Download Methodological Issues in Implantable Device Studies

Methodological Issues in
Implantable Medical Device(IMDs)
Studies
Abdallah ABOUIHIA
Senior Statistician, Medtronic
Objectives
 Introduction
 Key factors that impact IMD safety &
effectiveness
 Methodological issues related to Comparative
Studies of IMDs
 Summary & Recommendations
 Medical Device Working Group
Introduction
Medical Devices
Introduction
 IMDs are ubiquitous inside/ outside clinical
practice
 Use and complexity are increasing
 Device-related adverse events & Product
problems are significant
 Technical complexity & operator training have
great impact on public health
 Device-operator interaction plays an important role
in device safety and Effectiveness
Introduction
 Environment for evidence generation of IMD
outcome different than that for drug
 Trial is methodologically challenging:
randomization, blinding…..
 Design of trails requires care and innovative
approaches
 Observational studies of IMDs have their own
challenges
Key factors that impact IMD safety & effectiveness
Key factors that impact IMD Safety &
Effectiveness
 Selection of Comparator
 Characteristics of the Device
 Characteristics of the Operator
 Characteristics of the
Procedure/Intervention
 Characteristics of the Intervention Setting
 Characteristics of the Patient
 Selection and Measurement of Outcome
Selection of Comparator
 Choice has a critical implications and challenging
issues
 RCT: blinding and allocation concealment issues more
pronounced
 Observational study: blinding and allocation
concealment not possible
 Need more data on factors that drive therapeutic
choices
 Long term follow-up for device to optimal management
comparative studies
Characteristics of the Device
 Mechanical performance and reliability of the
device
 battery life duration, physical wear of the implant
 Identify variable associated with device failure
 IMDs continually evolve thus difficult to accurately
assess safety and effectiveness
Characteristics of the Operator
 Interpretation of evidence and preference are
critical factors for adopting a new technology
 Skill and learning curve are factors for adoption
and use
 Many large trials of IMDs procedure are conducted
by highly skilled operator
 Important implication in designing studies and
evaluating the evidence in real world
 Operator preferences driven by many factors:
training, institutional, reimbursement condition…
Characteristics of the Procedure/Intervention
 Understand various procedural factors related to
IMDs
 Documentation of surgical techniques/approaches
 Recognize and account for procedural choices,
invasiveness of treatment
Characteristics of the Intervention Setting
 Evaluate hospital and access factors
 Determine the groups of patients able to receive
IMDs
 Understand reasons for offering a particular IMD
technology
 Financial & organizational reasons behind hospital’s
choice
Characteristics of the Patient
 Learn about specific patient population
 Impact of diffusion of new technology on outcomes
of the IMD
 Real-world use might create difficulties in realworld evaluations of IMDs
 Patient outcomes important to the assessment of
Safety & Effectiveness not systematically collected
Selection and Measurement of Outcome
• Choice of outcome affect the strength of the
evidence
• Clinically meaningful outcomes are preferred
• Use of surrogate endpoint can very challenging
• Outcomes are combined into indexes due to small
sample sizes
• Use of disease specific measures of health status
become quit important
• Lack of information on QoL or Functional Status
Methodological issues related to Comparative
Studies of IMDs
• Blinding and Masking is difficult to achieve
• Inclusion/Exclusion is affected after initial
Intent/randomization
• Impacts analytical approach including Intention to
treat
• Learning curve issues affect the outcome of the
IMD
Methodological issues related to Comparative
Studies of IMDs
 Small Sample Size is a reality in IMDs
 Device modification and changes to medical
practice during study
 Potential for imbalances among treatment groups
 Generalizibility is affected when less sick patient,
Elite operators participate
Summary of Methodological issues and
recommendations
Medical Device Working Group
 Communication with decision makers in medical
device industry
 Lack of product development model similar to drug
business
 Review ICH and FDA guidelines to assess their
applicability to device setting
 Pursue methodological work on this area
Contact for membership:
[email protected]
[email protected]
Thank you