Rare diseases have the fewest providers and available treatments per affected individual. Each rare disease affects fewer than 200,000 individuals in the United States, but among the 7,000 rare diseases, these afflict 30 million people in the United States, or about 10% of the U.S. Population. Moreover, only 350 "common" rare diseases account for 80% of this number.
Remarkably, less than 5% of all rare diseases have a single FDA approved treatment. We are here to serve the rare disease community by bringing novel therapeutics to the rare disease community.
Because access to care is one of the major barriers in rare disease research, we partner with national and international foundations. Half of all rare diseases have a disease specific foundation supporting or researching their rare disease. Many foundations have laid the difficult groundwork necessary to move into the field of therapeutics. They have developed animal models of the disorder they focus on, performed preclinical studies, identified compounds with promising safety and therapeutic profiles, and established a relationship with families across the country, often maintaining detailed patient registries. With our resources, experience, and collaborators, CRND partners with both pharma and disorder-specific foundations to bridge the translational gap from investigational drug to FDA-approved treatment, on a timeline that works for investors, shareholders in publicly-traded companies, and patient groups alike.
We provide comprehensive clinical research services and clinical expertise in a number of rare neurological disorders. Because most physicians have little or no experience with rare diseases, we interface directly with the rare disease foundations and parent networks. Because translational research in rare diseases has typically been centered at major academic institutions, we maintain a close relationship with collaborators at several academic centers. We use these ties to support specialized research and development for pharmaceutical companies and foundations, including the development of specific outcome measures and biomarkers, tailored to the disease you are studying.
The founder of CRND has independently designed and carried out clinical trials in rare disease, and has served as principal investigator on a number of pharma-sponsored studies. CRND is currently a key part of a multi-site study bringing together 10 investigators, 7 coordinators, and 6 post-docs and assistants from 5 institutions around the country. The departments involved include both pediatric and adult neurology, neuropsychology, sleep medicine, genetics, biomedical engineering, and cell biology. CRND is also collaborating with both device manufacturers in industry and biomedical engineers in academia to develop new biotech which could improve detection of physiological changes in clinical trials.
Start-up time for a clinical trial at an academic institution ranges from 4-6 months, and quality of the team dedicated to the project varies widely. However, our patient-centered model guarantees consistent data collection by the same investigators, coordinators, therapists, and dieticians for the duration of the study. Moreover, our business model allows us to perform budget and contract negotiations and start recruiting for clinical trials 5 to 10 times faster than most academic institutions.
Our time from receiving a contract for review until recruitment of the first participant ranges from 2 to 6 weeks. We can achieve this because rare disease research is all we do, and we have the resources to meet your needs.
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