Orphan Drugs aus Sicht der forschenden Pharma- und Biotech-Firmen
Sign Up to like & getrecommendations! Published in 2019 at "Der Internist"
DOI: 10.1007/s00108-019-0579-6
Abstract: Bei dem vorliegenden Beitrag handelt es sich um eine Stellungnahme des Verbands forschender Pharma-Unternehmen. Weitere Sichtweisen zu diesem Thema finden Sie in den folgenden Beiträgen: 4 W.-D. Ludwig (2019) Orphan Drugs aus Sicht der Arzneimittelkommission… read more here.
Keywords: drugs aus; der forschenden; orphan drugs; sicht der ... See more keywords
Molecular similarity considerations in the licensing of orphan drugs.
Sign Up to like & getrecommendations! Published in 2017 at "Drug discovery today"
DOI: 10.1016/j.drudis.2016.11.024
Abstract: The large costs associated with modern drug discovery mean that governments and regulatory bodies need to provide economic incentives to promote the development of orphan drugs (i.e., medicinal products that are designed to treat rare… read more here.
Keywords: licensing orphan; similarity; similarity considerations; orphan drugs ... See more keywords
International differences in patient access to ultra-orphan drugs
Sign Up to like & getrecommendations! Published in 2017 at "Health policy and technology"
DOI: 10.1016/j.hlpt.2017.12.001
Abstract: Abstract Objectives Reimbursement recommendations on (orphan) drugs are usually made at a national level and this can lead to variation in patient access to the same drug in different countries. We compared differences in patient… read more here.
Keywords: status; orphan; orphan drugs; patient access ... See more keywords
Premarket safety population size associated with approval of expedited program drugs and orphan drugs.
Sign Up to like & getrecommendations! Published in 2019 at "Journal of clinical epidemiology"
DOI: 10.1016/j.jclinepi.2019.04.021
Abstract: OBJECTIVE For approved expedited program and orphan drugs, the safety populations used for premarket drug risk assessments are often limited in size. The study's objective was to determine whether large safety populations, with the capacity… read more here.
Keywords: expedited program; safety populations; safety; orphan drugs ... See more keywords
Orphan drugs—access and unmet needs in 194 countries and six regions: a comprehensive policy review with content analysis
Sign Up to like & getrecommendations! Published in 2019 at "The Lancet"
DOI: 10.1016/s0140-6736(19)32408-0
Abstract: Abstract Background Orphaned because of low prevalence and barely recoverable medical costs, patients with rare diseases are disproportionately deprived of life-saving treatment. In China, an estimated 16·8 million patients suffer from rare diseases. This substantial… read more here.
Keywords: review; drug; access; policy ... See more keywords
VP55 Health Technology Assessment Of Orphan Drugs: The Case Of Hereditary Angioedema In Italy
Sign Up to like & getrecommendations! Published in 2017 at "International Journal of Technology Assessment in Health Care"
DOI: 10.1017/s026646231700335x
Abstract: INTRODUCTION: The evaluation of orphan drugs raises both positive and normative issues. Due to the rarity of the disease, higher drug costs and greater uncertainty on effectiveness often result in incremental cost-effectiveness ratios (ICER) far… read more here.
Keywords: technology assessment; orphan drugs; hereditary angioedema; health ... See more keywords
Methods and criteria for the assessment of orphan drugs: a scoping review
Sign Up to like & getrecommendations! Published in 2022 at "International Journal of Technology Assessment in Health Care"
DOI: 10.1017/s0266462322000393
Abstract: Abstract Objectives This study aimed to identify different criteria for priority setting of rare diseases to help policy makers in making evidence-informed decisions. Methods A scoping review was conducted to comprehensively examine the existing various… read more here.
Keywords: methods criteria; scoping review; orphan drugs; priority setting ... See more keywords
Novel approach to decision making for orphan drugs
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DOI: 10.1017/s0266462323000053
Abstract: Abstract Background Out of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily… read more here.
Keywords: health; methodology; novel approach; orphan drugs ... See more keywords
Does an Orphan Drug Policy Make a Difference in Access? A Comparison of Canada and Australia
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DOI: 10.1177/0020731419886526
Abstract: Canada has been discussing whether to implement an orphan drug policy for more than 25 years. Recently, the federal government announced funding for orphan drugs starting in 2022, and the Canadian Senate has recommended that… read more here.
Keywords: policy; canada; drug policy; orphan drugs ... See more keywords
Rare pulmonary disease and orphan drugs: a path to the future
Sign Up to like & getrecommendations! Published in 2019 at "European Respiratory Review"
DOI: 10.1183/16000617.0115-2019
Abstract: In this issue of the European Respiratory Review (ERR), we are delighted to publish a series of articles on rare pulmonary diseases discussing hot topics presented at the 8th International Meeting on Pulmonary Rare Diseases… read more here.
Keywords: rare pulmonary; disease orphan; orphan drugs; european respiratory ... See more keywords
Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?
Sign Up to like & getrecommendations! Published in 2017 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-016-0551-7
Abstract: BackgroundMore than 6,800 rare diseases and conditions have been identified in the US, which affect 25–30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing… read more here.
Keywords: rare diseases; orphan drugs; drug; orphan drug ... See more keywords