Download clinical trial burden

Medtech in Transition:
Where are we now and what
lies ahead? – State of the industry
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Deepak Nath, Ph.D.
President, Abbott Vascular
Disclosure
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Agenda
REDUCING THE MEDICAL DEVICE
“CLINICAL TRIAL BURDEN”
– Least burdensome approach
– Clinical trial innovation
– Use of Real-World Evidence
OPPORTUNITIES WITH MARKET ACCESS
FOR MEDICAL DEVICES
– A Paradigm Shift
– Coverage with Evidence Development
– Local Coverage Determinations
– IDE Clinical Trial Coverage
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Least burdensome approach to evaluate effectiveness
Excerpt from the FD&C Act (Statute 360c(a)(3)D):
ii) Any clinical data, including one or more well-controlled investigations, specified
in writing by the Secretary for demonstrating a reasonable assurance of device
effectiveness shall be specified as result of a determination by the Secretary that
such data are necessary to establish device effectiveness. The Secretary shall
consider, in consultation with the applicant, the least burdensome appropriate
means of evaluating device effectiveness that would have a reasonable likelihood
of resulting in approval.
Language demonstrates FDA’s commitment to working with
the industry to reduce clinical trial burden while continuing to meet
requirements for safety and effectiveness
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Clinical trial innovation
Medical Device Innovation Consortium (MDIC) Clinical Trial
Science:
– Alternative, innovative trial designs
– Different models of data collection that consider data at multiple points in the
development of a new product
– Simplification of clinical trials design
FDA’s active engagement to working with stakeholders to reduce
clinical trial burden while continuing to meet requirements for safety
and effectiveness
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Use of real-world evidence to support regulatory
decision-making to reduce clinical trial burden
Proposed use of real-world evidence derived from real-world
data (the collection and use of data generated in the postmarket setting (e.g., EHRs, registries) may:
– Improve patient access to new therapies through efficient collection of data
– Evaluate the “real-world” safety and/or effectiveness of products
– Generate data to support coverage, reimbursement, and value analysis
Alternate methods to monitor the performance of technologies
reducing pre- and post-market burden for data collection
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Market access is no longer
just about regulatory approval
MARKET AUTHORIZATION
/APPROVAL
R&D/REGULATORY/CLINICAL
Safety
Efficacy
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Quality
MARKET PLACE CHALLENGES
Reimbursement/
HTA/Economic
Evaluation
Budgeting
Protocols and
Restrictions /
Hospital VACs
Patient Cost-Sharing
Value-Based Pricing
Transitioning to a value based model
OLD PARADIGM
Clinical innovation was sufficient for trial and increased adoption
INNOVATION
MODEL
Innovation
Safety &
Efficacy
Product
Experience
Adoption
NEW PARADIGM
• Decision making shifted from purely clinical decision to balance between clinical,
economic value and patient value
• Requires medical device manufacturers to demonstrate economic and patient
value to gain access
VALUE-BASED
MODEL
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Innovation
Safety &
Efficacy
Economic
& Patient Value
Market
Access
Abbott: Establishing value across CV portfolio
• MitraClip®
Transcatheter
Mitral Valve
Repair
CardioMEMS™
Heart Failure
System
OPTIS™ (FFR/OCT)
System
2.9
88%
73%
Low Hospital
Length of Stay1,2
88% of patients
discharged to home1,2
73% reduction in HF
hospitalization rate1,2
days
COST
QUALITY
• 2.3 more days alive outside
the hospital vs. SOC1
• Lowers patient hospitalization
rates2
• $8,506 reduction in
comprehensive HF
care cost5
Multivessel Disease:
FFR-guided PCI vs
Angiography
• 28% ↓ MACE1
• Saves hospitals $2,385 per
case2
©2017 Abbott. All rights reserved. SE2943747 Rev A.
