Workshops | Day 1 | Day 2

 

All Talks in the Main Auditorium unless noted.

September 14, 2018

8:00 AM

Registration and Continental Breakfast

9:00 AM

Welcome and Logistics

TRACK 4: Companion Diagnostics in the Clinic

9:05 AM

RNAscope ISH for CDx Biomarker Development Programs

James Wang, Business Development Executive

ACD Bio

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RNAscope ISH for CDx Biomarker Development Programs

There is an increasing need to stratify patients based on drug response, drug efficiency and risk of side effects. As a result, the need to develop RNA-based biomarkers and measure RNA expression has increased considerably. RNAscope® ISH technology enables tissue-based expression analysis in complex tumor microenvironments; providing the cellular resolution required to evaluate tissue heterogeneity and to potentially better screen and select patient specimens. ACD’s Pharma Assay Services offer a Feasibility and Prototype Biomarker Assay Development Program to assist laboratories in streamlining their assay development process; providing detailed optimized protocols, in-situ target verification, reproducibility testing and data analysis as required, on a number of platforms, sample-types, study criteria, as required.

9:30 AM

CDx/Rx Regulatory Pathways in the EU

Seamus Kearney, CEO

ARC Regulatory

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CDx/Rx Regulatory Pathways in the EU

In an era of rapidly advancing technologies in the field of companion diagnostics & precision medicine, how will EU lawmakers respond, to ensure that regulation does not restrict patient access to life-altering therapies?  As we enter a time that sees the US FDA recognizing the need for novel and innovative regulatory pathways for new Companion Diagnostics, we will explore the current law-making paradigm in the EU and whether it can keep pace with the rapidly evolving technological landscape for precision therapies.  We will examine the changes in EU regulation with specific focus on the main impacts on CDx/Rx collaborations and time to market, what is meant by “implementing acts” and, how they might be used to facilitate a more nimble regulatory approach. Finally, we consider how the effects of Brexit might impact on clinical research in the EU for CDx/Rx clinical investigations.

9:55 AM

Analytical Concordance between a Clinical Trial Assay and an IVD Companion Diagnostic Assay

Chris Major, Director of Oncology Diagnostics

Janssen Pharmaceuticals

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Analytical Concordance between a Clinical Trial Assay and an IVD Companion Diagnostic Assay

Commercial ready companion diagnostic assays are often not available to initiate potential registrational therapeutic trials. As such, it is often necessary to initiate registrational therapeutic trials using a prototype Clinical Trial Assay (CTA), followed by a migration to commercial ready Companion Diagnostic assay via a Bridging Study. This session will present results of an analytical concordance study between a CTA and a Market Ready IVD Assay as a Companion Diagnostic.

10:20 AM

How does Pharma address the challenges of CDx test adoption?

Flora Berisha, Director Companion Diagnostics

Daiichi Sankyo

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How does Pharma address the challenges of CDx test adoption?

While regulatory approval of the CDx is a critical and necessary step for commercial access, globally implementing the newly approved Companion Diagnostics is equally important. Pharma and diagnostic companies face significant challenges when commercializing their tests and in a global environment often dominated by local lab developed tests often called home-brewed assays. Pharma needs to respond to these CDx commercialization challenges in order to minimize the failure of biomarker-eligible patients to receive appropriate targeted therapy. To address this challenge, Pharma is improving the CDx commercialization process through a range of approaches which at times includes multiple partners.

10:45 AM

Networking Break

TRACK 5: Precision Diagnostics - Genomic Analysis, Digital Technologies and Digital Pathology Imaging

11:00 AM

Role of Digital Pathology in CDx and Immuno-oncology

George Lee, Digital Pathology Informatics Lead

Bristol-Myers Squibb

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Role of Digital Pathology in CDx and Immuno-oncology

In the era of precision medicine, the need for a scalable means of understanding how patients will respond to a plethora of therapies and combinations of therapies will be vital to its success. Digital pathology and artificial intelligence has the potential to transform what we know about immuno-oncology via high-throughput and exhaustive mining of tumor tissue and its micro-environment.

