FDA BRIEF: Week of June 12, 2017
FDA Science: Working at the Speed of Emerging Technologies
By: Luciana Borio, M.D., Acting Chief Scientist
Innovation is happening extraordinarily fast in biomedical sciences and at FDA
- FDA’s 11,000 scientists play essential role in advancing biomedical innovations
- Scientific research presented at Science Forum at FDA
- Research concentrates on developing knowledge to ensure medical products are safe and effective
2017 Science Forum topics
- Identification and Evaluation of New Biomarkers
- FDA Response to Urgent Public Health Needs
- Microbiome and Human Health
- Advanced Manufacturing and 3D Printing
- Omics Technologies at FDA
- Patient and Consumer Engagement and Communication
- Computational Modeling and Simulation at FDA
- Current Progress in Nanotechnology Research at FDA
Mobile communications also included
- Healthy Citizen @FDA to collaborate and communicate with citizens on public health outcomes
Fostering Medical Innovation: A Plan for Digital Health Devices
By: Scott Gottlieb, M.D., Commissioner, @SGottliebFDA
165,000 health-related apps with 1.7 billion downloads by 2017
- From mobile apps and fitness trackers to clinical decision support software
New Digital Health Innovation Plan
- To foster innovation at the intersection of medicine and digital health technology
- Novel, post-market approach for regulation and communicate them early
Guidances on products outside FDA scope
- Clarify provisions in 21st Century Cures Act about what falls outside FDA scope (e.g. clinical administrative support software, healthy lifestyle apps)
- Products with multiple software functions (some fall outside FDA scope)
- Other technologies with low risk with no pre-market regulatory requirements
Efficient, risk-based regulatory framework
- Third party certification program with no premarket review (low risk) or streamlined review (higher risk)
- Post-market collection of real-world data to support new product functions
- Leverage real-world data gathered through the National Evaluation System for health Technology (NEST)
Help innovators navigate a new, modern regulatory process
Statement from FDA Commissioner Scott Gottlieb, M.D. – FDA is taking new steps to help assess opioid drugs with abuse-deterrent properties
Everyone at FDA is committed to focusing on all aspects of the opioid epidemic
New policy steps
- Steering committee formation to examine regulatory and policy action
- Evaluate efforts to reduce number of new addiction cases
Abuse-deterrent opioid formulations and public health effect assessment
- Formulations intended to deter abuse
- However, effect in a real-world, meaningful decrease of opioid misuse/abuse unknown
- Public meeting on opioid medications with abuse-deterrent properties to discuss impact of these products in the real world
- Publicly available issues paper outlining existing regulatory and public health challenges
How Creative FDA Regulation Led to First-in-the-World Approval of a Cutting-Edge Heart Valve
FDA was first to approve Sapien 3 valve interatin to treat high-risk patients with transcatheter valve replacement (TAVR)
- For high-risk patients with worn out aortic or mitral bioprosthetic valves; Sapien 3 slips into these valves – “valve-in-valve” option
- FDA Heart Valve Review Team streamlined FDA’s nonclinical testing expectations
- More consistent, predictable, and transparent about clinical study expectations
- Industry collaboration on creative clinical trial designs and use of other sources of clinical evidence
Use of real-world evidence for approval
- Based on Transcatheter Valve Therapy (TVT) Registry, partnership of American College of Cardiology and Society of Thoracic Surgeons
- Collection of clinical data on TAVR performance both on-label and off-label uses
- 100,000 TAVR patients since 2011 first approval; >600 patients for off-label, valve-in-valve uses
- Reliance on real-world evidence to evaluate the benefits and risks of off-label use
FDA working to broaden and improve the opportunities to leverage real-world evidence
- Establishment of National Evaluation System for health Technology (NEST)
- Integrate data from clinical registries, electronic health records, and medical billing claims
Image credits: FDA