The impact of orphan drug policies in treating rare diseases

2019 ◽  
Vol 36 (2) ◽  
pp. 179-184 ◽  
Author(s):  
Shalini U. Weerasooriya
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Drug Policies ◽  
The Impact

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 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 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.

scholarly journals OP0082-PARE THE EFFECT COVID-19 HAS ON THE MENTAL HEALTH OF PEOPLE LIVING WITH RHEUMATIC DISEASES. FROM DATA TO INTERVENTIONS.

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 44-45
Author(s):  
S. Mingolla ◽  
A. Celano ◽  
M. Santopietro
Keyword(s):  
Mental Health ◽  
Rheumatic Diseases ◽  
Rare Diseases ◽  
Psychological Impact ◽  
Well Being ◽  
Total Sample ◽  
Support Service ◽  
Health Condition ◽  
National Association ◽  
The Impact

Background:Covid-19 has had an important impact on the mental health conditions of over 5 million Italians suffering from one of the over 150 rheumatic diseases. In order to understand the psychological impact of the Covid-19 emergency and the restrictions imposed to counter it, the Italian National Association of People with Rheumatic and Rare Diseases – APMARR APS launched the research “Living with a rheumatic pathology”.Objectives:Gather data directly from Italian patients about the impact of the COVID-19 and consequent restrictions on their mental health and feelings; evaluate the most effective intervention to be implemented to face the pandemic by Patients organization.Methods:A qualitative-quantitative survey was carried out through a questionnaire administered throughout the national territory to a sample of N = 1,001 people. The people invited to complete the questionnaire were women (55,9%) and men (44,1%), aged 18-85 years (age 18-41=26,7%; age 42-65=64%; age >65=9,3%) with at least one rheumatic pathology. The questionnaire was made up of 39 questions, of which 29 were closed and 10 were open. For the administration of the questionnaires, the CAWI (Computer Aided Web Interview) methodology of on-line survey was used. The 1,001 interviews were carried out from 7 to 14 August 2020.Results:More than 4 out of 10 people (total sample 44.2%; male 60%, female 35,7%; age 18-41=39,1%; age 42-65=45,9%; age >65 = 50%) declared that the emergency period has somehow caused a worsening of their health condition. People declared that the deterioration of their health is due to the emergency period for the following reasons: 1) Psychological: such as stress and anxiety: “Too much stress and anxiety made the symptoms worse.”; “The stress of the quarantine affected my problem”; “Insomnia. Nervousness. General ailments. Depression. Strong stress” 2) Inability to perform physiotherapy and motor activities due to the lockdown 3) Postponement of examinations, visits and checks 4) remote working, in some cases described as harmful for people’s mental and physical health: “Due to Covid19 I had to do remote working and I worked even 12 hours a day including holidays to the detriment of my family life”.Furthermore, from January 31, 2020 a significant increase emerged in communication problems with rheumatology specialist compared to the period before the emergency due to Covid-19. The sharp increase may be due to the situation of severe psychological stress to which also the doctors were subjected in the emergency phase: people could not find the comfort of being empathically listened to.Conclusion:The research shows that the most frequent symptoms among people with rheumatic diseases were depression and high levels of anxiety due to strong emotional stress. Psychological malaise caused direct effects in worsening the symptoms of rheumatic disease as well as other related effects, for example, insomnia. The forced isolation due to the lockdown has made people lack the social support that is fundamental for the psychological well-being especially for those suffering from some chronic pathology. Starting from the data collected, APMARR promptly activated a completely free psychological support service with 6 professional psychologists, two of them specialized in emergency psychology. The service is accessible online and is still going on for all who are not able to overcome the anxiety and fear related to the pandemic and its evolution. Thousands of accesses to the service have been measured to date.References:S Mingolla1, A Celano1, M Santopietro2[1]NATIONAL ASSOCIATION OF PEOPLE WITH RHEUMATIC AND RARE DISEASES - APMARR APS[2]WeResearch. Ricerche di marketingDisclosure of Interests:None declared

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.

Dare voce ai pazienti nella ricerca sulle malattie rare e sui famaci orfani / Giving patients a voice in the rare diseases and orphan drugs research

2018 ◽  
Vol 67 (1) ◽  
pp. 25-40
Author(s):  
Elena Mancini ◽  
Roberta Martina Zagarella
Keyword(s):  
Clinical Trials ◽  
Rare Diseases ◽  
Patient Reported Outcomes ◽  
Narrative Medicine ◽  
Orphan Drugs ◽  
New Drugs ◽  
Patient Centered ◽  
Patient Reported ◽  
Critical Issues ◽  
The Impact

L’articolo ha l’obiettivo di mettere in luce potenzialità e criticità dell’inclusione della prospettiva dei pazienti nella ricerca sulle malattie rare e sui farmaci orfani. A tal fine, nella prima parte, si propone un’analisi epistemologica dell’utilizzo dei racconti dell’esperienza individuale della malattia nella ricerca scientifica e nei trial clinici, facendo emergere, anche attraverso gli strumenti della medicina narrativa, le sfide teoriche e operative poste dall’inclusione della soggettività del paziente e del vissuto di malattia nonché l’importanza della valorizzazione della prospettiva del paziente, sia in generale sia nella ricerca sulle malattie rare e sui farmaci orfani. Nella seconda parte, il testo analizza in particolare il ruolo degli esiti riportati dai pazienti o Patient Reported Outcomes (PROs), misure per la valutazione complessiva della salute basate sulla prospettiva dei pazienti stessi, incentrandosi sulla sperimentazione clinica nel campo delle malattie rare. In questo contesto, infatti, i racconti di malattia, raccolti e valorizzati da fonti istituzionali e associazioni di pazienti, hanno contribuito a far emergere importanti questioni critiche e difficoltà nell’impiego di outcome centrati sul paziente nello sviluppo di nuovi farmaci e trattamenti, generando una serie di documenti e raccomandazioni relative al loro utilizzo per il benessere della comunità dei malati rari. ---------- This paper aims to highlight the potentiality and criticality of including patients’ perspective in rare diseases and orphan drugs research. In the first part, we propose an epistemological analysis of individual narrations of disease experience as they are used in scientific research and clinical trials. With the help of narrative medicine approach, this analysis points out theoretical and operational challenges of a perspective that includes patient’s subjectivity and illness experience. Furthermore, it reveals the significance of patients’ standpoints in general and in rare diseases as well as in the orphan drugs research. The second part of our article focuses on the role of the Patient reported Outcomes (PROs) – which are measures for the health’s overall assessment based on patient’s perspective – by investigating the impact on clinical trials for rare diseases. In this context, illness stories, which are collected and promoted by institutional sources and patients’ associations, contribute to underline important critical issues at stake in the employment of patient-centered outcomes both in new drugs and in the treatments development. Moreover, these stories are crucial to elaborate documents and recommendations concerning the use of PROs for the rare patients’ community welfare.

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