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Orphan drugs: major development challenges at the clinical stage.

07:00 EST 15th January 2019 | BioPortfolio

Summary of "Orphan drugs: major development challenges at the clinical stage."

Biotechnology has provided powerful tools to assist in research and development (R&D) for rare diseases. However, orphan drug development presents several major challenges and obstacles, such as low disease prevalence, disease severity, small and heterogeneous patient populations, difficulties in patient recruitment, and limited knowledge of the natural history of disease, among others. Several strategies can be used to plan for and overcome these clinical and regulatory challenges, namely improved clinical trial design, improved patient recruitment, and closer collaboration with the regulatory authorities and with patient associations. As growth in the orphan drug market is expected over the next few years, improving its relevance in the global pharmaceutical market, further challenges might present themselves in the development of orphan drugs.

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This article was published in the following journal.

Name: Drug discovery today
ISSN: 1878-5832
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Medical and Biotech [MESH] Definitions

An orphan nuclear receptor that is implicated in regulation of steroidogenic pathways. It is unlike most orphan nuclear receptors in that it appears to lack an essential DNA-binding domain and instead acts as a transcriptional co-repressor. Mutations in the gene Dax-1 cause congenital adrenal hypoplasia.

Severe distortions in the development of many basic psychological functions that are not normal for any stage in development. These distortions are manifested in sustained social impairment, speech abnormalities, and peculiar motor movements.

A family of cell surface receptors that were originally identified by their structural homology to neurotropic TYROSINE KINASES and referred to as orphan receptors because the associated ligand and signaling pathways were unknown. Evidence for the functionality of these proteins has been established by experiments showing that disruption of the orphan receptor genes results in developmental defects.

Drugs which have received FDA approval for human testing but have yet to be approved for commercial marketing. This includes drugs used for treatment while they still are undergoing clinical trials (Treatment IND). The main heading includes drugs under investigation in foreign countries.

Production of drugs or biologicals which are unlikely to be manufactured by private industry unless special incentives are provided by others.

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