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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.
This article was published in the following journal.
Name: Drug discovery today
In the development process for new drugs, dose-finding studies are of major importance. Absence of these studies may lead to failed phase 3 trials and delayed marketing authorization. In our study we ...
Rare diseases are an important global public health issue. One significant challenge is to ensure the access to orphan drugs for rare-disease patients. This study aims to evaluate the accessibility of...
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The objective of this project is to build a collection of biological samples consisting of fibroblasts and primary keratinocytes from patients with orphan monogenic diseases and to use the...
This is a clinical study to assess the safety and feasibility of Plasmodium vivax (P. vivax) blood-stage controlled human malaria infection (CHMI), by inoculation using a newly created sou...
Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in sub...
Orphan livers are organs that have been declined for clinical use by all centers due to their marginality. The current standard of care of liver preservation before transplant is cold stor...
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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.
BioPortfolio - life science, medical devices and pharmaceutical conference
BioPortfolio is a leading news, information and knowledge resource covering the global life science industries impacted on by biotechnology. The site aims to provide the lay person, the researcher and the management executive with a single location to so...
Clinical Approvals Clinical Trials Drug Approvals Drug Delivery Drug Discovery Generics Drugs Prescription Drugs In the fields of medicine, biotechnology and pharmacology, drug discovery is the process by which drugs are dis...