Chapter 4. Treating Rare Diseases: Business Model for Orphan Drug Development

2014 ◽  
pp. 83-111
Author(s):  
Cory Williams
Keyword(s):  
Drug Development ◽  
Business Model ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Orphan Drug Development

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 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 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.

scholarly journals New Financial and Research Models for Pediatric Orphan Drug Development: Focus on the NCATS TRND Program

2014 ◽  
Vol 28 (1) ◽  
pp. 1-6 ◽  
Author(s):  
John Shen ◽  
Gurmit Grewal ◽  
Andre M. Pilon ◽  
John C. McKew
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Orphan Drug Development
Sign in / Sign up

Export Citation Format

Share Document