scholarly journals Orphan Drugs, Compounded Medication and Pharmaceutical Commons

2021 ◽  
Vol 12 ◽  
Author(s):  
Kim Hendrickx ◽  
Marc Dooms
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Point Of Care ◽  
Orphan Drugs ◽  
Private Companies ◽  
Market Protection ◽  
Drug Regulations ◽  
Protection Mechanisms ◽  
Life Threatening ◽  
Low Prevalence

Regulatory agencies installed orphan drug regulations to stimulate research and development of new innovative treatments for life-threatening diseases with a low prevalence (rare diseases). We established a list of well-known food-related ingredients with clinical evidence for rare diseases in the open medical literature that obtained marketing authorization as an expensive “orphan drug”, protected by intellectual property (IP) rights. We show that these ingredients are part of an established practice of medicinal compounding—a form of point of care manufacturing. We argue that these ingredients should be considered as “pharmaceutical commons”, and that regulatory incentives for private companies and market protection mechanisms such as IP rights are not justified in this case.

scholarly journals Analysis of Patient Access to Orphan Drugs in Turkey

2020 ◽  
Author(s):  
GÜVENÇ KOÇKAYA ◽  
Sibel Atalay ◽  
Gulpembe Oguzhan ◽  
Mustafa Kurnaz ◽  
Selin Okcun ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Anatomical Therapeutic Chemical ◽  
Descriptive Analysis ◽  
Orphan Drugs ◽  
European Medicines Agency ◽  
Drug Status ◽  
Early Access ◽  
Life Threatening ◽  
Low Prevalence

Abstract Background: Rare diseases are life-threatening, serious, and chronic genetic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. Methods: The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey.Results: Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. Seventeen licensed products (74%) and 17 unlicensed products (35%) were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L –Antineoplastic and Immunomodulatory” agents.Conclusion: An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

Evaluation of Orphan Products by the U.S. Food and Drug Administration

1992 ◽  
Vol 8 (4) ◽  
pp. 647-657 ◽  
Author(s):  
Marlene E. Haffner ◽  
John V. Kelsey
Keyword(s):  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Marketing Approval ◽  
Drug Products ◽  
Governmental Agencies ◽  
Number Of Patients ◽  
Life Threatening ◽  
The U.S

AbstractOrphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy of orphan drugs. The U.S. Food and Drug Administration (FDA) has not established special criteria for evaluating orphan drugs per se, but the FDA has been flexible in evaluating drug products that present special problems, especially when these products are for treatment of serious of life-threatening illnesses. The FDA and other U.S. governmental agencies also have taken steps to promote the development and availability of drugs for rare diseases, including making these products available to patients who are in need, even before the drugs have full FDA marketing approval.

Orphan Drugs: The Question of Products Liability

1985 ◽  
Vol 10 (4) ◽  
pp. 491-513
Author(s):  
Susan F. Scharf
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Tort Law ◽  
Orphan Drugs ◽  
Products Liability ◽  
Liability Law ◽  
Design Defect ◽  
Liability Costs ◽  
Orphan Drug Development

AbstractOrphan drugs, essential for die treatment of persons widi rare diseases, generally are unprofitable for manufacturers to develop and market. While congressional and administrative efforts to promote die development of orphan drugs have met widi modest success, application of products liability doctrine to orphan drug sponsors could subvert those efforts. This Note describes die provisions of die Orphan Drug Act and analyzes products liability law with respect to orphan drug litigation. It argues that die goals of tort law support the imposition of liability for design defect, failure to warn and negligence in testing. Finally, die Note acknowledges diat liability costs create disincentives for orphan drug development and suggests mechanisms for reducing manufacturers’ liability concerns.

scholarly journals European and Italian regulation on orphan medicinal products

2013 ◽  
Vol 14 (2) ◽  
pp. 89-98
Author(s):  
Roberta Joppi
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Potential Benefit ◽  
Orphan Drugs ◽  
Pharmaceutical Companies ◽  
Drug Status ◽  
Medicinal Products ◽  
Eu Legislation ◽  
Orphan Medicinal Products ◽  
The Eu

