1. Clinical and Fiscal “Cost of Delay” – a statistician’s view
In spite of the cost of living, it's still popular.
-Kathleen Norris

2. Roseann M. White
Salary:
Abbott Vascular

3. Agenda Needs for evidence development from regulatory authorities
Needs for evidence development from both the public and private payors
Needs for evidence development from the sponsor

4. Delays related to evidence development from regulatory authorities (1 of 3)
Evolving knowledge in the field
Previous methods of evidence development (non-clinical, pre-clinical or clinical) are no longer sufficient
No established process how to modify, update existing information
Costs
Need to redo
Communicating to the patient and medical community
Continued use of potentially a less efficacious and/or cost-effective
Develop a processes using existing statistical mechanisms that would allow for updating information
Augmentation
Bayesian
Meta-analyses

5. Delays related to evidence development from regulatory authorities (2 of 3)
Evolving knowledge during the clinical trial population that would most benefit from this device
No established process how to modify, update existing information
Costs
Need to redo
Communicating to the patient and medical community
Continued use of potentially a less efficacious and/or cost-effective
Develop a processes using existing statistical mechanisms that would allow for optimizing the population during the course of the trial
Adaptive designs
Use of other data such as data repositories

6. Delays related to evidence development from regulatory authorities (3 of 3)
The benefits do not outweigh the risks
Decisions on approval are based the totality of the data
Need to develop a transparent and pre-specified mechanism for assessing benefit and riskBenefit
Costs
Need to redo
Communicating to the patient and medical community
Continued use of potentially a less efficacious and/or cost-effective
Benefit – Risk Project
EMA “Work package 2 report: Applicability of current tools and processes for regulatory benefit-risk assessment”

8. Delays related to evidence development from Public and Private Payors
Comparative Effectiveness
Evidence developed during the approval process is not sufficient for coverage
Evidence developed by two different sponsors with similar devices are inconsistent
Costs
Need to develop more evidence
Communicating to the patient and medical community
Continued use of potentially a less efficacious and/or cost-effective
Involving tech assessors in the process of developing the evidence for regulatory approval

9. Needs for evidence development from the sponsor
To paraphrase former President Clinton, "Intelligence, ability, and aspiration are evenly distributed across [medical device companies] – organization, investment, and opportunity are not.“
Evidence developed without protocols, statistical plans, appropriate monitoring, CEC, DSMB, etc.
Costs
Need to develop more evidence
Lower company evaluation and loss of funding
Continued use of potentially a less efficacious and/or cost-effective
Establish a minimum set of quality standards for developing evidence

10. Summary The costs of delay can include Delays can come from
Delay patient access to a cost effective devices
Redevelopment evidence that may or may not be possible for the sponsor
Communication as to reasons for the delay
Delays can come from
Regulatory Authorities
Payors
Sponsor
Develop methodologies to address potential
Adaptive/flexible designs
Data augmentations
Risk/Benefits analyses