IRB PRESENTATION REGULATORY PATHWAYS HDE – PMA William Hellenbrand MD Director – Pediatric Cardiology Columbia University College of Physicians & Surgeons.

Slides:



Advertisements
Similar presentations
Confidentiality, Consent and Data Protection Elizabeth M Robertson Deputy Medical Director Grampian University Hospitals Trust.
Advertisements

Regulatory Pathway for Platform Technologies
Susan Burner Bankowski, M.S., J.D. Chair, OHSU IRB
Regulation of Medical Devices Celia M. Witten, Ph.D., M.D. Director Division of General, Restorative and Neurological Devices Office of Device Evaluation.
Rare Diseases and FDASIA
Percutaneous Therapy of Pulmonic and Mitral Valve Disease Atman P. Shah MD FACC FSCAI Director, Coronary Care Unit Assistant Professor of Medicine The.
FDA Regulated Research:
Humanitarian Device Exemptions (HDE) 101 Elizabeth Hillebrenner, MSE Biomedical Engineer IDE and HDE Program Staff Center for Devices and Radiological.
Single Use Expanded Access IND/IDE: FDA and IRB Requirements Before and After Use IRB Webinar October 9,2014.
Clinical Trials Medical Interventions
CORE COMPETENCIES IN CLINICAL & TRANSLATIONAL RESEARCH: The Child Health Perspective I. Clinical & Translational Research Questions: Extract information.
CUMC IRB Investigator Meeting Special IND/IDE Considerations: Emergency Use of Investigational Product Compassionate Use & Emergency Research July 21,
Special Topics in IND Regulation
Medical Devices Approval Process
Cancer Clinical Trials: The Basics. 2 What Are Cancer Clinical Trials? Research studies involving people Try to answer scientific questions and find better.
+ Medical Devices Approval Process. + Objectives Define a medical device Be familiar with the classification system for medical devices Understand the.
1 THE UNIQUE ROLES OF IRB IN MEDICAL DEVICE CLINICALL TRIAL Chiu Lin, Ph.D. CITI, May, 2009 CITI, May, 2009.
Device Research Presented by Marian Serge, R.N.. Goals Identify devices Recognize difference between significant risk (SR) and non- significant risk (NSR)
Adverse Events, Unanticipated Problems, Protocol Deviations & other Safety Information Which Form 4 to Use?
The Medical Device Pathway as a Legal Onramp for Futuristic Persons THE FUTURE T HE M EDICAL D EVICE P ATHWAY AS A L EGAL.
Hormone Refractory Prostate Cancer A Regulatory Perspective of End Points to Measure Safety and Efficacy of Drugs Hormone Refractory Prostate Cancer Bhupinder.
Investigational Drugs in the hospital. + What is Investigational Drug? Investigational or experimental drugs are new drugs that have not yet been approved.
Humanitarian Use Devices September 23, 2011 Theodore Stevens, MS, RAC Office of Cellular, Tissue and Gene Therapies Center for Biologics Evaluation and.
بسم الله الرحمن الرحيم جامعة أم درمان الإسلامية كلية الطب و العلوم الصحية - قسم طب المجتمع مساق البحث العلمي / الدفعة 21 Basics of Clinical Trials.
Trial Design Issues Associated with Evaluation of Distal Protection Devices in Diseased Saphenous Vein Grafts Bram D. Zuckerman, MD, FACC Medical Officer,
Investigational Devices and Humanitarian Use Devices June 2007.
June 23, 2005Abiomed, H DRAFT. CIRCULATORY SUPPORT DEVICES PANEL Thursday, June 23, 2005 Abiomed, Inc. Abiocor® Implantable Replacement Heart HDE.
EVALUATING u After retrieving the literature, you have to evaluate or critically appraise the evidence for its validity and applicability to your patient.
Human and Animal Research 1. What issues does this raise? 2.
