Development of an Orphan Drug by a Start-Up Company: MetroGel® for Rosacea

1992 ◽  
Vol 8 (4) ◽  
pp. 566-572 ◽  
Author(s):  
Robert J. Borgman
Keyword(s):  
United States ◽  
Research And Development ◽  
Pharmaceutical Company ◽  
Orphan Drug ◽  
The United States ◽  
Orphan Drug Act ◽  
Start Up ◽  
Significant Research ◽  
Marketing Costs ◽  
Major Pharmaceutical Company

AbstractThe Orphan Drug Act of 1983, along with the discovery of a new use for a known drug and an investor willing to assume the necessary risk, brought about the formation of a start-up pharmaceutical company. The primary incentive of the Orphan Drug Act of seven years of marketing exclusivity provided the protection from competition necessary for recovery of the significant research and development and marketing costs. The orphan product, MetroGel®, for the treatment of rosacea, required approximately five years of development before it was approved for marketing by the Food and Drug Administration. MetroGel® has become the number one drug in the United States for the treatment of rosacea. It currently is marketed in other countries through a licensing agreement with a major pharmaceutical company.

Dercum’s Disease: Estimating the prevalence of a rare painful loose connective tissue disease

2020 ◽  
Author(s):  
Natalie Corina Munguia ◽  
B. Robert Mozayeni ◽  
Thomas Wright ◽  
Karen L. Herbst
Keyword(s):  
United States ◽  
Rare Disease ◽  
Orphan Drug ◽  
The United States ◽  
Rare Disorder ◽  
Quality Healthcare ◽  
Adiposis Dolorosa ◽  
Orphan Drug Act ◽  
Dercum’S Disease ◽  
The Uk

Abstract Background In the United States (US), the Orphan Drug Act of 1983 defines a rare disease as affecting less than 200,000 individuals. In Europe, a rare disease or disorder is defined as affecting 5 individuals in 10,000 which the European commission reports to be approximately 246,000 individuals. Dercum’s disease, also known as Adiposis Dolorosa, is a rare loose connective (fat) tissue disease that is characterized by painful lipomas (fatty masses). Orphanet, the National Organization for Rare Disease (NORD) and the United States National Institutes of Health (NIH) have classified Dercum’s disease as a rare disorder. The prevalence of Dercum’s disease is not well studied or understood. The objective of this paper is to estimate the prevalence of Dercum’s disease, whether it is in fact a rare disease, and increase awareness for this painful disease. Results An in-depth literature review was conducted in PubMed, the UK Biobank, the U.S. Agency for Health Research and Quality Healthcare Cost and Utilization, by incidence of disease in medical practice, in social media forums and by internet search in order to understand, estimate and determine the prevalence of Dercum’s disease. The prevalence of Dercum’s disease was found to range from 150 to 150,000 in the US population. Conclusion The data collected in this paper meet the requirements of the Orphan Drug Act which requires 200,000 individuals or less to be affected by a disorder for it to be classified as rare. These conclusions may apply to Europe as supporting data was utilized from both Sweden and the UK. Further research must be conducted to better understand subclassifications of this rare disorder.

scholarly journals Incentives for orphan drug research and development in the United States

2008 ◽  
Vol 3 (1) ◽  
Author(s):  
Enrique Seoane-Vazquez ◽  
Rosa Rodriguez-Monguio ◽  
Sheryl L Szeinbach ◽  
Jay Visaria
Keyword(s):  
United States ◽  
Research And Development ◽  
Orphan Drug ◽  
Drug Research ◽  
The United States

Assessment of Regulatory Strategies for Orphan Drug Development and Approval Process in USA & EU

2021 ◽  
Vol 12 (4) ◽  
pp. 2492-2500
Author(s):  
Sadiq Basha G ◽  
Srinivasan R
Keyword(s):  
United States ◽  
European Union ◽  
Drug Development ◽  
Rare Disease ◽  
Orphan Drug ◽  
Drug Approval ◽  
The United States ◽  
Pharmaceutical Drugs ◽  
The European Union ◽  
Orphan Drug Act