The paper presents an overview of the European and Italian Regulation on Orphan Medicinal Products (OMPs), along with some data on the OMPs licensed in the EU from 2000 to 2012. The EU legislation encourages pharmaceutical companies to develop drugs for rare diseases, so-called “orphan drugs”. The European Medicine Agency recognizes orphan drug status mainly on the basis of the prevalence of the disease (≤ 5/10,000), and potential benefit. Orphan status implies incentives for pharmaceutical companies. From 2000 up to 2012 890 candidate orphan drug designations received a positive opinion and the marketing authorization was granted to 72 OMPs corresponding to 80 different indications. Currently, 59 OMPs are available to Italian patients either because licensed to the market by the AIFA or included in the list of the L. 648/96. Despite of an encouraging regulation nearly all the currently estimated rare diseases still await treatments.

scholarly journals Analysis of patient access to orphan drugs in Turkey

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Güvenç Koçkaya ◽  
Sibel Atalay ◽  
Gülpembe Oğuzhan ◽  
Mustafa Kurnaz ◽  
Selin Ökçün ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Anatomical Therapeutic Chemical ◽  
Descriptive Analysis ◽  
Orphan Drugs ◽  
Orphan Drug Status ◽  
European Medicines Agency ◽  
Drug Status ◽  
The European Union ◽  
Early Access

Abstract Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. Methods The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey. Results Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L—Antineoplastic and Immunomodulatory” agents. Conclusion An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

scholarly journals Letter from the Editors

2020 ◽  
Vol 2 ◽  
pp. 1
Author(s):  
Editorial Office
Keyword(s):  
Clinical Trials ◽  
Medical Journal ◽  
Open Access ◽  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Case Reports ◽  
Orphan Drugs ◽  
Original Research ◽  
Orphan Drug Development

Last year we successfully introduced a new journal: The Journal of Rare Diseases and Orphan Drugs (JRDOD) is a peer-reviewed open-access medical journal that publishes original research, reviews, case reports, and letters covering a broad field of its specialty. We intend to publish articles stimulating to read, educate, and inform readers with the most up-to-date research in genetics, rare diseases, and new orphan drug development in different stages of clinical trials. Journal topics are centered on patients living with undiagnosed rare diseases, the importance of a diagnosis, individual approaches to treatments. We hope that this journal will increase awareness of many difficult to diagnosed and treat medical conditions.

Potential Interactions of the Orphan Drug Act and Pharmacogenomics: A Flood of Orphan Drugs and Abuses?

2005 ◽  
Vol 31 (2-3) ◽  
pp. 365-380 ◽  
Author(s):  
David Loughnot
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
The United States ◽  
New Drugs ◽  
United States Government ◽  
Orphan Drug Act ◽  
The Government ◽  
Effective Drugs ◽  
Potential Interactions

To overcome the unattractiveness of small markets, the United States government provides financial aid and incentives for drug manufacturers to create cures for rare diseases under the Orphan Drug Act (“the Act”). Recent research integrating genetic information and pharmacology holds promise for creating more effective drugs targeted at smaller populations than ever before. In the near future, it seems that a flood of new drugs targeted at small disease populations could take advantage of the government benefits under the Act. Drug applicants will include true orphan drugs along with “Trojan” applicants that seek to co-opt the benefits for drugs that should not qualify as orphans. Currently, the FDA appears ill prepared to discern between the two types of applicants and prevent abuse of the system.In 1983, the federal government passed the Act. Congress designed the Act and subsequent modifications to provide incentives for companies to bring drugs for rare diseases to market.

scholarly journals Letter of Editor

2020 ◽  
Vol 1 ◽  
Author(s):  
Editorial Office
Keyword(s):  
Rare Disease ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Publishing House ◽  
Orphan Drugs ◽  
New Drugs ◽  
Medical Community ◽  
Great Promise ◽  
Original Research ◽  
Small Contribution

