapes and sizes. Some registries are focused on data entered by the physician or researcher; others depend on information submitted by patients and caregivers, and some on both. A common aspect of both types of registries is that they use observational study methods to collect data (clinical or other) to evaluate outcomes for a specific population defined by a particular disease.

Here we will focus on a patient-driven registry. The backbone of such a registry is the natural history study. The aim of following patients through all disease phases is to understand the burden of the disease, elements of disease progression, disease heterogeneity, outcomes, and quality of life.

Registries are used to:

–         Better understand the disease course over time

–         Facilitate patient participation in research

–         Recruit patients for clinical trials

–         Collect patient-reported outcomes

–         Monitor and improve the quality of health care

–         Follow different treatments

–         Establish guidelines for treatment

–         Inform research priorities

–         Review data to advocate for expanded insurance coverage

–         Include the collection of tissue or blood samples to advance research

Participating in a registry requires a serious time commitment of patients and caregivers. They must consistently enter their data over a more extended period.  But this consistent commitment translates into advancing research toward treatments and a cure.

 

You can join the AEA Research Network today and start changing the course of AE. Click here to register.

In case you missed this morning’s post: