FDA-EMA Patient Engagement Cluster

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WHAT: Cluster to share best practices involving patients along drug and biologic regulatory lifecycles

WHY: Broadened approach to advance and strengthen international collaboration

WHO: FDA and European Medicines Agency (EMA)

HOW:  Meet up to four times per year

  • Learn how their respective patients are engaged and involved
  • Develop common goals of expanding future patient engagement activities



FDA NEWS: Rare Disease Grants, Idea Portal to Predict Future, FDA/EMA Clusters, Cyber Security Month

FDA BRIEF: Week of October 17, 2016 


 21 FDA grants to stimulate product development for rare diseases

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AWARD of 21 new clinical trial research grants totaling > $23 million over 4 years

  • Boost development of products for rare diseases
  • To Principal Investigators from academia and industry (drugs and devices)
  • For 21 different rare diseases with little or no available treatment
  • Through the Orphan Products Clinical Trials Grants Program
  • For clinical studies evaluating product safety and effectiveness contributing to FDA approval


  • 24  % for cancer particularly devastating brain cancer
  • 43% for  pediatric patients as young as newborns
  • 1 medical device for neuroprosthesis
  • Funding rate: 31%


Introducing FDA’s Emerging Sciences Idea Portal: Please Help Us Predict the Future

By: Donna L. Mendrick, Ph.D., Associate Director for Regulatory Activities, NCTR

Donna Mendrick


  • Representatives from FDA product and research centers & offices
  • Leverage scientific expertise and resources to conduct long-range horizon scanning
  • Emerging issues and cross-cutting scientific advances
  • FDA preparedness to proactively address  emerging issues and scientific advances


  • Identify areas not yet addressed in current products e.g. brain-computer interfaces
  • Develop science-based planning, programs, policies, reporting, communication


  • Cast a wide net – Within and outside FDA
  • Experts in  private sector


  • For science and technology experts outside of government
  • Submit predictions on the next new things in their field of specialization – will barely show up on a web search
  • NOT looking for advances that are already under discussion.



New FDA/EMA rare diseases and patient engagement clusters underway


Jonathan C. Goldsmith, M.D., FACP, Associate Director, Rare Diseases Program, CDER

Sandra Kweder, M.D., Deputy Director, EU Office, EMA Liaison

Dr. Jonathan Goldsmith

Sandra Kweder


  • FDA collaborates with other countries and international regulatory agencies for global development programs
  • European Medicines Agency (EMA) is valuable collaborator : 4,500 scientists, supervises medicines for > 500 million people in 31 countries


  • > 10 years
  • Work in groups called “clusters”
  • Summarized in EMA website.


  • To advance treatments for patients with rare diseases
  • Share information and review aspects of rare disease drug development programs
  • Identification and validation of trial end points
  • Potential trial designs
  • Flexibility in evaluation of drug development programs
  • Expediting review and approval


  • Incorporate the patient’s involvement and viewpoint in the drug development process
  • Develop common goals of expanding patient engagement activities
  • Finding patients as spokespersons
  • Train selected patients to effectively participate in agency activities
  • Report impact of patient involvement


 FDA & NAtional Cyber Security Awareness Month

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National Cybersecurity Awareness Month. Proclaimed by President Obama each year


  • Medical device cyber safety is large and shared responsibility
  • Requires diligence from all stakeholders: manufacturers, government agencies, health care organizations, health care professionals, cybersecurity researchers, and medical device user


  • Public and private sector organizations
  • Department of Health and Human Services, National Health Information Sharing and Analysis Center (NH-ISAC), Medical Device Innovation, Safety, and Security Consortium (MDISS), Department of Homeland Security’s Industrial Control Systems Cyber Emergency Response Team (ICS-CERT)

FDA RECOMMENDATIONS: Employing cybersecurity best practices and good cyber hygiene

RESOURCES: Stop.Think.Connect.™ campaignCDRH website.

Are you safe online? Visit the online resource guide to find out. Brought to you by DHS Stop.Think.Connect.




FDA BRIEF: Week of October 17, 2016

FDA approved

 LARTRUVO (Olaratumab) injection

Eli Lilly and Company, Indianapolis, Indiana, USA

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INDICATION:  In combination with doxorubicin, for the treatment of adult patients with soft tissue sarcoma (STS) with a histologic subtype for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery.


  • 12,310 new cases of STS and nearly 5,000 deaths  in 2016
  • New treatment option, added to doxorubicin,  for the initial treatment of soft tissue sarcoma since doxorubicin’s approval more than 40 years ago

REG PATHWAY:  Fast Track, Breakthrough Therapy Designation, Priority Review, Accelerated Approval, Orphan Drug Designation

  • Post Approval Commitment: Randomized, controlled trial to verify and further describe the clinical benefit is STS

MECHANISM OF ACTION: Human IgG1 antibody that binds platelet-derived growth factor receptor alpha (PDGFR-α), expressed on cells of mesenchymal origin and detected on tumor cells.


  • Open-label, randomized, active-controlled study (n=133), patients with  soft tissue sarcoma not amenable to curative treatment with surgery or radiotherapy,  LARTRUVO in combination with doxorubicin vs.  doxorubicin
  • Endpoints: Overall Survival (OS), Progression-Free Survival (PFS), Objective Response Rate (ORR) assessed by investigator and independent review using RECIST v1.1.
  • Statistically significant improvement in OS: 26.5 mo. vs. 14.7 mo. (p<0.05)
  • PFS: 8.2 mo. vs. 4.4 mo.
  • ORR: 18.2 % vs.  7.5 %



  • Serious risks: Infusion-related reactions and embryo-fetal harm
  • Most common side effects: Nausea, fatigue, low levels of white blood cells (neutropenia), musculoskeletal pain, inflammation of the mucous membranes (mucositis), hair loss (alopecia), vomiting, diarrhea, decreased appetite, abdominal pain, nerve damage (neuropathy) and headache.


BARRICOR Lithium Heparin Plasma Blood Collection Tubes  (BD Barricor™ Tubes) 

Becton, Dickinson and Company, USA

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INDICATION FOR USE: Collect, separate, process, transport and store venous blood samples for use in chemistry determinations, therapeutic drug monitoring (TDM), and zinc testing in plasma for in vitro diagnostic use. It is used in settings where a venous blood sample is collected by a trained healthcare worker.


  • Reduce the amount of blood drawn from each patient for testing
  • Reduce processing time in clinical labs.

REG. PATHWAY: 510(k) – Substantial Equivalence, CFR 862.1675 (Blood specimen collection devices), Class II, Product code: JKA

  • Predicate device: 3 BD Vacutainer® Brand PST™ Plasma Separation Tube


  • Sterile (interior), single-use, evacuated blood collection tubes with  mechanical separator (in place of gel), a low-zinc stopper and a plastic BD Hemogard™ color-coded lime green safety-engineered shield
  • Interior spray coated with lithium heparin anticoagulan
  • Novel separation technology which remains stable in its initial position, to enable the blood to be filled via current methods and subsequently creates a stable, robust barrier during processing



  •  Precision/Reproducibility, Traceability, Stability, Expected values

Comparison to Comparator:

  • 7 studies in healthy subjects and hospitalized patients
  • Blood samples collected into the BD BarricorTM tubes (candidate device) and the comparator tubes (BD PSTTM (k945952) for chemistry and BD Serum tube (k960250) for TDM)
  • Evaluations  on  selective common general chemistry analytes, immunology analytes, special chemistry analytes, cardiac markers, and therapeutic drug monitoring analytes on multiple instrument platforms
  •  62 analytes (55 chemistry and 7 TDM) evaluated
  • Comparable results between  candidate tube and the comparator tubes.

FDA Decision Summary

 TECENTRIQ (Atezolizumab) injection

Genentech, San Francisco, CA, USA.Image result for tecentriq

INDICATION:  Treatment of patients with metastatic non-small cell lung cancer (NSCLC) who have disease progression during or following platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving TECENTRIQ

REG PATHWAY: BLA, Added new indication

MECHANISM OF ACTION: Programmed death-ligand 1 (PD-L1) blocking antibody that previously received FDA accelerated approval for the treatment of locally advanced or metastatic urothelial carcinoma that has progressed after platinum-containing chemotherapy.


