scholarly journals Current Status and Trend of Clinical Development of Orphan Drugs in China

2021 ◽  
Author(s):  
Ziling Xiang ◽  
Wengao Jiang ◽  
Bo Yan ◽  
Junhao Jiang ◽  
Hang Zheng
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
Clinical Development ◽  
Current Status ◽  
Policy Makers ◽  
Critical Issues ◽  
Low Coverage ◽  
Orphan Drug Development

Abstract Background:Rare diseases have been increasingly recognized as medical and healthy burden worldwide, a growing demand for the development of orphan drugs emerges subsequently. Therefore, it is of great interest for both the regulatory agency and pharmaceutical companies to keep tract on the clinical orphan drug development in China.Objective and Method:This study aims to reveal the current situation and trend of the clinical development of orphan drugs in China, based on the data collected from the Platform for Drug Clinical Trials and Information Registration（http://www.chinadrugtrials.org.cn）of China Food and Drug Administration, dating from 2013 to March 8, 2021.Results:A total of 246 clinical trials for orphan drugs are extracted from the Platform, covering 22 rare diseases and 90 drugs. Among the 22 rare diseases, 3 (14 %) have more than 50 trials each , 17 (77%) had less than10 trials, and 10 (46%) only with one trial. Among 90 orphan drugs, 60 (67%) were chemical drugs, and 30 (33 %) were biological products. In addition, international multi-center trials accounts for nearly 10% of the total trials. The number of the trials with the Data Monitoring Security Committee (DMC) is 25 (10%) and the number of the trials with the trial injury insurance for subjects is 154 (63%). Furthermore, more than half of the total trials are carried in east (333, 30%) and north China (298, 27%), whereas a small portion are in the northwest (62, 6%) and northeast china (45, 4%).Conclusions:The clinical development of orphan drugs for rare diseases in China has made some progress in the passing decades. However, a couple of critical issues still need to be addressed, such as unmet needs for some rare diseases, low coverage of insurance and DMC, and uneven distribution of medical resources for clinical researches. Recommendations are put forward accordingly, which can provide improvement goals for policy makers and stakeholders involved in drug development for rare diseases.

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.

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.

scholarly journals Advances in Antifungal Drug Development: An Up-To-Date Mini Review

2021 ◽  
Vol 14 (12) ◽  
pp. 1312
Author(s):  
Ghada Bouz ◽  
Martin Doležal
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Clinical Development ◽  
Antifungal Drug ◽  
Current Status ◽  
Special Focus ◽  
High Toxicity ◽  
Clinical Stages ◽  
Promising Treatment ◽  
Novel Targets

The utility of clinically available antifungals is limited by their narrow spectrum of activity, high toxicity, and emerging resistance. Antifungal drug discovery has always been a challenging area, since fungi and their human host are eukaryotes, making it difficult to identify unique targets for antifungals. Novel antifungals in clinical development include first-in-class agents, new structures for an established target, and formulation modifications to marketed antifungals, in addition to repurposed agents. Membrane interacting peptides and aromatherapy are gaining increased attention in the field. Immunotherapy is another promising treatment option, with antifungal antibodies advancing into clinical trials. Novel targets for antifungal therapy are also being discovered, allowing the design of new promising agents that may overcome the resistance issue. In this mini review, we will summarize the current status of antifungal drug pipelines in clinical stages, and the most recent advancements in preclinical antifungal drug development, with special focus on their chemistry.

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.

scholarly journals Trends of Clinical Trials for Drug Development in Rare Diseases

2018 ◽  
Vol 13 (3) ◽  
pp. 199-208
Author(s):  
Ryuichi Sakate ◽  
Akiko Fukagawa ◽  
Yuri Takagaki ◽  
Hanayuki Okura ◽  
Akifumi Matsuyama
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Diseases

Development of Orphan Drugs under European Regulatory Incentives and Patent Protection

2017 ◽  
Vol 24 (3) ◽  
pp. 239-263 ◽  
Author(s):  
Mari Minn
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Patent Protection ◽  
Market Access ◽  
Orphan Drugs ◽  
Patient Access ◽  
Generic Market ◽  
European Regulatory ◽  
Orphan Drug Development ◽  
Regulatory Incentives

AbstractThis article analyses how the regulatory incentives provided by Regulation 141/2000 affect orphan drug development and generic market entry. It seeks to find out how the regulatory incentives work in combination with patent protection, and whether in combination they foster orphan drug innovation and overall patient access, or rather hinder the latter. The article argues that even though the regulatory incentives are targeted to fostering innovation and early generic market access, when combined with patent protection, the generic entry is likely blocked or delayed.

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