A Desk Top Allusion to The Rare Orphan Diseases and Orphan Drugs: Possessions to Discern by Every Healthcare Professional

This article gives a brief knowledge about orphan diseases and drugs. An orphan disease is a rare illness lack market and resource data for the treatment and the drugs used for their treatment are called orphan drugs. The pharmaceutical firms show less interest in the development of drugs to treat them, and lack profit without government assistance. The authors reviewed published scientific literature and grouped a broad list of Orphan diseases and orphan drugs. The article summarizes the entire list of orphan diseases and drugs with their description.

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Comparison and analysis of the fda approved orphane drugs registerand the vital and essential drugs list of the Russian Federation

Remedium Journal about the Russian market of medicines and medical equipment ◽

10.21518/1561-5936-2020-9-4-16 ◽

2020 ◽

pp. 4-16

Author(s):

D.S. Yurochkin ◽

◽

A.A. Leshkevich ◽

Z.M. Golant ◽

I.A. NarkevichSaint ◽

...

Keyword(s):

United States ◽

Russian Federation ◽

Rare Diseases ◽

Orphan Drugs ◽

The United States ◽

Orphan Diseases ◽

Essential Drugs ◽

The Russian Federation ◽

Comparison And Analysis ◽

The Government

The article presents the results of a comparison of the Orphan Drugs Register approved for use in the United States and the 2020 Vital and Essential Drugs List approved on October 12, 2019 by Order of the Government of the Russian Federation No. 2406-r. The comparison identified 305 international non-proprietary names relating to the main and/or auxiliary therapy for rare diseases. The analysis of the market of drugs included in the Vital and Essential Drugs List, which can be used to treat rare (orphan) diseases in Russia was conducted.

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Corporate Social Responsibility (CSR) of Innovative Pharmaceutical Corporations. The Case of BIOGEN

Comparative Economic Research ◽

10.2478/cer-2018-0018 ◽

2018 ◽

Vol 21 (3) ◽

pp. 45-62 ◽

Cited By ~ 1

Author(s):

Janina Witkowska

Keyword(s):

Corporate Social Responsibility ◽

Pharmaceutical Industry ◽

Social Responsibility ◽

Corporate Philanthropy ◽

Developed Countries ◽

Orphan Drugs ◽

Pharmaceutical Firms ◽

Life Saving ◽

Corporate Social

The aim of this paper is to discuss the common features and specificity of Corporate Social Responsibility (CSR) practices of innovative transnational corporations (TNCs) acting in the pharmaceutical industry. The innovativeness of pharmaceutical firms is understood here as their ability to make a breakthrough in the treatment of rare, incurable diseases. The examination of the issue leads to the conclusion that the specificity of CSR in this industry is related to the contradiction between the economic and social/ethical aspects of innovation processes in this field. A key issue of CSR in the innovative pharmaceutical industry seems to be the pricing of drugs, especially orphan and ultra‑orphan drugs, resulting in patients from less developed countries having limited access to life‑saving medicines or those that improve the quality of life. Corporations use their monopolistic position to set extremely high prices. However, without the market/marketing exclusivity offered to pharmaceutical firms by the law, orphan drugs would probably not be developed, produced and commercialized. Traditional CSR practices (corporate philanthropy, community and neighborhood programs, volunteerism etc.) cannot be treated as sufficient ‘compensation’ for the high prices of medicines. Real, true CSR in the innovative pharmaceutical industry requires either abandoning or reducing extreme monopolistic privileges and offering medicines for rare diseases at lower prices.

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Rare diseases, orphan drugs, and orphan diseases

BMJ ◽

10.1136/bmj.333.7559.127 ◽

2006 ◽

Vol 333 (7559) ◽

pp. 127 ◽

Cited By ~ 3

Author(s):

Jeff Aronson

Keyword(s):

Rare Diseases ◽

Orphan Drugs ◽

Orphan Diseases

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Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

European Journal of Clinical Pharmacology ◽

10.1007/s00228-012-1423-2 ◽

2012 ◽

Vol 69 (4) ◽

pp. 1009-1024 ◽

Cited By ~ 64

Author(s):

Roberta Joppi ◽

Vittorio Bertele’ ◽

Silvio Garattini

Keyword(s):

Orphan Drug ◽

Orphan Drugs ◽

Orphan Diseases ◽

Drug Legislation ◽

The Eu

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Billion Dollar Orphans

10.37419/lr.v6.i3.6 ◽

2019 ◽

Vol 6 (3) ◽

pp. 731-768

Author(s):

John Sheridan

Keyword(s):

Orphan Drug ◽

Orphan Drugs ◽

Federal Courts ◽

Pharmaceutical Companies ◽

Orphan Diseases ◽

Social Purpose

This Comment examines the extent to which Congress empowered the FDA to address the increase in petitions and the general accessibility of orphan drug remedies. Specifically, this Comment seeks to understand why the FDA’s interpretation of the purpose of the ODA seems to conflict with the statutory intent as interpreted by federal courts. This Comment considers a statute’s ultimate goal or social purpose to be the purpose of the statute, whereas the express mechanisms by which Congress seeks to bring about these goals is best understood as the statute’s intent. To understand the FDA and judiciary’s differing interpretations of the ODA, this Comment analyzes the language of the Statute, recent ODA litigation, FDA’s promulgated regulations, as well as recent response to pharmaceutical companies’ increase in designation requests for orphan drugs. Ultimately, this Comment strives to determine whether or not the ODA can effectively achieve the goals Congress set forth in 1983.This Comment conducts a statutory analysis of the ODA and closely examines how courts, the FDA, and litigant pharmaceutical companies interpret the Statute differently. This Comment argues that Congress’s intent in passing the ODA was to create lucrative incentives for the development of drugs for orphan diseases. But, Congress’s purpose in drafting the ODA was to ensure the drugs became available to patients. The incentives serve as a tool to achieve the purpose of the ODA: to treat patients suffering from rare diseases.This Comment concludes that to better effectuate this purpose, Congress must amend the ODA or pass other legislation empowering the FDA to promulgate regulations that alter the schedule and administration of the ODA’s lucrative “basket of goodies.”

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