  • 2  randomized, open-label clinical trials (n=1137) , patients with NSCLC, Compared  atezolizumab vs docetaxel
  • Primary endpoint: Overall Survival (OS)
  • Median OS: 13.8 mo. vs.  9.6 mo, ( p=0.0004, Study 1),  12.6 mo. vs.  9.7 mo (Study 2)


  • Most common adverse reactions: Fatigue, decreased appetite, dyspnea, cough, nausea, musculoskeletal pain, and constipation
  • Clinically significant immune-related adverse events: Pneumonitis, hepatitis, colitis, and thyroid disease.
Sensor Monitored Alimentary Restriction Therapy (SMART) Device
Scientific Intake, Atlanta, GA, USA
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INDICATION FOR USE: Intended to aid in weight management in overweight to obese individuals. The device is indicated for individuals with a body mass index (BMI) in the range of 27-35 kg/m2 in conjunction with behavioral modification instruction.
REG. PATHWAY: De Novo Request, 21 CFR§ 876.5981
  • Regulation Name: Oral removable palatal space occupying device for weight management and/or weight loss Regulatory
  • Classification: II
  • Product Code: ONY


  • Oral removable palatal space occupying device
  • Worn during meals to limit bite size, thereby reducing the amount of food that is consumed
  • Recording sensors for monitoring patient use





  1. A way to send information to FDA
  2. A way to get safety information from FDA
  • Who, How, When What to report
  • High Quality Report Authoring : Tutorials, practices
  • Facilitated reporting: Online, Mail/Fax, Phone, Mobile

FDA Review and Actions from Reports : Signal Detection based on reports, Safety communications, Labeling changes

Resources :






Guidances: Blood Glucose Monitoring Systems, Software as Medical Device, Custom Device Exemptions

FDA BRIEF: Week of Oct 10, 2016



SCOPE: Studies and Criteria for 510(k)s) for blood glucose monitoring systems (BGMSs)

  • Product Codes: CGA (glucose oxidase method) CFR (hexokinase method)  LFR (glucose dehydrogenase method)
  • Studies to demonstrate performance in diverse professional healthcare settings
  • Intended use patients in all professional healthcare settings, patients in specific healthcare settings (e.g., in emergency response vehicles), patients in long-term care facilities, or patients at a physician’s office
  • Account for factors such as disease state, patient condition, physiological state, medications that might affect device performance in intended use population


  • REDUCING THE RISK OF BLOODBORNE PATHOGEN TRANSMISSION: Validated cleaning and disinfection, Robustness of cleaning
  • PERFORMANCE EVALUATION FOR PRESCRIPTION-USE: Precision, Linearity, Method Comparison/User Evaluation,  Interference Evaluation, Flex Studies, Meter Calibration and Quality Control



SCOPE: Studies and Criteria  for 510(k)s) for self-monitoring blood glucose test systems (SMBGs) which are for over-the-counter (OTC) home use by lay-users

  • Regulated under 21 CFR 862.1345, Glucose Test System, product code NBW
  • Use capillary whole blood from fingertip
  • NOT intended for use in healthcare or assisted-use settings


  • REDUCING THE RISK OF BLOODBORNE PATHOGEN TRANSMISSION: Validated cleaning and disinfection, Robustness of cleaning
  •  PERFORMANCE EVALUATION AND CRITERIA: recision, Linearity, Method Comparison/User Evaluation,  Interference Evaluation, Flex Studies, Meter Calibration and Quality Control



SCOPE:  To establish the scientific validity, clinical performance, and analytical validity for a Software as a Medical Device (SaMD)

  • Prepared by IMDRF- voluntary group of global medical device regulators including FDA FDA’s IMDRF webpage.
  • Globally harmonized, risk-based principles of  clinical evaluation
  • Clinical evaluation is assessment and analysis of clinical data pertaining to a medical device in order to verify the safety, effectiveness and performance. Ongoing process conducted during the lifecycle of device.


  • DEFINITIONS:: Clinical Validity,  Scientific Validity,  Clinical Performance,  Analytical Validity of a SaMD
  • GENERAL PRINCIPLES: Clinical Evaluation,
  • CLINICAL EVALUATION METHODS, EVIDENCE, APPRAISAL: Evidence Goals,  Required Level of Clinical Evaluation,  Scientific Validity Evidence,  Generating Analytical Validity Evidence,Generating Clinical Performance Evidence,  Appraisal of Clinical Evaluation Evidence
  • LEVEL OF EVIDENCE ACCORDING TO SaMD CATEGORY: Categories, Importance of Clinical Evidence and Expectations by Category, Importance of Independent Review of Evidence by Category, Pathway for Continuous Learning Leveraging Real World Clinical Evidence


