Mar. 27, 2020
Posted by Laura Mullen
The National Organization for Rare Disorders (NORD®) is excited to announce that a new peer-reviewed manuscript written by members of our research team has been published as a leading article by the journal Pharmaceutical Medicine.
The paper, entitled “Establishing Patient Registries for Rare Diseases: Rationale and Challenges,” begins by highlighting common barriers experienced by the rare disease community including small and geographically dispersed patient populations, restricted research funding and diagnostic delays. These diagnostic delays are often due to disease complexities, limited medical training for practitioners and an evolving understanding of disease characterizations.
In an effort to overcome these challenges, the research team explores current trends towards innovative and patient-centered healthcare that are allowing patient registries to emerge as valuable resources within the world of rare disease research and drug development.
Patient registries are effective tools that center patients as research partners and bring rare communities together around a common initiative. The data collected in the registries has the potential to inform clinicians and researchers of the natural progression of a disease, assist with clinical trial recruitment, monitor clinical trial treatments in patients, and provide support for the establishment of disease-specific standards and care.
Throughout the paper, the NORD research team describes the value of and rationale for establishing rare disease patient registries while explaining the considerations and challenges that researchers, industry, health care providers and patient community organizations, may encounter. This includes the importance of clearly defining registry study objectives, ensuring that the registry is ethically governed, developing comprehensive data analysis plans and ensuring that sustainability and transition protections are in place.
It is NORD’s aim to provide the rare disease community with valuable information and the support needed to begin the journey towards establishing patient registries. Please contact the team at [email protected] to learn more about NORD’s IAMRARE™ registry program or with questions or comments.
“Establishing Patient Registries for Rare Diseases: Rationale and Challenges” is available in its entirety via open access here.