Over the past few years, digital patient registries have become a popular tool for patient communities fighting rare diseases. As you may have already heard, the FD Foundation is helping build the FD/MAS Patient Registry, an upcoming research project that will collect medical history information from people with fibrous dysplasia or McCune-Albright syndrome.
Public health experts are extremely supportive of patient registry projects. The National Organization for Rare Disorders, European Organisation for Rare Diseases, and the Canadian Organization for Rare Disorders released a joint statement calling for patient registries to be recognized as a global priority in the field of rare diseases. The leader of the Food and Drug Administration’s Center for Drug Evaluation and Research encourages the use of digital data collection tools to learn more about the experiences of people living with rare diseases. The National Institutes of Health offers resources to help rare disease patient organizations launch their patient registries.
But why do so many experts agree that patient registries are hugely important projects? Here are three major ways that patient registries can benefit rare disease patients.
Modern, digital rare disease patient registries are websites where people with a particular rare disease can go to tell their story. After a patient or parent registers on the website, he or she can complete surveys that systematically capture that person’s history of symptoms, treatments and outcomes.
Researchers are ready and waiting to analyze that information, and to learn more about which treatments seem to get the best results in which situations. The researchers share those results with patients and families, who have better information about which treatments are best in which situations.
#2 Registries amplify your voice
In addition to asking about the patient’s history of symptoms, treatments and outcomes, a lot of patient registries ask you to give your point of view on important matters! For example, some registries let you share your point on view on which symptoms are most important symptoms to find treatments for, or which existing treatments you wish there was better information on.
Sharing your point of view is extremely powerful, because it helps research funding agencies, pharmaceutical companies, regulatory agencies and patient organizations learn how they can best serve you and your interests. Requiring researchers to listen to and serve the interests of the patient is becoming more and more common before funding can be won, or drugs can be approved.
#3 Registries can improve chances of drug development
When a drug company learns that a rare disease community already has a rare disease registry with useful data, that company sees a lot of potential savings, which makes the company more likely to pursue drug development.
For example, recruiting patients with rare diseases to participate in clinical trials is usually very expensive for pharmaceutical companies. It’s very hard for the companies to get information about the clinical trial to the right people. If there is a rare disease registry run by a patient organization, when patients sign up, each patient can indicate whether they want to learn about clinical trials that are recruiting. Then, once it’s time to start a clinical trial, the patient organization can send the information about the clinical trial to people who have opted-in for information, without bothering people who aren’t interested, or sharing patient contact information with the drug company. This approach is much less expensive for a drug company than trying to find the contact information for hundreds of patients overnight, many of whom might not be interested in participating in research at all!
Statistics that come from registry research can also be very helpful to drug companies. The FDA often wants drug companies to provide independent verification of information about symptoms or patient preferences. When a patient registry already exists, with lots of patients participating, the drug companies know that there is an unbiased data source that they can apply to access for drug development purposes.
There’s a lot to be said about patient registries
Patient registries are very important projects, with a lot of potential benefits for any rare disease community, but there are important things to consider before you join any research study, including a patient registry. Once the FD/MAS Patient Registry is launched, the study’s investigators will be available to answer specific questions you might have about that research study, including potential risks and benefits.