FAST Congress

Pre-conference Events*

Wednesday, September 19

Workshop: Clinical Assay Development*

7:30-9:00 am Registration for Pre-Conference Workshop

9:00-9:05 Chairperson’s Opening Remarks

9:05-9:30 Clinical Assay Development: Priorities, Resources and Regulations

J. Milburn Jessup, M.D., Chief, Diagnostics Evaluation Branch, Cancer Diagnosis Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health

Discovery and research assays are increasingly being converted into clinically useful assays that then are used in clinical trials to improve patient outcome and to support drug development. This process requires that laboratory directors dedicate resources to develop such assays as well as to prioritize what assays should be developed. As assays become clinically useful and are used in trials, a series of regulations must be met that consume more resources. This presentation will discuss these issues in the context of a new National Cancer Institute Clinical Assay Development Program (CADP) that may assist this process.

9:30-9:55 Talk Title to be Announced

Herbert A. Fritsche, Ph.D., Senior Vice President and CSO, Health Discovery Corporation

9:55-10:20 Strategies for Transforming Commercial Biomarker Assays into Assays Suitable for Clinical Trials

Paul Rhyne, Ph.D., Senior Director, Tandem Labs

Currently, there are numerous biomarker assays available for purchase that are sold as “research use only,” many of which are not suitable for use in clinical trials. These commercial assays are typically developed and validated on biomarker samples that are made with commonly used laboratory buffers such as phosphate-buffered saline containing bovine serum albumin and tween-20. However these assays often perform quite differently when used on clinical samples. There are many strategies that can be used to overcome these issues including new sample preparation procedures, incorporation of new reference standards, arrangement of samples on assay plates, and use of different technologies. This talk will focus on the main issues encountered with commercial kits and discuss different strategies to help overcome them to allow use in the clinic.

10:20-10:45 Coffee Break

10:45-11:10 Assessment of the Analytical and Clinical Performance of Biomarker Assays: When Are Biomarkers Ready for Prime Time?

Gene Pennello, Ph.D., Team Leader, Division of Biostatistics, U.S. Food and Drug Administration

Many biomarkers require an in vitro diagnostic assay for measurement. For example, companion in vitro diagnostic assays measure biomarkers that provide information that is essential for the safe and effective use of a specific therapeutic product. For such biomarkers, clinical utility is only as good as assay quality of measurement. Quality of measurement is characterized by analytical validation studies of accuracy, reproducibility, and limit of detection, for example. In this presentation, I’ll review the design and statistical evaluation of common analytical validation studies. For companion in vitro diagnostic assays, I’ll link analytical performance with clinical performance in drug trials. To illustrate concepts, I’ll provide many examples.

11:10-11:35 Combined CLIA and IVD Strategy Accelerates Timing and Mitigates Risk for Companion Diagnostics

Andrew Grupe, Ph.D., Senior Director, Pharmacogenomics, Celera/Quest Diagnostics

Personalized medicines are heralded as the future of drug development. Bringing a targeted medicine to market requires the concerted efforts of a drug developer and a diagnostics partner. There is no one size fits all approach for these partnerships, and often a tailored relationship is forged between the two partners either before or during a pivotal Phase III trial. Late-stage partnerships increase the drug candidate’s risk profile when it is deemed to need a predictive biomarker for a pivotal Phase III trial. This presentation will provide specific examples that illustrate the benefits of working with a diagnostics organization with both experienced IVD manufacturing and an extensive CLIA laboratory infrastructure. The tangible benefits that mitigate the drug development risk and may be attractive to both partners if relationship starts before Phase II will be described.

11:35-12:00 From Site to Sample: Innovative Strategies for Non-Invasive Diagnostic Development

Kathleen A. Mandziuk, M.P.H., R.N., Senior Scientific Affairs Director, Late Phase Services, PRA International

This presentation will focus on proven and effective techniques to obtain primary minimal risk diagnostic samples and corresponding data from clinical practices. These techniques can be used from every phase of discovery development, as well as post-approval positioning. The presentation will focus on each component of the process starting with selecting the right sites through statistical analytics.

12:00-1:00 pm Luncheon Technology Showcase (Sponsorship Opportunities Available)

Contact Ilana Quigley at or 781-972-5457

1:00-1:25 Laboratory-Developed Tests in the Genomic Medicine Era: Validation, Regulation and Challenges Faced by New Technologies and Clinical Applications

Andrea Ferreira-Gonzalez, Ph.D., Professor and Chair, Division of Molecular Diagnostics; Director, Molecular Diagnostics Laboratory, Department of Pathology, Virginia Commonwealth University

Laboratory-developed tests are those tests developed, validated and performed by clinical laboratories. There are standards and regulations in place for the validation of these tests before they are introduced into clinical practice. This presentation will discuss the process of validation under the current regulatory framework, and regulatory challenges posed by new technologies such as NGS and its clinical applications.

1:25-1:50 LDTs and Public Policy: Critical Issues for Patient Care and Innovative Test Development

Mary Steele Williams, Executive Director, Association for Molecular Pathology

In 2009, the U.S. Food and Drug Administration announced an intention to abandon its regulatory policy of enforcement discretion toward some laboratory developed tests. In response, a number of medical and business organizations put forth various paradigms, and several legislative proposals have been discussed or introduced for strengthening LDT regulation. The Association for Molecular Pathology (AMP) is recognized internationally as the experts in molecular diagnostics and has been involved in LDT policy discussions from the beginning. The issues are complex and the importance for patient care and innovative test development is critical.

1:50-2:15 FDA-Approved LDT: Accelerating the Path to Commercialization while Mitigating Risk and Cost

Alan Wookey, Executive Director, Companion Diagnostics, LabCorp Clinical Trials, Laboratory Corporation of America

Pharmaceutical companies seek biomarkers that correlate with drug response. If a biomarker is used to stratify a patient or to determine the appropriate dose of drug, the FDA requires that a companion diagnostic be developed to support testing in the healthcare setting. LabCorp’s laboratory approval LDT approach represents a viable alternative to the traditional IVD kit route, thereby saving pharmaceutical companies significant time and expense. LabCorp’s approach has been well received by regulatory agencies, and several case studies will be presented. Using the LabCorp network to support clinical trials or diagnostic testing offers an attractive option, which has culminated in 510(k) and pre-market approval submissions to the U.S. Food and Drug Administration.


*Separate Registration Required