Managing Biomarker-Driven Clinical Trials

The concept of personalized or precision medicine, with medical decisions, practices, and/or products being tailored to the individual patient, has brought to life several types of clinical trials that involve biomarkers. Effective management of these trials can be complicated and requires specific operational approaches. CHI’s Symposium“Managing Biomarker-Driven Clinical Trials”is designed as an educational event to discuss solutions to overcome operational and scientific challenges with various types of studies including trials with biomarker-based stratified trials, trials for biomarker discovery and trials with biomarkers as end points. Study design specifics and operational challenges in biomarker-involved clinical trials will be discussed by experts from pharmaceutical companies and academic centers of clinical research excellence.

Tuesday, February 23

7:15 am Registration and Morning Coffee

8:25 Opening Plenary Keynotes - View Details

9:45 Grand Opening Coffee Break in the Exhibit Hall


10:45 Chairperson’s Remarks

Rebecca Blanchard, Ph.D., Executive Director, Genetics and Pharmacogenomics, Head, Clinical Pharmacogenomics, Merck & Co., Inc.


Bardia_AkbariBardia Akbari, Pharm.D., Vice President, Product Global Development, Oncology Genentech, Inc.

Advancements in diagnostic technologies and greater understanding of the underlying molecular pathophysiology of diseases has led to propagation of discovery and development of targeted therapies.  Despite the potential to clear higher efficacy bar, early and late stage development of target therapies in biomarker selective patient populations introduces unique scientific, operational, regulatory, and commercial challenges. In this talk we will examine some of these challenges.

11:30 Pharmacogenetics as Innovative Clinical Drug Development

Rebecca_BlanchardRebecca Blanchard, Ph.D., Executive Director, Genetics and Pharmacogenomics, Head, Clinical Pharmacogenomics, Merck & Co., Inc.

Our understanding of the consequences of human genetic variation, on the practice of interventional therapeutic medicine, is maturing at a rapid pace. Today, many drug development efforts include evaluation of genetic determinants of drug response. Pharmacogenetic (PGx) data are now being used to impact drug development strategy and the clinical use of drugs. This presentation will highlight methods used to leverage PGx in clinical research and thereby generate data that create new scientific and commercial opportunities.

12:10 pm Interactive Discussion: Clinical Operations to Adjust to the Concept of Personalized Medicine

Topics to be discussed include but are not limited to the following:

  • Applying the concept of personalized/precision medicine to clinical development
  • Unique operational challenges of early and late stage development of therapies in biomarker selective patient population
  • Leveraging pharmacogenomics in clinical research
  • Operationalizing biomarker-based randomization
  • Multi-drug multi-sponsor trials: new paradigm leads to new challenges
  • Collaboration and exchange of information regarding molecular profiling and treatment selection
  • Regulatory challenges and impact on FDA submission strategies
  • Commercial challenges and solutions

12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:20 Coffee and Dessert in the Exhibit Hall


2:00 Chairperson’s Remarks

Kris Kokomoor, Specimen Management Product Owner, Clinical Trial Solutions, Operations CoE, Pfizer Worldwide Research and Development

2:05 Operationalizing Precision Medicine

Brenda_YanakBrenda Yanak, Ph.D., Director, Precision Medicine Leader, Clinical Innovation, Pfizer

This presentation will cover logistics considerations related to sample management and dealing with central laboratories, as well as clinical informatics requirements and adjustments required for clinical trials in the era of precision medicine. Both US and global trials will be discussed.

2:35 Technology Framework for Sample and Biospecimen Management and Tracking

Kris Kokomoor, Specimen Management Product Owner, Clinical Trial Solutions, Operations CoE, Pfizer Worldwide Research and Development

Biomarkers become an integral part of clinical trials. In order to run a biomarker-driven trial we need to put in place an effective system of sample and biospecimen management and tracking. IT technologies come instrumental in this process. This talk will discuss the use of a specimen management framework to operationalize biomarker- focused clinical research and the precision medicine development.

3:05 Clinical Sample Tracking and Compliance to Enable Data-Driven Drug Development

Daniel Joelsson, Director, Global Business Planning & Operations, MedImmune

MedImmune is implementing a solution to track clinical samples across the complex system of labs and other service providers that support our studies. Knowing what samples we have at any time as well as being able to reconcile what samples we should have is a prerequisite to support biomarker-driven clinical trials for precision medicine. This talk will highlight the capabilities of our approach as well as some of the lessons learned during implementation.

3:35 Interactive Discussion: Biospecimen Handling Technology and IT Framework in Biomarker-Driven Trials

Topics to be discussed include, but are not limited to, the following:

  • Clinical informatics solutions for additional logistics challenges of biomarker-driven clinical trials
  • Managing sample collection to support clinical trials
  • Assuring the quality and the sample – test – result flow
  • Addressing the issues related to global trials
  • Widely available and in-house IT application for sample management


3:55 Find Your Table and Meet Your Moderator

4:00 Interactive Breakout Discussion Groups

Concurrent breakout discussion groups are interactive, guided discussions hosted by a facilitator or set of co-facilitators to discuss some of the key issues presented earlier in the day’s sessions. Delegates will join a table of interest and become an active part of the discussion at hand. To get the most out of this interactive session and format please come prepared to share examples from your work, vet some ideas with your peers, be a part of group interrogation and problem solving, and, most importantly, participate in active idea sharing.

