header


BALLROOM

9:00 AM - 3:00 PM
CVCT MASTERCLASS HOW TO REPORT AND CRITIQUE MAJOR TRIALS IN CARDIOLOGY FROM A STATISTICAL PERSPECTIVE, INCLUDING RECENT STATISTICAL ADVANCES

  • By: Stuart Pocock (London, GBR); John Gregson (London, GBR)
  • Objectives
    To provide a comprehensive review of major clinical trials in cardiology, as regards to their reporting and critical appraisal, including a “cardiologist friendly” update on recent statistical advances.
  • Target audience
    The content will be aimed at cardiologists, regulators, academic researchers, industry scientists and statisticians: indeed all health professionals wanting to expand their understanding of clinical trial reports and their statistical methods.
  • Course Description
    The course will comprise three 90 minute sessions, covering key aspects of statistical analysis, reporting and critical appraisal of major trials in cardiology. This includes some recent advances in analysis of event outcomes and implications for future trial design. All will be presented in a non-technical manner, comprehensible to non-statisticians. The two lecturers will actively encourage audience participation in a lively discussion, facilitated by an expert cardiologist as lead discussant. Topics will be illustrated by real examples of cardiovascular trials. The goal is to structure the whole experience to be of direct practical value. Appropriate references to enhance further knowledge will be provided.
  • Part 1: 9:00 AM to 10:30 AM
    Moderator: Bernard Gersh (Rochester, USA)
    A critique of some key recent clinical trials in cardiology from a statistician’s perspective:
    Bernard Gersh (Rochester, USA)
  • Part 2: 11:00 AM to 12:30 PM
    Moderator: Scott Solomon (Boston, USA)
    a) Analysis and interpretation of event outcomes: life beyond the hazard ratio
    John Gregson (London, GBR)
    b) When to stop (and not stop) a trial early for efficacy, safety, or futility
    Stuart Pocock (London, GBR)
    c) Patient viewpoint
    Jillianne Code (Vancouver, CAN)
  • Part 3: 1:30 PM to 3:00 PM
    Moderator: Jan Tijssen (Amsterdam, NED)
    a) The value of using repeat events in cardiology trials and how to allow for the competing risk of death
    John Gregson (London, GBR)
    b) How to handle the trade-off between efficacy and safety: what’s best for the individual patient
    John Gregson (London, GBR)


BALLROOM

9:00 AM – 12:15 PM
NOVEL IMPLEMENTATION TRIALS: MAXIMIZING RIGOR & PRAGMATISM A CVCT-NIH Joint Session

  • The last several decades of clinical research have resulted in clinically important advances in cardiovascular research, yet much remains lost in translation. For example, although high-quality practice guidelines are available, effective strategies for sustained behavior change at multiple levels (patient, provider, community, health system) in diverse populations and conditions are still elusive. To close the research to translation gap, we need innovative implementation trials incorporating multilevel strategies that are designed for rigor, pragmatism, and sustainability. The purpose of this session is to highlight several innovative implementation design strategies that are rigorous and pragmatic and discuss challenges and strategies for moving cardiovascular implementation science forward.


BALLROOM

8:30 AM - 10:30 AM
PATIENT – TRIALIST FORUM EMPOWERING PATIENTS TO BE ACTIVE PARTICIPANTS IN CLINICAL TRIALS

  • Introduction:
    Moderators: Cynthia Chauhan (Wichita, USA); Denis Janssen (Zuid, NED)
    Cynthia Chauhan (Wichita, USA)
    The aim of this session is to continue the discussion of contributions, concerns, and viewpoints of patients through meaningful dialogue on the issue of patient engagement in the development of and participation in clinical trials. The outcome of this session would include deeper understanding by researchers of the issues patients consider important and better understanding by patients of the hurdles researchers see in engaging patients in research development and participation. The eventual goal is improved patient engagement and better accrual with less early withdrawal from trials.
  • FDA Perspective on Meaningful Patient Engagement: Successes and Failures
    Theresa Mullin (FDA, USA)
  • The role of societies in clinical trials. The patient engagement initiative at HFSA.
    Mitchell Psotka (Washington, USA)
  • The Patient Perspective
    Empowering Patients as Active Participants in Clinical Trials
    Denis Janssen (Zuid, NED); Robin Martinez (Denver, USA)
    Patients with Comorbidities and Trials
    Cynthia Chauhan (Wichita, USA)
    Inclusion of Minorities in Trials
    Patrick Gee (Chesterfi eld, USA)
    Why Patient Reported Outcomes Matter
    Susan Quella (Rochester, USA); Greg Merritt (Ann Arbor, USA)
    The Role of Mental Health Studies in Cardiovascular Trials
    Jillianne Code (Vancouver, CAN); Penilla Gunther (Stockholm, SWE)
    Patient Representation in Industry
    Barry Liden (Edwards, USA)
  • Patient Representation in Industry
    Tamara Krcmar (Servier, FRA)
  • The CVCT Forum: Moderated multi-stakeholder panel discussion
    Moderators: Cynthia Chauhan (Wichita, USA); Denis Janssen (Zuid, NED)
  • Panelists: Jillianne Code (Vancouver, CAN); Patrick Gee (Chesterfi eld, USA); Penilla Gunther (Stockholm, SWE); Denis Janssen (Zuid, NED); Tamara Krcmar (Servier, FRA); Barry Liden (Edwards, USA); Robin Martinez (Denver, USA); Greg Merritt (Ann Arbor, USA); Theresa Mullin (FDA, USA) Susan Quella (Rochester, USA)


BALLROOM

3:30 PM – 7:00 PM
ARRHYTHMIAS AND ELECTROPHYSIOLOGY TRIALS A CVCT-HRS-EHRA Joint Session


9:00 AM – 10:30 AM
Part I: Implementation Strategies in Health Care and Other Settings

  • Implementation Strategies Embedded in the Health Care Setting
    Moderators: George A. Mensah (NIH, USA); Catherine M. Stoney (NIH, USA)
    Part I includes presentations of rigorous and innovative on-going work from implementation scientists to illustrate novel design and implementation strategies embedded in a variety of settings including urban and metropolitan health care systems, rural settings, worksites, and community health systems. Two use cases of completed studies are presented to underscore the impact of implementation science on clinical outcomes.
    Implementation Designs embedded within the health care system
    Bijal Balasubramanian (Dallas, USA); Deborah J. Cohen (Portland, USA)
    Implementation Designs embedded within rural health care settings
    Korey Kennelty (Iowa City, USA)
    Use case: Implementation Designs embedded within the health care system
    Rachel Gold (Portland, USA)
  • Implementation Strategies Embedded in the Other Settings
    Implementation Designs within Worksites
    Rajesh Vedanthan (New York, USA)
    Implementation Designs within Community Settings
    Lisa Cooper (Baltimore, USA)
    Use Case: The Barbershop Study
    Keith Ferdinand (New Orleans, USA)
  • Patient viewpoint
    Penilla Gunther (Stockholm, SWE)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion Part I: Implementation Strategies Embedded in Various Settings
    Moderators: George A. Mensah (NIH, USA); Catherine M. Stoney (NIH, USA)

  • Panelists: Bijal Balasubramanian (Dallas, USA); Deborah J. Cohen (Portland, USA); Lisa Cooper (Baltimore, USA); Keith Ferdinand (New Orleans, USA); Korey Kennelty (Iowa City, USA); Rachel Gold (Portland, USA); Rajesh Vedanthan (New York, USA)


BALLROOM

11:00 AM – 12:30 PM
INNOVATIVE APPROACHES TO INFORMED CONSENT IN PRAGMATIC TRIALS SOCIETY OF CLINICAL TRIALS (SCT)-CVCT Joint Session

  • Introduction
    Moderators: Yves Rosenberg (NIH, USA)
    Charles Weijer (London, CAN)
    Pragmatic trials seek to evaluate health interventions in real world conditions to inform decision making by patients, health providers, and health system managers. Pragmatic trials commonly involve comparisons of usual care interventions, take place in primary care settings, and use routinely collected health data.

    While informed consent remains a central protection for research participants, trialists and ethicists have questioned whether standard approaches to written informed consent are desirable or required for pragmatic trials.

    In this session, we critically assess several innovative approaches to informed consent

    Integrated consent was first proposed by Kim and Miller (2014) and it allows the health provider to obtain verbal consent to research participation guided by a script and documenting consent in the electronic health record.

    Trials within cohorts were first described by Relton and colleagues (2010). This novel pragmatic trial design uses a cohort of patients with a chronic disease as a platform for multiple randomized trials. Broad consent is obtained from participants for data collection and future trials while specific informed consent is obtained from participants selected for the experimental condition in trials.

    Electronic consent, while standard for the growing number of health apps, is new to pragmatic trials. E-consent offers the opportunity to change the way that participants enroll in research and permits flexibility in information delivered.

    The opportunities and limitations of each of these innovative approaches to informed consent in pragmatic trials will be discussed.

  • Integrated consent in pragmatic trials
    Scott Kim (NIH, USA)
  • The role of broad consent in trials within cohorts
    Clare Relton (London, GBR)
  • E-consent: regulatory, ethical and practical issues
    Holly Fernandez Lynch (Philadelphia, USA)
  • A trialist’s perspective on innovative approaches to consent
    Dean Fergusson (Ottawa, CAN)
  • Industry viewpoint
    Helina Kassahun (Amgen, USA)
  • Regulatory viewpoint
    Kolbeinn Gudmundsson (CHMP, EMA, ICE); Ellis Unger (FDA, USA)
  • Patients viewpoint
    Jacqueline Alikhaani (Los Angeles, USA)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion Making innovative approaches to consent actionable
    Moderators: Yves Rosenberg (NIH, USA)

  • Panelists: Jacqueline Alikhaani (Los Angeles, USA); Dean Fergusson (Ottawa, CAN); Kolbeinn Gudmundsson (CHMP, EMA, ICE); Helina Kassahun (Amgen, USA); Scott Kim (NIH, USA); Holly Fernandez Lynch (Philadelphia, USA); Clare Relton (London, GBR); Ellis Unger (FDA, USA)


3:30 PM – 5:00 PM
Part I: Risk stratification for sudden arrhythmic death: Is there a role for machine learning, big data, or other options?