• 37% ↓ HF
Hospitalizations1
• 58% ↓ 30-day
Readmissions3
• Patient Quality of Life
improvement at 6 mos1
OUTCOMES
• 37% ↓ HF Hospitalizations1
• 57% ↓ Mortality over SOC
Pts on GDMT managed with
PA pressures4
Stable CAD:
FFR-guided PCI + OMT vs.
OMT Alone
FFR Guidance: Intermediate,
Obstructive/Significant or
Indeterminate severity
Lesions
• 86% ↓ risk of unplanned
hospitalizations for
revascularization3
• FFR meets appropriate use
criteria for lesion severity4
• Documented FFR may reduce
exposure to audits
See Important Safety Information referenced within
Coverage with Evidence Development (CED):
Ensure balance burden/cost with value of data
• Supports coverage approach that permits access to innovative technologies
while evidence is gathered in a systematic manner
• CED, when integrated with FDA post-approval commitments, can be a costeffective way to gather data on real-world use
• CED should be reserved for unique technologies and circumstances. When data
are robust, CED may be inappropriate
• A goal of any CED program should be to cover all indicated patients and expand
access to new therapies
For each CED consider:
• Will data collected via CED be valuable for clinicians and decision makers?
• Does the CED program strike the right balance between the usefulness of data
vs. cost and provider burden to collect and report?
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Local Coverage Determination (LCD) opportunities
• Most technologies are subject to the local coverage process
administered by Medicare Administrative Contractors (MACs)
Opportunities to improve the process:
• Technologies with New Technology
Add-on Payment and/or Transitional
Pass-through Payment have inconsistent
coverage by MACs
Medicare Administrative Contractors
(MAC) map of jurisdictions
• Category III CPT codes receive blanket
non-coverage policies. MACs need to
consider available evidence when
making coverage decisions
• Enhance transparency and stakeholder
access in coverage process
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Squares/rectangles indicate A/B Mac Jurisdictions
that have NOT yet transitioned to A/B MACs
Circles indicate A/B MAC Jurisdictions that have
transitioned to A/B MACs
IDE Clinical Trial Reimbursement:
Positive advances in last 2 years
BEFORE 2015: Coverage was
not automatic or consistent
BEGINNING IN 2015: Centralized
Reimbursement Process
• Each clinical investigator site had to notify
their local Medicare contractor (MAC) of
their intent to enroll Medicare patients into
the clinical study and wait for approval
• Medicare coverage now determined
centrally based on Sponsor application;
Manufacturer applies not Investigators
• Submission packet had to be prepared
by Each clinical investigator and
submitted as far in advance of enrolling
Medicare patients
• Part A (Hospital claims) and Part B
(Physician services) are separate and
required independent contractor approval.
• Process took 3 weeks to 6 months
©2017 Abbott. All rights reserved. SE2943747 Rev A.
• Most patients will be covered for standard
of care treatment at current payment level
• Application straight forward
• CMS updates list of approved trials weekly
• Currently, 106 approved studies. 2 – 6
weeks. Have been up to 3months.
• Uncertainty regarding early feasibility and
continued access studies
Expand Use of New Data Sources for Approval,
Reimbursement & Post-Market Surveillance
Randomized
Controlled
Trials
•
•
•
•
•
Randomized Controlled Trials
Registries
Real World Registries
Claims Analysis
Electronic Health Records
Work together to determine how to utilize expanded data sources and big
data to better understand new technologies and serve patients
©2017 Abbott. All rights reserved. SE2943747 Rev A.
Sources for Slide 9
MitraClip® Transcatheter Mitral Valve Repair
1. MitraClip Clip Delivery System Instructions for Use
2. Lim D.S., Reynolds M.R., Feldman T., et al. Improved functional status and quality of life in prohibitive surgical risk patients with
degenerative mitral regurgitation following transcatheter mitral valve repair with the MitraClip® system. J Am Coll Cardiol. 2014;64:182–
192.
