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Yale-Mayo Clinic Centers of Excellence in Regulatory Science and Innovation (CERSI) B12 Pediatric Device Survey
Online SURVEY COMPONENT 1
We are conducting a study of pediatric medical device development with funding and support from the U.S. Food and Drug Administration (or FDA). You have been identified as having valuable expertise in this field and we are seeking your assistance with this inquiry. The study has two components: one completed on-line, the second with an option to complete either on-line or over the phone (or video call). This on-line component of the survey should require no more than 60 minutes to complete.
Your responses are confidential; if content from any individual responses are reported in publications emerging from this project, they will be attributed to an anonymous study ID number, rather than an identified respondent. Participation in this study is completely voluntary. You are free to decline to participate, to end participation at any time for any reason, or to refuse to answer any individual question without penalty.
Our research team is affiliated with the Yale University-Mayo Clinic Center of Excellence in Regulatory Science & Innovation (CERSI). Our findings may help to inform policy and practice at the FDA; however the agency will not be privy to the primary data collected through this study, will not have access to the identity of any respondents, nor will be directly involved in the analysis of the data collected.
Background
Please enter your study ID number included in the email inviting you to participate in this study: ______________ [Have auto-filled if possible]
Which sectors have you worked in? Select all that apply.
Clinical Care
Medical Device Manufacturer
Trade Organization/Industry Representative
Regulatory Affairs
Payer
Investor
Other ________________________ [please fill in]
For what type(s) of conditions and indications do you have experience in the development or promotion of pediatric medical devices? Select all that apply.
□ Cardiovascular
□ Orthopedic
□ Diabetes Mellitus
□ Neurology
□ Other: _______________________ [please fill in]
For how many devices that you have been involved with over the past ten years has there been some consideration of submitting for FDA review for pediatric uses?
□ None
□ One
□ Two
□ Three
□ Four
□ Five or more
How many devices that you have been involved with over the past ten years have actually been submitted for FDA review of pediatric uses?
□ None
□ One
□ Two
□ Three
□ Four
□ Five or more
Barriers
We would first like your perceptions of factors that create the largest impediments to developing new products for pediatric use and submitting those products for FDA review. Please consider how these factors influence the medical device industry as a whole, not simply your own company.
From your perspective, how frequently are the following barriers encountered to developing products for the pediatric population? (
[Response Scale:1=Never 2=Rarely 3=Sometimes 4=Often 5=Always]
Economic Incentives and Market Conditions
Low priority within existing medical device companies
Lack of funding from external investors
Small pediatric market generates inadequate revenue
Reimbursement challenges related to insurance coverage (coding and coverage)
Challenges associated with the sales and marketing of pediatric devices
Engineering
Engineering challenges associated with creating devices for children (e.g., creating devices for bodies that grow, lack of appropriate animal models, determining appropriate bench testing)
Clinical Trials
Regulation
Burdensome requirements during FDA regulatory review process
Challenges associated with obtaining clearance/approval for multiple device sizes
Lack of international regulatory harmonization
Liability/Reputation Issues
Increased product liability profile associated with pediatric devices and trials
Potential negative publicity if a child is harmed by an approved device
Which of the following is the most consequential barrier to developing products for the pediatric population? Select one.
Low priority within existing medical device companies
Lack of funding from external investors
Small pediatric market generates inadequate revenue
Reimbursement challenges related to insurance coverage (coding and coverage)
Challenges associated with the sales and marketing of pediatric devices
Costs of marketing pediatric devices
Engineering challenges associated with creating devices for children
Difficulty conducting pediatric clinical trials
Burdensome requirements during FDA regulatory review process
Challenges associated with obtaining clearance/approval for multiple device sizes
Lack of international regulatory harmonization
Increased product liability profile associated with pediatric devices and trials
Potential negative publicity if a child is harmed by an approved device
Relative to each other, how consequential are each of the following broad categories of factors in preventing companies from submitting devices for pediatric review? Please allocate 10 points among the categories, with a higher number of points for the more consequential barriers.
Economic Incentives (Market Conditions) ___
Engineering Devices ___
Logistics of Clinical Trials ___
Regulation ___
Liability and Reputation Issues ___
Company culture or historical focus ___
TOTAL ____ [auto sum column; if not = 10 provide
error message].
Incentives
We would now like your perceptions of the incentives or policies that could most effectively motivate the development of medical devices for pediatric use or expand labeling to pediatric populations for devices that have already been developed for the adult population.
How much impact would you expect each of the following incentives or policies to have?
(From 1-5, where 1 is no impact and 5 is very large impact.)