  • Imaging (MIBG, MRI) for risk stratification of sudden arrhythmic death
    Moderators: Thomas Deering (Atlanta, USA); Gerhard Hindricks (Leipzig, GER)
    Jeroen Bax (Leiden, NED)
  • Trials to prospectively validate risk-guided ICD implantation: Unmet needs, challenges and opportunities
    Nikolaos Dagres (Leipzig, GER)
  • Case Studies: Slow Trial Enrollment Resulting in Trial Termination: MADIT S-ICD, ADMIRE – ICD, STAR-VT
    Valentina Kutyifa (Rochester, USA)
  • Industry viewpoint
    Alan Cheng (Medtronic, USA)
  • FDA viewpoint
    George Van Hare (CDRH, FDA, USA)
  • Payer viewpoint: What is needed to approve reimbursement?
    Steve Farmer (CMS, USA)
  • Patient viewpoint
    Juddson Rupp (Charlotte, USA); Patricia Vlasman (Amsterdam, NED)
  • The CVCT Forum: Moderated multi-stakeholder panel discussion Part I: How to make risk-guided ICD a reality
    Moderators: Thomas Deering (Atlanta, USA); Gerhard Hindricks (Leipzig, GER)

  • Panelists: Jeroen Bax (Leiden, NED); Alan Cheng (Medtronic, USA); Nikolaos Dagres (Leipzig, GER); Valentina Kutyifa (Rochester, USA); Jessica Paulson (FDA, USA); Jonathan Piccini (Durham, USA); Juddson Rupp (Charlotte, USA); George Van Hare (CDRH, FDA, USA); Patricia Vlasman (Amsterdam, NED); Bram Zuckerman (FDA, USA)


BALLROOM

11:00 AM - 12:15 PM
Part II: Communication Challenges and Opportunities

  • Part II includes presentations of the challenges and opportunities in communicating implementation science fi ndings in high impact journals.
    Moderators: Harlan Krumholz (New Haven, USA); Brian Mittman (Pasadena, USA)
    Joseph Hill (Circulation, USA)
    John Jarcho (NEJM, USA)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion and concluding remarks Part II: Communicating implementation science fi ndings
    Moderators: Harlan Krumholz (New Haven, USA); Brian Mittman (Pasadena, USA)
  • Panelists: Joseph Hill (Circulation, USA); John Jarcho (NEJM, USA)


BALLROOM

2:00 PM - 6:30 PM
PRECISION MEDICINE FOR FUTURE CARDIOVASCULAR CLINICAL TRIALS

  • Heterogeneity of treatment eff ects is one of the most critical problems in study interpretation today, and may be leading to very erroneous decision making when applying study results to individual patients by clinicians. This is true across all disciplines, not just cardiology, but may be most visible in cardiac, stroke, and diabetes research because of the large volume of trials in these areas, and the infl uence of these trial results on clinicians.

    According to the National Institute of Health (NIH), Precision Medicine is “an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment and lifestyle for each person.”

    Thus far in most cardiovascular trials all comers have been included but increasing evidence over the last couple of years has suggested that this is no longer the appropriate strategy to reduce residual risk, mainly because background therapy in cardiovascular patients nowadays has become very strong and powerful. Thus, it will become more and more diffi cult to improve risk of recurrent events in this group of patients if one does not focus on specifi c high risk subgroups. It is fairly evident that patients after myocardial infarction with stable CHD represent a heterogenous group regarding their future risk for recurrent events. For example, in the large SMART cohort study a score has been developed based on clinical and laboratory variables and has shown a wide distribution of risk, e.g. 18% over 5 years had a risk < 10% for recurrent events while on the other side 22% had a > 30% risk over 5 years.

    In heart failure, especially HFpEF, the all comers approach has failed so far. Also, future trials of prevention of heart failure can only be aff ordable if targeting selected patients, stratifi ed according bioprofi les most likely to respond to targeted therapies.
    Today, precision medicine is far more advanced in haematology and oncology than in cardiovascular disease. One reason is most likely being related to the underlying multifactorial causes of cardiovascular disease with many diff erent pathways being involved compared to oncology. So basic questions to ask are: Is precision medicine feasible at all in cardiovascular diseases? Are deep phenotyping and pharmacophenomics helpful?


BALLROOM

5:30 PM – 7:00 PM
Part II: Have Rhythm Management randomized clinical trials died: who killed them and where are the alternatives?

  • AFib ablation trials tribulations– Is CABANA an exception or a warning of the future?
    Moderators: John Camm (London, GBR); Yves Rosenberg (NIH/NHLBI, USA)
    Bernard Gersh (Rochester, USA)
  • Have we lost our ability to execute electrophysiology clinical trials? If so, what can we do about it?
    John Camm (London, GBR)
  • Are there alternatives? Value of registries/real world evidence to supplement or even replace randomized clincial trials
    Kenneth Dickstein (Stavenger, NOR)
  • NIH Perspective
    Yves Rosenberg (NIH/NHLBI, USA)
  • FDA viewpoint
    George Van Hare (CDRH, FDA, USA)
  • Patient viewpoint
    Juddson Rupp (Charlotte, USA)
  • Industry viewpoint
    Kenneth Stein (Boston Scientifi c, USA)
  • The CVCT Forum: Moderated multi-stakeholder panel discussion Part II: How to make EP more evidence-based
    Moderators: John Camm (London, GBR); Yves Rosenberg (NIH/NHLBI, USA)

  • Panelists: Bernard Gersh (Rochester, USA); Kenneth Dickstein (Stavenger, NOR); Jessica Paulson (FDA, USA); Jonathan Piccini (Durham, USA); Juddson Rupp (Charlotte, USA); Kenneth Stein (Boston Scientifi c, USA); George Van Hare (CDRH, FDA, USA); Bram Zuckerman (FDA, USA)


BALLROOM

2:00 PM – 6:30 PM
LEVERAGING, MANAGING, VALIDATING, AND PROTECTING REAL WORLD DATA TO FACILITATE THE TRANSFORMATION OF CLINICAL EVIDENCE A CVCT- DCRI Think Tank Joint Session

  • Moderators: Patrick Gee (Chesterfield, USA); Helina Kassahun (Amgen, USA); Stefan James (Stockholm, SWE); Matthew Roe (Durham, USA)
    Cardiovascular disease has historically been the subject of a wide variety of study designs falling under the RWE umbrella, including long-term endpoint trials, pragmatic clinical trials, and health-economic evaluations.

    Given the increasing interest in real-world evidence (RWE) generation to inform the development of new therapeutics, the underlying data sources, technology approaches, and study solutions for pragmatic trials with regulatory implications are evolving rapidly.

    Real-world data (RWD) obtained from electronic health records (EHRs), administrative claims databases, or directly from patients via digital health applications and/or biosensors/wearables are increasingly being utilized to design, plan, and execute clinical trials and observational studies.

    However, recent experiences with pragmatic trials and observational studies using RWD sources and technology advances have highlighted several important considerations including how to best access, utilize, and protect RWD during all phases of a clinical trial, opportunities for patients to directly acquire and share their health data from RWD sources to increase opportunities for clinical trial participation, and methodologies for assessing data quality with RWD sources.

    The 21st Century Cures Act in the US has increased FDA acceptance of RWE and given rise to the term “regulatory-grade RWE.” But how is the regulatory-grade criterion for regulators and payers defined and what does it take for RWE to meet it?

    Issues of data validity, data provenance, and data protection related to RWE continue to evolve.

    This issue is complicated by variations in real world data RWD quality, rigor of RWD validation, analysis, and evidentiary standards for different regulatory and reimbursement decisions (post-marketing safety assessments, changes to product labeling, comparative effectiveness).

    Strong collaborations among all stakeholders will be needed to conduct the clinical trials and observational studies of the future.



2:00 PM – 3:30 PM
Part I: THE ROADMAP FOR PRECISION MEDICINE

  • Precision Medicine in Cardiovascular Disease. Do we have a roadmap?
    Moderators: Wolfgang Koenig (Munich, GER); Sanjiv Shah (Chicago, USA)
    Joseph Loscalzo (Boston, USA)
  • Lumpers and Splitters: The Bumby Road to Precision Medicine
    Thomas Lüscher (Zurich, CH)
  • Heterogeneity of treatment effect and risk-stratified approach for reporting/ implementing clinical trial results
    Speaker:
    David Kent (Boston, USA)
    Discussant:
    John Gregson (London, GBR)
  • Genomics and precision medicine: Are polygenic scores the breakthrough?
    Geneticist viewpoint
    Heribert Schunkert (Munich, GER)
    Basic scientist viewpoint
    Geoff rey Pitt (New York, USA)
  • CVCT Multi-stakeholders moderated panel discussion Part I: THE ROADMAP FOR PRECISION MEDICINE
    Moderators: Wolfgang Koenig (Munich, GER); Sanjiv Shah (Chicago, USA)
  • Panelists: John Gregson (London, GBR); David Kent (Boston, USA); Joseph Loscalzo (Boston, USA); Thomas Lüscher (Zurich, CHE); Geoff rey Pitt (New York, USA); Heribert Schunkert (Munich, GER)