HUD and Emergent Use Walter Kraft. Device Classification Significant risk – Often involve an invasive procedure for implantation or use – Requires IDE.
Human Specimen Repositories Requirements of 21 CFR Parts 50 & 56 PRIM & R May 5, 2004 Sally A. Hojvat, Ph.D. Director of Microbiology Devices Office of.
Making Randomized Clinical Trials Seem Less Random Andrew P.J. Olson, MD Assistant Professor Departments of Medicine and Pediatrics University of Minnesota.
Purpose of Clinical Trials Assess safety and efficacy of Experimental treatments New combinations of drugs New approaches to surgery or radiation therapies.
Radiology Advisory Panel Meeting Radiology Advisory Panel Meeting Computer-Assisted Detection (CADe) Devices Joyce M. Whang Deputy Division Director Radiological.
Innovation in Pediatric Medical Devices: Thinking Outside The Box Gwenyth Fischer University of Minnesota Masonic Children’s Hospital.
November 9, 2015 February 20, 2017 Using real world evidence – industry perspective Pma indication expansion Melissa hasenbank, phd Sr. Clinical Research.
Rachel Neubrander, PhD Division of Cardiovascular Devices
PERCUTANEOUS PULMONARY VALVE REPLACEMENT:
The CRT of EFS Where We’ve Been and Where We’re Going
Use of Postmarket Data to Support Premarket Approvals
GCP AND MEDICAL DEVICES
Clinical Trial Design for Second Generation TAVI - Academic View
What Are the FDA Requirements for Submitting an IDE?
Division of Cardiovascular Devices
Adherence to the Labeling
FDA’s IDE Decisions and Communications
Balancing Pre and Postmarket Requirements Different Scenarios
Balancing Regulation and Innovation: An FDA Division of Cardiovascular Devices Perspective Bram Zuckerman, MD, FACC Director, FDA Division of Cardiovascular.
Insights from the Melody Pulmonic Transcatheter Valve Panel
University of Pennsylvania
To start the presentation, click on this button in the lower right corner of your screen. The presentation will begin after the screen changes and you.
Reasonable Assurance of Safety and Effectiveness: An FDA Division of Cardiovascular Devices Perspective Bram Zuckerman, MD, FACC Director, FDA Division.
Clinical Trials Medical Interventions
Martha Carvour, MD, PhD March 2, 2017
Deputy Director, Division of Biostatistics No Conflict of Interest
Patent Foramen Ovale Devices and Trials Update: Is the Current Data Sufficient for Approval? CRT 2017 Feb18-21, 2017 Steven L. Goldberg, MD Medical.
Introduction of New Technology: An FDA Division of Cardiovascular Devices Perspective Bram Zuckerman, MD, FACC Director, FDA Division of Cardiovascular.
Medical Device Regulatory Essentials: An FDA Division of Cardiovascular Devices Perspective Bram Zuckerman, MD, FACC Director, FDA Division of Cardiovascular.
Structural Heart Disease and Devices in Japan and USA
Early Feasibility in the USA –An Academic View
Erica Takai, PhD for Andrew Farb, M.D.
Clinical Trials.
To start the presentation, click on this button in the lower right corner of your screen. The presentation will begin after the screen changes and you.
Speeding access to therapies
Streamlining IRB Procedures for Expanded Access
Issues in Hypothesis Testing in the Context of Extrapolation
Implantable Medical Devices: Accelerating Standards Development to Streamline Regulation Joshua Price | August 2,
Opening an IND: Investigator Perspective
Ethical Considerations for Pediatric Clinical Investigations
Research, Experimentation, & Clinical Trials
Presentation transcript:

IRB PRESENTATION REGULATORY PATHWAYS HDE – PMA William Hellenbrand MD Director – Pediatric Cardiology Columbia University College of Physicians & Surgeons Morgan Stanley Children’s Hospital of New York-Presbyterian

DISCLOSURE Medtronic –Investigator –Consultant –Proctor

REGULATORY PATHWAYS Paucity of high level evidence for therapy in congenital heart disease(CHD) –Reviewed 3317 articles over 3 year period( ) related to therapy for CHD –Surgery (49%), Catheter Interventions (23%), medications (9%)… –Case reports & series (52%), non randomized - controlled series (45%), RCT (3%) –Over a 10 year period there were 213 RCTs in pediatric cardiology, 3263 in neonatology & 5394 in adult ischemic heart disease Senthilnathan, Selvi ; Postgraduate course in Pediatric Cardiovascular Disease; Feb 2008

REGULATORY PATHWAYS  Randomization:  Reduces the likelihood of patient selection bias  Enhances likelihood that comparable groups of subjects are actually compared  Supports use of common statistical tests Randomization is the gold standard for evaluating new Device and Drug therapies Zuckerman TCT 2005

ADVANTAGES OF MULTI- CENTER TRIALS No single center has the resources to answer a question Collaboration will speed the resolution of the research question Collaboration will resolve the question more authoritatively

REGULATORY PATHWAYS Barriers to randomized multi-center trials –Congenital cardiovascular diseases are rare and diverse disorders –Randomization is difficult –Proper endpoints are difficult –Lack of clinical equipoise –Lack of equal maturity of the techniques being compared

RANDOMIZATION IS DIFFICULT Many medical devices are already approved and available Off label use is not regulated as the IRB does not require informed consent if we promise not to collect data or learn anything about the procedure We have to have informed consent if we randomize and perform the procedure in only half the patients instead of all of them

RANDOMIZATION IS DIFFICULT Due to all of these issues and patient knowledge from physicians and especially the internet; patient entry into these clinical trials may preclude adequate patient numbers

ENDPOINTS ARE DIFFICULT They must be important –The FDA traditionally interested in mortality(do you live longer) or feel better (symptoms) They must be achievable –Outcomes must be measurable in a reasonable time frame or have surrogate markers that accurately predict outcome

ENDPOINTS ARE DIFFICULT Patients are not likely to die (30% of the population and 2% of the deaths) Symptoms are difficult to quantify Patients are a moving target as they grow and develop

HDE VS PMA How does the FDA view these two different regulatory pathways and the need for multi-center randomized trials in a population of diverse disorders and a small number of patients

HDE The regulation provides for the FDA submission of a humanitarian device exemption (HDE) application, which is similar in both form and content to a premarket approval (PMA) application. However, unlike a PMA application, an HDE application is not required to contain the results of scientifically valid clinical investigations demonstrating that the device is effective for its intended purpose. The HDE application must, however, contain sufficient information for the FDA to determine that the device does not pose an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use; taking into account the probable risks and benefits of currently available devices or alternative forms of treatment. Additionally, the HDE applicant must demonstrate that no comparable devices are available to treat or diagnose the disease or condition, and that the device could not otherwise be brought to market unless it is granted HUD status.

OFF LABEL USE OF AN APPROVED DEVICE Off-Label Use of a Humanitarian Use Device in Emergency or Compassionate Situations: It is recognized that there may be circumstances in which “off-label” use of a HUD may be necessary to save the life or protect the well-being of a given patient. Under either of these situations, the involved physician and manufacturer of the device should, on a case-by-case:

MELODY ® - TPV

INDICATIONS & INTENDED PERFORMANCE Indications RVOT conduit regurgitation RVOT conduit stenosis Intended Performance Restore pulmonary valve competence Relieve conduit stenosis without inducing regurgitation Prolong conduit life

MELODY ® - TPV RVOT conduits used to palliate complex congenital heart defects RVOT conduit dysfunction is common  Functional lifespan of RVOT conduits limited due to progressive regurgitation & stenosis Current options for management – Medical management – Surgical pulmonary valve replacement – Transcatheter management

MELODY ® - TPV

OFF LABEL USE Why is there a difference in off label use of a PMA vs an HDE approved device With a PMA approved device it is not regulated by the FDA or the IRB and left to clinical judgment