Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities

2021 ◽  
Author(s):  
Bilal Abuasal ◽  
Mariam A. Ahmed ◽  
Priyank Patel ◽  
Salwa Albusaysi ◽  
Sreedharan Sabarinath ◽  
...  
Keyword(s):  
Clinical Pharmacology ◽  
Drug Development ◽  
Rare Diseases

Chronic Cannabis Users: A New Special Population to Consider for Drug Development

2021 ◽  
Vol 16 ◽  
Author(s):  
Gina Pastino ◽  
Diana Shuster
Keyword(s):  
Clinical Pharmacology ◽  
Drug Development ◽  
Cannabis User ◽  
Special Population ◽  
Safety And Efficacy ◽  
New Drug ◽  
Metabolic Interactions ◽  
Widespread Availability ◽  
Baseline Characteristics ◽  
Efficacy Profile

The use and acceptance of cannabis, either medically or recreationally, has substantially outpaced the collection of data necessary to evaluate its use in any population. However, the mere widespread availability does not imply the absence of risk or confirmation of efficacy and should not be treated as such. There is enough data to suggest that not only does the potential for pharmacokinetic and metabolic interactions exist, but also that baseline characteristics for a given population could be different in chronic cannabis users. Either or both of these may impact the safety and efficacy profile for any new drug in development. As such, we encourage drug developers to consider that the cannabis user may very well be a special population that warrants its own clinical pharmacology evaluation.

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.

Crowdfunding drug development: the state of play in oncology and rare diseases

2014 ◽  
Vol 19 (11) ◽  
pp. 1775-1780 ◽  
Author(s):  
Nick Dragojlovic ◽  
Larry D. Lynd
Keyword(s):  
Drug Development ◽  
Rare Diseases ◽  
The State

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

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.

Clinical Pharmacology Modeling and Simulation in Drug Development

2021 ◽  
pp. 243-256
Author(s):  
Ann-Charlotte Egnell ◽  
Susanne Johansson ◽  
Chao Chen ◽  
Alienor Berges
Keyword(s):  
Clinical Pharmacology ◽  
Drug Development ◽  
Modeling And Simulation

Role of Guidance and Policy in Enhancing the Impact of Clinical Pharmacology in Drug Development, Regulation, and Use

2020 ◽  
Vol 108 (4) ◽  
pp. 710-715
Author(s):  
Rajanikanth Madabushi ◽  
Elimika Pfuma Fletcher ◽  
Jeffry Florian ◽  
Lauren Milligan ◽  
Anuradha Ramamoorthy ◽  
...  
Keyword(s):  
Clinical Pharmacology ◽  
Drug Development ◽  
The Impact ◽  
Development Regulation

Orphan drug development: the increasing role of clinical pharmacology

2019 ◽  
Vol 46 (5) ◽  
pp. 395-409 ◽  
Author(s):  
Mariam A. Ahmed ◽  
Malek Okour ◽  
Richard Brundage ◽  
Reena V. Kartha
Keyword(s):  
Clinical Pharmacology ◽  
Drug Development ◽  
Orphan Drug ◽  
Orphan Drug Development
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