AUDITORIUM

9:00 AM - 3:00 PM
DIABETES CARDIORENAL OUTCOME TRIALS


BALLROOM

2:00 PM – 3:30 PM
Part I: Continuous Learnings from Digital Health and RWD-Enabled Trials

  • Apple Heart Study
    Moderators: Patrick Gee (Chesterfield, USA); Helina Kassahun (Amgen, USA); Stefan James (Stockholm, SWE)
    Chris Granger (Durham, USA)
  • ADAPTABLE
    Matthew Roe (Durham, USA)
  • The SWEDEHEART experience
    Thomas Jernberg (Stockholm, SWE)
  • Real-world data, to predict randomized trial results before they’re done; The RCT DUPLICATE initiative
    Nicolle Gatto (Aetion, USA)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion Part I: Continuous Learnings from Digital Health and RWD-Enabled Trials
    Moderators: PatrickGee (Chesterfield, USA); Helina Kassahun (Amgen, USA); Stefan James (Stockholm, SWE)
  • Panelists: Nadia Giannetti (Montreal, CAN); Chris Granger (Durham, USA); Thomas Jernberg (Stockholm, SWE); Jack Lawrence (Janssen, USA); Nicolle Gatto (Aetion, USA)


BALLROOM

4:00 PM – 6:30 PM
Part II: BIOMARKER BIOPROFILING TO ENABLE PRECISION CARDIOLOGY TRIALS

  • Precision post-ACS: Interaction between ADCY9 and CETP genes, from ACCELERATE and REVEAL to DalGenE trial
    Moderators: Jim Januzzi (Boston, USA); Jean-Claude Tardif (Montreal, CAN)
  • Speaker:
    Jean-Claude Tardif (Montreal, CAN)
  • Discussant:
    Jemma Hopewell (Oxford, GBR)
  • Biomarker bioprofiling to enable precision cardiology trials
    Proteomics approach in CAD
    Martin Magnusson (Lund, SWE); Erik Stroes (Amsterdam, NED)
    Metabolomics and risk profiling
    Manuel Mayr (London, GBR)
    Multi-omics in heart failure
    Joao Ferreira (Nancy, FRA)
  • Currently available “usual suspect” biomarkers. How useful to guide therapy?
    Jim Januzzi (Boston, USA)
  • Radiomics bioprofiling to enable precision cardiology trials
    Charalambos Antoniades (Oxford, GBR)
  • Regulatory viewpoint
    Christian Grimstein (FDA, USA); Andrea Laslop (CHMP, EMA, AUT)
  • Industry viewpoint
    Agim Beshiri (Abbott Diagnostics, USA)
    Industry viewpoint
    Reijo Laaksonen (Zora Biosciences, FIN)
    Industry viewpoint
    Fouzia LaghrissiThode (DalCor, CHE)
    Industry viewpoint
    Evan Mills (OLINK, USA)
    Industry viewpoint
    Rachel Ostroff (Somalogic, USA)
  • Payer viewpoint
    Omar Ali (Portsmouth, GBR)
  • Patient viewpoint
    Penilla Gunther (Stockholm, SWE); Robin Martinez (Denver, USA)
  • CVCT Multi-stakeholders moderated panel discussion Part II: BIOMARKER PROFILING TO ENABLE PRECISION CARDIOLOGY TRIALS
    Moderators: Jim Januzzi (Boston, USA); Jean-Claude Tardif (Montreal, CAN)

  • Panelists: Omar Ali (Portsmouth, GBR); Kirkwood Adams (Chapel Hill, USA); Charalambos Antoniades (Oxford, GBR); Agim Beshiri (Abbott Diagnostics, USA); Joao Ferreira (Nancy, FRA); Christian Grimstein (FDA, USA); Penilla Gunther (Stockholm, SWE); Jemma Hopewell (Oxford, GBR); Jim Januzzi (Boston, USA); Fouzia Laghrissi-Thode (DalCor, CHE); Reijo Laaksonen (Zora Biosciences, FIN); Andrea Laslop (CHMP, EMA, AUT); Martin Magnusson (Lund, SWE); Robin Martinez (Denver, USA); Manuel Mayr (London, GBR); Evan Mills (OLINK, USA); Rachel Ostroff (Somalogic, USA); Erik Stroes (Amsterdam, NED


9:00 AM - 10:30 AM
DIABETES CARDIORENAL OUTCOME TRIALS Part I: Claims and approvable indications. How to adapt to the changing landscape?

  • GLP1 receptor agonist trials across the CV risk spectrum
    Moderators: Adrian Hernandez (Durham, USA); Muthiah Vaduganathan (Boston, USA)
    Robert Mentz (Durham, USA)
  • Clinical significance of CREDENCE: A cardiorenal composite outcome trial
    Meg Jardine (Sydney, AUS)
  • Targeting diabetes kidney disease. Insight from FIGARO, FIDELIO and other trials
    Georges Bakris (Chicago, USA)
  • Endothelin receptor antagonists. The SONAR trial
    Georges Bakris (Chicago, USA)
  • How to design outcomes trials when standards of care are changing
    Darren McGuire (Dallas, USA)
  • Statistical viewpoint
    David DeMets (Madison, USA)
  • DIABETES CARDIORENAL OUTCOME TRIALS The CVCT Forum: Multi-stakeholders moderated panel discussion Part I: Claims and approvable indications. How to adapt to the changing landscape?
    Moderators: Adrian Hernandez (Durham, USA); Muthiah Vaduganathan (Boston, USA)
  • Panelists: Georges Bakris (Chicago, USA); David DeMets (Madison, USA); Meg Jardine (Sydney, AUS); Hiddo Lambers Heerspink (Groningen, NED); Robert Mentz (Durham, USA); Darren McGuire (Dallas, USA)


BALLROOM

4:00 PM – 5:00 PM
Part II: Data Provenance and Protection with Real World Data, Practical aspects of implementing credible Real World Evidence

  • Patients to acquire and share their EHR and claims data. The Blue Button initiative.
    Moderators: Patrick Gee (Chesterfi eld, USA); Helina Kassahun (Amgen, USA); Matthew Roe (Durham, USA)
    Health and Human Services viewpoint
    Robert Tagalicod (Clarksburg, USA
    Patient viewpoint
    Patrick Gee (Chesterfi eld, USA); Penilla Gunther (Stockholm, SWE)
    Investigator viewpoint
    Harlan Krumholz (New Haven, USA)
    Industry viewpoint
    Helina Kassahun (Amgen, USA); Theodore Lystig (Medtronic, USA)
    Regulatory viewpoint
    Krishna Prasad (CVWP, EMA, GBR)
    Media viewpoint:
    Larry Husten (New York, USA)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion
    Part II: Data Provenance and Protection with Real World Data, Practical aspects of implementing credible Real World Evidence

    Moderators: Patrick Gee (Chesterfi eld, USA); Helina Kassahun (Amgen, USA); Matthew Roe (Durham, USA)
  • Panelists: Nadia Giannetti (Montréal, CAN); Penilla Gunther (Stockholm, SWE); Helina Kassahun (Amgen, USA); Harlan Krumholz (New Haven, USA); Theodore Lystig (Medtronic, USA); Carolyn Magill (Aetion, USA); Robert Tagalicod (Clarksburg, USA)


AUDITORIUM

9:00 AM – 3:30 PM
HEART FAILURE WITH PRESERVED EJECTION FRACTION LIGHT AT THE END OF THE TUNNEL?

  • • Often, when a clinical trial does not meet its primary end point, we learn more from the secondary analyses than with a successful intervention.

    • “The totality of evidence must be carefully considered when evaluating the results of a trial. In this trial, the investigators set a high bar for success and identifi ed some signals suggestive of benefi t, despite a neutral result for the primary end point.”

    Chris O’Connor NEJM editorial of October 24th 2019


AUDITORIUM

11:00 AM – 12:30 PM
DIABETES CARDIORENAL OUTCOME TRIALS Part II: Interpretation and reporting issues

  • Moderators: Robert Mentz (Durham, USA); Julio Rosenstock (Dallas, USA)
    Industry viewpoint
    Pete DiBattiste (Raritan, USA)
    Industry viewpoint
    Jyothis George (Boehringer Ingelheim, GER)
    Industry viewpoint
    Stephen Gough (Novo Nordisk, DEN)
    Industry viewpoint
    Jeff rey Riesmeyer (Eli Lilly, USA)
    Industry viewpoint
    Lothar Roessig (Bayer, GER)
  • Regulatory viewpoint. When can we rely on surrogate renal endpoints in pivotal trials for approval?
    Angeles Alonso (MHRA-EMA, GBR)
  • Journal Editors viewpoint
    John Jarcho (NEJM, USA)
  • CARDIORENAL OUTCOME TRIALS Part II: Interpretation and reporting issues The CVCT Forum: Multi-stakeholders moderated panel discussion
    Moderators: Robert Mentz (Durham, USA); Julio Rosenstock (Dallas, USA)

  • Panelists: Angeles Alonso (MHRA-EMA, GBR); Pete DiBattiste (Raritan, USA); Jyothis George (Boehringer Ingelheim, GER); Stephen Gough (Novo Nordisk, DEN); John Jarcho (NEJM, USA); Jeff rey Riesmeyer (Eli Lilly, USA); Lothar Roessig (Bayer, GER)


