Phase-0 Microdosing Network

Enabling Human-Based Translation in Drug Development

New York, NY   |  https://www.phase-0Microdosing.org

Mission

The Phase-0/Microdosing Network is a non-profit organization, a consortium dedicated to the education and research of Phase-0 approaches. Phase-0 approaches, which include microdosing, are novel drug development approaches that allow testing of new drugs in humans using smaller doses than traditional approaches. Because of that Phase-0 approaches are safer to the human volunteers who participate in their testing, are quicker and cheaper to test, are consistent with the 3R's of animal research, leading to acceleration of drug development and reduction of the use of animals in research. This international consortium includes stakeholders who are experts in the field or who have interest in the safety, effectiveness, and productivity of drug development, and/or the protection of animals.

Ruling year info

2019

President/CEO

Dr. Tal Burt

Professor of Clinical Pharmacology and pediatric intensivist at Radboud University & Erasmus MC Sophia Children's Hospital, Rotterdam, the Netherlands

Dr. Saskia de Wildt

Main address

1214 5th Ave.

New York, NY 10029 USA

Show more contact info

EIN

83-2794849

NTEE code info

Alliance/Advocacy Organizations (H01)

IRS filing requirement

This organization is required to file an IRS Form 990-N.

Communication

Blog

Programs and results

What we aim to solve

SOURCE: Self-reported by organization

Human drug development is a lengthy and expensive process with low success rates that puts humans and animals at risk during testing of novel drugs. One of the most challenging steps in drug development is the transition or translation of pre-clinical data into human applications. This transition, which involves testing of the novel drug in animals and humans is fraught with risks due to the large amount of unknowns about the novel drug. Phase-0 ap

Our programs

SOURCE: Self-reported by organization

What are the organization's current programs, how do they measure success, and who do the programs serve?

Intra-Target Microdosing (ITM) Task Force

The Intra-Target Microdosing (ITM) Task Force is a Phase-0/Microdosing Network program aimed at advancing research and disseminating information about this novel drug development approach. ITM improves and expands the scope of Phase-0 approaches in drug development by enabling the collection of therapeutic data after the administration of a safe, sub-therapeutic doses. This is achieved by administering the safe, sub-therapeutic dose (microdose) only into a small area of the body thereby limiting the duration and extent of these exposures while obtaining all the scientific benefits. The approach allows safer, quicker, and cheaper testing of new drug in humans, including the testing of old and new drugs in vulnerable populations (such as children and pregnant women) who are routinely excluded from drug development research. The approach also extends the ability of Phase-0 approaches to reduce the use of animals in human drug development.

Population(s) Served
People with diseases and illnesses
Researchers
Activists
Caregivers

The Phase-0/Microdosing Network provides regular and periodic educational activities to its members. These consist of quarterly teleconference, a yearly stakeholder meeting, an online blog, and regular updates by email n progress in the field and any relevant events. In addition, there are specialized task forces aimed at advancing specialized topics within the Phase-0 field.

Population(s) Served
Academics
Activists
Health

Where we work

Our results

SOURCE: Self-reported by organization

How does this organization measure their results? It's a hard question but an important one.

Number of free participants in conferences

This metric is no longer tracked.
Totals By Year
Population(s) Served

Activists, Academics

Related Program

Member Education Program

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

The 1st Annual Stakeholder Meeting was held in 2019, the 2nd was online in 2021, the 3rd an in-person/online hybrid in 2022, in addition to quarterly triple teleconferences and ad-hoc meetings.

Number of clients participating in educational programs

This metric is no longer tracked.
Totals By Year
Population(s) Served

Academics, Activists

Related Program

Member Education Program

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

This metric is the number of participants in 12 quarterly teleconferences to members of the Phase-0/Microdosing Network. The teleconferences include one for board members and two for general members.

Number of new champions or stakeholders recruited

This metric is no longer tracked.
Totals By Year
Population(s) Served

Academics, Activists

Related Program

Member Education Program

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

This metric reflects the number of active members in the Phase-0/Microdosing Network. These members receive regular updates by email, through website, blog, stakeholder meetings, and webinars.