CardioMEMS™ Heart Failure System
1. Abraham WT, Adamson PB, Bourge RC, et al. Wireless pulmonary artery hemodynamic monitoring in chronic heart failure: A randomized
controlled trial. Lancet. 2011;377(9766);658-66.
2. Adamson PB, et al. Cost Effectiveness Assessment of Pulmonary Artery Pressure Monitoring for Heart Failure Management. HRS 2015,
Boston, MA. AB36-01
3. Adamson, P. B., et al. (2014). Impact of wireless pulmonary artery pressure monitoring on heart failure hospitalizations and 30-day
readmissions in Medicare-eligible patients with NYHA class III heart failure: results from the CHAMPION trial. Circulation, 133, A16744
4. Abraham, W. T., et al. (2015, March). Pulmonary artery pressure management in heart failure patients with reduced ejection fraction
significantly reduces heart failure hospitalizations and mortality above and beyond background guideline-directed medical therapy.
Abstract 902-04 presented at ACC 2015, San Diego, CA.
5. Medicare claims analysis from June 2014 – June 2016
OPTIS™ (FFR/OCT) System
1. Tonino PA, De Bruyne B, Pijls NH, et al. Fractional flow reserve versus angiography for guiding percutaneous coronary intervention. N Engl
J Med. 2009;360(3):213-24.
2. Fearon WF, Bornschein B, Tonino PA, et al. Economic evaluation of fractional flow reserve-guided percutaneous coronary intervention in
patients with multivessel disease. Circulation. 2010;122(24):2545-50.
3. De Bruyne B, Pijls NH, Kalesan B, et al. Fractional flow reserve-guided PCI versus medical therapy in stable coronary disease. N Engl J Med.
2012;367(11):991-1001.
4. Patel MR, Smith PK, Spertus JA, et al. ACCF / SCAI / STS / AATS / AHA / ASNC / HFSA / SCCT 2012 Appropriate Use Criteria for coronary
revascularization: Focused update. J Am Coll Cardiol. 2012;59(9):857-81.
©2017 Abbott. All rights reserved. SE2943747 Rev A.
IMPORTANT SAFETY INFORMATION
MITRACLIP® NT CLIP DELIVERY SYSTEM
INDICATION FOR USE
The MitraClip® NT Clip Delivery System is indicated for the percutaneous reduction of significant
symptomatic mitral regurgitation (MR ≥ 3+) due to primary abnormality of the mitral apparatus
[degenerative MR] in patients who have been determined to be at prohibitive risk for mitral valve
surgery by a heart team, which includes a cardiac surgeon experienced in mitral valve surgery and a
cardiologist experienced in mitral valve disease, and in whom existing comorbidities would not
preclude the expected benefit from reduction of the mitral regurgitation.
CONTRAINDICATIONS
The MitraClip® NT Clip Delivery System is contraindicated in DMR patients with the following
conditions:
• Patients who cannot tolerate procedural anticoagulation or post procedural anti-platelet
regimen
• Active endocarditis of the mitral valve
• Rheumatic mitral valve disease
• Evidence of intracardiac, inferior vena cava (IVC) or femoral venous thrombus
WARNINGS
• DO NOT use MitraClip® NT outside of the labeled indication. Treatment of non-prohibitive risk
DMR patients should be conducted in accordance with standard hospital practices for surgical
repair and replacement.
• MitraClip® NT is intended to reduce mitral regurgitation. The MitraClip® NT procedure is
recommended to be performed when an experienced heart team has determined that reduction
of MR to ≤2+ is reasonably expected following the MitraClip® NT. If MR reduction to ≤2+ is not
achieved, the benefits of reduced symptoms and hospitalizations, improved quality of life, and
reverse LV remodeling expected from MitraClip® NT may not occur.
