Tagged: Rare Diseases

SCIO is critical for accelerating approval and significantly reducing the costs of drug and medical device development. One of the major challenges facing this industry is the rising cost of clinical trials. Thus, it is crucial to ensure that trials are significantly more efficient and cost-effective with the involvement of strategy at the time of […]

Pediatric Rare diseases and Rare Pediatric Disease Designation FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified. Section 529 of the FD&C Act is intended to encourage development of new drug and biological products for the prevention and treatment of rare pediatric diseases. Section […]

If you’re interested in learning more about drug repurposing to treat rare disease, BBCR can help with your regulatory consulting needs. We offer expertise in the following areas: FDA meeting and submission Pre-IND integrated development plan Clinical consultant Clinical study design and protocol CRO and project management Study remediation and rescue    

Rare Diseases are conditions that affect less than 200,000 people in the U.S. However, over 7,000 Rare Diseases affect more than 400,000,000 people worldwide, including ~25 million in the U.S. and ~30 million in Europe (1). In recent years, the number of orphan drugs approved has increased significantly. However, developing and marketing orphan drugs remains […]

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Biomarkers are now a routine part of drug development In rare diseases and precision medicine, implementation of biomarkers during product translation into clinic and early clinical development, moves treatment to market faster.

Drug repurposing acts to lower the need for early stage clinical trials and can help identify new uses for existing drugs. People tend to believe that a repurposed therapy can never be truly novel or transformative. Nothing could be further from the truth. One attractive option of Drug Repurposing is to use a scientific approach […]

Gene Therapy improved vision in patients with leber hereditary optic neuropathy (LHON) By: Dr. Maria Niu Mitochondria, powerhouses of eukaryotic cells, is a certain kind of cytoplasmic organelle and plays a critical role in energy production. Mitochondria dysfunction results in a broad spectrum of multisystem disorders. LHON is a mitochondrial neurodegenerative disease typically caused by […]

Biologic treatments show promise in providing clinical solutions to a variety of diseases including rare cancers and precision medicine. Services include: Indications analysis and prioritization Strategic drug assessment Clinical study design and protocol Biomarker strategy Early Clinical Development FDA meeting and submission Pre-ND integrated development plan CRO and project management Study remediation and rescue   […]

Biomarkers are now a routine part of drug development The FDA recognizes biomarker development as a high priority area for future research. The FDA and European Medicines Agency (EMA) have developed similar processes for the qualification of biomarkers intended for use as companion diagnostics or for development and regulatory approval of a drug or therapeutic. […]