AUDITORIUM

9:00 AM - 10:30 AM
Part I: Understanding and Interpreting the results of PARAGON

  • PARAGON main results
    Moderators: Lars Lund (Stockholm, SWE); Marc Pfeffer (Boston, USA)
    Scott Solomon (Boston, USA)
  • Target population and insight from subgroup analyses
    Carolyn Lam (Singapore, SIN)
    Target population and insight from subgroup analyses
    Faiez Zannad (Nancy, FRA)
  • Renal Outcomes
    Finnian McCausland (Boston, USA)
  • Biomarker results and mechanistic insight?
    Faiez Zannad (Nancy, FRA)
  • Statistical viewpoint
    Frank Rockhold (Durham, USA)
  • CVCT Multi-stakeholders moderated panel discussion Part I: Interpreting the results of PARAGON
    Moderators: Lars Lund (Stockholm, SWE); Marc Pfeffer (Boston, USA)
  • Panelists: Paul Armstrong (Alberta, CAN); Carolyn Lam (Singapore, SIN); Finnian McCausland (Boston, USA); Milton Packer (Dallas, USA); Frank Rockhold (Durham, USA); Scott Solomon (Boston, USA); Faiez Zannad (Nancy, FRA)


BALLROOM

5:00 PM – 6:30 PM
Part III: Data Authenticity and Data Quality. How to meet regulatory requirements for advanced Real World Evidence

  • Trial Designs to Leverage Real World Data
    Moderators: Stefan James (Stockholm, SWE); Matthew Roe (Durham, USA)
    Peter Jüni (Toronto, CAN); Tariq Ahmad (New Haven, USA)
  • AI techniques to enrich and validate RWD
    Dan Riskin (Verantos, USA)
  • Regulatory Perspectives on Assessing RWD Quality
    Jacqueline Corrigan-Curay (FDA, USA); Krishna Prasad (MHRA, EMA, GBR)
  • Industry Perspective
    Laura Mauri (Medtronic, USA); David Thompson (Syneos Health, USA)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion Part III: Data Authenticity and Data Quality. How to meet regulatory requirements for advanced Real World Evidence
    Moderators: Stefan James (Stockholm, SWE); Matthew Roe (Durham, USA)
  • Panelists: Tariq Ahmad (New Haven, USA); Jacqueline Corrigan-Curay (FDA, USA); Nadia Giannetti (Montreal, CAN); Peter Jüni (Toronto, CAN); Jack Lawrence (Janssen, USA); Laura Mauri (Medtronic, USA); Krishna Prasad (MHRA, EMA, GBR); Dan Riskin (Verantos, USA); David Thompson (Syneos Health, USA)


AUDITORIUM

1:30 PM – 3:00 PM
CARDIORENAL OUTCOME TRIALS Part III: Implementation issues. How to facilitate adoption?

  • How guidelines are to adapt to the rapidly changing landscape? Who will be at the driving seat?
    Moderators: Darren McGuire (Dallas, USA); Christoph Wanner (Würzburg, GER)
    The Diabetologist?
    Julio Rosenstock (Dallas, USA)
    The Cardiologist?
    Muthiah Vaduganathan (Boston, USA)
    The Nephrologist?
    Christoph Wanner (Würzburg, GER)
  • Payer viewpoint
    Bruno Flamion (Namur, BEL)
  • Patient viewpoint
    Denis Janssen (Zuid, NED); Susan Quella (Rochester, USA)
  • CARDIORENAL OUTCOME TRIALS The CVCT Forum: Multi-stakeholders moderated panel discussion Part III: Implementation issues. How to facilitate adoption?
    Moderators: Darren McGuire (Dallas, USA); Christoph Wanner (Würzburg, GER)

  • Panelists: Bruno Flamion (Namur, BEL); Denis Janssen (Zuid, NED); Susan Quella (Rochester, USA); Julio Rosenstock (Dallas, USA); Muthiah Vaduganathan (Boston, USA)


AUDITORIUM

9:00 AM - 3:30 PM
SGLT2 INHIBITORS HEART FAILURE TRIALS

  • Type 2 diabetes mellitus (T2DM) increases the risk of HF, frequently occurs concomitantly with HF, and worsens the prognosis of HF. Some anti-hyperglycemic medications have been associated with worse HF outcomes. Sodium glucose co-transporter 2 (SGLT2) inhibition reduce CV risk in T2DM.

    Across three large SGLT2 inhibitor (SGLT2i) CV outcome trials, EMPA-REG Outcomes, CANVAS) Program and DECLARE -TIMI 58 trials, SGLT2is reduced the risk of the composite of CV death or hospitalization for HF (HHF). CV benefits were more related to a reduction in incident HF events as opposed to ischemic vascular endpoints. HF prevention in T2DM may become a new indication for SGLT2is.

    Several mechanisms have been put forward to explain the HF benefits of SGLT2is and these drugs may emerge as therapies in the prevention of HF, but also for the treatment of patients with established heart failure in patients with or without diabetes.

    Several large trials are currently exploring this potential treatment opportunity for patients with HF. DAPA-HF is the first such trial with results available short before (3 weeks) the CVCT 2019 Forum.

    Beyond and after the main results of the DAPA-HF trial there is an important need for understanding the clinical significance, the practical consequences in terms of change of drug label, new label claims, change in international guidelines, implementation issues, and consequences on ongoing similar trials.

    These issues will be discussed from all stakeholders’ perspectives which can be assembled only at CVCT meetings, including the principal investigators of DAPA-HF and of all other major trials in this space, clinicians and guideliners with cardiology, diabetology and nephrology respective backgrounds, statisticians, industry R&D experts, key regulatory people from FDA, EMA and Japan PMDA, major journal editors, patients, health technology agencies and payers.



AUDITORIUM

11:00 AM – 12:30 PM
Part II: Implementing the results of PARAGON

  • Sacubutril Valsartan across the spectrum of LV ejection fractions
    Moderators: Carolyn Lam (Singapore, SIN); Lars Lund (Stockholm, SWE)
    Marc Pfeff er (Boston, USA)
  • PARAGON in context; Revisiting the evidence so far with RAAS inhibitiors
    Sverre Kjeldsen (Oslo, NOR)
  • MRA therapy in HFpEF, insight from PARAGON, and expectations from SPIRIT, SPIRRIT and FINE-ARTS-HF
    Faiez Zannad (Nancy, FRA)
  • The CVCT Forum. Moderated Multi-stakeholders panel discussion.
    Moderators: Carolyn Lam (Singapore, SIN); Lars Lund (Stockholm, SWE)
    Part II: Implementing the results of PARAGON
    Eric Velasquez (New Haven, USA)
    Part II: Implementing the results of PARAGON
    Felipe Martinez (Cordoba, ARG)
    Part II: Implementing the results of PARAGON
    Faiez Zannad (Nancy, FRA)
  • Panelists: Sverre Kjeldsen (Oslo, NOR); Carolyn Lam (Singapore, SIN); Felipe Martinez (Cordoba, ARG); Marc Pfeff er (Boston, USA); Cristina Rabadan-Diehl (Washington, USA); Eric Velasquez (New Haven, USA); Faiez Zannad (Nancy, FRA


AUDITORIUM

3:30 PM – 7:00 PM
HOW CAN WE DO A BETTER JOB MAXIMIZING THE CHANCES OF SUCCESS IN PROGRESSING TO PHASE III TRIALS?


AUDITORIUM

9:00 AM - 10:30 AM
Part I: Understanding and Interpreting the results of DAPA-HF

  • DAPA-HF main results
    Moderators: Naveed Sattar (Glasgow, GBR); Scott Solomon (Boston, USA)
    John McMurray (Glasgow, GBR)
  • Target population and insight from subgroup analyses
    Scott Solomon (Boston, USA)
  • Renal endpoints. Time to mind the kidney?
    Scott Solomon (Boston, USA)
  • Safety considerations
    Piotr Ponikowski (Wroclaw, POL)
  • Statistical viewpoint
    David DeMets (Madison, USA)
  • Mechanistic insight. What can be learnt from DAPA-HF and other mechanistic studies?
    Subodh Verma (Toronto, CAN)
  • The CVCT Forum. Moderated Multi-stakeholders panel discussion. Part I: Interpreting the results of DAPA-HF
    Moderators: Naveed Sattar (Glasgow, GBR); Scott Solomon (Boston, USA)
  • Panelists: David DeMets (Madison, USA); Hiddo Lambers Heerspink (Groningen, NED); John McMurray (Glasgow, GBR); Piotr Ponikowski (Wroclaw, POL); Scott Solomon (Boston, USA); Subodh Verma (Toronto, CAN)


AUDITORIUM

2:00 PM – 3:30 PM
Part III: Stakeholders Viewpoint

  • Regulatory viewpoint
    Moderators: Kenneth Dickstein (Stavanger, NOR); Scott Solomon (Boston, USA)
    Krishna Prasad (MHRA-EMA, GBR); Bob Temple (FDA, USA)
  • Industry viewpoint
    Marty Lefkowitz (Novartis, USA)
  • Patient viewpoint
    Cynthia Chauhan (Wichita, USA); Nick Harshorne-Evans (London, GBR)
  • Epilogue
    Marc Pfeff er (Boston, USA)
  • The CVCT Forum. Moderated Multi-stakeholders panel discussion. Part III: Stakeholders Viewpoint
    Moderators: Kenneth Dickstein (Stavanger, NOR); Scott Solomon (Boston, USA)
  • Panelists: Cynthia Chauhan (Wichita, USA); Nick Harshorne-Evans (London, GBR); Marty Lefkowitz (Novartis, USA); Marc Pfeff er (Boston, USA); Krishna Prasad (MHRA-EMA, GBR); Bob Temple (FDA, USA)


3:30 PM – 5:00 PM
Part I: Early strategies for downstream success

  • Limitations and advantages of preclinical animal models
    Moderators: Jane Freedman (Worcester, USA); Hani Sabbah (Detroit, USA)
    Hani Sabbah (Detroit, USA)
  • Human-induced pluripotent stem cell model
    Joseph Wu (Stanford, USA)
  • Biomarker guided early development
    Francis Spinale (Columbia, USA)
  • Basic science journal editor viewpoint
    Jane Freedman (Circulation Research, USA)
  • Industry viewpoint
    Fady Malik (Cytokinetics, USA)
  • Industry viewpoint
    Benoît Tyl (Servier, FRA)
  • Patient viewpoint
    Mariette Verbakel (Nijmegen, NED)
  • The CVCT Forum: Moderated Multi-stakeholders panel discussion Part I: Reassessing upstream strategies for success
    Moderators: Jane Freedman (Worcester, USA); Hani Sabbah (Detroit, USA)