Custom Device Exceptions

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FDA guidance on CUSTOM DEVICE EXCEPTIONS, explains the new statutory provisions and defining certain terms

  • AMENDMENT to introduce new concepts and procedures

Some of the clauses  for“custom device” qualification:

  • created or modified in order to comply with order of individual physician
  • not generally available in the US
  • designed to treat a unique pathology or physiological condition that
    no other device is domestically available to treat
  • assembled from components or manufactured and finished on a case-by-
    case basis to accommodate unique needs
  • treating a “sufficiently rare condition, such that conducting clinical
    investigations on such device would be impractical;’
  • production `limited to no more than five units per year of a particular device type
  • annual report to FDA


FDA News: Xarelto AF Trial, FDASIA 907 Progress, IT Security Program

FDA BRIEF: Week of Oct 10, 2016


Cardiobeat Masthead

FDA analyses conclude that XARELTO clinical trial results were not affected by faulty monitoring device

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Direct Factor Xa inhibitor, Xarelto (rivaroxaban), approved to reduce the rates of stroke and blood clots in patients with non-valvular atrial fibrillation (NVAF)

  • Alere INRatio device, used to monitor warfarin therapy in control group of pivotal ROCKET-AF clinical trial,  recalled due to potential to generate inaccurate results

FDA analyses to assess impact of faulty monitoring device on ROCKET-AF study results

  • Quite likely patients in warfarin arm  received higher doses of warfarin
  • Increased intensity of anticoagulation had modest effect on clinical outcomes
  • Accordingly, the FDA benefit-risk  conclusion for 2011 approval  not changed

Recommendations: No changes in rivaroxaban labeling


Where We Are/What We Have Done – Two Years After Releasing Our FDASIA 907 Action Plan

By: Janice Soreth, M.D.,  Chair FDASIA 907 Steering Committee, Acting Associate Commissioner for Special Medical Programs

Janice Soreth

SECTION 907 of FDASIA- Advance inclusion of diverse populations in clinical trials


  • Action Plan identified 27 discrete actions  within the three priority areas
    • improving data quality
    • encouraging greater clinical trial participation
    • ensuring more data transparency.
  • Draft guidance (2016) “Evaluation and Reporting of Race and Ethnicity Data in Medical Device Clinical Studies.”
  • Update guidanse (2005): “Guidance for Industry Collection of Race and Ethnicity Data in Clinical Trials.”
  • Drug Trials Snapshots, provide information about clinical trials participants and  whether differences in benefits and side effects among sex, race, and age groups
  • Initiative on “Diverse Women in Clinical Trials”  to increase awareness
  • Public service announcements on FDA’s YouTube channel



FDA’s IT Security Program

This chart shows 12(80%) of the 15 Program Recommendations have been remediated.3 of the Program Recommendations are in progress.

Government Accountability Office (GAO), August 2016 report, outlined recommendations for Information security and the protection of industry and public health information

FDA fully implemented

  • 80% GAO’s program recommendations
  • 61% GAO’s technical recommendations
  • Anticipate completing remaining recommendations within next tear

Also implemented processes, procedures and tools to ensure the deterrence, prevention, detection and correction of incidents

Working with the Energy and Commerce Committee and the GAO to ensure the timely closure of their findings.




Dealing with ADHD

Dealing with ADHD: What You Need to Know

boy struggling in class (600x400)


  • Children being diagnosed with ADHD continues to increase
  • 11% (6.4 million, 4 – 17 yr ) in 2011, up 7.8% in 2003
  • Boys (13.2%) were more likely than girls (5.6%)


FDA Approved Treatments 

Reduce symptoms and improve functioning in children as young as age 6.

  • Stimulants :Calming effect, increase brain levels of dopamine
  • Non-stimulants: Useful alternative for children who do not tolerate stimulants well
  • In addition to medication,  behavioral therapy and  community support groups

Testing in Younger Kids

  • FDA-approved medications tested for safety and effectiveness in clinical trials of children 6 and older
  • Post-Approval: . FDA asking for clinical trials with children 4- 5


Adults and ADHD

  • 4% of adults may have ADHD
  • Poor time management skills, trouble with multitasking, restless, lack of concentration


FDA Webinar on Patient Preference Information





  • Provide context and overview of Patient Preference Information Final Guidance
  • Describe key updates to the Benefit-Risk worksheet in the “Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications” Guidance
  • Differences between Patient Preference vs. patient Reported Outcome


