Characteristics of rare disease marketing applications associated with FDA product approvals 2006–2010

Pariser, Anne; Slack, Daniel J.; Bauer, Larry; Warner, Catherine A.; Tracy, LaRee

doi:10.1016/j.drudis.2012.04.011

Cited by 19 publications

(12 citation statements)

References 16 publications

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“…Since 1983, approximately 400 orphan products have been approved by the FDA compared to 10 drugs that received marketing approval between 1973 and 1983. Ninety percent of these were approved by the FDA's Centre for Drug Evaluation and research (who process the above NDAs) and the remaining 10% by FDA's Center for Biologics Evaluation and Research (who process BLAs) 11 .…”

Section: Fda Oddpmentioning

confidence: 99%

“…11 June 1996 Treatment of cystic hydatid disease of the liver, lung and peritoneum, caused by the larval form of the dog tapeworm, Echinococcus granulosus. ose (cysts in spinal fluid) neurocysticercosis in symptomatic cases and(2)prophylaxis of epilepsy and other sequelae in asymptomatic neurocysticercosis11 June 1996 Treatment of parenchymal neurocysticercosis due to active lesions caused by larval forms of the pork tapeworm, Taenia solium. Part of combination therapy in adults (aged 18 years or older) with pulmonary multidrug resistant tuberculosis (MDR-TB).…”

mentioning

confidence: 99%

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The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases

Sachs‐Barrable

Conway

Gershkovich

et al. 2014

Drug Development and Industrial Pharmacy

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Neglected tropical diseases (NTDs) are infections which are endemic in poor populations in lower- and middle-income countries (LMIC). Approximately one billion people have now or are at risk of getting an NTD and yet less than 5% of research dollars are focused on providing treatments and prevention of these highly debilitating and deadly conditions. The United States Food and Drug Administration (FDA) Orphan Drug Designation program (ODDP) provides orphan status to drugs and biologics, defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and/or disorders that affect fewer than 200 000 people in the United States, or that affect more than 200 000 persons but are not expected to recover the costs of developing and marketing a treatment drug. These regulations have led to the translation of rare disease knowledge into innovative rare disease therapies. The FDA Guidance for Industry on developing drugs for the treatment and prevention of NTDs describes the following regulatory strategies: Orphan Product Designation, Fast Track Designation, Priority Review Designation, Accelerated Approval and Tropical Disease Priority Review Voucher. This paper will discuss how these regulations and especially the ODDP can improve the clinical development and accessibility of drug products for NTDs.

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Section: Fda Oddpmentioning

confidence: 99%

mentioning

confidence: 99%

The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases

Sachs‐Barrable

Conway

Gershkovich

et al. 2014

Drug Development and Industrial Pharmacy

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“…The contribution of regulators in scientific advice/protocol assistance increases the chance of a successful development of the orphan medicinal product [4]. The experience with authorizations of medicines for rare diseases shows that even though marketing authorization applicants are dealing with extremely complicated development programs and regulatory authorities are faced with limited data in comparison with frequent diseases, it is still feasible to bring to the market safe and effective products for rare diseases in ratios that are comparable with nonrare diseases [5,6].…”

Section: Regulatory Supportmentioning

confidence: 99%

Promoting the development of drugs against rare diseases: what more should be done?

Westermark¹,

Llinares²

2012

Expert Review of Pharmacoeconomics & Outcomes Research

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“…Notably, more than half of the 26 new biologic applications submitted to CDER were for rare disease indications. Furthermore, approval rates for marketing applications for rare and common disease indications were similar (77% approval rate for rare disease products and 71% approval for non-rare disease products) [27–29]. For IEM, there were six products approved during this same time period (2006–2010).…”

Section: Current Federal Research Infrastructure For Rare Disordermentioning

confidence: 99%

Expanding research to provide an evidence base for nutritional interventions for the management of inborn errors of metabolism

Camp

Lloyd-Puryear

Yao

et al. 2013

Molecular Genetics and Metabolism

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A trans-National Institutes of Health initiative, Nutrition and Dietary Supplement Interventions for Inborn Errors of Metabolism (NDSI-IEM), was launched in 2010 to identify gaps in knowledge regarding the safety and utility of nutritional interventions for the management of inborn errors of metabolism (IEM) that need to be filled with evidence-based research. IEM include inherited biochemical disorders in which specific enzyme defects interfere with the normal metabolism of exogenous (dietary) or endogenous protein, carbohydrate, or fat. For some of these IEM, effective management depends primarily on nutritional interventions. Further research is needed to demonstrate the impact of nutritional interventions on individual health outcomes and on the psychosocial issues identified by patients and their families. A series of meetings and discussions were convened to explore the current United States’ funding and regulatory infrastructure and the challenges to the conduct of research for nutritional interventions for the management of IEM. Although the research and regulatory infrastructure are well-established, a collaborative pathway that includes the professional and advocacy rare disease community and federal regulatory and research agencies will be needed to overcome current barriers.

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Characteristics of rare disease marketing applications associated with FDA product approvals 2006–2010

Cited by 19 publications

References 16 publications

The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases

The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases

Promoting the development of drugs against rare diseases: what more should be done?

Expanding research to provide an evidence base for nutritional interventions for the management of inborn errors of metabolism

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