  • Panelists: Fady Malik (Cytokinetics, USA); Hani Sabbah (Detroit, USA); Francis Spinale (Columbia, USA); Benoît Tyl (Servier, FRA); Mariette Verbakel (Nijmegen, NED); Joseph Wu (Stanford, USA)


AUDITORIUM

11:00 AM – 3:30 PM
Part II: Implementing the results of DAPA-HF

  • What’s next? The consequence of DAPA-HF on the other ongoing SGLT2i HF trials
    Moderators: Piotr Ponikowski (Wroclaw, POL); Faiez Zannad (Nancy, FRA)
    Milton Packer (Dallas, USA); Bertram Pitt (Ann Arbor, USA)
  • Implementation issues in HFrEF. How to accommodate novel therapies in an ever-increasing polypharmacy burden
    Faiez Zannad (Nancy, FRA)
  • Globalisation Issues
    Felipe Martinez (Cordoba, ARG)
  • Industry viewpoint
    Jyothis George (Boehringer, GER)
    Industry viewpoint
    Anna Maria Langkilde (AstraZeneca, SWE)
  • Payer viewpoint
    Bruno Flamion (Namur, BEL)
  • Patient viewpoint
    Susan Quella (Rochester, USA)
  • The CVCT Forum. Moderated multi-stakeholder panel discussion. Part II: Implementing the results of DAPA-HF
    Moderators: Piotr Ponikowski (Wroclaw, POL); Faiez Zannad (Nancy, FRA)

  • Panelists: Bruno Flamion (Namur, BEL); Robert Cody (Flemington, USA); Jyothis George (Boehringer, GER); Anna Maria Langkilde (AstraZeneca, SWE); Felipe Martinez (Cordoba, ARG); Milton Packer (Dallas, USA); Bertram Pitt (Ann Arbor, USA); Susan Quella (Rochester, USA); Bob Temple (FDA, USA); Bart Van der Schueren (CVWP, CHMP, BEL)


AUDITORIUM

4:00 PM – 6:30 PM
International Society of Cardiovascular Pharmacology DIET AND CV PREVENTION TRIALS CVCT-ISCP Joint Session

  • The New Prevention Guidelines. How was evidence graded?
    Moderators: Bertram Pitt (Ann Arbor, USA); Hector Ventura (Miami, USA)
    ACC/AHA
    Erin Michos (Baltimore, USA)
    ESC
    Naveed Sattar (Glasgow, GBR)
  • Are dietary guidelines for cardiovascular prevention evidence-based?
    Salim Yusuf (Hamilton, CAN)
  • Medical misinformation as a barrier to evidence based preventive medicine
    Joseph Hill (Circulation, USA)
  • Media viewpoint. “The Big Fat Surprise About Diet and Nutrition”
    Nina Teicholz (New York, USA)
  • How powerful is population-based prevention through changes in lifestyle and environment? The Finnish experience
    Erkki Vartiainen (Helsinki, FIN)
  • Weight gain after smoking cessation
    Koji Hasegawa (Kyoto, JAP)
  • Is decrease in CV risk score an approvable indication?
    Regulatory viewpoint
    Fred Senatore (FDA, USA); Emmanouil Zouridakis (MHRA, GBR)
    Patient viewpoint
    Jillianne Code (Vancouver, CAN); Marietta Verbakel (Nijmegen, NED)
  • CVCT Multi-stakeholder panel moderated discussion
    Moderators: James Januzzi (Boston, USA), Jean-Claude Tardif (Montreal, CAN)

  • Panelists: Jillianne Code (Vancouver, CAN); Koji Hasegawa (Kyoto, JAP); Joseph Hill (Circulation, USA); Larry Husten (New York, USA); Muhammad Shahzeb Khan (Chicago, USA); Erin Michos (Baltimore, USA); Naveed Sattar (Glasgow, GBR); Fred Senatore (FDA, USA); Nina Teicholz (New York, USA); Erkki Vartiainen (Helsinki, FIN); Marietta Verbakel (Nijmegen, NED); Salim Yusuf (Hamilton, CAN); Emmanouil Zouridakis (MHRA, GBR)


AUDITORIUM

5:30 PM – 7:00 PM
Part II: Are ongoing early trials designed to optimally transition to phase III? RAPID FIRE CASE STUDIES (5 min presentations)

  • BAY 1753011Dual Vasopressin antagonist
    Moderators: Christopher O’Connor (Washington, USA)
    Wilfried Dinh (Bayer, GER)
  • Valproic Acid repurposing
    Björn Dahlöf (Cereno, SWE)
  • HNO donors
    Maria Borentain (BMS, USA)
  • Cardiac myosin inhibitor
    Fady Malik (Cytokinetics, USA)
  • Aldose reductase inhibitors
    Francesca Lawson (Applied Therapeutics, USA)
  • Cardiac myosin modulators
    Amy Sehnert (Myokardia, USA)
  • Galectin-3 inhibitors for treatment and prevention of cardiovascular disease
    Pieter Muntendam (G3Pharma, USA)
  • Calcium antagonists for valve disease
    Geoff rey Pitt (New York, USA)
  • Noncoding RNA therapeutics
    Thomas Thum (Hanover, GER)
  • Anti IL-6 tocilizumab in myocardial infarction
    Ola Kleveland (Oslo, NOR)
  • New P2Y12 receptor antagonist
    Sebastien Roux (Idorsia, CHE)
  • The CVCT Forum: Moderated Multi-stakeholders panel discussion Part II: Are we creative enough in dealing with early STAGE DEVELOPMENTS?
    Moderators: Christopher O’Connor (Washington, USA)

  • Panelists: Maria Borentain (BMS, USA); Björn Dahlöf (Cereno, SWE); Wilfried Dinh (Bayer, GER); Ola Kleveland (Oslo, NOR); Francesca Lawson (Applied Therapeutics, USA); Fady Malik (Cytokinetics, USA); Pieter Muntendam (G3Pharma, USA); Geoff rey Pitt (New York, USA); Sebastien Roux (Idorsia, CHE); Amy Sehnert (Myokardia, USA); Thomas Thum (Hanover, GER)


AUDITORIUM

4:00 PM – 6:30 PM
NEW ANTIHYPERTENSIVE STRATEGIES

  • Low cost polypill approach towards hypertension control and lowering CVD risk
    Moderators: George Bakris (Chicago, USA); Patrick Rossignol (Nancy; FRA)
    Salim Yusuf (Hamilton, CAN)
    Several polypill formulations have been developed and trials results have proven the feasibility, safety and efficacy in reducing risk factor levels of the polypill in individuals at moderate risk. Many challenges remain relative to the content and the formulation of polypills, the long-term safety and tolerability, the efficacy in reducing risk factor levels and cardiovascular events, physician, patient and societal acceptability, adherence, regulatory requirements, cost, and impact on lifestyle habits.

    Hypertension HT remains poorly controlled worldwide, and its prevalence is growing. Not all HT pathophysiological mechanisms are mitigated by the current classes of antihypertensive treatment currently available.

    This session will focus on the unmet needs and the development of new antihypertensive drugs acting on new targets, and the challenges related to the market pressure of cheap generic drugs.

    The results of early uncontrolled and unblinded trials reported large reductions in blood pressure following renal denervation in patients with uncontrolled hypertension were negated by the results of the randomized, sham-controlled SYMPLICITY HTN-3 trial. Non-adherence to antihypertensive medications, patient selection, renal denervation technologies, and center effect might have influenced these results. New trials using modern technologies and different target populations are reviving interest in renal denervation.

    The rigor of developing device-based treatments as well as technological modifications of existing devices is a matter of debate.

  • Resistant hypertension
    Patrick Rossignol (Nancy, FRA)
    Resistant hypertension
    Hector Ventura (Jeff erson, USA)
  • Non steroidal mineralocorticoid receptor antagonists
    Bertram Pitt (Ann Arbor, USA)
  • Renal denervation revival: Ongoing and recent trials
    Hypertension specialist viewpoint
    Michel Azizi (Paris, FRA)
    Interventional cardiologist viewpoint
    David E Kandzari (Atlanta, USA)
  • Carotid barostimulation: internal or external stimulation?
    George Bakris (Chicago, USA)
  • Industry perspective
    Sidney Cohen (Medtronic, USA)
    Industry perspective
    Fred Yang (KBP Biosciences, USA)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion
    Moderators: George Bakris (Chicago, USA); Patrick Rossignol (Nancy, FRA)
  • Panelists: Michel Azizi (Paris, FRA); Sidney Cohen (Medtronic, USA); David E Kandzari (Atlanta, USA); Bertram Pitt (Ann Arbor, USA); Hector Ventura (Jeff erson, USA); Fred Yang (KBP Biosciences, USA); Salim Yusuf (Hamilton, CAN)


8:30 AM – 6:30 PM
INTERVENTIONAL CARDIOVASCULAR CLINICAL TRIALISTS (ICVCT) FORUM
Program Directors: C. Michael Gibson (Boston, USA); Roxana Mehran (New York, USA)


TOCQUEVILLE

9:00 AM – 10:30 AM
AN ARTERY CVCT FOCUSED WORKSHOP ARTERIAL STIFFNESS/AORTIC PRESSURE WAVEFORM ANALYSIS. HOW HELPFUL FOR CV TRIALS?

