FDA BRIEF: Week of July 29, 2016
SCOPE:
Real-World Data (RWD): Data collected from sources outside of traditional clinical
trials
Real-World Evidence (RWE): Evidence derived from aggregation and analysis of
RWD elements.
FDA’s National Evaluation System leverages RWD to identify safety problems, to better understand the benefit-risk profile, reduce the time and cost of evidence generation
RWE:
- Of sufficient quality to provide confidence in analyses for regulatory decision making
- May be used in total product life cycle e.g. generating clinical study hypotheses, historical control, concurrent control group, expand labeling, public health surveillance
RWD:
- Relevance: Ddata adequately addresses applicable regulatory question/ requirement
- Reliability: Data accrual, Data adequacy, Data assurance
Comment period : End October.
SCOPE:
Defines expected content and forms of the Unique Device Identifier (UDI) to assist both labelers and FDA-accredited issuing agencies
UDI Rule:
- Create a standardized identification system for medical devices in US
- Adequately identify devices through distribution and use
UDI Forms:
- Easily readable plain-text
- AIDC
UDI Content, Data delimiters
Comment period: End September
SCOPE:
How to plan and implement adaptive designs for clinical studies when used in medical device development programs
Adaptive Designs:
- prospectively planned modifications based on accumulating study data without undermining the study’s integrity and validity
- efficient, saving time, money, and resources
- can improve the chance of trial success by employing sample size reassessment
- may facilitate transition from premarket to postmarket follow-up
- can enhance patient protection
- can include a plan to modify the patient population during the study
- can improve decision-making at milestones
- Limitations: more effort at the design stage, can introduce operational or statistical bias, may confound the interpretation,
Principles:
- control of the chance of erroneous conclusions (positive and negative)
- minimization of operational bias
Adaptations Using Unblinded Data:
- Group Sequential Designs, Sample Size Reassessment, Bayesian Sample Size Adaptation, Group Sequential Designs etc.
Special Considerations:
- Not Preplanned chnages, Simulations, Safety Endpoints, Open-Label Randomized Studies, Observational Comparative Studies, One-Arm Studies without a Control
Challenges:
- Data Monitoring Committees, Minimize Operational Bias, IRBs, Logisitcs
Regulatory Considerations:
- FDA interactions, Sponsor Monitoring, Protect Study Blinding
POLICY for low risk general wellness products
CDRH does not intend to examine
- whether they are devices
- whether they comply with the premarket review and post-market regulatory requirements for devices
- whether they are implementing regulations : registration and listing, premarket notification, labeling, good manufacturing practice, Medical Device Reporting
Must meet factors
- intended for only general wellness use AND
- present a low risk to the safety of users and other persons
Intended Use
- Maintaining or encouraging a general state of health or a healthy activity
- Relates the role of healthy lifestyle with helping to reduce the risk or impact of certain chronic diseases or conditions
CATEGORIES
Sustaining or offering general improvement to functions with general health
- weight management, physical fitness, relaxation or stress management, mental acuity, self-esteem, .sleep management, sexual function
Sustaining or offering general improvement to functions associated with a general state of health while making reference to diseases or conditions
- may help to reduce the risk of certain chronic diseases or conditions
- may help living well with certain chronic diseases or conditions
QUESTIONNAIRE to determine fit