May 31, 2019 | At the Next Generation Dx Summit, a full program will explore point-of-care diagnostics, immuno-oncology, digital pathology and AI, and all of the accompanying regulatory and reimbursement questions.
If you need professional education, there are training seminars focused on liquid biopsy for cancer, image analysis and deep learning, NGS lab issues. Three special forums focusing on Point-of-Care diagnostics will cover product strategies, the expanding portfolio for veterinary applications, and point-of-care in the pharmacy.
The main conference itself—three days covering six programs—is full of speakers and topics informing and forming the diagnostics landscape. Here's what we've flagged so far.—The Editors
Dig Deep Into Regulatory Issues
In the plenary presentation, Tim Stenzel, Director, Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health, US Food and Drug Administration, will introduce the new Office Director of OIR and give updates on precision medicine and other initiatives at the FDA.
Following Stenzel, a panel will discuss proposals and solutions for diagnostic reform including oversight of laboratory developed tests. Cynthia A. Bens, Personalized Medicine Coalition, will moderate the discussion and Julie Khani, (American Clinical Laboratory Association), Donald E. Horton, Jr., (Laboratory Corporation of America Holdings), and Susan Van Meter (AdvaMedDx) will join her. The panel will discuss how stakeholders influence congressional activity on the Verifying Accurate Leading-edge IVCT Development (VALID) Act, how the VALID Act will change the current oversight landscape for diagnostics, including LDTs, how policymakers are addressing the role of CMS and CLIA in the VALID Act, what impact changes in diagnostics regulation and oversight will have on patient care, and more.
If you need regulatory insight, there's a whole host of FDA speakers in addition to Tim Stenzel's plenary slot. Other sessions focusing on regulatory issues include:
Direct-to-consumer or over-the-counter genetic tests—initiated by consumers/patients rather than clinical physicians—are the most disruptive health technology of this decade. Following FDA's landmark authorization of 23andMe's DTC pharmacogenetic test system, establishing the federal regulatory framework for marketing "clinical grade" in vitro diagnostic genetic test kits marketed directly to consumers/patients, the Agency stepped up its enforcement activity against clinical laboratories and digital health companies with safety alerts warning physicians and patients about patient initiated genetic tests that have not undergone FDA review. In an April 2019 Warning Letter to Inova Genomics Laboratory, FDA declared that certain LDTs ordered by the company/laboratory physicians "pose significant risks to patient safety." In that strongly worded letter, FDA further emphasized that, to protect public health, it can and will take enforcement action against LDTs for failing to comply with "premarket review and other FDA legal requirements that do apply to LDTs." As 23andMe's external FDA regulatory counsel and nationally recognized FDA regulatory advisors for LDTs, digital health/AI, and NGS companion diagnostics, Goldbug Strategies is at the forefront of the evolving FDA regulatory landscape. Sheila D. Walcoff, former FDA Associate Commissioner and the CEO and Founder of Goldbug Strategies, will discuss the evolving FDA regulatory landscape for commercializing genomic testing and information services. Wednesday, August 21
Roger D. Klein of Arizona State University will moderate a discussion covering differences between in vitro diagnostic test kits and LDTs, current legislative proposals that would revamp FDA's regulatory paradigm for in vitro diagnostic tests, ways to modernize CLIA to meet perceived regulatory gaps, regulatory approaches to test regulation in Europe, and more. Joining Klein will be FDA's Stenzel; Eric Konnick (University of Washington), Brad Spring (BD Diagnostics), and Paul Gerrard (Palmetto GBA). Wednesday, August 21
Real Talk on Reimbursement
Reimbursement questions are challenging, and many talks across the conference will share the latest in reimbursement regulations, tips, and models.