  • State of the art: Arterial stiffness/Aortic pressure waveform analysis in health and disease
    Moderators: Pierre Boutouyrie (Paris, FRA); Arshed Quyyumi (Atlanta, USA)
    Pierre Boutouyrie (Paris, FRA)
    FOCUSED
    WORKSHOP Focused CVCT Workshops are organized as campfire-sessions, fit for smaller focused groups: the experts and participants brainstorm around a U shape-table, as equals. These are high caliber faculty with a typical mix of stakeholders from academia, regulatory, payers, industry, patients and journal editors. The moderated panel discussion aims at fostering exchanges, potentially shifting the lines and reaching operational conclusions.
  • Is arterial stiffness a potential target for therapy?
    Julio Chirinos (Philadelphia, USA)
  • Could arterial stiffness become a surrogate for CV protection?
    Kennedy Cruickshank (London, GBR)
  • Regulatory viewpoint: Are subclincial disease endpoints approvable?
    Norman Stockbridge (FDA, USA)
  • Investigator viewpoint
    Arshed Quyyumi (Atlanta, USA)
  • Industry viewpoint
    Wilfried Dinh (Bayer, GER); Jon Plehn (Covance, USA); Bobby Stutz (AtCor, USA)
  • CVCT Multi-stakeholders moderated panel discussion
    Moderators: Pierre Boutouyrie (Paris, FRA); Arshed Quyyumi (Atlanta, USA)
  • Panelists: Julio Chirinos (Philadelphia, USA); Kennedy Cruickshank (London, GBR); Wilfried Dinh (Bayer, GER); Louise Marais (Issy les Moulineaux, FRA); Jon Plehn (Covance, USA); Norman Stockbridge (FDA, USA); Bobby Stutz (AtCor, USA)


TOCQUEVILLE

9:00 AM – 12:15 PM
FOCUSED CVCT WORKSHOP WHAT ARE THE DATA NEEDS OF REGULATORS, PAYERS, GUIDELINES TO ESTABLISH POTASSIUM BINDERS AS RAASI ENABLERS?

  • Reassessing the risk associated with dyskalemia
    Moderators: Bertram Pitt (Ann Arbor, USA); Patrick Rossignol (Nancy, FRA)
    Ileana Piña (Detroit, USA)
    FOCUSED WORKSHOP
    Focused CVCT Workshops are organized as campfire-sessions, fit for smaller focused groups: the experts and participants brainstorm around a U shape-table, as equals. These are high caliber faculty with the typical mix of stakeholders from academia, regulatory, payers, industry, patients and journal editors. The moderated panel discussion aims at fostering exchanges, potentially shifting the lines and reaching operational conclusions.
    Development of hyperkalemia is associated with higher risk of mortality

    The concern about inducing or worsening hyperkalemia in patients eligible to or receiving RAS inhibitors and MRAs is the main driver of poor optimization of these therapies.

    The availability of new safe and well tolerated potassium-lowering agents may reduce the risk of hyperkalemia associated with RAASi and MRA use and could potentially enable better longterm use of these life saving medications.

    How best to assess the long-term risks and benefits of strategies using potassium-lowering agents is the focus of this session.

  • Strategies for mitigating the risk of hyperkalemia, and where potassium binders may fit?
    Patrick Rossignol (Nancy, FRA)
  • Initiating clinical trials in patients with heart failure with reduced ejection fraction undergoing chronic hemodialysis to ascertain whether treatment with a K+ binder can ENABLE sustained RAASi therapy
    Murray Epstein (Miami, USA)
  • RAASi enabling and cardiovascular/kidney disease outcome trials for new potassium binders. Why and when are they needed?
    Javed Butler (Jackson, USA)
  • What efficacy endpoints, other than CV outcomes? The importance of Patient Reported Outcomes, quality of life measures and dietary modification
    Jean-Claude Tardif (Montreal, CAN)
  • What safety endpoints and what should new potassium binders be compared to?
    Manish Sood (Ottawa, CAN)
  • End stage renal disease trials
    George Bakris (Chicago, USA)
  • Industry viewpoint
    Industry viewpoint
    Elisabeth Björk (AstraZeneca, SWE)
    Industry viewpoint
    Andrey Gurevich (Vifor Fresenius, CHE)
  • Payer viewpoint
    Omar Ali (Portsmouth, GBR)
  • Regulatory viewpoint
    Aliza Thompson (FDA, USA)
  • Patient viewpoint
    Patrick Gee (Chesterfi eld, USA); Nick Hartshorne-Evans (London, GBR); Greg Merritt (Ann Arbor, USA)
  • The CVCT Forum: Multi-stakeholder moderated panel discussion
    Moderators: Bertram Pitt (Ann Arbor, USA); Patrick Rossignol (Nancy, FRA)

  • Panelists: Omar Ali (Portsmouth, GBR); George Bakris (Chicago, USA); Elisabeth Björk (AstraZeneca, SWE); Javed Butler (Jackson, USA); Murray Epstein (Miami, USA); Patrick Gee (Chesterfi eld, USA); Nick Hartshorne-Evans (London, GBR); Andrey Gurevich (Vifor Fresenius, CHE); Greg Merritt (Ann Arbor, USA); Ileana Piña (Detroit, USA); Manish Sood (Ottawa, CAN); Jean-Claude Tardif (Montreal, CAN); Aliza Thompson (FDA, USA)


Inaugural Event
Welcome to iCVCT Bertram Pitt and Faiez Zannad CVCT Chairpersons


TOCQUEVILLE

11:00 AM – 3:00 PM
CVCT FOCUSED WORKSHOP eTECHNOLOGY AND OTHER CREATIVE SOLUTIONS FOR OPTIMIZING RANDOMIZED CLINICAL TRIAL EXECUTION A CVCT- ACRP (ASSOCIATION OF CLINICAL RESEARCH PROFESSIONALS) Joint Session

  • FOCUSED
    WORKSHOP Focused CVCT Workshops are organized as campfire-sessions, fit for smaller focused groups: the experts and participants brainstorm around a U shape-table, as equals. These are high caliber faculty with the typical mix of stakeholders from academia, regulatory, payers, industry, patients and journal editors. The moderated panel discussion aims at fostering exchanges, potentially shifting the lines and reaching operational conclusions. potentially shifting the lines and reaching operational conclusions.
    The cost of conducting CV clinical trials is the rising cost of drug development, and has increased at a much higher rate than inflation during the past 25 years.

    The total development costs for a drug have risen nearly 13-fold ($100 million in 1975 to $1.3 billion in 2005 in constant dollars), as a consequence of increases in the size and length of trials.

    However, major duration and cost drivers are work required by study personnel, the number of trial visits and procedures, the type and amount of data collected, and site monitoring.

    These considerations have resulted in some pharmaceutical companies shifting away from the cardiovascular arena beceause they perceive a relatively greater potential return from investments in non-cardiovascular areas.

    Many creative solutions for optimizing randomized clinical trial execution are being offered, tested and implemented.

    This multi-stakeholder session will examine the progress so far, successes and failures, expectations and challenges, hype and hope.



TOCQUEVILLE

2:00 PM – 3:30 PM
FOCUSED CVCT WORKSHOP IRON IN HEART FAILURE – PATIENT REPORTED OUTCOMES TO MORTALITY MORBIDITY TRIALS

  • Iron therapy for heart failure: What is the rationale and mechanistic background?
    Moderators: Gerasimos Filippatos (Athens, GRE); Robert Mentz (Durham, USA)
    Pieter van der Meer (Groningen, NED)
    FOCUSED WORKSHOP
    Focused CVCT Workshops are organized as campfire-sessions, fit for smaller focused groups: the experts and participants brainstorm around a U shape-table, as equals. These are high caliber faculty with the typical mix of stakeholders from academia, regulatory, payers, industry, patients and journal editors. The moderated panel discussion aims at fostering exchanges, potentially shifting the lines and reaching operational conclusions.
  • Iron therapy for heart failure: the evidence so far
    Is oral iron out?
    Javed Butler (Jackson, USA)
    Is intravenous iron in?
    Gerasimos Filippatos (Athens, GRE)
  • Iron therapy for heart failure: navigating from patient reported outcomes to morbidity-mortality trials
    Robert Mentz (Durham, USA)
  • Industry viewpoint
    Claudio Mori (Vifor, CHE)
    Industry viewpoint
    Linda Mundy (American Regent, USA)
  • Regulatory viewpoint
    Andrea Laslop (CHMP, EMA, AUT)
    Regulatory viewpoint
    Noman Stockbridge (FDA, USA)
  • The CVCT Forum: Moderated multi-stakeholder panel discussion Moving from patient reported outcome trials to cardiovascular outcome trials
    Moderators: Gerasimos Filippatos (Athens, GRE); Robert Mentz (Durham, USA)
  • Panelists: Javed Butler (Jackson, USA); Nick Hartshorne-Evans (London, GBR); Claudio Mori (Vifor, CHE); Linda Mundy (American Regent, USA); Andrea Laslop (CHMP, EMA, AUT); Noman Stockbridge (FDA, USA); Patricia Vlasman (Amsterdam, NED); Pieter van der Meer (Groningen, NED)