FDA Approval, Classification: ILARIS, EPIC CLEARVIEW

FDA BRIEF: Week of October 3, 2016

FDA approved

ILARIS (canakinumab) injection

Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA

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  • Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS) ILARIS is indicated for the treatment of Tumor Necrosis Factor (TNF) receptor Associated Periodic Syndrome (TRAPS) in adult and pediatric patients.
  • Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) ILARIS is indicated for the treatment of Hyperimmunoglobulin D (Hyper-IgD) Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) in adult and pediatric patients
  • Familial Mediterranean Fever (FMF) ILARIS is indicated for the treatment of Familial Mediterranean Fever (FMF) in adult and pediatric patients.


  • New indications are for rare and serious auto-inflammatory diseases in adult and pediatric patients:
  • Hereditary diseases characterized by fever and inflammation, severe muscle pain
  • No previously approved therapies for TRAPS or HIDS/MKD.


Supplemental BLA. Previously approved for another periodic fever syndrome Cryopyrin-Associated Periodic Syndromes (CAPS) and for active systemic juvenile idiopathic arthritis.


  • 4-Part study (TRAPS, HIDS/MKD, and FMF Study 1) consisting of three separate, disease cohorts (TRAPS, HIDS/MKD and FMF)
  • n=185 patients, age: > 28 days, Ilaris vs Placebo
  • Primary Endpoint:  Complete Response (Resolution of Index Flare by Day 15 and Maintained Through Week 16)
  • TRAPS: 45.5% vs. 8.3%), p= 0.005
  • HIDS/MKD: 35.1% vs. 5.7%, p=0.002
  • FMF:61.3% vs 6.3%, p <0.0001


  • Most common adverse reactions: Injection site reactions, cold susceptibility
  • Serious side effects: Risk of serious infections




EPIC ClearViewTM

EPIC Research & Diagnostics, Scottsdale, AZ , USA

Image result for EPIC ClearView


CODE: 21 CFR 882.1561.

GENERIC NAME: Evoked photon image capture device – e.g. EPIC ClearView

INTENDED USE: Non-invasive measurement tool that applies electricity to detect electrophysiological signals emanating from the skin, which are reported numerically and as images without clinical interpretation. The device is not intended for diagnostic purposes.


  • Adverse tissue reaction, electromagnetic incompatibility, and electromagnetic malfunction (e.g., shock)
  • Not safe for use except under the supervision of a practitioner licensed by law
  • Prescription device and must satisfy prescription labeling requirements ( 21 CFR 801.109 Prescription devices)



Guidances: Tropical Diseases Voucher

FDA BRIEF: Week of Oct 3, 2016

fda guidances


FDAAA (Food and Drug Administration Amendments Act of 2007), SECTION 524

  • Priority review vouchers to sponsors of certain tropical disease product applications
  • Further explanation of Section 524 c


  • Definitions: Tropical Disease Product Application and Tropical Disease
  • Priority Review Vouchers: General Information
  • Priority Review Vouchers: Eligibility
  • Priority Review Vouchers: Transferability
  • Priority Review Voucher Fees and Use
  • Relationship Among the Priority Review Voucher Program and Other Programs


Medical Device Misconnections

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Reducing Risks Associated with Medical Device Misconnections

FDA News: precisionFDA, Patient Voice, Drug Safety Priorities, Priority Review Vouchers, Open Positions-Consumer & Patient Representatives

FDA BRIEF: Week of October 3, 2016


precisionFDA’s Next Challenge? Conduct an App-a-Thon!


Zevana Tezak

Zivana Tezak, Ph.D.,  Associate Director, Science and Technology, CDRH

Elaine Johanson

Elaine Johanson,  precisionFDA Project Manager, Deputy Director Office of Health informatics


  • Online research portal
  • FDA scientists + Silicon Valley minds
  • Part of President Obama’s Precision Medicine Initiative (PMI)


  • Advance the use of next generation sequencing (NGS)
  • Map entire human genome; data, analytics, and sequencing tools
  • Open source cloud-based space for data, ideas, and methodologies sharing
  • > 1,600 participants


 “App-a-Thon”: Closes Oct 28, 2016

  • Add NGS software apps for simulations, benchmarking, data integration, mapping portions of the genome, or identifying genetic variants
  • Add to the precisionFDA app library
  • FDA provides framework and materials, storage, and compute capacity
  • Results to be highlighted by FDA Commissioner at World Precision Medicine Congress on Nov. 14, 2016 in Washington D.C

App-a-Thon in a Box toolkit.


