Phase-0 / Microdosing Approaches

Intra-Target Microdosing (ITM):

Revolutionizing Clinical Pharmacology and Drug Development

Join our:

4th Annual International Phase-0/Microdosing Stakeholder Meeting 2023

Monday, April 24, 2023, 8:00 am - 5:00 pm,

Wyndham Boston Beacon Hill, Boston, MA, USA (and online)

See program below

Register at:  tburt@Phase-0Microdosing.org

Check our recent AAPS webinar on Phase-0 approaches:

Phase-0/Microdosing: Time for mainstream application in drug development

https://www.pathlms.com/aaps/events/2332/video_presentations/189322

And respective publication at: https://rdcu.be/cxlbW

Phase-0 Including Microdosing Approaches

Phase-0 drug development trials are clinical development approaches that include microdosing trials. These approaches are sometimes called ‘Exploratory Clinical Trials’ ‘Exploratory Investigational New Drug (eIND)’ applications. Phase-0 studies are aimed at providing human data to support selection of preclinical drug candidates prior to entering clinical development.
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Phase-0 approaches, also called 'Exploratory Clinical Trials', and 'exploratory Investigational New Drug' (eIND) applications:

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Regulatory Framework

The International Conference on Harmonization (ICH) M3 Guidelines outline the regulatory position on Phase-0 including Microdosing approaches. In addition, examples of 5 Phase-0 approaches are provided.

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Operational Factors

Phase-0 approaches offer many operational advantages over traditional Phase-1 approaches. These advantages enable arrival at meaningful developmental decisions in a safer, quicker, and cheaper manner than is possible with traditional Phase-1.

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Application Criteria

The Phase-0 application criteria are developmental scenarios in which Phase-0 may provide advantages over traditional Phase-1. These criteria are the product of Phase-0/Microdosing Network discussions and stakeholder meetings.

Meetings

4th Annual Phase-0/Microdosing Stakeholder Meeting: Intra-Target Microdosing (ITM): Revolutionizing Clinical Pharmacology and Drug Development

In-person / online meeting

Monday, April 24, 2023 (8:00 am - 5:00 pm Eastern Standard Time) 
Wyndham Boston Beacon Hill
Boston, MA, USA

Intra-Target Microdosing (ITM): Revolutionizing Clinical Pharmacology and Drug Development

Intra-Target Microdosing (ITM): Revolutionizing Clinical Pharmacology and Drug Development

In-person and online meeting

Monday, April 24, 2023

Organized by: Phase-0/Microdosing Network (phase-0microdosing.org) 

Chair: Tal Burt, MD - President, Phase-0/Microdosing Network and Burt Consultancy, LLC

Organizing Committee:

Organizing Committee:

Yuichi Sugiyama, PhD, - Special Professor Emeritus in Josai Interanational University.

Kirsten Anderson

Markus Weiss, PhD - Director in clinical pharmacokinetics, Novartis Institutes for Biomedical Research 

Joseph DiMasi, PhD, - Director of Economic Analysis, Tufts Center for the Study of Drug Development, Tufts University

Kev Dhaliwal, MD, PhD - Professor of Molecular Imaging & Healthcare Technology, University of Edinburgh, UK

Go van Dam, MD, PhD - CEO & Co-Founder, TRACER

Intra-Target Microdosing (ITM): Revolutionizing Clinical Pharmacology and Drug Development

In-Person and Online Meeting

Monday, April 24, 2023, Boston, MA, USA

Organized by: Phase-0/Microdosing Network (phase-0microdosing.org) 

Chair: Tal Burt, MD - President, Phase-0/Microdosing Network and Burt Consultancy, LLC.
Co-Chair: Saskia de Wildt, MD, PhD - Professor of Clinical Pharmacology and pediatric intensivist at Radboud University Medical Center.

Organizing Committee:

Chair: Tal Burt, MD - President, Phase-0/Microdosing Network and Burt Consultancy, LLC

Yuichi Sugiyama, PhD, - Special Professor Emeritus in Josai Interanational University.

Kirsten Anderson

Markus Weiss, PhD - Director in clinical pharmacokinetics, Novartis Institutes for Biomedical Research

Joseph DiMasi, PhD, - Director of Economic Analysis, Tufts Center for the Study of Drug Development, Tufts University

Kev Dhaliwal, MD, PhD - Professor of Molecular Imaging & Healthcare Technology, University of Edinburgh, UK

Go van Dam, MD, PhD - CEO & Co-Founder, TRACER

Goals:

  1. Formulate guidelines for the application of ITM and other Phase-0/Microdosing
  2. Establish recommendations for further research and development

Objectives:

  1. Provide update on validation, methodology, applications, and
  2. Obtain input from stakeholders (regulatory, academia, industry, CROs, non-profit, patient advocacy) on the value, prospects, and challenges facing these approaches.
  3. Establish consensus statements on future directions in research and applications 

