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

Chemists characterize the fatal fungus among us

Life-threatening fungal infections affect more than two million people worldwide. Effective antifungal medications are very limited. Until now, one of the major challenge...

FDA approves first cancer drug through new oncolog…

The U.S. Food and Drug Administration today approved Kisqali (ribociclib) in combination with an aromatase inhibitor for the treatment of pre/perimenopausal or postmenopa...

Discovery of kidney cancer driver could lead to ne…

University of North Carolina Lineberger Comprehensive Cancer Center scientists have uncovered a potential therapeutic target for kidney cancers that have a common genetic...

FDA approves first targeted treatment for patients…

The U.S. Food and Drug Administration today approved Tibsovo (ivosidenib) tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (...

Women and older people under-represented in drug t…

Trying to determine how best to treat a patient, doctors often look to randomized clinical trials to guide their choice of what drug to prescribe. One of the most common ...

Novartis renews drug donation of Egaten® (triclabe…

Novartis reaffirms its commitment to the fight against liver fluke (fascioliasis), signing a renewed memorandum of understanding with the World Health Organization (WHO) ...

Novartis marks a new era for migraine patients wit…

Novartis announced today that the European Commission (EC) approved Aimovig® (erenumab) for the prevention of migraine in adults experiencing four or more migraine days p...

Sanofi and REVOLUTION Medicines launch global part…

Sanofi and REVOLUTION Medicines, Inc. today announced an exclusive worldwide partnership to develop and commercialize targeted therapies, based on the biology of the cell...

Mushrooms of the Far East hold promise for the ant…

Mushrooms from the Far East area contain the natural chemical compounds, which could be used for the design of the novel drugs with highly specific anti-tumor activities ...

FDA grants Breakthrough Therapy Designation for Ro…

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tecentriq® (atezolizumab) i...

New findings suggest allergic responses may protec…

The components of the immune system that trigger allergic reactions may also help protect the skin against cancer, suggest new findings. The research, led by Imperial Col...

Atacand to be divested to Cheplapharm in Europe

AstraZeneca has agreed to sell the commercial rights to Atacand (candesartan cilexetil) and Atacand Plus (fixed-dose combination of candesartan cilexetil and hydrochlorot...