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How to Make Postmarket Surveillance More Cost Effective
June 16, 2016
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Introduction
Post Market Surveillance Status in the Industry
Value Based Healthcare and Real World Evidence
Rising Costs and Opportunities for Cost Effectiveness
Case Studies
Agenda
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Vicki Anastasi
Vice President and Global Head Medical Device and Diagnostics Research
ICON Medical Device and Diagnostics Research Group
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4,300 + Europe, Middle East, Africa
2,000 + Asia Pacific
4,900 + The Americas
ICON: A market leader positioned for continued growth
Operating from 90 offices in 37 countries with 11,900 employees
PHD or Higher - 9%
Master Degree - 24%
Bachelor Degree - 39%
Diploma/Certificate - 13%
Other - 15%
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Seren Phillips HTA training slides
ICON Expertise Over the Medical Device Lifecycle
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Medical Device Clearance / Approval Process
Concept & Design
( 12 mo)
Pre-Clinical Development ( 24-36 mo)
Clinical Trials Proof of
Concept / Pivotal Trial
(Timing varies)
FDA Review (510K3-5 mo) (PMA 12 -24
mo)
Reimbursement Assignment ( 0-24 mo)
Post Market Activities
Regulatory strategy Pre-submission meeting Quality System support
Pre-clinical test plans Human factors testing
Study conduct & report preparation
IDE Submission 510K / PMA Submission
FDA discussion / negotiations
Post Market studies Registry studies
Product enhancements / new submissions Technology
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Rise of Post Market Surveillance Global Impact
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50% increase in the number of PMS studies conducted by top 5 medical device manufacturers between 2009 and 2015 (ClinicalTrials.gov analysis)
Worldwide requirements from regulators, payers To intensify under EU MDR/IVDR
Major changes proposed by the Commission Impact on non-grandfathered CE-approved products already on the market Effect of changes on diagnostics and companion diagnostics Changes in clinical evidence requirements Impact on the industry of increased requirements, as the number of Notified Bodies
is reduced
Costs are not the only challenge! Viability of product in the value-based healthcare environment will become paramount.
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Value-Based Healthcare is Driving Increase in Post-Market Data
Global market launch needs to be aligned with future requirements
Requires a different framework to define value and commercial success
Bundled payment model: CMS Comprehensive Care for Joint Replacement
Model program delivers on CMS pledge to shift 50% of Medicare payments to ACOs and bundles by 2018
Expect the next area, based on volume and variance in per-procedure costs, to be cardiovascular
How does your product fit in to these programs? What is next?
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Global Rationale of Value Driven Data Collection
What are the effects? (e.g., providers have targeted manufacturers for price concessions first, before reforming care protocols or delivery)
Lessons from UK [e.g., payments differ between indications, thus requiring careful choice of patient population (e.g., co-morbidities) as hospitals become smarter about codes]
Start earlier to accelerate true time-to-market: Arrival at regulatory clearance with limited evidence of real-world cost effectiveness and a suboptimal prioritization of payer targets will delay market access, restrain early revenue potential, and concede control of a products value story.
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Broadening Opportunities for Real World Evidence
The "top programmatic priority" for the US Food and Drug Administration
(FDA), under Commissioner Robert Califf, is to leverage real world evidence
from the healthcare system to inform FDA decision making
Prospectively designed registries and cohort studies in the context of clinical practice are highly valuable, and randomized trials conducted in the context of clinical practice, often called a pragmatic clinical trial may be the most important source of knowledge in the future Robert Califf, FDA Commissioner
Food and Drug Law Institute's Annual Conference, May, 2016
First, both data sources and research methods must be optimized
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http://www.raps.org/Regulatory-Focus/News/2016/03/10/24517/Real-World-Evidence-Can-it-Support-New-Indications-Label-Expansions/
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Seren Phillips HTA training slides
Real World Evidence Solutions
As healthcare accelerates towards a patient-centric, value based approach, a new set of questions are emerging from key stakeholders : How will patients respond to your product when it enters the real world? How does its value proposition hold up within the competitive set and current
standards of care? Which subset of patients are likely to see the greatest benefit?