On January 1, 2019 outpatient clinical laboratory tests received the second of three expected 10% reimbursement cuts under the Protecting Access to Medicare Act (PAMA). In addition, Medicare contractor Palmetto GBA issued local coverage determinations (LCDs) that limited coverage for syndromic multiplexed molecular infectious diseases tests. Kimberly Hanson, ARUP Laboratories and an AMP member, will describe recent reimbursement changes under the Centers for Medicare and Medicaid Services (CMS) and discuss the potential impact on clinical laboratories. Wednesday, August 21
A panel will take on reimbursement and pricing considerations as well, outlining reimbursement models in point-of-care testing, how to balance value and price in test development, tips for working with reimbursement and regulatory agencies, and reimbursement planning throughout the product development process. Victoria Pratt, Indiana University School of Medicine and president of the Association of Molecular Pathology, and Mark Girardi, Boston Healthcare Associates, will lead the discussion. Tuesday August 20
The approval of tissue-agnostic drugs such as Keytruda and Vitrakvi, for example, will force payers to figure out policies for pan-cancer indications and associated genetic testing, while the approval of cellular-based CAR-T therapies and gene therapies such as Luxturna, Kymriah, and Yescarta are challenging the traditional way that high-cost therapeutics can be reimbursed. Daryl Pritchard, Personalized Medicine Coalition, leads the discussion. Panelists include Kristine Bordenave (Humana), Eugean Jiwanmall (Independence Blue Cross), Katherine B. Szarama (CMS), and Robert Dumanois (Thermo Fisher Scientific). The panel will take on the key reimbursement challenges for advanced diagnostic tests, which strategies are emerging for the coverage and reimbursement of cell-based and genetic therapies, and what evidence is necessary to demonstrate the value of personalized medicine technologies to payers and providers. Wednesday, August 21
Real-world evidence (RWE) is increasingly developed to support both regulatory and reimbursement decisions for new health technologies. Wearable sensors that collect RWE can help diagnostic manufacturers demonstrate the utility and value of their diagnostic tests. Anita Chawla, Analysis Group, will review recent examples of wearable data capture to generate evidence supporting coverage and reimbursement of diagnostics, considering how wearable technology is used today, and may be used in the future. Tuesday, August 20
Artificial Intelligence's Role In Diagnostics
Several sessions dig deeply into how artificial intelligence and machine learning are finding a role in diagnostics.
James Courtney Fackler, Johns Hopkins Medicine, believes the opportunity for improved health care value lies in early diagnosis and intervention. The key, however, to delivering this value will be optimizing and managing the symbiosis between the AI machines and the expert clinician. Thursday, August 22
Mahdi Sarmady, The Children's Hospital of Philadelphia, will present a model for automated screening of genomic variants using machine learning. Genomic diagnostics in clinical laboratories increasingly relies on high-throughput technologies and therefore is subject to associated complexities. Challenges include inherent artifacts in the results as well as size of data. Despite advances in genomic databases to help with interpretation, the process still involves manual curation of genomic variants. Sarmady will present a machine learning model to use the curated information to be able to screen somatic variants automatically. Wednesday, August 21
Artificial intelligence promises to increase healthcare efficiencies and accuracy and reduce cost. Its development, however, has been generally outside of any regulatory environment, and little legal scholarship about it currently exists. Timothy Craig Allen, The University of Mississippi Medical Center, argues that successful artificial intelligence regulation will require an evolving regulatory strategy that abandons traditional normative boundaries, and instead will require shared governmental and stakeholder involvement. Medical malpractice liability theory will also require careful consideration and evolution as artificial intelligence evolves. Tuesday, August 20
Much, Much More
The event breadth goes far beyond just these areas though. We've flagged sessions across the six programs. Here is more of what we've marked so far.
James H. Nichols, Vanderbilt University School of Medicine, will explore opportunities for point-of-care testing in modern healthcare. With the advantage of rapid turnaround time and device portability, POCT is finding new applications as healthcare expands into the community. Nichols will look at the ways POCT is being deployed and finding new avenues for delivering faster testing for improved patient management. Tuesday, August 20
Nanopore sequencing is a paradigm-shifting technology, says Charles Chiu, UCSF School of Medicine. The technology enables real-time, comprehensive analysis of clinical samples for potential pathogens and interrogation of the host response by RNA sequencing (RNA-Seq). Chiu will discuss current and future applications of nanopore sequencing for diagnosis for viral infections in low-resource and POC settings. Tuesday, August 20
Amy J. Mathers, University of Virginia School of Medicine, takes on antimicrobial stewardship. With several novel and emerging technologies entering the clinical microbiology field to assist with decreasing the time to identification and antimicrobial susceptibility testing, it can be challenging to navigate the potential application and barriers for each one. Mathers will explore some of the technical hurdles found in the clinical microbiology laboratory as well as some of the deployment challenges with getting results to expedite changes in patient care. Wednesday, August 21