Development Incentives
Tax credit incentives totaling 25% of development costs of the pediatric application for FDA-approved device use (i.e., pediatric R&D tax credits)
Tax credit incentives totaling 50% of development costs of the pediatric application
(i.e., more generous pediatric R&D tax credits)
Tax credit incentives totaling 10% of development costs of the adult application
(i.e., indirect R&D tax credits)
Tax credit incentives totaling 20% of development costs of the adult application
(i.e., more generous indirect R&D tax credits)
Regulatory Review
Fees and Timing
Elimination of device user fee for devices developed for both adult and pediatric use
Elimination of user fee for supplements to expand adult indications to pediatric populations
Review Time
Defined/mandated timelines for device review / approval process
Expedited regulatory review for all pediatric submissions
Expedited review for a follow- up adult indication if a company first develops a pediatric device and obtains clearance/approval for pediatric indication
Review Process and Logistics
Greater acceptance of pre-clinical modeling
“Reverse” extrapolation (i.e., extrapolate data from pediatric submissions to adult submissions)
Use of small confirmatory pediatric trials with supportive data from registries, adult data extrapolation or real-world evidence
Greater emphasis on post-market data collection or monitoring in lieu of pre-market data
All reviewers for pediatric applications selected to have expertise in the pediatric population specific expertise
Progressive Review
Progressive review for all devices
Progressive review limited to pediatric devices
Progressive review limited to pediatric devices used in particular settings
Coverage
Mandated coverage by Medicaid at the national level for all devices receiving FDA review and approval for pediatric use.
Mandated private insurance coverage for all devices receiving FDA review and approval for pediatric use.
Breakthrough designation following review process for Medicaid coverage, which would result in a coverage bonus of 15% of otherwise reimbursable costs
Breakthrough designation following review process for Medicaid coverage, which would result in a coverage bonus of 25% of otherwise reimbursable costs
Evidence Generation
Single contract access to a national network of pediatric academic medical centers
Single IRB review established for such a national network of pediatric academic medical centers
Comparisons to Pharmaceutical Development
Some past policies have incorporated combinations of incentives to motivate drug development for particular populations. We would like you to consider four such policies, first considering their suitability for application to pediatric devices, then their likely impact if adapted.
Each of the following policies/practices have been adopted for pharmaceuticals. How applicable would these be for pediatric device development? (From 1-5, where 1 is not at all applicable and 5 is very applicable.)
Orphan Drug Act: tax credits for 50% of development costs, 7-year market exclusivity, and waived user fees.
Best Pharmaceuticals for Children Act (BPCA): 6-month market exclusivity to a drug/biologic that conducts testing in children in response to a written request from the FDA.
Pediatric Research Equity Act (PREA): mandated pediatric studies for drugs in which the condition exists in children.
Priority Review Voucher: transferrable vouchers for expedited review of some other drug after approval of a pediatric drug. These vouchers can be resold and used for any product, including those deployed exclusively in adult populations.
How large of an impact would these policies likely have on pediatric device development? (From 1-5, where 1 is no impact and 5 is very large impact.)
Orphan Drug Act: tax credits for 50% of development costs, 7-year market exclusivity, and waived user fees.
Best Pharmaceuticals for Children Act (BPCA): 6-month market exclusivity to a drug/biologic that conducts testing in children in response to a written request from the FDA.
Pediatric Research Equity Act (PREA): mandated pediatric studies for drugs in which the condition exists in children.
Priority Review Voucher: transferrable vouchers for expedited review of some other drug after approval of a pediatric drug. These vouchers can be resold and used for any product, including those deployed exclusively in adult populations.
Mandates and Regulatory Requirements
A final category of policies involve a variety of regulatory requirements for device manufacturers. These requirements could be coupled with incentives to enhance the feasibility of implementation. We would like your assessment of both their general acceptability within the industry, as well as your prediction of the likely impact that they would have on pediatric device development.
How acceptable to the device industry would the following be?
Response Scale [1=very unacceptable, 2=somewhat unacceptable, 3=Neutral
4=somewhat acceptable, 5=very acceptable]
Regulatory Mandates Alone
Require submission of devices for pediatric review if the conditions appear in children above some threshold prevalence
Mandate that all off-label uses of medical devices in children by reported to a national registry
Regulatory Mandates Combined with Incentives
Mandatory submission for pediatric review combined with a tax credit for 25% of development costs
Mandatory submission for pediatric review combined with a tax credit of 50% of development costs
Mandatory submission for pediatric review combined with a requirement that Medicaid cover all FDA approved pediatric devices
Mandatory submission for pediatric review combined with a requirement that Medicaid and private insurance cover all FDA approved pediatric devices
How much of an impact would this have on pediatric device development?
Response Scale: [1= significantly decrease, 2=somewhat decrease, 3=unclear impact
4= somewhat increase, and 5 =is significantly increase]
Regulatory Mandates Alone
Require submission of devices for pediatric review if the conditions appear in children above some threshold prevalence
Mandate that all off-label uses of medical devices in children by reported to a national registry
Regulatory Mandates Combined with Incentives
Mandatory submission for pediatric review combined with a tax credit for 25% of development costs
Mandatory submission for pediatric review combined with a tax credit of 50% of development costs
Mandatory submission for pediatric review combined with a requirement that Medicaid cover all FDA approved pediatric devices
Mandatory submission for pediatric review combined with a requirement that Medicaid and private insurance cover all FDA approved pediatric devices
What else would you like us to know about the current state of pediatric device development or potential changes that could be made to current practices? (open-ended box)
Thank you for taking time to respond to our survey!
| File Type | application/vnd.openxmlformats-officedocument.wordprocessingml.document |
| Author | Emily Boudreau |
| File Modified | 0000-00-00 |
| File Created | 2024-09-09 |