11:25 AM

The Promise and Challenges of Pathology Imaging: Artificial Intelligence as a Companion Diagnostic for Immuno-oncology

Mike Montalto, Executive Director & Head of Translational Pathology & Clinical Biomarker Laboratories in Translational Medicine

Bristol-Myers Squibb

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The Promise and Challenges of Pathology Imaging: Artificial Intelligence as a Companion Diagnostic for Immuno-oncology

Understanding the role of the tumor micro environment is paramount to the success of patient selection strategies in immune-oncology drug development.   This talk will explore the role of automated image analysis and artificial intelligence for pathology samples in I-O drug development. We will also discuss the technical, commercial and regulatory challenges of this platform for companion diagnostics.

11:50 AM

Artificial Intelligence for Immuno-oncology Pathology: From Discovery to AI-powered Companion Diagnostics

Andy Beck, Co-founder and CEO

PathAI

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Artificial Intelligence for Immuno-oncology Pathology: From Discovery to AI-powered Companion Diagnostics

Pathologic analysis of patient tissue specimens plays a central role in the field of immuno-oncology (IO). Recent advances in artificial intelligence and computer vision offer tremendous potential for discovering new pathologic mechanisms of IO treatment response and identifying new signatures for patient selection. We will discuss these new advances and their potential for accelerating progress in the development and approval of IO therapies for cancer patients.

12:15 PM

The Role of Laboratory Developed Tests in Clinical Diagnosis

Oscar Puig, Chief Scientific Officer

Phosphorus

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The Role of Laboratory Developed Tests in Clinical Diagnosis

Laboratory developed tests (LDTs) are “diagnostic tests that are developed, validated and performed by individual laboratories”. LDTs are developed only for in-house diagnostic use and cannot be commercially distributed to other laboratories. LDTs are currently regulated by the Centers for Medicare and Medicaid as high-complexity tests under the Clinical Laboratory Improvement Amendments (CLIA), which follows strict standards of analytical and clinical validation. Phosphorus has developed Next Generation Sequencing-based LDTs for different disease areas (cancer, cardiovascular, infertility, pharmacology, ophthalmology, neurology), together with a clinical exome. This presentation will focus on the complexities that LDTs present as diagnostic tests, and the different measures that labs take to ensure assay performance and patient safety.

12:40 PM

Lunch and Networking

TRACK 6: Regulatory Issues and Launching CDx into the Clinic

1:45 PM

Companion Diagnostics in Immuno-Oncology: Global Commercial and Partnership Consideration

Joseph V. Ferrara, CEO

Boston Healthcare Associates

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Companion Diagnostics in Immuno-Oncology: Global Commercial and Partnership Consideration

The immuno-oncology testing paradigm is increasingly complex, moving beyond PD-L1 to include mismatch repair, microsatellite instability, tumor mutational burden, and others. Key commercialization considerations for drug and test innovators, including balancing test access and quality, and embedding CDx global commercial considerations in pharma and diagnostic company partnerships will be highlighted.

2:10 PM

Can We Use Clinical Validated Data To Substitute For Clinical Utility? What Are Alternatives To Coverage With Evidence Development?

Gary Spitzer, Partner & Consultant

Strategic Medical Testing Services

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Can We Use Clinical Validated Data To Substitute For Clinical Utility? What Are Alternatives To Coverage With Evidence Development?

There are unfortunately only two avenues of proceeding after the discovery and locked-down descriptive phase of a test, to establish a high level of clinical validity. Frequently the development has been established on specimens of convenience, poorly defined intended use populations. These are

  1. Material derived from a prospective trial with blinded retrospective analysis of the test result, with no modification of the test before analysis or after. Specimens of this type are difficult to come by, and many samples may be missing or depleted. Because of the importance these specimens it is becoming routine to collect biospecimens, in clinical trials, but the trial has to match what the intended use of the test would be, and specimens collected at correct time points.
  2. Prospective Observational Trial in a strictly defined intended use population, with a practical short-term outcome.

 

However, even a prospective observational single arm study requires considerable resources, stretching the viability of small companies, and potentially receiving a death blow if coverage is not obtained after. The clinical utility endpoints need to be very well developed before embarking on the initial development. The intended use should be an unmet need, which will not evaporate with changing clinical practice, and potentially apply to s significant patient population.