• The MitraClip® NT Device should be implanted with sterile techniques using fluoroscopy and
echocardiography (e.g., transesophageal [TEE] and transthoracic [TTE]) in a facility with on-site
cardiac surgery and immediate access to a cardiac operating room.
• Read all instructions carefully. Failure to follow these instructions, warnings and precautions
may lead to device damage, user injury or patient injury. Use universal precautions for
biohazards and sharps while handling the MitraClip® NT System to avoid user injury.
• Use of the MitraClip® NT should be restricted to those physicians trained to perform invasive
endovascular and transseptal procedures and those trained in the proper use of the system.
• The Clip Delivery System is provided sterile and designed for single use only. Cleaning, resterilization and/or reuse may result in infections, malfunction of the device or other serious
injury or death.
©2017 Abbott. All rights reserved. SE2943747 Rev A.
PRECAUTIONS
• Patient Selection:
– Prohibitive risk is determined by the clinical judgment of a heart team, including a cardiac
surgeon experienced in mitral valve surgery and a cardiologist experienced in mitral valve
disease, due to the presence of one or more of the following documented surgical risk factors:
• 30-day STS predicted operative mortality risk score of
– ≥8% for patients deemed likely to undergo mitral valve replacement or
– ≥6% for patients deemed likely to undergo mitral valve repair
• Porcelain aorta or extensively calcified ascending aorta.
• Frailty (assessed by in-person cardiac
surgeon consultation)
• Hostile chest
• Severe liver disease/cirrhosis
(MELD Score >12)
• Severe pulmonary hypertension
(systolic pulmonary artery pressure >2/3 systemic pressure)
• Unusual extenuating circumstance, such as right ventricular dysfunction with severe
tricuspid regurgitation, chemotherapy for malignancy, major bleeding diathesis,
immobility, AIDS, severe dementia, high risk of aspiration, internal mammary artery (IMA)
at high risk of injury, etc.
– Evaluable data regarding safety or effectiveness is not available for prohibitive risk DMR
patients with an LVEF < 20% or an LVESD > 60mm. MitraClip® NT should be used only when
criteria for clip suitability
for DMR have been met.
– The major clinical benefits of MitraClip® NT are reduction of MR to ≤2+ resulting in reduced
hospitalizations, improved quality of life, reverse LV remodeling and symptomatic relief in
patients who have no other therapeutic option. No mortality benefit following MitraClip® NT
therapy has been demonstrated.
• The heart team should include a cardiac surgeon experienced in mitral valve surgery and a
cardiologist experienced in mitral valve disease and may also include appropriate physicians to
assess the adequacy
of heart failure treatment and valvular anatomy.
• The heart team may determine an in-person surgical consult is needed to complete the
assessment of prohibitive risk. The experienced mitral valve surgeon and heart team should take
into account the outcome of this surgical consult when making the final determination of patient
risk status.
IMPORTANT SAFETY INFORMATION
(CONTINUED)
• For reasonable assurance of device effectiveness, pre-procedural evaluation of the mitral valve
and underlying pathologic anatomy and procedural echocardiographic assessment are essential.
• Note the “Use by” date specified on the package.
• Inspect all product prior to use. Do not use if the package is open or damaged, or if product is
damaged.
POTENTIAL COMPLICATIONS AND ADVERSE EVENTS
The following ANTICIPATED EVENTS have been identified as possible complications of the
MitraClip® NT procedure.
