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The Orphan Drug Act of 1983 (ODA) put in place a set of financial and marketing incentives to stimulate the development of drugs to treat rare diseases, and since its passage more than 600 orphan drug and biologic products have been brought to market in the United States. Rapid growth in orphan drug approvals in conjunction with high orphan drug prices have triggered concern that drug makers are exploiting certain aspects of the ODA for financial gain and that some pharmaceutical drugs are receiving orphan status where it is not warranted. The landscape of approved therapies for rare skin diseases has not been well described. In this article, we provide a descriptive analysis of the Food and Drug Administration-approved orphan drugs for the treatment of rare dermatologic conditions and skin-related cancers since the enactment of the ODA. We discuss policy issues that emerge from the analysis and suggest areas for future research. Next, we elucidate ODA loopholes using dermatologic drugs as examples and propose potential reforms. Finally, we consider future directions for orphan drug development in the field of dermatology.
This article was published in the following journal.
Name: Journal of the American Academy of Dermatology
Rare diseases provide a challenge in the evaluation of new therapies. However, orphan drug development is of increasing interest because of the legislation enabling facilitated support by regulatory a...
There are nearly 30 million Americans that suffer from at least one of the more than 7000 rare diseases identified to date. Therapies for treating, preventing, or diagnosing rare diseases have been li...
Most rare diseases still lack approved treatments despite major advances in research providing the tools to understand their molecular basis, as well as legislation providing regulatory and economic i...
As the calendar year comes to an end, this article reviews unique issues in dermatology that made the news in 2019: chemical sunscreen, the measles outbreak, drug approvals for pediatric dermatology a...
Drug development for rare diseases, classified as diseases with a prevalence of
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 study will help the investigator understand the pathogenesis of different rare genetic conditions and to establish database of rare genetic databases. This would ultimately help to pr...
The DiRiP study will enroll patients (n = 3500) with unclear rare diseases and suspected genetic reasons. In group 1 (n = 500) subjects are clinically characterized in the context of outpa...
The incidence of rare diseases is extremely low, the disease is numerous, the symptoms are serious, and the detection technology is complicated. Countries have different definitions of rar...
RATIONALE: Evaluating patients who have skin diseases may help doctors plan more effective treatment and follow-up for skin diseases and systemic diseases that show up in the skin. PURPOS...
The practice of prescribing or using a drug outside the scope of the drug's official approved label as designated by a regulatory agency concerning the treatment of a particular disease or condition.
Prenatal interventions to correct fetal anomalies or treat FETAL DISEASES in utero. Fetal therapies include several major areas, such as open surgery; FETOSCOPY; pharmacological therapy; INTRAUTERINE TRANSFUSION; STEM CELL TRANSPLANTATION; and GENE THERAPY.
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.
The science concerned with the benefit and risk of drugs used in populations and the analysis of the outcomes of drug therapies. Pharmacoepidemiologic data come from both clinical trials and epidemiological studies with emphasis on methods for the detection and evaluation of drug-related adverse effects, assessment of risk vs benefit ratios in drug therapy, patterns of drug utilization, the cost-effectiveness of specific drugs, methodology of postmarketing surveillance, and the relation between pharmacoepidemiology and the formulation and interpretation of regulatory guidelines. (Pharmacoepidemiol Drug Saf 1992;1(1); J Pharmacoepidemiol 1990;1(1))
Therapies using arts or directed at the senses.
Pharmacy is the science and technique of preparing as well as dispensing drugs and medicines. It is a health profession that links health sciences with chemical sciences and aims to ensure the safe and effective use of pharmaceutical drugs. The scope of...