Number of conferences held

This metric is no longer tracked.
Totals By Year
Population(s) Served

Academics, Activists

Related Program

Member Education Program

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

This metric reflects the number of activities arranged by the Phase-0/Microdosing Network for its members. These events include 1 Stakeholder Meetings and 2 webinars in 2021

Our Sustainable Development Goals

SOURCE: Self-reported by organization

Learn more about Sustainable Development Goals.

Goals & Strategy

SOURCE: Self-reported by organization

Learn about the organization's key goals, strategies, capabilities, and progress.

Charting impact

Four powerful questions that require reflection about what really matters - results.

Goals:
1. Enhance human-based translation and accelerate drug development by enabling safer, quicker, and more-cost effective transition between non-clinical and clinical stages of human drug development
2. Increase the application and utilization of Phase-0 approaches in human drug development
3. Advance research and knowledge dissemination pertaining to Phase-0
4. Reduce the use of animals in human drug development as a result of the application of Phase-0 approaches

The strategic plan of the Phase-0/Microdosing Network is consistent with its remit to use educational and research methods to advance the utilization of microdosing and other Phase-0 approaches in human drug development. The plan covers the period 2019 – 2024.

Objectives:
1. Enhance the familiarity of drug development stakeholders with Phase-0 approaches
2. Formulate guidelines for the application of Phase-O/Microdosing approaches
3. Establish recommendations for further research and development

Strategies:
1. Disseminate information about Phase-0 approaches and, specifically, provide update to drug development stakeholders on validation, methodology, applications, and research relevant to Phase-0 approaches
2. Obtain input from stakeholders (regulatory, academia, industry, CROs, patients, patient advocacy, and activists) on the value, prospects, and challenges facing these approaches
3. Establish consensus statements on future directions in education, research, and applications pertaining to Phase-0 approaches

Tactics:
1. Actual drug development (collaborations as sponsors, funders, CROs, and expert supporters of Phase-0 application)
2. Investigative Medicine (advancing the field of medicine, including ‘Blue Sky’ research with no immediate or tangible commercial goals)
3. Research advancing the science and methodology of Phase-0/Microdosing approaches. This line of research aims at expanding applicability and validity and therefore the utility of these approaches:
a) Novel applications (e.g., in hepatically- and renally-impaired, women of childbearing age, extreme environments)
b) Novel methodological approaches (e.g., adaptive Phase-0/Phase-1 designs, Intra-Target Microdosing [ITM])
c) Novel modeling and simulation approaches to increase the extrapolation validity of Phase-0/Microdosing study data (e.g., using PKPD modeling and in-vivo-in-vitro modeling and simulation, microdose vs. full dose in animal models)
d) Economic analyses – to determine what developmental scenarios favor the use of Phase-0/Microdosing approaches, what are the cutoff parameters for applicability and profitability

Our organization is composed of key global experts in the drug development field. They represent a wide range of stakeholders with interest in safe, effective, and productive drug development process. These stakeholders include industry, academia, regulatory, CROs, non-profit, patient advocacy, and the public at large. Through their connections, publications, research, education, and advocacy work, our organization and its members have the capability to improve global drug development. Specifically, by disseminating information on innovations and applications of microdosing and other Phase-0 approaches, our organization has the potential to enhance and improve the safety, speed, and cost-effectiveness of drug development.

The Phase-0/Microdosing Network has operated since 2012 to increase awareness about and impact of Phase-0 including microdosing approaches. It has disseminated information about this approaches to an increasing membership of global expert stakeholder, engaged in research on application and methodologies of Phase-0 approaches to increase their applicability and impact, and generated key publications of the topic such as those listed below. The Network holds quarterly teleconferences and annual meetings to update its members and the drug development community at large about progress in the field and engage these stakeholders in consensus-building discussion regarding the future of the field.

