Rare Diseases

Biography

Biography: Ginger Spitzer

Abstract

Workshop will review how to create a patient registry, outlining major elements. Then participants will work together and with presenter to identify specific needs (based on resources, objectives) and review options and recommendations. In the age of “big data” needs, it is becoming apparent that patient groups, foundations, non-profits, etc. can provide significant content to the research field by having access to patients that pharma and academia may not. This is especially true within the rare disease field where patient access and data availability are common barriers to advancing research. Of the roughly 7000 rare diseases identified by NIH, best estimates are that fewer than 20% of rare diseases have patient registries. Most of these are operated by patients' organizations or academic researchers, states The Office of Rare Diseases Research (ORDR), a component NCATS, NIH. For many groups building a registry is a major undertaking. While smaller patient groups, foundations, non-profits often provide patient education and support, many want to add resources to accelerate research in their specific disease. One form of research to provide is a patient registry that gathers and shares data. Many of these groups need information and guidance on how their particular group could launch a registry.

The proposed workshop will outline:

1)Types of registries

2)Identify which type is best

3)IRB process

4)HIPPA and privacy Issues

5)Resources and partners/venders needed

6)Designing the registry (functionality and appearance)

7)Development process

8)Involving the scientific community

9)Technology to host a registry

10)User-ability

11)Sponsorships/Funding

12)Educating the Patients, Physicians, and other stakeholders

13)Launching the Registry

14)Patient Engagement and Marketing

15)Tracking and communicating with participants

16)Building data-sharing agreements

17)Disseminating to scientific community