5:00 Welcome Reception in the Exhibit Hall

6:30 Close of Day

Wednesday, February 24

7:15 am Registration and Morning Coffee


8:25 Chairperson’s Remarks

8:30 Statistical Designs of Master Protocols and on the Design of the SWOG Lung Master Protocol (S1400; Lung-Map)

Mary_RedmanMary Redman, Ph.D., Lead Statistician, Lung Cancer Committee Southwest Oncology Group, Lead Statistician, Lung Map Trial, Fred Hutchinson Cancer Research Center

The Master Protocol concept has been around for a long time. The idea behind a master protocol is to gain efficiencies by utilizing one protocol that can be updated as new studies or objectives arise. However, the perceived utility of master protocols has been limited until recently. Genomic characterization studies conducted over the last 10 years have revealed a number of therapeutically targetable alterations, many of which have received therapeutic validation. This has led to a transformation of our standard of care approach for these patients that are now routinely molecularly genotyped in an attempt to pair the identified mutation with the appropriate targeted therapy.

The rate of potentially drug-able target identification and associated therapies is increasing and general consensus is that now is the time for master protocols to facilitate speed in development of new therapies and to bring new drugs to patients more quickly. Master protocols are often separated into two categories: umbrella protocols which evaluate multiple drugs and targets among patients with the same disease type and basket protocols which evaluate drugs/target pairs among patients defined by their genetic alternation and across disease type (so-called histology agnostic trials). However, the underlying statistical design of such studies can be broadly categorized into confirmatory and discovery-focused design.

The specific details of the design are motivated by the objectives of the study and study population. In this talk we will discuss the basic design options and possible design features for master protocols. The SWOG Lung-MAP protocol will be used to motivate the different decision points and trade-offs between different designs.

9:10 Design and Analysis of Biomarker-Driven Clinical Trials

Robert_BigelowRobert Bigelow, Ph.D., Associate Director, CT Statistics, Duke Clinical Research Institute

Biomarkers can be useful in disease prognosis and prediction of treatment outcome, giving physicians the ability to more precisely tailor the therapeutic approach to individual patients. While a biomarker may have a plausible biological mechanism, demonstration of its prognostic and predictive accuracy poses numerous statistical challenges, including appropriate use of randomization, multiplicity, stratification and statistical interaction and use of surrogate endpoints.

This session presents design strategies in biomarker-driven studies.

9:50 Interactive Discussion: Statistical Concepts for Biomarker-Driven Trials

Topics to be discussed include but are not limited to the following:

  • Stratified trials (or master protocols) can be described by a common underlying framework
  • Broadly speaking these designs can be classified as either confirmatory or discovery-based
  • The overall goal of the trial determines the design
  • We will discuss the relative merits and trade offs for different designs

10:10 Coffee Break in the Exhibit Hall


11:10 Chairperson’s Remarks

Paul S. Savuto, President & CFO, Blinded Diagnostics

11:15 Novel Application of Point-of-Care Testing (POCT) in a Global Pharmaceutical Clinical Trial

John J. Brennan, Ph.D., Senior Project Director, Global Pharmaceutical Research and Development, Abbvie

To reduce screen failures, a global pharmaceutical company deployed point-of-care testing (POCT) systems to pre-screen subjects for registration in a new drug trial prior to central lab screening. The sponsor will present data and analysis to show how well POCT correlates to Central Lab and the financial impact this application had on the study budget.

11:35 Evolution of POCT Use in Pharmaceutical Clinical Trials and How to Use Effectively

Paul_SavutoPaul S. Savuto, President & CFO, Blinded Diagnostics

Point-of-care diagnostic testing (POCT) has been utilized in pharmaceutical clinical trials since the late 1990s. Successful application of this technology relies heavily on the selection of the test device, training of investigators, logistics, data collection and support. Published data, POCT technology options, encryption and case studies will be presented along with outcomes.

11:50 Interactive Discussion: Enabling Clinical Research with the Latest Diagnostics Advances

Topics to be discussed include but are not limited to the following

  • Are there any members of the audience that have used POCT in a clinical trial? If so, please share your experience.
  • What regulatory challenges might the audience expect to face using POCT in a trial?
  • What internal challenges might the audience expect to deal with in proposing POCT use?
  • When and what type of trial applications would fit into projects you are initiating now?
  • What is the difference between wearable biometric devices and POCT diagnostic devices?

12:10 pm Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

12:50 Coffee and Dessert in the Exhibit Hall

1:30 Plenary Keynotes

3:00 Refreshment Break in the Exhibit Hall (Last Chance for Viewing)


4:00 Chairperson’s Remarks

Richard Scheyer, M.D., Vice President, Medical Affairs, Medpace

4:05 JOINT PRESENTATION: Collaboration of CRO, Laboratory and Sponsor to Support Biomarker-Driven Clinical Trials

Richard_ScheyerRichard Scheyer, M.D., Vice President, Medical Affairs, Medpace

Matthew_KelsoMatthew Kelso, Pharm.D., Ph.D., Associate Director, Medpace Reference Laboratories

Abdel_HalimAbdel Halim, Pharm.D., Ph.D., Vice President, Translational Medicine, Biomarkers and Diagnostics, Celldex Therapeutics

Incorporating biomarkers into clinical trials requires coordination of study sites, CRO, lab(s) and sponsor(s). This is critical when rapid turn-around is required for patient eligibility or dose-adjustment. Presenters will share examples from the sponsor, CRO, and laboratory perspectives addressing lab and kit selection, turn-around, and mid-trial changes in assay kit. The challenge of multiple protocols and sponsors, such as when the laboratory is providing safety and efficacy testing for the therapeutic and the companion diagnostic, will also be discussed.

5:05: Interactive Discussion: The Art, Science and Logistics of Sponsor-CRO-Lab Collaboration

Topics to be discussed include but are not limited to the following

  • Accelerating assay turnaround in global trials
  • Ensuring assay quality in face of extended trial and evolving technology
  • Balancing interests and priorities of diagnostic and therapeutic sponsors

5:45 Close of Symposium

Thursday, February 25

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