Allergic reaction (anesthetic, contrast, Heparin, nickel alloy, latex); Aneurysm or pseudo-aneurysm;
Arrhythmias; Atrial fibrillation; Atrial septal defect requiring intervention; Arterio-venous fistula;
Bleeding; Cardiac arrest; Cardiac perforation; Cardiac tamponade/Pericardial Effusion; Chordal
entanglement/rupture; Coagulopathy; Conversion to standard valve surgery; Death; Deep venous
thrombus (DVT); Dislodgement of previously implanted devices; Dizziness; Drug reaction to antiplatelet/ anticoagulation agents/contrast media; Dyskinesia; Dyspnea; Edema; Emboli (air,
thrombus, MitraClip® NT Device); Emergency cardiac surgery; Endocarditis; Esophageal irritation;
Esophageal perforation or stricture; Failure to deliver MitraClip® NT to the intended site; Failure to
retrieve MitraClip® NT System components; Fever or hyperthermia; Gastrointestinal bleeding or
infarct; Hematoma; Hemolysis; Hemorrhage requiring transfusion; Hypotension/hypertension;
Infection; Injury to mitral valve complicating or preventing later surgical repair; Lymphatic
complications; Mesenteric ischemia; MitraClip® NT erosion, migration or malposition; MitraClip®
NT Device thrombosis; MitraClip® NT System component(s) embolization; Mitral stenosis; Mitral
valve injury; Multi-system organ failure; Myocardial infarction; Nausea/ vomiting; Pain; Peripheral
ischemia; Prolonged angina; Prolonged ventilation; Pulmonary congestion; Pulmonary thromboembolism; Renal insufficiency or failure; Respiratory failure/atelectasis/pneumonia; Septicemia;
Shock, Anaphylactic or Cardiogenic; Single leaflet device attachment (SLDA); Skin injury or tissue
changes due to exposure to ionizing radiation; Stroke or transient ischemic attack (TIA); Urinary
tract infection; Vascular trauma, dissection or occlusion; Vessel spasm; Vessel perforation or
laceration; Worsening heart failure; Worsening mitral regurgitation; Wound dehiscence
STEERABLE GUIDE CATHETER
INDICATION FOR USE
The Steerable Guide Catheter is used for introducing various cardiovascular catheters into the left
side of the heart through the interatrial septum.
©2017 Abbott. All rights reserved. SE2943747 Rev A.
CONTRAINDICATIONS
• Patients who cannot tolerate procedural anticoagulation or post procedural anti-platelet
regimen
• Evidence of intracardiac, inferior vena cava (IVC) or femoral venous thrombus.
WARNINGS
• Read all instructions carefully. Failure to follow these instructions, warning and precautions may
lead to device damage, user injury or patient injury. Use universal precautions for biohazards
and sharps to avoid user injury.
• Use the Steerable Guide Catheter with sterile techniques using fluoroscopy and
echocardiography (e.g., transesophageal [TEE] and transthoracic [TTE]) in a facility with on-site
cardiac surgery and immediate access to a cardiac operating room.
• The Steerable Guide Catheter is designed for single use only. Cleaning, re-sterilization and/or
reuse may result in infections, malfunction of the device or other serious injury or death.
• Patients with the following considerations in whom the Steerable Guide Catheter is used may
have an increased risk of having a serious adverse event which may be avoided with
preoperative evaluation and proper device usage.
– Previous interatrial septal patch or prosthetic atrial septal defect (ASD) closure device which
could result in significant difficulty in visualization or technical challenges during transseptal
puncture and/or introducing the SGC into the left atrium.
– Known or suspected unstable angina or myocardial infarction within the last 12 weeks could
increase the procedural morbidity and mortality, due to increased hemodynamic stress
secondary to general anesthesia.
– Patients with active infection have an increased risk of developing an intraoperative and/or
postoperative infection, such as sepsis or soft tissue abscess.
– Known or suspected left atrial myxoma could result in thromboembolism and tissue injury
due to difficulty with device positioning.
– Recent cerebrovascular event (CVA) may increase the procedural morbidity associated with a
transcatheter intervention, such as recurrent stroke.
PRECAUTIONS
• NOTE the “Use by” date specified on the package.
• Inspect all product prior to use. Do not use if package is opened or damaged.
Prior to use, please reference the Instructions for Use at www.abbottvascular.com/ifu for more
information on indications, contraindications, warnings, precautions, and adverse events.
