Every Patient Counts: How to Make the Most of Rare Disease Registries
February 23, 2015
Rare Disease Day 2015 is February 28, and as the day approaches I find myself thinking about how we as a biopharmaceutical industry can help the millions of patients living with a rare disease. There are more than 6,000 rare diseases affecting hundreds of millions of people around the globe. Yet because the population of sufferers from each disease is small and often widely dispersed, it can be difficult for researchers to connect with physicians and recruit enough patients to perform clinical trials. It is also much more difficult for them to get a sense of common treatment patterns, how those treatments are working and where treatment gaps exist – all of which is vital to the research process.
These barriers become much more manageable through the use of patient registries.
Rare disease registries, where physicians, patients and caregivers record information and track details of the patients’ diagnosis, condition and treatment, can address many of the scientific and communication challenges faced by rare disease researchers. Patient and caregiver reported outcomes provide a valuable glimpse into how various treatment strategies impact their quality of life. These insights, which can’t be gleaned from labs or physicians assessments, often shape the direction of their investigations and help them validate the choice of clinical outcomes. Registries also give researchers access to the stakeholder community, where they can connect with physicians, patients and even advocacy groups, to share information about their studies, build rapport with the broader patient community, and more efficiently and cost-effectively recruit participants.
But these benefits can only be achieved if researchers make the most of this valuable clinical tool. To ensure the success of your registry, consider doing the following:
Registries are an invaluable tool to any clinical research program, but for rare disease research, they can act as a lifeline between patients and the researchers trying to help them. By following these guidelines you ensure everyone derives the greatest return on their investment in the registry community. For more insights into patient registries, read about the Registry of Patient Registries.