Our 20th Patient-Focused Drug Development meeting: Enhancing the patient’s voice in FDA’s approach to drug review and development


Theresa Mullin

Theresa M. Mullin, Ph.D., Director, Office of Strategic Programs, CDER

Patient Focused Drug Development (PFDD)  program launched as part of PDUFA V

  • PFDD meetings helps FDA strengthen  understanding of targeted disease areas and hear directly from patients, families, and caregivers about symptoms that matter most to them
  • 20th PFDD public meeting  with organ transplant patients occurred Sept 27th
  • All meetings chronicled in Voice of the Patient reports
  • Plan to hold 4 more PFDD meetings in 2017

Externally-led Patient-Focused Drug Development Meetings

  • To expand PFDD benefits, FDA welcomes patient-focused meetings organized by the patient groups themselves
  • Interested patient groups can submit a letter of intent

Externally-led Patient-Focused Drug Development Meetings


2016-2017 CDER Drug Safety Priorities: Initiatives and Innovation


CDER’s programs to ensure safety of drug products and evolving drug safety enterprise

Miletones and broad picture of FDA’s multidisciplinary collaborations and partnership

Drug Safety Oversight Across the Product Lifecycle

Advancing Drug Safety Science

  • JumpStart – Enhancing Pre-Market Drug Review with Digital Tools
  • Postmarketing Safety Surveillance and Oversight
  • Improving Drug Safety through Research
  • Advancing Drug Safety Science through Public-Private Partnerships

Improving Operations and Management in Support of Drug Safety

  • The 2015 GAO Report: A Call To Improve Data on Safety Issues
  • Opioid Addiction and Abuse: Addressing a National Crisis
  • Safe Use Initiative
  • Working to Ensure Drug Product Quality
  • Compounded Drugs and Drug Supply Chain Security

 Communicating Drug Safety: A Global Public Interface

  • Expert Responses to Public Queries
  • Social Media and Online Tools
  • Drug Safety Communications
  • Safety Labeling Changes
  •  Risk Communications Research


FY 2017 User Fees for Priority Disease Review Vouchers

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Rare Pediatric Disease Priority Review Voucher 

Tropical Disease Priority Review Voucher 

 OPEN- Consumer Representative Positions on FDA Advisory Committee

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  • Represent consumer perspective on issues and actions before Advisory Committee
  • Serve as liaison between Committee and interested consumers, associations, coalitions, and consumer organizations
  • Facilitate dialogue with Committees on scientific issues that affect consumers.. FDA is currently recruiting to fill

Consumer Representative positions.

OPEN- Nominations for Voting Members for CDRH Patient Engagement Advisory Committee


  • Improve communication of benefits, risks, clinical outcomes
  • Increase integration of patient perspective in regulatory process
  • Identify new approaches, unforeseen risks, unintended consequences resulting from FDA policy

CDRH Patient Engagement member




FDA BRIEF: Week of September 26, 2016

FDA approved


Vela Diagnostics USA, Inc. Fairfield, NJ, USA

Image result for SENTOSA ZIKA test image

INTENDED USE: The Sentosa® SA ZIKV RT-PCR Test is a real-time RT-PCR test intended for the qualitative detection of RNA from the Zika virus in serum, EDTA plasma or urine (collected alongside a patient-matched serum or plasma specimen) from individuals meeting Centers for Disease Control and Prevention (CDC) Zika virus clinical criteria (e.g., clinical signs and symptoms associated with Zika virus infection) and/or CDC Zika virus epidemiological criteria (e.g., history of residence in or travel to a geographic region with active Zika transmission at the time of travel, or other epidemiological criteria for which Zika virus testing may be indicated). Testing is limited to U.S. laboratories that are certified under the Clinical Laboratory Improvement Amendments of 1988 (CLIA), 42 U.S.C. §263a, to perform high complexity tests, and similarly qualified non-U.S. laboratories

REG PATHWAY: Emergency Use Authorization (EUA)

DESCRIPTION: Uses the following materials

  • Enzyme Mix
  • Zika Virus Primer/Probe Mix
  • Extraction Control Primer/Probe Mix
  • Extraction Control
  • Positive Control
  • Negative Control (nuclease free water)


Medtronic MiniMed, Inc., Northridge, CA, USA

Picture of the device.