IMPORTANT SAFETY INFORMATION
CARDIOMEMS
Brief Summary:Prior to using these devices, please review the User’s Manual for a complete
listing of indications, contraindications, warnings, precautions, potential adverse events and
directions for use.
CAUTION:FEDERAL LAW (USA) RESTRICTS THIS DEVICE TO SALE, DISTRIBUTION AND USE BY
OR ON THE ORDER OF A PHYSICIAN.
INDICATIONS AND USAGE:
The CardioMEMS HF System is indicated for wirelessly measuring and monitoring pulmonary
artery (PA) pressure and heart rate in New York Heart Association (NYHA) Class III heart failure
patients who have been hospitalized for heart failure in the previous year. The hemodynamic
data are used by physicians for heart failure management and with the goal of reducing heart
failure hospitalizations.
CONTRAINDICATIONS:
The CardioMEMS™ HF System is contraindicated for patients with an inability to take dual
antiplatelet or anticoagulants for one month post implant.
POTENTIAL ADVERSE EVENTS:
Potential adverse events associated with the implantation procedure include, but are not
limited to the following: Infection, Arrhythmias, Bleeding, Hematoma, Thrombus, Myocardial
infarction, Transient ischemic attack, Stroke, Death, and Device embolization.
Refer to the User’s Manual for detailed indications, contraindications, warnings, precautions
and potential adverse events.
©2017 Abbott. All rights reserved. SE2943747 Rev A.
IMPORTANT SAFETY INFORMATION
CARDIOMEMS
BRIEF SUMMARY:
Prior to using these devices, please review the User’s Manual for a complete listing of
indications, contraindications, warnings, precautions, potential adverse events and directions
for use.
CAUTION:FEDERAL LAW (USA) RESTRICTS THIS DEVICE TO SALE, DISTRIBUTION AND USE BY
OR ON THE ORDER OF A PHYSICIAN.
INDICATIONS AND USAGE:
The CardioMEMS HF System is indicated for wirelessly measuring and monitoring pulmonary
artery (PA) pressure and heart rate in New York Heart Association (NYHA) Class III heart failure
patients who have been hospitalized for heart failure in the previous year. The hemodynamic
data are used by physicians for heart failure management and with the goal of reducing heart
failure hospitalizations.
CONTRAINDICATIONS:
The CardioMEMS™ HF System is contraindicated for patients with an inability to take dual
antiplatelet or anticoagulants for one month post implant.
POTENTIAL ADVERSE EVENTS:
Potential adverse events associated with the implantation procedure include, but are not
limited to the following: Infection, Arrhythmias, Bleeding, Hematoma, Thrombus, Myocardial
infarction, Transient ischemic attack, Stroke, Death, and Device embolization.
Refer to the User’s Manual for detailed indications, contraindications, warnings, precautions
and potential adverse events.
©2017 Abbott. All rights reserved. SE2943747 Rev A.
OPTIS INTEGRATED SYSTEM
BRIEF SUMMARY:
Please review the Instructions for Use prior to using these devices for a complete listing of
indications, contraindications, warnings, precautions, potential adverse events and directions
for use.
Thank you
Deepak Nath, Ph.D.
President, Abbott Vascular
Abbott Vascular
3200 Lakeside Dr., Santa Clara, CA. 95054 USA, Tel: 1.800.227.9902
Caution: This product is intended for use by or under the direction of a physician. Prior to use, reference the Instructions for Use provided inside the
product carton (when available) or at www.abbottvascular.com/ifu for more detailed information on Indications, Contraindications, Warnings, Precautions
and Adverse Events.
Illustrations are artist’s representations only and should not be considered as engineering drawings or photographs. Photos taken by and on file at Abbott
Vascular.
MitraClip, CardioMEMs, and OPTIS are trademarks of the Abbott Group of Companies.
www.AbbottVascular.com
©2017 Abbott. All rights reserved. SE2943747 Rev A.