We need to push for the development of novel approval processes, which have occurred in drug development. Here approvals are generated from single-arm studies, and confirmatory data is gathered after marketing. Frankly, the FDA is still the only body with the bandwidth to do this.  After the discovery data a pre-submission meeting with the FDA for feedback, just like drug development to address 1) the strength of the discovery data and 2) does the discovery data have the potential to reach, if clinically validated by a prospective material, obtain a Clinical Utility rating. If the FDA agrees the discovery set is promising for the intended use, the prospective validation, with its consequent costs will proceed. A cleared test should be submitted to CMS for coverage and post-approval data collected by simplified real-world outcomes, or even obtained through claims data initiated, but with the simultaneous reimbursement of the test . Safety and efficiency should be equivalent to Clinical Utility. Some of the approaches to make the equivalent of Coverage with Evidence Development (CED) will be discussed.

2:35 PM

Successes and challenges in pre-market registration and approval of Companion Diagnostic in global markets

Xiaolei Xu, Director, Global Regulatory Affairs/Companion Diagnostics

Agilent

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Successes and challenges in pre-market registration and approval of Companion Diagnostic in global markets

Regulatory landscape continues to evolve in US and global markets for Companion/Complementary Diagnostic. Successful pre-market activities require frequent communication with regulatory agencies and constant adjustment of regulatory strategies.

 

This talk will plan to cover

  • Agilent experiences and alignment with co-development guidance in obtaining pre-market approvals with original submissions as well as expansion of indications
  • CDx Regulatory considerations in global markets
  • Challenges in assay development and registration to meet pharma partners needs

3:00 PM

Connecting Patients with Biomedical Research - Biospecimen Basics

Jeff Allard, President

Lakeside Life Science

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Connecting Patients with Biomedical Research - Biospecimen Basics

This presentation will explore various aspects of obtaining well-annotated patient biospecimens for development of Companion Diagnostic tests.  We will focus on methods to shorten the timing and decrease the cost to initiate and manage clinical studies. Our objective will be to provide a clear and simple primer for current routes of obtaining patient biospecimens, highlight the obstacles that need to be overcome, and to propose alternative strategies to ensure high numbers of enrolled patients.

 

We will begin with a discussion of various modes of collecting patient biospecimens for research and development of companion diagnostics:

  • What are Remnant Biospecimens?
  • What are Archived Biospecimens?
  • What are Prospectively Collected Patient Biospecimens ?

 

We will describe the Patient Biospecimen Regulatory concerns:

  • What requires consent and what does not?
  • Lengthy Informed Consent Forms
  • Do all collections require IRB approval?
  • Are remnant or archived samples a useful alternative to prospective studies?

3:25 PM

PANEL: Connecting Patients with Biomedical Research: Enrollment and Acquiring Biospecimens

Panel chair: Jeff Allard, President

Lakeside Life Science

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PANEL: Connecting Patients with Biomedical Research: Enrollment and Acquiring Biospecimens

Many recent publications have addressed the issue of obtaining quality well characterized patient biospecimens.  We will discuss alternatives to address:

  • Why is it so hard to get quality samples?
  • Why do so few patients enroll in clinical studies?
  • Advantages and risks to the different modes of collecting patient samples

 

The regulatory literature is replete with discussions, warnings and predictions on the issue of obtaining quality patient biospecimens.  How do we get the right samples for the right studies? We will discuss the legality, the ethics and the regulations around the different approaches with emphasis on:

  • How do accelerate and increase patient enrollment
  • How do we involve patients more deeply?
  • How do we gain patient’s trust, and thereby, commitment?
  • Why is our current enrollment and collection model failing us?
  • What can we do to shorten the time and lower the cost of prospective studies?

Joseph Ferrara, CEO

Boston Healthcare Associates

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Adriane Zernhelt, Translational Biomarkers Operations

Merck & Co.

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3:55 PM

Closing Comments

Tom Fare, Director, Strategic Alliances

PlanetConnect

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Closing Comments

TBA