LIBRARY

8:30 AM – 10:30 AM
iCVCT Session 1 PCI PHARMACOLOGY TRIALS/DEVICE DRUG INTERACTIONS

  • DAPT duration Trials: Short DAPT in High Bleeding Risk Patients who receive Stents: Strengths and Weaknesses
    Moderators: C. Michael Gibson (Boston, USA); Roxana Mehran (New York, USA)
    David J. Cohen (Kansas City, USA)
  • ASA Withdrawal Studies: When Bleeding becomes an effectiveness endpoint
    Chris Granger (Durham, USA)
  • Drug v. Drug Studies in ACS and PCI: ISAR REACT trial, Role of ISS studies and future needs
    C. Michael Gibson (Boston, USA)
  • Inflammation in ACS: Highlights of the COLCOT trial
    Jean-Claude Tardif (Montreal, CAN)
  • Regulatory viewpoint
    Adrian Magee (FDA, USA)
  • Industry viewpoint
    Sidney Cohen (Medtronic, USA)
  • The iCVCT Forum: Multi-stakeholder moderated panel discussion PCI PHARMACOLOGY TRIALS/DEVICE DRUG INTERACTIONS ANTITHROMBOTIC DILEMMAS IN PCI: AFIB, HBR, AND DAPT DURATION
    Moderators: C. Michael Gibson (Boston, USA); Roxana Mehran (New York, USA)
  • Panelists: Jeff Borer (New York,USA); David J Cohen (Kansas City, USA); Sidney Cohen (Medtronic, USA); Darrel Francis (London, GBR); Natasha Giordano (PLx Pharma, USA); Chris Granger (Durham, USA); Susheel Kodali (New York, USA); Adrian Magee (FDA, USA); David Maron (Stanford, USA); Ileana Piña (Detroit, USA); Yves Rosenberg (NIH, USA); Tabassome Simon (Paris, FRA); Jean-Claude Tardif (Montreal, CAN); Ellis Unger (FDA, USA); Martin Unverdorben (Daiichi-Sankyo, USA); Emmanouil Zouridakis (MHRA, GBR)


TOCQUEVILLE

11:00 AM – 12:30 PM
Part I: eSolutions for clinical trials

  • Use of Electronic Medical Records for patient screening, recruitment, and follow up
    Moderators: Jim Kremidas (Alexandria, USA); Harlan Krumholz (New Haven, USA)
    Stefan James (Stockholm, SWE); Harlan Krumholz (New Haven, USA)
  • Artificial Intelligence decision tool for echocardiography in clinical trials
    Yoran Hummel (eki.ai, SIN)
  • Machine learning for medical research
    Maxime He (Owkin, FRA)
  • Decentralized follow up with trained healthcare professionals. The HEROs experience
    Jodi Akin (Hawthorne Effect, USA)
  • Deriving Real-World Insights From Real-World Data: Statistician viewpoint
    Frank Rockhold (Durham, USA)
  • The CVCT Forum: Multi-stakeholders moderated panel discussion Part I: eSolutions for clinical trials
    Moderators: Jim Kremidas (Alexandria, USA); Harlan Krumholz (New Haven, USA)
  • Panelists: Jodi Akin (Hawthorne Eff ect, USA); Maxime He (Owkin, FRA); Stefan James (Stockholm, SWE); Yoran Hummel (eki.ai, SIN); Frank Rockhold (Durham, USA)


TOCQUEVILLE

4:00 PM - 6:30 PM
HEART FAILURE DEVICE THERAPY. CASE STUDIES OF RECENT THE NEW FDA BREAKTHROUGH APPROVALS

  • The FDA CV device breakthrough program. Rationale and guidance
    Moderators: Ileana Piña (Detroit, USA); Michael Zile (Charlestone, USA)
    Bram Zuckerman (FDA, USA)
    On April 13, 2015, the Expedited Access for Premarket Approval and De Novo Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions document was issued as guidance for industry and FDA Center for Devices and Radiological Health staff.

    The Expedited Access Pathway (EAP) was designed as a new program for medical devices to address unmet medical needs for life-threatening or irreversibly debilitating conditions.

    The FDA Center for Devices and Radiological Health intended the EAP program to “help patients have more timely access to medical devices by expediting their development, assessment, and review, while preserving the FDA’s statutory standard for premarket approval.” This guidance document also described the types of clinical evidence that might be used to support approval of a device under the EAP and, in particular, discussed the use of intermediate and surrogate endpoints.

    Bayesian adaptive trial designs are particularly well suited to studies with a primary clinical endpoint and related intermediate endpoints. During trial updates, interim data are analyzed using a statistical model that estimates the treatment effect(s), as well as the strength of the relationship between the primary and intermediate endpoints. Provided that early trial data confirm that the intermediate endpoints are closely related to primary outcomes, the intermediate endpoints can assist in predicting the outcome of the eventual primary analysis, potentially reducing the number of patients enrolled and improving the study conclusions.

    A number of HF devices have recently been approved for premarket access using this new FDA breakthrough devices program, based on beneficial effects in intermediate endpoints trials embedded in still ongoing hard outcome trials.

    The objectives of this session are:

    • to understand and discuss the value of the evidence generated using the EAP, by clinicians and HF drug trialists/statisticians more familiar with frequentist statistics, regulators behind the EAP new guidance and the recent approvals, editors of journals to report related trials, payers, HTA experts, and patients
    • to discuss opportunities and challenges for implementation in clinical practice

  • Design and methodology of expedited access pathway HF trials
    Intermediate endpoints used for pre-market access
    Faiez Zannad (Nancy, FRA)
    Adaptive design and the use of Bayesian methodology
    Rajat Mukherjee (Cytel, USA)
  • Question and Answers
  • Case Studies
    Cardiac Contractility Modulation. Optimizer Smart System. The FIX-HF-5 trial
    Martin Borggrefe (Heidelberg, GER)
    Baroreflex stimulation. Barostim. The BEAT-HF trial
    Michael Zile (Charlestone, USA)
    Vagal nerve stimulation. Vitaria. ANTHEM-HFrEF Pivotal Study
    Marvin Konstam (Boston, USA)
  • Question and Answers
  • Multi-stakeholders viewpoints
    Industry viewpoints
    Lorenzo A. DiCarlo (Livanova, USA); Liz Galle (CVRx,USA)
    Regulatory viewpoint
    Andrew Farb (FDA, USA)
    Payer viewpoint
    Joseph Hutter (CMS, USA)
    Patient viewpoints
    Sadegh Alikhaani (Los Angeles, USA); Mariette Verbakel (Nijmegen, NED)
  • The CVCT Forum. Moderated Multi-stakeholders panel discussion. HF BREAKTHROUGH DEVICES. POST APPROVAL AND IMPLEMENTATION ROADMAP
    Moderators: Ileana Piña (Detroit, USA); Michael Zile (Charlestone, USA)
  • Panelists: Sadehgh Alikhaani (Los Angeles, USA); Martin Borggrefe (Heidelberg, GER); Lorenzo A. DiCarlo (Livanova, USA); Andrew Farb (FDA, USA); Joseph Hutter (CMS, USA); Rajat Mukherjee (Cytel, USA); Liz Galle (CVRx,USA); Leonardo Guimarães (Quebec City, CAN); Marvin Konstam (Boston, USA); Mariette Verbakel (Nijmegen, NED); Faiez Zannad (Nancy, FRA); Michael Zile (Charlestone, USA); Bram Zuckerman (FDA, USA)


LIBRARY

11:00 AM – 12:30 PM
iCVCT Session 2: TAVR PHARMACOLOGY TRIALS

  • TAVR in Patients at Low Risk for Surgery: Public Health Significance and Implications for Antithrombotic Therapie
    Moderators: George Dangas (New York, USA); Ileana Piña (Detroit, USA)
    George Dangas (New York, USA)
  • Regulatory viewpoint
    Emmanouil Zouridakis (MHRA, GBR)
  • Industry viewpoint
    Martin Unverdorben (Daiichi-Sankyo, USA)
  • The iCVCT Forum: Multi-stakeholder moderated panel discussion ANTITHROMBOTIC STRATEGIES IN THE TAVR ENVIRONMENT
    Moderators: George Dangas (New York, USA); Ileana Piña (Detroit,USA)

  • Panelists: Jeff rey Borer (New York,USA); Sidney Cohen (Medtronic, USA); George Dangas (New York, USA); Darrel Francis (London, GBR); Chris Granger (Durham, USA); C. Michael Gibson (Boston, USA); Susheel Kodali (New York, USA); Adrian Magee (FDA, USA); David Maron (Stanford, USA); Roxana Mehran (New York, USA); Mauro Moscucci (FDA, USA); Dragica Paunovic (Terumo, USA); Tabassome Simon (Paris, FRA); Ellis Unger (FDA, USA); Martin Unverdorben (Daiichi-Sankyo, USA); Bernard Vasseur (FDA, USA); Patrick Verta (Edwards, USA); Emmanouil Zouridakis (MHRA, GBR); Bram Zuckerman (FDA, USA)


TOCQUEVILLE

1:30 PM – 3:00 PM
Part II: Creative operational solutions for trial execution

  • What went wrong with phase III “failed” trials?
    Moderators: Gadi Cotter (Momentum Research, USA); Mona Fiuzat (Washington, USA)
    Gadi Cotter (Momentum Research, USA); Beth Davison (Momentum Research, USA)
  • The role of collaborative investigator networks. Experience from Heart Failure Collaboratory
    Mona Fiuzat (Washington, USA)
  • Transitioning to single institutional review boards
    Orly Vardeny (Minneapolis, USA)
  • Trials in resource limited settings
    Yan Lijing (Kunshan, CHN)
  • Site clinical research professional view
    Jim Kremidas (Alexandria, USA)
  • Industry viewpoint
    Uli Broedl (Boehringer, CAN)
  • Patient viewpoint
    Robin Martinez (Denver, USA); Greg Merritt (Ann Arbor, USA)
  • The CVCT Forum: Multi-stakeholders moderated panel discussion Part II: Creative operational solutions for trial execution
    Moderators: Gadi Cotter (Momentum Research, USA); Mona Fiuzat (Washington, USA)

  • Panelists: Uli Broedl (Boehringer, CAN); Beth Davison (Momentum Research, USA); Jim Kremidas (Alexandria, USA); Yan Lijing (Kunshan, CHN); Greg Merritt (Ann Arbor, USA); Orly Vardeny (Minneapolis, USA)