Reasons for optimism Digitization of healthcare continues to increase New technologies improving data capture, connectivity and access Numerous RWE data sources available
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Seren Phillips HTA training slides
Robust RWE Strategy Drives Commercial Success
Preliminary Objectives Situational Assessment
Disease and subtype dynamics Treatment Landscape
Current standard of care, treatment algorithms Clinical and in market targets with indications current and anticipated profiles for
safety and efficacy Review of clinical research programs
Determine Opportunities and Potential Challenges How can these be better identified, quantified exploited and mitigated through RWE What is of primary importance and what can be addressed by secondary RWE data
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Seren Phillips HTA training slides
Real World Evidence Solutions
Herein lies the challenge with Real World Evidence
Industry has a plethora, perhaps too many, real world evidence options to consider
How should companies go about navigating what should be generated de novo primary RWE (i.e. Registries) versus sourced, secondary RWE (available data)
Determining a sound RWE strategy requires a multifaceted approach
You need expertise in both realms as well as clinical, medical, operational, data and outcomes expertise, to define your RWE strategy
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Real World Evidence Case Study
Maestro Rechargeable Systems approved based on Patient Preference Information
Real World Intelligence (RWE): drive R&D with insights from RWE that is
provided earlier in the process and across the usual clinicalmedical affairs
commercial divide (facilitates deeper payer and patient-centric insights into
optimal target indications, opportunities to improve care management, product
feature profiles that align to potentially non-obvious patient populations, viable
protocol designs, endpoint sensitivity, or recruitment strategies)
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Key market Strategy Registry Plan: Design, Execution, and Communication
REPORTS
LEGAL, REGULATORY, IRB REVIEW
MATERIALS PRODUCTION AND DISTRIBUTION SITE RECRUITMENT AND TRAINING
PATIENT ENROLLMENT, OUTCOMES TRACKING,
DATA COLLECTION
SITE SUPPORT
ANALYSES
NEWSLETTERS
PUBLICATIONS ABSTRACTS, PRESENTATIONS
MEETINGS
STRATEGY
ANALYSIS PLAN COMMUNICATIONS PLAN
DATA COLLECTION FORMS, PROCESSES, AND LOGISTICS SCIENTIFIC ADVISORY PANEL
SITE IDENTIFICATION (FIELD INVOLVEMENT)
Sheet1
SITE SUPPORTPUBLICATIONSABSTRACTS, PRESENTATIONSREPORTS
ANALYSES
PATIENT ENROLLMENT, OUTCOMES TRACKING, DATA COLLECTIONMEETINGS
NEWSLETTERS
MATERIALS PRODUCTION AND DISTRIBUTIONSITE RECRUITMENT AND TRAINING
LEGAL, REGULATORY, IRB REVIEW
DATA COLLECTION FORMS, PROCESSES, AND LOGISTICSSCIENTIFIC ADVISORY PANELSITE IDENTIFICATION (FIELD INVOLVEMENT)
ANALYSIS PLANCOMMUNICATIONS PLAN
STRATEGY
Sheet2
Sheet3
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Methodological Considerations: Patient Selection, Size & Scope
Target Population
Assessable Population
Intended Population
The population to which the study
findings are meant to apply
Subset of the target population available
for the study
Subset of the population sampled
according to the registry design
Patients who actually participate in the
registry
Identification of the target population All patients
Actual Population
Defining Registry Size & Scope: Design Considerations
Duration of the registry o Time needed to assess outcomes o Plan for evolution?
How many patients per site? o Size of overall patient population o Enrollment timeframe expectations o Level of engagement o Not too many to bias analysis, but enough to be interesting to sites o 5-10 is generally the rule
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Patient registry success primarily hinges on a well-reasoned design and good data collection practices
Success Factors for Patient Registries Broad entry criteria Make sure entry criteria is not limited to specific treatments to facilitate comparisons
Ideal ration of incident vs. prevalent cases
It is key to strike the right balance between methodological purity (only newly diagnosed) vs. practicality (more eligible patients, if you allow previously diagnosed)
Targeted data collection
While you may have the ability to collect large amounts of data, sometimes it is a good idea to collect only what you know you plan to analyze
However, often very interesting questions can be addressed when collecting extra information (the drawback is that you risk losing sites and/or patients)
It is important to get both clinical data and patient-reported data, because neither can substitute for the other
Data collection automation Get objective medical information (e.g. labs, utilization information, Rx refill information, etc.) in an automated way, because it will greatly enhance your registry at a relatively low cost
Address known biases Address all known biases at the design stage, including Patient selection within sites and systematic
differences between sites (academic/community), because these may subsequently affect interpretation of the results
Data control Control access to the data by having a single team of analysts this guards against misinterpretation of the data and poor-quality analyses
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Costs are Accelerating Best Practices for Management
Post-Market surveillance budgets rival clearance/approval budgets
Build an evidence generation roadmap
Consider all stakeholders needs are considered
Leverage Payer and Market Access available data
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Opportunities to Increase Cost Effectiveness
Operational differences for post-market data collection
Site selection
Use of networks and EMR
Enrollment
Site management
Continual training
Monitoring
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Opportunities to Increase Cost Effectiveness Technology
Site training Risk based monitoring Ease the burden of patient participation
Truly informed consent/eConsent Better informed participants
Reduce visits with mobile, cloud and wearables Types of sensors Data gathering Apps Patients vs healthy individuals what is available
Outsourcing is it a solution for your program?
