The Rare Diseases Clinical Research Network: a model for clinical trial readiness
Joanne M Lumsden1, Tiina K Urv2
1Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences, National Institutes of Health, 6801 Democracy Boulevard, Bethesda, MD 20892-0001, USA.
Background:
The current road to developing treatments for rare diseases is often slow, expensive, and riddled with risk. Change is needed to improve the process, both in how we think about rare disease treatment development and the infrastructure we build to support ongoing science. The National Institutes of Health (NIH)-supported Rare Diseases Clinical Research Network (RDCRN) was established to advance the diagnosis, management, and treatment of rare diseases and to promote highly collaborative, multi-site, patient-centric, translational, and clinical research. The current iteration of the RDCRN intends to build upon and enhance successful approaches within the network while identifying innovative methods to fill gaps and address needs in the approach to the rare disease treatment development process through innovation, collaboration, and clinical trial readiness.
Objective:
The objective of this paper is to provide an overview of the productivity and influence of the RDCRN since it was first established 20 years ago.
Design And Methods:
Using a suite of tools available to NIH staff that provides access to a comprehensive, curated, extensively linked data set of global grants, patents, publications, clinical trials, and FDA-approved drugs, a series of queries were executed that conducted bibliometric, co-author, and co-occurrence analysis.
Results:
The results demonstrate that the entire RDCRN consortia and network has been highly productive since its inception. They have produced 2763 high-quality publications that have been cited more than 100,000 times, expanded international networks, and contributed scientifically to eight FDA-approved treatments for rare diseases.
Conclusion:
The RDCRN program has successfully addressed some significant challenges while developing treatments for rare diseases. However, looking to the future and being agile in facing new challenges that arise as science progresses is important.
Insights
The Rare Diseases Clinical Research Network (RDCRN) has significantly advanced rare disease treatment development over 20 years. This network has produced high-impact publications and contributed to eight FDA-approved treatments, demonstrating its crucial role in rare disease research.
Area of Science:
- * Rare disease research and treatment development.
- * Translational and clinical research infrastructure.
- * Biomedical research productivity and influence analysis.
Background:
- * Developing treatments for rare diseases is challenging, requiring improved processes and infrastructure.
- * The National Institutes of Health (NIH)-supported Rare Diseases Clinical Research Network (RDCRN) was established to enhance rare disease diagnosis, management, and treatment.
- * The RDCRN promotes collaborative, multi-site, patient-centric research and aims to innovate approaches for treatment development.
Purpose of the Study:
- * To provide an overview of the productivity and influence of the RDCRN over its 20-year history.
- * To assess the network's contributions to rare disease research and treatment development.
Main Methods:
- * Bibliometric, co-author, and co-occurrence analyses were conducted.
- * Data from a comprehensive, curated dataset of global grants, patents, publications, clinical trials, and FDA-approved drugs were utilized.
- * Tools available to NIH staff were employed for data analysis.
Main Results:
- * The RDCRN has been highly productive, generating 2763 high-quality publications cited over 100,000 times.
- * The network has expanded international collaborations and scientific contributions.
- * RDCRN research has contributed to eight FDA-approved treatments for rare diseases.
Conclusions:
- * The RDCRN program has successfully overcome significant challenges in rare disease treatment development.
- * Continued agility and innovation are essential to address future challenges in advancing rare disease science.
Related Concept Videos
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:12Hazard Ratio
For example, in a clinical trial...
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...