LIBRARY

2:00 – 3:30 PM
iCVCT Session 3: STRUCTURAL HEART INTERVENTIONS: DISSECTING THE TRIALS

  • “Proportionate” vs “Disproportionate” mitral regurgitation. Reconciling COAPT and Mitral FR
    Moderators: Jeff Borer (New York, USA); Susheel Kodali (New York, USA)
    Milton Packer (Dallas, USA)
  • Tricuspid Valve: Not so Forgotten-Trial designs and future Directions
    Susheel Kodali (New York, USA)
  • Non Interventional Clinician viewpoint
    Jeff Borer (New York, USA)
  • Regulatory viewpoint
    Bernard Vasseur (FDA, USA)
  • Industry viewpoint
    Patrick Verta (Edwards, USA)
  • Payer viewpoint
    Joseph Hutter (CMS, USA)
  • iCVCT Session 3 The CVCT Forum: Moderated Multi-stakeholder panel discussion TAVR AND TMVR AND TRICUSPID REPAIR. HOW TO BRIDGE THE GAPS IN EVIDENCE?
    Moderators: Jeff Borer (New York, USA); Susheel Kodali (New York, USA
  • Panelists: Alaide Chieff o (Milano, ITA); Sidney Cohen (Medtronic, USA); George Dangas (New York, USA); Darrel Francis (London, GBR); C. Michael Gibson (Boston, USA); Chris Granger (Durham, USA); David Maron (Stanford, USA); Roxana Mehran (New York, USA); Mauro Moscucci (FDA, USA); Milton Packer (Dallas, USA); Ileana Piña (Detroit, USA); Yves Rosenberg (NIH, USA); Tabassome Simon (Paris, FRA); Ellis Unger (FDA, USA); Martin Unverdorben (Daiichi-Sankyo, USA); Bernard Vasseur (FDA, USA); Patrick Verta (Edwards, USA); Emmanouil Zouridakis (MHRA, GBR); Bram Zuckerman (FDA, USA)


TOCQUEVILLE

3:30 PM – 7:00 PM
A KINEXUM CVCT FOCUSED WORKSHOP WHAT CAN RARE CV DISEASE TRIALISTS AND COMMON CV DISEASE TRIALISTS LEARN FROM EACH OTHER?

  • Objectives
    • Discuss issues, opportunities and solutions associated with rare cardiovascular diseases and their small to tiny clinical trials.

    • Promote exchange of resourceful approaches and best practices in trials and evidence generation for therapies targeting rare cardiovascular diseases

    • Case studies and examples from emerging companies working on rare cardiovascular diseases will serve for a robust multi-stakeholder discussion involving small and large pharma (and device) companies, R&D experts, small and large trialists, methodologists, statisticians, NIH, EMA and FDA experts, as well as input from payers, lawyers, and importantly from “patients trialists,” a mix unique to CVCT meetings.
  • FOCUSED WORKSHOP
    Focused CVCT Workshops are organized as campfire-sessions, fit for smaller focused groups: the experts and participants brainstorm around a U shape-table, as equals. These are high caliber faculty with the typical mix of stakeholders from academia, regulatory, payers, industry, patients and journal editors. The moderated panel discussion aims at fostering exchanges, potentially shifting the lines and reaching operational conclusions.
    Orphan and rare cardiovascular diseases include rare diseases of systemic or pulmonary circulation, cardiomyopathies, congenital or genetic cardiovascular diseases, arrhythmogenic disorders, cardiac tumors and cardiovascular disease in malignancy, cardiovascular diseases in pregnancy, and subsets of common cardiovascular conditions (e.g. heart failure with preserved ejection fraction).

    There are distinctive features of clinical and regulatory development of drugs in such rare diseases, compared to standard common CV diseases. This includes tiny patient pools, challenging recruitment strategies, “creative” adaptive design and biostatistical approaches adapted to “small” samples, more reliance on biomarkers, surrogate endpoints, and patient reports outcomes endpoints and frequent use of post-approval safety and Real-World Evidence data collection. Topics worth discussing are whether i) there are lessons Small Trialists may offer to Large Trialists, and vice-versa, ii) innovations of rare disease trials may or may not be extendable to large trials, iii) common diseases will approach rare diseases with the advent of precision medicine, iv) FDA and EMA may act synergistically and better align in more impactful, respective, or common guidance documents.


4:00 PM – 6:30 PM
iCVCT Session 4 REVASCULARIZATION STRATEGIES PCI V. CABG V. MEDICAL THERAPY

  • Moderators: C. Michael Gibson (Boston, USA); Chris Granger (Durham, USA)
    ISCHEMIA and COURAGE: Where do we stand for Chronic Coronary Syndromes?
    Darrel Francis (London, GBR)
    ISCHEMIA and COURAGE: Where do we stand for Chronic Coronary Syndromes?
    Bernard Gersh (Rochester, USA)
    ISCHEMIA and COURAGE: Where do we stand for Chronic Coronary Syndromes?
    David Maron (Stanford, USA)
  • PCI v. CABG: EXCEL, NOBLE, SYNTAXES
    Roxana Mehran (New York, USA)
  • Multi-Vessel Disease in STEMI and Shock: COMPLETE or Culprit only PCI?
    David J. Cohen (Kansas City, USA)
  • Mechanical Support Devices in Complex PCI- Hope or Hype?
    Kendra Grubb (Atlanta, USA)
  • Evidence for Support Devices in STEMI and Shock- What have we learned?
    Mina Karami (Amsterdam, NED)
  • The NHLBI viewpoint
    Yves Rosenberg (NIH, USA)
  • Regulatory viewpoint
    Mauro Moscucci (FDA, USA)
  • Industry viewpoint
    Kenneth Stein (Boston Scientific, USA)
  • Patient viewpoint
    Brenda Alsemgeest (Amsterdam, NED)
  • iCVCT Session 4 The CVCT Forum: Moderated Multi-stakeholder panel discussion HOW WILL THE CLINICIAN KNOW WHOM TO TREAT?
    Moderators: C. Michael Gibson (Boston, USA); Chris Granger (Durham, USA)

  • Panelists: Brenda Alsemgeest (Amsterdam, NED); Jeff rey Borer (New York, USA); David J. Cohen (Kansas City, USA); Sidney Cohen (Medtronic, USA); George Dangas (New York, USA); Andrew Farb (FDA, USA); Darrel Francis (London, GBR); Kendra Grubb (Atlanta, USA); David Holmes (Rochester, USA); Mina Karami (Amsterdam, NED); Susheel Kodali (New York, USA); David Maron (Stanford, USA); Roxana Mehran (New York, USA); Mauro Moscucci (FDA, USA); Ileana Piña (Detroit, USA); Yves Rosenberg (NIH, USA); Tabassome Simon (Paris, FRA); Kenneth Stein (Boston Scientifi c, USA); Ellis Unger (FDA, USA); Martin Unverdorben (Daiichi-Sankyo, USA); Bernard Vasseur (FDA, USA); Bram Zuckerman (FDA, USA)


TOCQUEVILLE

3:30 PM – 5:00 PM
Part I: Creative solutions for rare disease trials

  • Clinical and Regulatory challenges/Opportunities in Rare Cardiovascular Diseases
    Moderators: Kimberly Chapman (Washington, USA); Janet Wittes (Washington, USA)
    Kimberly Chapman (Washington, USA)
  • Innovative design and statistical solutions for small clinical trials
    Janet Wittes (Washington, USA)
  • Innovative operational solutions enabling trials in rare/orphan disease
    Marlene Haffner (Haffner Associates, USA)
  • Challenges of matching patients, clinical trials, and practitioners
    Sameer Ather (Xpert Dox, USA)
  • Case Study: Advanced Therapy for Rare CV Disease
    Douglas Losordo (Caladrius, USA)
  • The CVCT Forum: Moderated multi-stakeholder panel discussion Part I: Creative solutions for rare disease trials
    Moderators: Kimberly Chapman (Washington, USA); Janet Wittes (Washington, USA)
  • Panelists: Sameer Ather (Xpert Dox, USA); Marlene Haff ner (Haff ner Associates, USA); Douglas Losordo (Caladrius, USA)


Adjourn C. Michael Gibson


TOCQUEVILLE

5:30 PM – 7:00 PM
Part II: The innovations of rare disease trials and how they may or may not be extendable to large trials

  • Are FDA and EMA guidance aligned regarding clinical development of drugs for rare/orphan diseases?
    Moderators: Alexander Fleming (Kinexum, USA); Marlene Haffner (Haffner Associates, USA)
    Kolbeinn Gudmundsson (CHMP, EMA, ICE); Ilan Irony (FDA, USA)
  • Statistical viewpoint
    Janet Wittes (Washington, USA)
  • Industry viewpoint
    Jay Edelberg (Myokardia, USA); Alexander Fleming (Kinexum, USA)
  • Payer viewpoint
    Joe Hutter (CMS, USA)
  • Patient viewpoint
    Brenda Alsemgeest (Amsterdam, NED); Robin Martinez (Denver, USA)
  • The CVCT Forum: Moderated multi-stakeholder panel discussion Part II: The innovations of rare disease trials and how they may or may not be extendable to large trials
    Moderators: Alexander Fleming (Kinexum, USA), Marlene Haffner (Haffner Associates, USA)
  • Panelists: Brenda Alsemgeest (Amsterdam, NED); Philippe Brudi (Kinexum, USA); Jay Edelberg (Myokardia, USA); Alan Fisher (Kinexum, USA); Kolbeinn Gudmundsson (CHMP, EMA, ICE); Joe Hutter (CMS, USA); Ilan Irony (FDA, USA); Robin Martinez (Denver, USA); Manal Morsy (Athersys, USA); Janet Wittes (Washington, USA)