Recent Network member publications:
Burt, T., Roffel, A. F., Langer, O., Anderson, K., & DiMasi, J. (2022). Strategic, feasibility, economic, and cultural aspects of phase 0 approaches: Is it time to change the drug development process in order to increase productivity? Clin Transl Sci, 15(6), 1355-1379. doi:10.1111/cts.13269
Burt, T., Young, G., Lee, W., Kusuhara, H., Langer, O., Rowland, M., & Sugiyama, Y. (2020). Phase 0/microdosing approaches: time for mainstream application in drug development? Nat Rev Drug Discov. doi:10.1038/s41573-020-0080-x
Gundle, K. R., Deutsch, G. B., Goodman, H. J., Pollack, S. M., Thompson, M. J., Davis, J. L., . . . Maki, R. G. (2020). Multiplexed Evaluation of Microdosed Antineoplastic Agents In Situ in the Tumor Microenvironment of Patients with Soft Tissue Sarcoma. Clin Cancer Res. doi:10.1158/1078-0432.Ccr-20-0614
Kim, J., Ahn, S. W., Deans, K., Thompson, D., Ferland, B., Divakar, P., . . . Jonas, O. (2022). Intratarget Microdosing for Deep Phenotyping of Multiple Drug Effects in the Live Brain. Front Bioeng Biotechnol, 10, 855755. doi:10.3389/fbioe.2022.855755
Mooij, M. G., van Duijn, E., Knibbe, C. A., Allegaert, K., Windhorst, A. D., van Rosmalen, J., . . . de Wildt, S. N. (2017). Successful Use of [14C]Paracetamol Microdosing to Elucidate Developmental Changes in Drug Metabolism. Clin Pharmacokinet. doi:10.1007/s40262-017-0508-6
Okour, M., Derimanov, G., Barnett, R., Fernandez, E., Ferrer, S., Gresham, S., . . . Haselden, J. (2017). A Human Microdose Study of the Anti-Malarial GSK3191607 in Healthy Volunteers. Br J Clin Pharmacol. doi:10.1111/bcp.13476
Rowland, M. (2016). Microdosing of protein drugs. Clin Pharmacol Ther, 99(2), 150-152. doi:10.1002/cpt.275
van Groen, B. D., Vaes, W. H., Park, B. K., Krekels, E. H. J., van Duijn, E., Korgvee, L. T., . . . Turner, M. A. (2019). Dose-linearity of the pharmacokinetics of an intravenous [(14) C]midazolam microdose in children. Br J Clin Pharmacol. doi:10.1111/bcp.14047
Vlaming, M., van Duijn, E., Dillingh, M. R., Brands, R., Windhorst, A. D., Hendrikse, N. H., . . . Vaes, W. (2015). Microdosing of a Carbon-14 Labeled Protein in Healthy Volunteers Accurately Predicts Its Pharmacokinetics at Therapeutic Dosages. Clin Pharmacol Ther, 98(2), 196-204. doi:10.1002/cpt.131

Financials

Phase-0 Microdosing Network
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Operations

The people, governance practices, and partners that make the organization tick.

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Phase-0 Microdosing Network

Board of directors
as of 08/30/2022
SOURCE: Self-reported by organization
Board chair

Dr. Tal Burt

Burt Consultancy, LLC

Term: 2013 -

Malcolm Rowland

University of Manchester

Yuichi Sugiyama

Josai International University

Saskia de Wildt

Radboud University Medical Center

Oliver Langer

Medical University of Vienna

Wouter Vaes

Netherlands Organization for Applied Scientific Research (TNO)

Joseph DiMasi

Tufts Center for the Study of Drug Development, Tufts Medical School, Tufts University

Kirsten Anderson

Presage Biosciences

Organizational demographics

SOURCE: Self-reported; last updated 12/7/2021

Who works and leads organizations that serve our diverse communities? GuideStar partnered on this section with CHANGE Philanthropy and Equity in the Center.

Leadership

The organization's leader identifies as:

Race & ethnicity
White/Caucasian/European
Gender identity
Male
Disability status
Decline to state

The organization's co-leader identifies as:

Race & ethnicity
White/Caucasian/European
Gender identity
Female

Race & ethnicity

Gender identity

 

Sexual orientation

No data

Disability

No data