The health challenges of China offer big opportunities for multinational pharma companies. Cases of chronic diseases are on the rise in the region, as are lung, gastric and liver cancers. China alone is likely to be the world’s third-largest pharmaceutical market with sales of more than $50 billion. Marielena Mata with Pfizer explores the large opportunity for oncology drugs in China, but also the challenges of conducting the clinical trials needed for registration. Changing regulations, restrictions for the exportation of samples, IP requirements and availability of CROs represent a few of the obstacles that need to be overcome to successfully conduct global registration studies in China. Tuesday, August 20
Precision medicine continues to transform treatment paradigms through new biomarkers and interpretation of clinical data, says George Green, Bristol-Myers Squibb. Testing for Immuno-Oncology biomarkers, such as PD-L1 and tumor mutational burden, may help optimize treatment decisions when assessed individually or in combination. The advancement of increasingly-complex biomarkers also brings a need for advanced diagnostic tools. Green will discuss biomarkers, their dynamic monitoring, and assessment. Wednesday, August 21
Steven A. Soper presents a new paradigm for managing cancer diseases. We are generating innovative microfluidic tools for selecting circulating markers from whole blood and determining the presence/absence of disease-specific molecular signatures secured from the liquid biopsy markers to guide therapy for a patient, says Soper, with University of Kansas, Lawrence and the NIH Biotechnology Resource Center of BioModular Multi-Scale Systems for Precision Medicine. Soper will discuss the exosome isolation chip, and its use in several clinical examples and securing molecular information from the affinity-selected exosomes. Tuesday, August 20
With the increasing interest in treatment assessment using liquid biopsy and circulating DNA, sensitive and multiplexed detection of tumor-derived alterations in blood is needed. G. Mike Makrigiorgos, Dana Farber Cancer Institute and Harvard Medical School, outlines novel forms of digital PCR, as well as mutation enrichment-based real time PCR methods that enable several orders of magnitude improvement of detecting mutations or microsatellite instability than currently possible; are highly multiplex-able; reduce cost of analysis. (Read more about PCR's future at Diagnostics World News) Tuesday, August 20
Michael R. Speicher, Medical University of Graz, and Brian Dougherty, AstraZeneca, present a pair of keynote sessions focused on liquid biopsy. Genome-driven oncology may have enormous potential to change the clinical management of patients with cancer, says Speicher. He'll present current and future perspectives of liquid biopsies and their impact on genomics-driven oncology. Dougherty will share how AstraZeneca has been investigating the use of ctDNA testing for patient selection, drug resistance emergence, and patient monitoring. Additional studies have extended studies into exosomal DNA and transcriptome sequencing. I will also present a recently published orthogonal bench-marking study of commercial ctDNA assays that determined most tumor-plasma discordance is due to assay technical performance, not tumor heterogeneity or clonal hematopoiesis. Thursday, August 22
Academic Industry Partnerships (AIPs) are key to successful "innovation cycles" within academic health systems (AHCs). Michael J. Becich, University of Pittsburgh School of Medicine, will describe the rules of engagement for faculty innovators and AHCs to evolve "best practices" for AIPs. Keys to successful efforts involve basic/translational researchers, technology transfer and university/health systems offices of research/compliance as well as industry relations officers. Becich will discuss guidelines for innovators establishing AIPs and appropriate management of conflict of interest policies. Tuesday, August 20
Data scientists are from Mars, clinicians are from Venus, quips David Ledbetter of Children's Hospital Los Angeles. Clinicians and data scientists come from very different backgrounds and speak different languages. Bridging that gap is a critical component to being able to successfully execute projects in healthcare. Ledbetter will discuss some of the successful strategies for improving communication to foster collaboration between interdisciplinary teams including embedding data scientists in a clinical environment, embedding clinicians in data analysis, and providing clinical education to aspiring data scientists. Wednesday, August 21
It has been almost a decade since we started treating leukemia patients with chimeric antigen receptor (CAR)-engineered T cells, notes Joseph Melenhorst, University of Pennsylvania. General principles of efficacy and toxicity are starting to take shape. These principles have altered the way we design trails, but also engineer CAR T cells. Melenhorst will provide examples of such findings and novel, unpublished findings shaping this rapidly evolving field. Tuesday, August 20
Successful use of immune checkpoint blockade for the treatment of patients with previously intractable advanced cancers has led to widespread enthusiasm for therapeutic approaches that are immunomodulatory. Challenges remain, but Ana I. Robles, National Institutes of Health, believes proteomics can complement genomics for the development of biomarkers that predict which patients will most likely respond to immune-based therapy, and support informatic strategies that reveal which peptides translated from mutated sequences are promising neoantigens for use in immunotherapy protocols. Wednesday, August 21