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Patient registries offer specific benefits to manufacturers developing treatments for rare diseases, providers treating patients with these conditions, and the patients themselves. For a drug or device manufacturer developing a product for a rare disease, a registry can be useful in both the pre- and post-approval phases. In the pre-approval phase, a disease registry may be used to advance the understanding of the disease; for example, the registry could provide new insights into the course of the disease or disease sub types, or provide more information on the burden of the disease and its effect on patient quality of life. This information may be important to designing and operating successful clinical trials to test a potential treatment. From a marketing standpoint, a disease registry can help to identify treating physicians and centers with large numbers of patients and to establish distribution channels prior to the launch of a new product.
In the post-approval phase, a product registry might demonstrate the performance of a product in the real world, provide information on product use patterns, or meet a post-marketing study commitment. Disease registries may also be useful in the post-approval phase, helping manufacturers better understand and separate the effects of the new treatment from the symptoms of the disease. A manufacturer with an existing disease registry may also consider adapting the registry into a product registry after the launch of a new treatment. This process could involve limiting enrollment of new patients to those using the approved treatment. Alternately, the registry could continue to enroll all patients with the disease, but collect additional data on patients using the new treatment.
For providers, a patient registry offers a tool for learning more about the progression and treatment of a rare disease. Both disease and product registries can help treating physicians connect with a community of their peers, where they can compare their patients progress and trends to others and to the aggregate. These registries can help foster the formation of an international community of expert physicians who can collaboratively develop recommendations on the clinical management of patients. Evidence has shown that collective clinical experience can lead to the development of recommendations for evaluation and monitoring of patients with rare diseases. The analysis of registry data on treatment outcomes can facilitate the establishment of therapeutic goals for patients with rare diseases. This builds a foundation for a consensus and the foundation for a consensus- and evidence-based disease management approach
Patients with a rare disease may also benefit from participating in a patient registry. Registries can provide patients with valuable sources of information on their disease. For example, the registry may develop and distribute educational materials to patients. The registry may also use information collected at each visit to develop custom reports for patients; these reports could track the patient's progress over time on key indicators. Some patients may find satisfaction in participating in a project that will improve the understanding of their disease.