We are pleased to announce a new addition to SciencePower Publishing House, LLC journal collection: Journal of Rare Diseases and Orphan Drugs (JRDOD), a peer-reviewed open-access medical journal that publishes original research, reviews, case reports, and letters covering a broad field of its specialty. Why do we need a new journal? Because the science moves fast all around the Planet and new technology opened new horizons for the prevention and treatment of rare and genetically based disorders. Rare disease and disorders often need a multidisciplinary care team, deep knowledge of healthcare providers, and extensive patients’ and patient families’ support. It may take years until a disease is recognized and/or diagnosed. Spreading knowledge about rare disease conditions among biomedical scientists and physicians from the different subspecialty fields including genetics, pediatrics, cardiology, pulmonary, gastroenterology, rheumatology, neurology, dietary science, and other related disciplines is one of the goals of this journal, which we will be widely advertised within the medical community. Patients with special conditions could be “Rare, but Not Alone” and we want to add our voice to those patient advocacies. We also want to spread knowledge about orphan drugs. Well known fact that an orphan drug is a medication (pharmaceutical) that remains underdeveloped due to the lack of a company to find the drug profitable because the numbers of patients, who will benefit from the treatment, are small and so the potential market for new drugs to treat these rare diseases is also small. This situation fortunately somewhat changed in 1983, when the U.S. Congress passed the Orphan Drug Act. It facilitated and charged the orphan drugs' developmental landscape. Nowadays the FDA established the Office of Orphan Product Development (OOPD) to help with the development of orphan drugs (and other medical products for rare disorders), including FDA support for research grants. The international medical community has recognized the need to increase research and development of orphan drugs. We hope that our journal will be a small contribution to the promotion of wellbeing of people with rare disease conditions. Our publisher motto is "Superbi progressu aspiramus ad maximum" (the proud aspire to great promise). Please, consider submitting your next manuscript to the Journal of Rare Diseases and Orphan Drugs.

Talking About Rare Diseases

2020 ◽  
pp. 1-86
Author(s):  
Sonia M. Rodrigues Oliveira
Keyword(s):  
Rare Disease ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Financial Incentives ◽  
Orphan Drugs ◽  
Neglected Diseases ◽  
Orphan Diseases ◽  
Health Burden ◽  
Proper Diagnosis ◽  
Orphan Drug Development

Originally, rare diseases and orphan diseases were not synonyms. Rare diseases became known as orphan diseases because pharmaceutical companies were not interested in developing treatments for them. The Orphan Drug Act (USA) used financial incentives for orphan drug development. Herewith, a definition for rare disease was also established. This differs slightly across countries. Some rare diseases respond to drugs that are not orphan drugs. Orphan diseases refer to neglected diseases and even common disorders as endometrial cancer and infantile diabetes. Nonetheless, nowadays very often rare and orphan are used as synonyms. The causes for rare diseases are very diverse. The majority are thought to be genetic. Most lack proper diagnosis, treatment, or known therapeutic targets. However, rare disease patients account for a huge proportion of the health burden. This chapter discusses rare (and orphan) diseases, what has been reported about them, and how they appear to the general public. The different categories of rare diseases are introduced.

scholarly journals Orphan Drug Regulation In Japan and Australia

2020 ◽  
Vol 11 (2) ◽  
pp. 1831-1839
Author(s):  
Nagaraja S ◽  
Balamuralidhara V ◽  
Jyothi M S ◽  
Ragunandhan H V
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Essential Medicine ◽  
Orphan Drugs ◽  
Small Population ◽  
Regulatory Body ◽  
Life Saving ◽  
Current Economic Situation ◽  
New Business ◽  
Pharmaceutical Industries

Due to various reasons, the pharmaceutical industries growth has slowed in recent years. The current economic situation has, therefore, shifted the pharmaceutical companies ' focus from essential medicine to the new business model called "orphan drugs”. Healthcare standards are among the world's highest in Japan. For about 99 percent of its citizens, the Japanese government provides health insurance. Rare diseases fall under ‘intractable diseases (Nanbyo)' in Japan. Japan's regulatory body PMDA is providing all guidance and instructions on orphan drugs. In Australia, Australia’s regulatory body providing all guidance on orphan drugs to the health care system. To be named an orphan drug in Australia, the pharmaceutical company that developed the medication will show to the Orphan Drug Program that the drug is not commercially viable for such a small population of patients. Often, the Orphan Drug System reduces the amount of time it usually takes for the TGA to test a drug. Once a drug is approved as an orphan drug, it is easier for the drug to become available, and it can be obtained through the Life Saving Drugs Program in certain situations. This review study provides comparative study of guidelines for orphan drug and rare diseases regulations in Japan and Australia.

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