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 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.

Development of Orphan Drugs under European Regulatory Incentives and Patent Protection

2017 ◽  
Vol 24 (3) ◽  
pp. 239-263 ◽  
Author(s):  
Mari Minn
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Patent Protection ◽  
Market Access ◽  
Orphan Drugs ◽  
Patient Access ◽  
Generic Market ◽  
European Regulatory ◽  
Orphan Drug Development ◽  
Regulatory Incentives

AbstractThis article analyses how the regulatory incentives provided by Regulation 141/2000 affect orphan drug development and generic market entry. It seeks to find out how the regulatory incentives work in combination with patent protection, and whether in combination they foster orphan drug innovation and overall patient access, or rather hinder the latter. The article argues that even though the regulatory incentives are targeted to fostering innovation and early generic market access, when combined with patent protection, the generic entry is likely blocked or delayed.

scholarly journals Rare Genetic Diseases with Defects in DNA Repair: Opportunities and Challenges in Orphan Drug Development for Targeted Cancer Therapy

Cancers ◽  
2018 ◽  
Vol 10 (9) ◽  
pp. 298 ◽  
Author(s):  
Sonali Bhattacharjee ◽  
Saikat Nandi
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Genome Stability ◽  
Rapid Development ◽  
Genetic Diseases ◽  
Cancer Therapeutics ◽  
Disease Genes ◽  
Synthetic Lethal ◽  
Orphan Drug Development

A better understanding of mechanistic insights into genes and enzymes implicated in rare diseases provide a unique opportunity for orphan drug development. Advances made in identification of synthetic lethal relationships between rare disorder genes with oncogenes and tumor suppressor genes have brought in new anticancer therapeutic opportunities. Additionally, the rapid development of small molecule inhibitors against enzymes that participate in DNA damage response and repair has been a successful strategy for targeted cancer therapeutics. Here, we discuss the recent advances in our understanding of how many rare disease genes participate in promoting genome stability. We also summarize the latest developments in exploiting rare diseases to uncover new biological mechanisms and identify new synthetic lethal interactions for anticancer drug discovery that are in various stages of preclinical and clinical studies.

Orphan drugs: trends and issues in drug development

2018 ◽  
Vol 29 (5) ◽  
pp. 437-446 ◽  
Author(s):  
Proteesh Rana ◽  
Shalini Chawla
Keyword(s):  
Pharmaceutical Industry ◽  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Drug Market ◽  
Medical Needs ◽  
Common Diseases ◽  
Pharmaceutical Industries ◽  
The Government ◽  
Orphan Drug Development

Abstract Research in rare diseases has contributed substantially toward the current understanding in the pathophysiology of the common diseases. However, medical needs of patients with rare diseases have always been neglected by the society and pharmaceutical industries based on their small numbers and unprofitability. The Orphan Drug Act (1983) was the first serious attempt to address the unmet medical needs for patients with rare diseases and to provide impetus for the pharmaceutical industry to promote orphan drug development. The process of drug development for rare diseases is no different from common diseases but involves significant cost and infrastructure. Further, certain aspect of drug research may not be feasible for the rare diseases. The drug-approving authority must exercise their scientific judgment and ensure due flexibility while evaluating data at various stages of orphan drug development. The emergence of patent cliff combined with the government incentives led the pharmaceutical industry to realize the good commercial prospects in developing an orphan drug despite the small market size. Indeed, many drugs that were given orphan designation ended up being blockbusters. The orphan drug market is projected to reach $178 billion by 2020, and the prospects of research and development in rare diseases appears to be quite promising and rewarding.

scholarly journals Current Status and Trend of Clinical Development of Orphan Drugs in China

2021 ◽  
Author(s):  
Ziling Xiang ◽  
Wengao Jiang ◽  
Bo Yan ◽  
Junhao Jiang ◽  
Hang Zheng
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
Clinical Development ◽  
Current Status ◽  
Policy Makers ◽  
Critical Issues ◽  
Low Coverage ◽  
Orphan Drug Development

Abstract Background:Rare diseases have been increasingly recognized as medical and healthy burden worldwide, a growing demand for the development of orphan drugs emerges subsequently. Therefore, it is of great interest for both the regulatory agency and pharmaceutical companies to keep tract on the clinical orphan drug development in China.Objective and Method:This study aims to reveal the current situation and trend of the clinical development of orphan drugs in China, based on the data collected from the Platform for Drug Clinical Trials and Information Registration（http://www.chinadrugtrials.org.cn）of China Food and Drug Administration, dating from 2013 to March 8, 2021.Results:A total of 246 clinical trials for orphan drugs are extracted from the Platform, covering 22 rare diseases and 90 drugs. Among the 22 rare diseases, 3 (14 %) have more than 50 trials each , 17 (77%) had less than10 trials, and 10 (46%) only with one trial. Among 90 orphan drugs, 60 (67%) were chemical drugs, and 30 (33 %) were biological products. In addition, international multi-center trials accounts for nearly 10% of the total trials. The number of the trials with the Data Monitoring Security Committee (DMC) is 25 (10%) and the number of the trials with the trial injury insurance for subjects is 154 (63%). Furthermore, more than half of the total trials are carried in east (333, 30%) and north China (298, 27%), whereas a small portion are in the northwest (62, 6%) and northeast china (45, 4%).Conclusions:The clinical development of orphan drugs for rare diseases in China has made some progress in the passing decades. However, a couple of critical issues still need to be addressed, such as unmet needs for some rare diseases, low coverage of insurance and DMC, and uneven distribution of medical resources for clinical researches. Recommendations are put forward accordingly, which can provide improvement goals for policy makers and stakeholders involved in drug development for rare diseases.

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 The role of globalization in drug development and access to orphan drugs: orphan drug legislation in the US/EU and in Latin America

F1000Research ◽  
2015 ◽  
Vol 4 ◽  
pp. 57 ◽  
Author(s):  
Renée J.G. Arnold ◽  
Lida Bighash ◽  
Alejandro Bryón Nieto ◽  
Gabriela Tannus Branco de Araújo ◽  
Juan Gabriel Gay-Molina ◽  
...  
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Latin American ◽  
Rare Diseases ◽  
Market Access ◽  
Orphan Drugs ◽  
The United States ◽  
Drug Status ◽  
Drug Legislation ◽  
The Us

Compared to a decade ago, nearly three times as many drugs for rare diseases are slated for development. This article addresses the market access issues associated with orphan drug status in Europe and the United States in contrast to the legislation in five Latin American (LA) countries that have made strides in this regard--Mexico, Brazil, Colombia, Chile and Argentina. Based on the success of orphan drug legislation in the EU and US, LA countries should strive to adopt similar strategies with regard to rare diseases and drug development. With the implementation of new targeted regulations, reimbursement strategies, and drug approvals, accessibility to treatment will be improved for people afflicted with rare diseases in these developing countries.

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