International agreement that human-relevant research is needed to enhance drug discovery

The average pre-approval cost of research and development for a successful drug is estimated to be US$2.6 billion and the number of new drugs approved per billion US dollars spent has halved roughly every 9 years since 1950. More than 90% of drug candidates entering clinical trials fail to gain regulatory approval, mainly as a result of insufficient efficacy and/or unacceptable toxicity, because of the limited predictive value of preclinical, animal-based studies. Without significant intervention, the pipeline responsible for new drug production is predicted to dry up completely within 50 years.

However, great advances have been made in life science technologies and computer science, increasing our ability to generate and analyze data, and there is a growing recognition that, to improve the success rate, a stronger focus on human-relevant data is needed. Proceedings of a multistakeholder workshop co-organized by The Humane Society of the United States, Humane Society International and the National Institutes of Health under the auspices of the global BioMed21 Collaboration (biomed21.org) is published in Drug Discovery Today, presenting a comprehensive overview of existing efforts to prioritize human-based biology for health research and proposing key recommendations required to revitalize the drug discovery process.

Report co-author Dr Kate Willett, Senior Director for Science and Regulatory Affairs for HSUS and HSI, said: "Through the BioMed21 collaboration, we are stimulating strategic scientific dialogue on regional and global levels, bringing key stakeholders together to explore and develop consensus recommendations around barriers, opportunities, and priorities for future research funding. Improvements to the drug development process are possible, but stakeholders need to work together to shift toward improved understanding of disease pathways and networks in humans, together with continued development and exploitation of human-relevant enabling technologies such as microphysiological systems and computational systems biology."

In 2007, the National Academy of Sciences first articulated how a transition to an approach based on explicit delineation of biological pathways could improve chemical safety assessment. Since then, the Adverse Outcome Pathway (AOP) framework has developed into a central tool for realizing this vision, and beyond. Such a framework could provide a more predictive and effective rubric for understanding disease pathophysiology across levels of biological organization, and for targeting and evaluating new interventions using the growing toolbox of modern, human-specific tools for biomedical research.

The forthcoming publication makes key recommendations for enhancing drug discovery and development including the need for interdisciplinary and international collaboration and cooperativity. Workshop participants - which included experts from 6 NIH institutes, 5 FDA centers, and other key stakeholders - were in agreement that, in order to incentivize global data sharing, there is a need for standardized data and consistent ontologies and that global funding calls should prioritize the human-based methods, such as induced pluripotent stem cells, organoids and organs-on-chips.

Marshall LJ, Austin CP, Casey W, Fitzpatrick SC, Willett C.
Recommendations toward a human pathway-based approach to disease research.
Drug Discov Today. 2018 Jun 2. doi: 10.1016/j.drudis.2018.05.038.

Most Popular Now

Most popular vitamin and mineral supplements provi…

The most commonly consumed vitamin and mineral supplements provide no consistent health benefit or harm, suggests a new study led by researchers at St. Michael's Hospital...

AstraZeneca heads to 2018 ASCO Annual Meeting with…

AstraZeneca and MedImmune, its global biologics research and development arm, head to the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, US...

Tiny particles could help fight brain cancer

Glioblastoma multiforme, a type of brain tumor, is one of the most difficult-to-treat cancers. Only a handful of drugs are approved to treat glioblastoma, and the median ...

New approach to immunotherapy leads to complete re…

A novel approach to immunotherapy developed by researchers at the National Cancer Institute (NCI) has led to the complete regression of breast cancer in a patient who was...

Spiolto® Respimat® enables greater physical activi…

Boehringer Ingelheim announced data which add to the growing body of evidence that show Spiolto® (tiotropium/olodaterol) Respimat® enables greater physical activity in pa...

Amgen Foundation and Harvard team up to offer free…

The Amgen Foundation and Harvard University today announced plans to launch a free online science education platform uniquely designed to level the playing field for aspi...

The Pfizer Foundation announces $5 million in gran…

The Pfizer Foundation announced a new $5 million grant commitment to initiatives in low- and middle-income countries that provide family planning access and education for...

Study finds antioxidant-enriched vitamin reduces r…

Researchers at Children's Hospital Colorado (Children's Colorado) and the University of Colorado School of Medicine have found that taking a specially formulated antioxid...

New drugs could also be deployed against lung and …

A new anti-cancer drug may be effective against a wider range of cancers than previously thought. Using a mouse model and samples taken from cancer patients, a team from ...

What would help or hinder patient participation in…

As clinical trials gear up with the aim of attaining the first FDA-approved treatments for mitochondrial disease, a new study reports for the first time what patients and...

Pfizer to expand venture investing with $600 milli…

Pfizer Inc. (NYSE:PFE) today announced it plans to invest $600 million in biotechnology and other emerging growth companies through Pfizer Ventures, the company’s venture...

Update on Phase III clinical trials of lanabecesta…

AstraZeneca and Eli Lilly and Company (Lilly) are discontinuing the global Phase III clinical trials of lanabecestat, an oral beta secretase cleaving enzyme (BACE) inhibi...