Policy Implications of the Orphan Drug Designation for Remdesivir to Treat COVID-19.
Sign Up to like & getrecommendations! Published in 2020 at "JAMA internal medicine"
DOI: 10.1001/jamainternmed.2020.2759
Abstract: On March 23, 2020, the US Food and Drug Administration (FDA) granted Gilead Science an orphan drug designation for remdesivir to treat coronavirus disease 2019 (COVID-19), even though this status is usually reserved for drugs… read more here.
Keywords: drug; drug designation; orphan drug; designation remdesivir ... See more keywords
Drug Development for Rare Paediatric Epilepsies: Current State and Future Directions
Sign Up to like & getrecommendations! Published in 2019 at "Drugs"
DOI: 10.1007/s40265-019-01223-9
Abstract: Rare diseases provide a challenge in the evaluation of new therapies. However, orphan drug development is of increasing interest because of the legislation enabling facilitated support by regulatory agencies through scientific advice, and the protection… read more here.
Keywords: drug development; development rare; drug; orphan drug ... See more keywords
Opportunities and barriers for innovation and entrepreneurship in orphan drug development
Sign Up to like & getrecommendations! Published in 2020 at "Technological Forecasting and Social Change"
DOI: 10.1016/j.techfore.2020.120333
Abstract: Abstract Orphan diseases pose both a challenge to the global medical community and an opportunity for it to focus on global peace engineering and innovation. Where, any single orphan disease is rare, when taken as… read more here.
Keywords: drug development; orphan drug; field; innovation entrepreneurship ... 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
Drugmakers accused of exploiting orphan drug incentives, fueling price problem
Sign Up to like & getrecommendations! Published in 2017 at "Nature Biotechnology"
DOI: 10.1038/nbt0417-301
Abstract: In January Kaiser Health News published a data-rich report focusing on the increase in approvals of orphan disease drugs, detailing how even high-volume products have reaped the tax breaks and market exclusivity that come with… read more here.
Keywords: drug prices; nat biotechnol; drug; price ... See more keywords
Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH Pave-GT Experience.
Sign Up to like & getrecommendations! Published in 2023 at "Human gene therapy"
DOI: 10.1089/hum.2022.232
Abstract: Orphan drug designation (ODD) is an important program intended to facilitate the development of orphan drugs in the US. An orphan drug benefitting pediatric patients can qualify as a drug for a Rare Pediatric Disease… read more here.
Keywords: orphan drug; gene; rpdd; gene therapy ... See more keywords
Does an Orphan Drug Policy Make a Difference in Access? A Comparison of Canada and Australia
Sign Up to like & getrecommendations! Published in 2019 at "International Journal of Health Services"
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
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
Investigating the landscape of US orphan product approvals
Sign Up to like & getrecommendations! Published in 2018 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-018-0930-3
Abstract: BackgroundThe Orphan Drug Act was enacted in 1983 to encourage the development of drugs for rare diseases. Previous research has attempted to examine the impact of the Act by assessing either the number of orphan… read more here.
Keywords: act; time; landscape; drug ... See more keywords
Estimating the clinical cost of drug development for orphan versus non-orphan drugs
Sign Up to like & getrecommendations! Published in 2019 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-018-0990-4
Abstract: BackgroundHigh orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the… read more here.
Keywords: drug development; drug; orphan drug; non orphan ... See more keywords
How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Pharmacology"
DOI: 10.3389/fphar.2022.902150
Abstract: Introduction: The expansion of orphan drug treatment at increasing prices, together with uncertainties regarding their (cost-)effectiveness raises difficulties for decision-makers to assess these drugs for reimbursement. The present qualitative study aims to gain better insight… read more here.
Keywords: drugs reimbursement; decision makers; value assessment; orphan drug ... See more keywords