The Current Status of Drug Discovery and Development as Originated in <scp>United States</scp> Academia: The Influence of Industrial and Academic Collaboration on Drug Discovery and Development

Takebe, Tohru; Imai, Ryoka; Ono, Shunsuke

doi:10.1111/cts.12577

Cited by 179 publications

(109 citation statements)

References 32 publications

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“…Some of the drugs developed by large companies that originate from small companies, including start-ups and academia, may tend to have a higher probability of success. 23 The number of patients was negatively associated with a likelihood of approval (Table 2); that is, designated orphan drugs with fewer patients were more likely to be approved. This may conflict with our general intuition that difficulties in clinical development increase in disease areas with a small number of patients.…”

Section: Discussionmentioning

confidence: 99%

Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval

Harada

Toriyabe

Ono

2019

The Journal of Clinical Pharma

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The basic components of regulatory and supporting policies for orphan drug development appear similar between the United States and Japan, but drugs designated as orphan drugs have been different between the 2 countries. The probabilities of development success (ie, marketing approval) in designated orphan drugs have also been significantly different. In this study, we analyzed recent outcomes of development for orphan drugs designated from 1993 to 2017 in Japan, considering their development and approval status in the United States. Our analysis showed that success for orphan drug development in Japan was apparently associated with prior approval status in the United States. Company size, orphan development experience, and patient enrichment were also positively associated with successful marketing approval. Although similar designations and priority review systems for orphan drugs have been enacted, economic incentives and regulatory conditions provided by the systems seem to be different between the 2 countries, which may lead to varied performance in orphan designation and approval. We need to pay close attention to the impact of industrial global development strategies when comparing the outcomes and performance of different orphan drug promotion systems.

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Section: Discussionmentioning

confidence: 99%

Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval

Harada

Toriyabe

Ono

2019

The Journal of Clinical Pharma

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“…As the complexity of biomedical research questions has increased, so too has the need to bring together expertise and resources from multiple disciplines and organizations (Cooke et al, 2015). Consequently, several articles by thought leaders have called for more collaboration in the drug development process (Altshuler et al, 2010;Dahlin et al, 2015;Alteri and Guizzaro, 2018;Takebe et al, 2018;Chaturvedula et al, 2019).…”

Section: Drug Discovery Consortiamentioning

confidence: 99%

Accelerating Therapeutics for Opportunities in Medicine: A Paradigm Shift in Drug Discovery

Hinkson¹,

Madej²,

Stahlberg³

2020

Front. Pharmacol.

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Conventional drug discovery is long and costly, and suffers from high attrition rates, often leaving patients with limited or expensive treatment options. Recognizing the overwhelming need to accelerate this process and increase success, the ATOM consortium was formed by government, industry, and academic partners in October 2017. ATOM applies a team science and open-source approach to foster a paradigm shift in drug discovery. ATOM is developing and validating a precompetitive, preclinical, small molecule drug discovery platform that simultaneously optimizes pharmacokinetics, toxicity, protein-ligand interactions, systems-level models, molecular design, and novel compound generation. To achieve this, the ATOM Modeling Pipeline (AMPL) has been developed to enable advanced and emerging machine learning (ML) approaches to build models from diverse historical drug discovery data. This modular pipeline has been designed to couple with a generative algorithm that optimizes multiple parameters necessary for drug discovery. ATOM's approach is to consider the full pharmacology and therapeutic window of the drug concurrently, through computationally-driven design, thereby reducing the number of molecules that are selected for experimental validation. Here, we discuss the role of collaborative efforts such as consortia and public-private partnerships in accelerating cross disciplinary innovation and the development of opensource tools for drug discovery.

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“…Some studies may be conducted in people with the target disease if the mechanism of action is specific to the condition, e.g., chronic endobronchial infection with Pseudomonas aeruginosa in people with CF (16). Up to 75% of drug candidates move on to Phase II trials (17).…”

Section: Clinical Developmentmentioning

confidence: 99%

Drug Development for Cystic Fibrosis

Sanders

Chmiel

2020

Preprint

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The first drug specific for cystic fibrosis (CF) was approved in 1993, and since then several other drugs have been approved. Median predicted survival in people with CF has improved from approximately 30 years to 44.4 years over that same period. In 2020, highly-effective modulators of the cystic fibrosis transmembrane conductance regulator became available to approximately 90% of people with CF ages 12 years and older. These transformative therapies will surely reduce morbidity and further extend longevity. The drug development pipeline is filled with therapies that address most aspects of CF disease. As survival and CF therapies have advanced, and the complexity of CF care increases, the process of drug development has become more sophisticated. In addition, detecting meaningful changes in clinical outcomes has become more difficult as the health status of people with CF improves. Design of clinical trials in CF has become more complex, and innovative approaches are required to continue to advance drug development. This review provides a general overview of drug development from the pre-clinical phase through Phase IV. Special considerations with respect to CF are integrated into the discussion of each phase of drug development. The dawn of a new era has arrived for people with CF. As CF care evolves, drug development must continue to evolve as well, until a one-time cure is available to all people with CF.

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The Current Status of Drug Discovery and Development as Originated in United States Academia: The Influence of Industrial and Academic Collaboration on Drug Discovery and Development

Cited by 179 publications

References 32 publications

Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval

Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval

Accelerating Therapeutics for Opportunities in Medicine: A Paradigm Shift in Drug Discovery

Drug Development for Cystic Fibrosis

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