Orphan medicines are pharmaceutical drugs or vaccines supposed to deal with, preventing or diagnose of a rare disease (viz., ailment, Huntington's disease, syndrome, etc.). The meaning of rare sicknesses varies throughout some jurisdictions, however, generally contemplates the ailment of occurrence, severity, and life of alternate healing options. An uncommon ailment isn't worldwide and depends on the rules and guidelines officially adopted and approved via each region or U.S.A. The Orphan Drug Act, 1983 (ODA) has been recognized and adopted in numerous nations, international (United States of America (USA) and European Union (EU).) in the preceding 35 years, and has effectively advanced R and D ventures to widen new pharmaceuticals for the remedy of rare sickness. The rate of occurrences of such diseases were outpaced at an extra pace than the speed with which medicines are researched and developed to treat rare diseases. One of the main reasons is that the pharmaceutical industry is not interested in researching the enhancement of orphan medicines since they no longer have a significant large market. Despite the multiple incentives provided by the orphan drug act, this is the current reality. However, in this article, we've tried to concentrate on the current regulatory framework, current concepts of rare disease, regulatory challenges for rare disease drug development, and orphan drug approval in the United States & the European Union.

A History of Geothermal Energy Research and Development in the United States. Reservoir Engineering 1976-2006

2010 ◽  
Author(s):  
B. Mack Kennedy ◽  
Karsten Pruess ◽  
Marcelo J. Lippmann ◽  
Ernest L. Majer ◽  
Peter E. Rose ◽  
...  
Keyword(s):  
United States ◽  
Research And Development ◽  
Geothermal Energy ◽  
The United States ◽  
Reservoir Engineering ◽  
Energy Research ◽  
History Of

A HISTORICAL REVIEW OF THE ACCOUNTING TREATMENT OF RESEARCH AND DEVELOPMENT COSTS

1992 ◽  
Vol 19 (1) ◽  
pp. 51-78 ◽  
Author(s):  
Paul E. Nix ◽  
David E. Nix
Keyword(s):  
United States ◽  
Research And Development ◽  
Alternative Treatment ◽  
Historical Review ◽  
The United States ◽  
Current Treatment ◽  
Social Consequences ◽  
Financial Accounting ◽  
Development Costs ◽  
Accounting Rules

This study reviews the literature and the practice of accounting for research and development (R&D) costs from the first reference in 1917 to the current treatment. The conceptual treatment of R&D is compared to current financial accounting rules and explanation of the evolution of the current rules is presented. The economic and social consequences of the current rules which require R&D costs to be expressed are examined. The paper explores possible alternative treatment of R&D costs. As a contrast to U.S. practice, the accounting treatment of R&D costs in other countries is discussed. Given the findings of this paper, a strong case can be made for changing the way that R&D costs are accounted for in the United States.

From Start-Up to Scale-Up

2018 ◽  
Author(s):  
Gilles Duruflé ◽  
Thomas Hellmann ◽  
Karen Wilson
Keyword(s):  
United States ◽  
Conceptual Framework ◽  
Public Policies ◽  
Scale Up ◽  
The United States ◽  
Smart Money ◽  
Start Up ◽  
The Us ◽  
The Creation

This chapter examines the challenge for entrepreneurial companies of going beyond the start-up phase and growing into large successful companies. We examine the long-term financing of these so-called scale-up companies, focusing on the United States, Europe, and Canada. The chapter first provides a conceptual framework for understanding the challenges of financing scale-ups. It emphasizes the need for investors with deep pockets, for smart money, for investor networks, and for patient money. It then shows some data about the various aspects of financing scale-ups in the United States, Europe, and Canada, showing how Europe and Canada are lagging behind the US relatively more at the scale-up than the start-up stage. Finally, the chapter raises the question of long-term public policies for supporting the creation of a better scale-up environment.

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