Optimal Cost-Effective Go–No Go Decisions in Clinical Development

Author(s):  
Cong Chen ◽  
Robert A. Beckman ◽  
Linda Z. Sun
2016 ◽  
Vol 1 (1) ◽  
pp. 14
Author(s):  
Lynn Marks

As the economic pressures increase on healthcare systems around the world due to aging populations, chronic diseases, expanding patient populations in emerging markets and advances in medical technology, it is crucial that we focus on developing and delivering innovative and quality medicines with true medical value to patients around the world in a more collaborative, quality-focused and cost-effective manner.An important component to this mission across the biopharmaceutical industry is identifying and solving common issues that compromise the success of a clinical development program – the shared pathway to safer and more clinically meaningful medicines. However, subject recruitment challenges, data collection and follow-up issues, identification of high-quality trial sites, and lack of successfully achieving study timelines continue to stress clinical trial operations teams across companies. Although there has been progressing across this range of roadblocks by individual companies, the underlying economics continue to threaten the research and development (R&D) business model.Failure to solve these key issues will affect all parties involved in the clinical trial enterprise: patients, clinical inves-tigators, health authorities, academia, tax-payers and the sponsor companies. The question remains whether a deep and broad collaborative effort that stretches across the clinical development arena—one that is charged with a common goal of improving quality, enhancing the investigator and patient experience, reducing costs and sharing data—can be a catalyst for success. With encouraging signs already realised, the operation of TransCelerate BioPharma Inc., a non-profit organisation created to improve the health of people around the world by accelerating and enhancing the R&D of innovative new therapies will test this premise.


2021 ◽  
Vol 8 (3) ◽  
pp. 579-587
Author(s):  
Parisa Sabbagh

In this article, a new optimal cost-effective data carrier architecture for smart contracts in the blockchain-enabled IoT environment proposed. This method requires communication with external off-chain data. Three steps consist of this model such as Mission Manager, Task Publisher, and Worker which formulated with the ACO algorithm. Based on this method, some of the best solutions for filtering smart contract events and decoding event logs to fit different requirements are presented adequately. The proposed system is designed to minimize contract deployment costs and monitor contract events without subscribing to any filter at the Ethereum node. In the evaluation, we show that it will save about 21USD deployment costs for average by our data carrier system.


2021 ◽  
Author(s):  
Moataz Dowaidar

Recent breakthroughs in clinical research and deployment of RNAi therapeutics have validated siRNA's promise to cure human diseases. RNAi therapy has been proven to effectively alter the expression of human-related target genes, including cancer. It has the potential to regulate oncogenes not addressed by standard treatment, such as oncogenic lncRNAs, to treat cancer more successfully. Due to their intrinsic liver affinity, successful RNAi therapies in clinical development primarily target liver diseases. Systemic dispersion of therapeutic siRNAs is an effective cancer therapy technique, especially for advanced diseases. Despite recent advances in siRNA delivery technologies, a problem remains with efficiently distributing siRNAs into solid tumors and cancer cells. To overcome several challenges to the dispersion of siRNA in cancer cells' cytoplasm, novel and highly effective delivery systems need to be devised. Delivery devices' ability to sense and adjust to environmental changes throughout the delivery process can make cytosolic distribution into cancer cells more efficient and accurate. Due to their well-defined and simple chemical structures and their multifunctionalities to respond to environmental changes to facilitate efficient cytosolic transport, environment-responsive lipids are promising platforms for clinical development to deliver siRNA. The primary benefit of simple, well-defined lipid structures over complex systems for cost-effective CMC and clinical translation is the simple, well-defined lipid structures. Environment-responsive lipids might be considered as simple, clever siRNA delivery methods that can overcome delivery difficulties for successful cancer treatment.


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