scholarly journals Should rare diseases get special treatment?

2021 ◽  
pp. medethics-2021-107691
Author(s):  
Monica Magalhaes
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Drug Policy ◽  
Developing World ◽  
Current System ◽  
Special Treatment ◽  
Common Diseases ◽  
Drug Policies

Orphan drug policy often gives ‘special treatment’ to rare diseases, by giving additional priority or making exceptions to specific drugs, based on the rarity of the conditions they aim to treat. This essay argues that the goal of orphan drug policy should be to make prevalence irrelevant to funding decisions. It aims to demonstrate that it is severity, not prevalence, which drives our judgments that important claims are being overlooked when treatments for severe rare diseases are not funded. It shows that prioritising severity avoids problems caused by prioritising rarity, and that it is compatible with a range of normative frameworks. The implications of a severity-based view for drug development are then derived. The severity-based view also accounts for what is wrong with how the current system of drug development unfairly neglects common diseases that burden the developing world. Lastly, the implications of a severity-based view for current orphan drug policies are discussed.

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.

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 Orphan drug policy

2020 ◽  
Author(s):  
Roshani goel
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Access To Health Care ◽  
Health Care Services ◽  
Developed Countries ◽  
Family Welfare ◽  
Pharmaceutical Companies ◽  
Care Services ◽  
Drug Policies ◽  
The Cost

Millions of people in developing countries do not have access to health care services and medications which are available in developed countries. Most of the rare diseases are genetic in nature. They disproportionately impact children: 50% of new cases are in children and are responsible for 35% of deaths before the age of 1 year, 10% between the ages of 1 and 5 years and 12% between 5 and 15 years (Ministry of Health and Family Welfare, 2017, p.9). One of the main problems is that doctors are not able to study the disease as it is genetic in nature. Despite having the medication, they do not know how to track and treat the disease. However, patients suffering from rare diseases have same rights of care like any other patient. The major concern related to it is of accessibility and awareness. This shows an obvious link between the disease and poverty which raises a serious question on the cost of a treatment which is unaffordable by the patient. Development of Orphan drug policies are least of pharmaceutical companies. They work on the calculation of number of vaccines manufactured on cost of per vaccination. Market is a precious venture for pharmaceutical companies. They are hesitant on the sale of the product. Research done for the development of are diseases and money invested in it will not be recovered by market sales. Thus, government has asked ministries to draft a policy balancing both the issue simultaneously.

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.

scholarly journals Decolonizing drug policy

2021 ◽  
Vol 18 (1) ◽  
Author(s):  
Colleen Daniels ◽  
Aggrey Aluso ◽  
Naomi Burke-Shyne ◽  
Kojo Koram ◽  
Suchitra Rajagopalan ◽  
...  
Keyword(s):  
Drug Policy ◽  
Current System ◽  
Control Policy ◽  
Mass Incarceration ◽  
Drug Control ◽  
People Who Use Drugs ◽  
Power Structures ◽  
Drug Law ◽  
Drug Law Enforcement ◽  
Drug Policies

AbstractThis paper reviews evidence of how drug control has been used to uphold colonial power structures in select countries. It demonstrates the racist and xenophobic impact of drug control policy and proposes a path to move beyond oppressive systems and structures. The ‘colonization of drug control’ refers to the use of drug control by states in Europe and America to advance and sustain the systematic exploitation of people, land and resources and the racialized hierarchies, which were established under colonial control and continue to dominate today. Globally, Black, Brown and Indigenous peoples are disproportionately targeted for drug law enforcement and face discrimination across the criminal system. These communities face higher arrest, prosecution and incarceration rates for drug offenses than other communities, such as majority populations, despite similar rates of drug use and selling among (and between) different races. Current drug policies have contributed to an increase in drug-related deaths, overdoses and sustained transnational criminal enterprises at the expense of the lives of people who use drugs, their families and greater society. This review provides further evidence of the need to reform the current system. It outlines a three-pillared approach to rebuilding drug policy in a way that supports health, dignity and human rights, consisting of: (1) the decriminalization of drugs and their use; (2) an end to the mass incarceration of people who use drugs; (3) the redirection of funding away from ineffective and punitive drug control and toward health and social programs.

scholarly journals Rare Diseases, Drug Development, and AIDS: The Impact of the Orphan Drug Act

1995 ◽  
Vol 73 (2) ◽  
pp. 231 ◽  
Author(s):  
Peter S. Arno ◽  
Karen Bonuck ◽  
Michael Davis
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Orphan Drug Act ◽  
The Impact

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.

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