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Evidence Generation Roadmap: The role of real world data in the product lifecycle
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LIFECYCLE
POST MARKET
NO PHASE RESEARCH
CONCEPT
PRECLINICAL & PHASE I
PRE-CLINICAL & FEASIBILITY
PHASE II
FIRST IN MAN
PHASE III
PILOT
REGISTRATION
PIVOTAL
Strategy Development
Strategy Development
Clinical Outcomes Assessment
Peri-Approval & Observational Research / Medical Device & Diagnostic Research
Health Economics & Epidemiology
PRODUCT LIFECYCLE
Patient / Physician surveys
Observational studies
Preference Studies (DCE / WTP)
Select or develop PROs / ClinROs / ObsROs, including linguistic validation
PRO measurement strategy - Efficacy endpoints and labelling
Regulatory interactions / submissions - Dossier development
PRO dissemination strategy
Regulated and non regulated clinical studies
Phase IIIB/IV Prospective Trials
Directed Use Programs
Registries (pharma /device/ commercial strategy)
Interventional / Non-Interventional Research
PASS, PAES
Comparative Effectiveness Research
Burden of Illness
Resource Use Studies
Epidemiology Characterisation of Disease & Treatment Patterns
Dynamic Transition Modelling
Economic Modelling
Meta-Analysis, NMA
PERI-POST APPROVAL
POST-APPROVAL
PHARMA:
DEVICE:
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Case Study: Seamless Transition from PMA to Post-Market Study
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Case Study: Rapid Post-Market Study Start-up
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Accelerating Time to Market and Eliminating $5 Million in Development Costs through Adaptive Design
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Your New Prescription for
Commercial Success
Peri-Approval & Observational Research
Global Pricing & Market Access
Healthcare Communications
Medical Device and Diagnostics Research
Clinical Outcomes Assessment
Market Research
Creative Digital Language Services
Content Call Center
ICON Commercialisation & Outcomes
Health Economics & Epidemiology
Real World Evidence
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How to Make Postmarket Surveillance More Cost EffectiveOMTEC-2016_Anastasi_Postmarket-SurveillanceHow to Make Postmarket Surveillance More Cost EffectiveAgendaICON Medical Device and Diagnostics Research GroupICON: A market leader positioned for continued growthICON Expertise Over the Medical Device LifecycleMedical Device Clearance / Approval ProcessRise of Post Market Surveillance Global ImpactValue-Based Healthcare is Driving Increase in Post-Market DataGlobal Rationale of Value Driven Data CollectionBroadening Opportunities for Real World EvidenceReal World Evidence SolutionsRobust RWE Strategy Drives Commercial SuccessReal World Evidence SolutionsReal World Evidence Case StudyKey market Strategy Registry Plan: Design, Execution, and CommunicationMethodological Considerations: Patient Selection, Size & ScopePatient registry success primarily hinges on a well-reasoned design and good data collection practices Costs are Accelerating Best Practices for Management Opportunities to Increase Cost EffectivenessOpportunities to Increase Cost Effectiveness TechnologyEvidence Generation Roadmap: The role of real world data in the product lifecycleCase Study: Seamless Transition from PMA to Post-Market StudyCase Study: Rapid Post-Market Study Start-upAccelerating Time to Market and Eliminating $5 Million in Development Costs through Adaptive DesignSlide Number 25
Online closing side OMTEC 2016 speaker presentations