AGENDA

8:00 - 12:00 : Plenary session:

TIME

SPEAKER

TITLE

8:00-8:25

Tal Burt, MD

Introduction to Intra-Target Microdosing (ITM)

8:25-8:50

Oliver Jonas, PhD

Lab-in-a-Patient Microdevices for Multiple Drug Screening in Oncology

8:50-9:15

Nathan Schauer, PhD

CIVO: Bridging the Translational Divide in Oncology

9:15-9:45

Yuichi Sugiyama, PhD & Yasunori Aoki, PhD

ITM Modeling and Simulations

9:45-10:00

Break

10:00-10:25

Kev Dhaliwal, MD, PhD

Pulmonary ITM

10:25-10:55

Technology panel

Technology Aspects of ITM and Other Phase-0 Approaches

10:55-11:35

Regulatory panel

Regulatory Aspects of ITM and Other Phase-0 Approaches

11:35-12:00

Joseph DiMasi, PhD

The Economics of Implementing Phase 0 Techniques – Methods for Measuring Financial Value


TimeSpeakerTitle
8:00-8:25Tal Burt, MDIntroduction to Intra-Target Microdosing (ITM)
8:25-8:50Oliver Jonas, PhDLab-in-a-Patient Microdevices for Multiple Drug Screening in Oncology
8:50-9:15Nathan Schauer, PhDCIVO: Bridging the Translational Divide in Oncology
9:15-9:45Yuichi Sugiyama, PhD + Yasunori Aoki, PhDITM Modeling and Simulations
9:45-10:00Break
10:00-10:25Kev Dhaliwal, MD, PhDPulmonary ITM
10:25-10:55Technology PanelTechnology Aspects of ITM and Other Phase-0 Approaches
10:55-11:35Regulatory Aspects of ITM and Other Phase-0 ApproachesRegulatory Panel
11:35-12:00Joseph DiMasi, PhDThe Economics of Implementing Phase 0 Techniques – Methods for Measuring Financial Value

12:00 - 13:00 : Luncheon seminar (Lunch time talks by sponsors) and break
13:00 - 15:00 : Breakout Sessions:

TIME

Moderators

Group

Discussion item

13:00-15:00

Kev Dhaliwal, MD, PhD

1

ITM

13:00-15:00

Oliver Jonas, PhD

2

Technology 

13:00-15:00

Tal Burt, MD

3

Regulatory 

13:00-15:00

Nathan Schauer, PhD

4

Strategy and execution 


TimeModeratorsGroupDiscussion item
13:00-15:00Kev Dhaliwal, MD, PhD1ITM
13:00-15:00Oliver Jonas, PhD2Technology
13:00-15:00Tal Burt, MD3Regulatory
13:00-15:00Nathan Schauer, PhD4 Strategy and Execution

15:00 - 15:15 : Break
15:15 - 17:00 : Colseout Session: Summary of breakout sessions, consensus statements, and action items (Chairs: Tal Burt & Kev Dhaliwal)

Abstract

Concerns about the costs and duration of drug development projects and the risks of exposing animals, in early work, and humans to novel chemical entities, have led to efforts to improve drug development with limited exposure studies using microdosing and other Phase-0 approaches. Common to these approaches is the implied safety of limited exposures to the investigational drug. For example, with microdosing the dose is less than 100 µg or 1/100th of the anticipated therapeutic dose. With Intra-Target Microdosing (ITM) the microdose is administered directly into an area about 1/100th of the body mass, momentarily generating therapeutic-level exposures. Such approaches allow safer and earlier entry into human testing, and human-based selection from preclinical candidates. These approaches are also called Exploratory Investigational New Drug (eIND) applications and exploratory clinical trials and are regulated under the internationally harmonized ICH M3 guidance.

Phase-0 approaches allow study of drug pharmacokinetic (PK) and pharmacodynamic (PD) properties. Specific applications include use in target localization, drug-drug interactions (DDIs), effects in vulnerable populations (e.g., pediatric), and ITM. The sub-therapeutic doses in Phase-0/Microdose studies require the use of sensitive analytic tools such as Accelerator Mass Spectrometer (AMS), Positron Emission Tomography (PET) and Liquid Chromatography Tandem Mass Spectrometry (LC-MS/MS). These tools are used to study disposition, effects, and metabolites of the novel drug under study.

Recent research has advanced the validity and applicability of microdosing and other Phase-0 approaches. The approaches can accelerate drug development timelines and reduce developmental attrition by increasing the quality of candidates entering clinical development and by reducing the time to ‘go-no-go’ decisions. The meeting will engage participants in discussion of the recent advances, implementation challenges, and future directions in research and applications of these approaches.

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