Conference Day 2 - November 8, 2017 

Day Two


Chairman's opening remarks


Iris Grossman

Innovation in Drug Discovery and Development: The Role of Genetics

  • Why the push towards precision medicine will continue to increase and why policymakers must align their efforts
  • Solving the issue of reimbursement for diagnostics
  • How definitions of precision value in Europe and the USA have diverged
Panel discussion

PANEL SESSION: Pricing and reimbursement: guaranteeing market access for precision drugs

  •     Collaborating with payers in order to ensure cost containment in personalized health does not affect expanded patient access
  •     Optimizing value propositions to allay payer and provider scepticism around the cost-benefit balance of precision medicine 
  •     The role of pharmacy benefit managers and pay-for-play programs in precision medicine pathways 
Ruslan Novosiadly

Supporting immunotherapy development with pre-clinical biomarker research

  •     How biomarker discovery teams can work with clinical counterparts for a streamlined development strategy
  •     Modelling biomarker development in the pre-clinical stage of research 




Discovery & Development

The next wave of gene editing in a multi-stakeholder environment

  •     Circumventing ethical concerns around gene editing of early human cells
  •     Payers: Will insurance companies pay for personalized gene editing, and where funding will come from if not 
  •     Alternate uses for CRISPR: controlling genes rather than editing them 
Diagnostics & Data

More than a match for cancer: outcomes and developments of the NCI’s MATCH program

  •     Hear how the NCI has developed a manageable strategy for patient recruitment in the MATCH program
  •     An analysis of the quid pro quo needed to build collaboration between the NCI and the pharmaceutical industry 
  •     Why the program aims for a 25% rate of rare cancers in patient recruitment and what results this has yielded 
Discovery & Development

Tissue-based cell therapy: a new route for precision med manufacturing

  • Delivering functional, architectural tissue to replace or augment organ function
  • Permitting delivery of gene therapy / iPSC / CART modified cells to solid organ targets
  • Providing a highly reproducible, automated way to enable tissue engraftment and vascularization into target organs
Diagnostics & Data

My Research Legacy – a project at the intersection of big data, genomics and medicine

  •     Building a platform for millions of patients by curating strategic partnerships in tech
  •     Driving personalized medicine and patient adherence solutions through the AHA’s big data platform 
Discovery & Development

Short-term costs of integrating whole genome sequencing into cardiology and primary care.

  • Variant interpretation, confirmation and disclosure add substantially to the cost of sequencing
  • The impact of sequencing on downstream health care costs may be minimal
  • Larger, long-term studies are needed to understand the full cost and health impact
Kurt Christensen, Instructor in Medicine, Brigham and Women's Hospital
Diagnostics & Data

The Use of Long Read Sequencing to Investigate the HER2 Locus in Breast Cancer

  • HER2 is amplified in many breast cancers, but we do not know truly understand the structure of the amplified sequence
  • We have used long read Oxford Nanopore Sequencing to Investigate the HER2 Locus
  • These long reads have given us an enhanced view of the genetics and can help us better understand ways to treat Breast Cancer
Jeffrey Rosenfeld, Assistant Professor of Pathology and Laboratory Medicine, Rutgers University
round tables


  • Adaptive trials – where companion diagnostics fit in
  • Animal models in pre-clinical biomarker exploration
  • Epigenetics - modifying gene expression and studying the outcomes
  • Finding funding – lobbying for increased federal support for precision medicine
  • Genetic data for drug discovery
  • Human Cell Atlas – discussing the possibilities and pitfalls
  • International data sharing – the obstacles and the opportunities
  • Monica Mallampalli

    Monica Mallampalli, Women's Health & Board of Directors,, SCAD Alliance

  • Medical devices – the interplay between test developer and drug developer
  • Mental health – are genomics enough of a variable?
  • Patient adherence – is it achievable and is it valuable?
  • Prenatal diagnostics – considerations around counselling and education
  • Psychological aspects of genomic screening for patients
  • The Journey to Building a Comprehensive Precision Oncology Program at an Academic Medical Center
  • Bat-ami Katzman Gordon

    Bat-ami Katzman Gordon, Project manager of Precision Medicine at the Sylvester Comprehensive Cancer Center, University of Miami





    Introduction to I-PWG and our intent for the workshop
    In this introduction, we will describe I-PWG, our composition, mission, and goals.  We will describe pharmacogenomics and its importance in drug development, activity within the industry to facilitate the study of pharmacogenomics and the modalities that we leverage to contribute to the development of precision medicines.  

    Moderator: Michelle Penny, Senior Director, Head of Computational Biology and Genomics
    Talk 1: NGS and its evolving application in pharmacogenomics 
    Learn how companies are utilizing NGS to inform the discovery and development of safe and effective medicine. Data from an I-PWG survey on NGS practices will be shared.
    Presenter:  Charles Paulding, Director Pharmacogenomics, Regeneron Pharmaceuticals, Inc.
    Talk 2: Optimizing Pharmacogenomics Sampling in Clinical Trials: Current Practices for collecting DNA
    Hear about current solutions within industry, best practices, and challenges, including country specific examples,  to obtaining PGx populations that are representative of the global clinical trial population.
    Presenter: Anita Nelsen, Head of Genomic Medicine, PAREXEL International
    Talk 3: Closing the knowledge gap: 
    Pharmacogenomics education and resources for the patient, site staff and ethics committees
    This presentation will highlight the need for pharmacogenomics education and describe some of the resources available through I-PWG.
    Presenter: Sandra Prucka, Director of Genetic Counseling for Clinical Services, Department of Medical & Molecular Genetics, Indiana University, School of Medicine
    Panel Discussion- Hot topics

    Michelle Penny, Senior Director, Head of Computational Biology and Genomics, Biogen Idec
    Charles Paulding, Director Predictive Medicine, Genetics, Regeneron Pharmaceuticals
    Anita Head, Head of Genomic Medicine, PAREXEL International
    Sandra Prucka, Director of Genetic Counseling for Clinical Services, Department of Medical & Molecular Genetics, Indiana University, School of Medicine
    Rebecca Blanchard, Head of Clinical Pharmacogenomics, Executive Director, MSD
    Kechi Anyadike, Senior Manager, Precision Medicine, Regeneron Pharmaceuticals
    Jean-Claude Marshall, Head of Clinical Genetics and Biospecimens, Pfizer  
    Kechi Anyadike, Senior Manager of Precision Medicine, Regeneron

    Harnessing patient advocacy in the effort to accelerate precision medicine discovery and development

    •     Rare disease: how the burden is an obstacle to patient engagement and how patient advocacy can overcome it
    •     Patient privacy and how to empower patient advocates and caregivers to champion genomic research
    •     How pharma can build stronger relationships with rare disease and precision medicine foundations 

    Enabling patients to engage with their data

    •     Why offering patient choice leads to increased engagement and adherence with an economic benefit for pharma and physicians
    •     How unprecedented privacy controls in the Reg4All registry allay patient concerns and enable future research

    Multi-omics: integrating multiple data sources into one precision medicine proposal

    •     Combining multiple ‘omics’ into a clear value proposition for patient personalization
    •     Understanding the value of advanced analytical technology in order to integrate data for decision-making processes
    •     Leveraging patient-centered data in a meaningful way by understanding physician needs  

    Wearables for precision medicine

    •     Promoting the use of wearables for acute and chronic illness detection
    •     Collaborating with tech and academia to enable investment from pharma 
    •     Reimbursement: plotting value propositions for companion wearables to fund future advances  



    last published: 31/Oct/17 23:05 GMT