INDICATION FOR USE:  For continuous delivery of basal insulin (at user selectable rates) and administration of insulin boluses (in user selectable amounts) for the management of Type 1 diabetes mellitus in persons, fourteen years of age and older, requiring insulin as well as for the continuous monitoring and trending of glucose levels in the fluid under the skin. The MiniMed 670G System includes SmartGuard technology, which can be programmed to automatically adjust delivery of basal insulin based on Continuous Glucose Monitor sensor glucose values, and can suspend delivery of insulin when the sensor glucose value falls below or is predicted to fall below predefined threshold values.


  • Need to advance the development of an artificial pancreas device system
  • Automatically monitors blood glucose and provides appropriate insulin doses in people with diabetes who use insulin.

REG PATHWAY: PMA, Device Procode: OZP,

  • Artificial pancreas device system, single hormonal control
  • Priority Review because device is novel technology and availability is in patients’ best interest.
  • Insulin reservoirs and infusion sets used with the 670G System are the same as those currently used with the MiniMed 530G System (P120010)

DEVICE DESCRIPTION: System consists of:

  • MiniMed 670G insulin pump
  • Guardian Link Transmitter
  • Guardian Sensor
  • One-Press Serter
  • Contour NEXT Link 2.4 Glucose Meter


  • Pivotal study: Safety Evaluation of the Hybrid Closed Loop (HCL) System in Type 1 Diabetes
  • Performance Evaluation of the Enlite® 3 Sensor to Support a Full 168 hours (7 Days) of Use
  • Established sensor performance across the claimed measuring range (40 to 400 mg/dL glucose), the precision, and the calibration frequency (calibrate minimally every 12 hours or 3-4 times a day) of the 7 day wear period for the Guardian sensor
  • Established the performance of the alarms and alerts


  • Risks of the Auto Mode feature
  • Risks of the predictive suspend feature
  • Risks of the pump hardware problems
  • Potential device-related non-serious events : Skin irritation, Infection, Pain, Discomfort, Bruising, Edema, Rash, Induration of skin, Allergic reaction to adhesives, Hematoma, Unnecessary fingersticks, Hyperglycemia, Ketosis, Sensor may break


The Artificial Pancreas Device Systems Website


WHAT: Website  on FDA’s Efforts to Advance Artificial Pancreas Device Systems. Sometimes an artificial pancreas device system is referred to as a “closed-loop” system, an “automated insulin delivery” system, or an “autonomous system for glycemic control.” NOTE: The Artificial Pancreas Device Systems described on this site do not involve biomaterial, synthetic or artificial tissue or organs.

WHY: To  advance the development of an artificial pancreas device system, an innovative device that automatically monitors blood glucose and provides appropriate insulin doses in people with diabetes who use insulin.

WHO:  FDA along with diabetes patient groups, diabetes care providers, medical device manufactures, and researchers 

WHEN:  On September 28, 2016, the FDA approved the first hybrid closed loop system, the Medtronic’s MiniMed 670G System, intended to automatically monitor blood sugar and adjust basal insulin doses in people with type 1 diabetes. 



FDA News: Payer Communication Task Force, Clinical InvestigatorTraining, BEST, Symbols in Labeling

FDA BRIEF: Week of September 26,2016


Payer Communication Task Force (PCTF)

CDRH initiative to  shorten the time between FDA approval or clearance and device coverage

  • PCTF for public/private payer organizations  to  provide input on clinical evidence to support coverage decisions.
  • Centers for Medicare and Medicaid Services (CMS) : Parallel Review Pilot Program initiated
  • Private payers (Tabulated below): Expressed interest in attending Pre-Submission meetings to provide clinical evidence input

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FDA’s Clinical Investigator Training Helps Support the Drug Development Process


Leonard Sacks, M.D., Associate Director for Clinical Methodologies, Office of Medical Policy, CDER

Mili Duggal, Ph.D., M.P.H., ORISE Fellow, Office of Medical Policy, CDER 


Leonard Sacks

Mili Duggal

7th Annual Clinical  Investigator Training Course

  • For physicians, nurses, pharmacists, and other healthcare professionals
  • Design, conduct, and evaluation of clinical trials
  • Training by senior FDA experts and guest speakers
  • Investigators could learn directly from FDA staff and interact with them


Biomarkers, EndpointS, and other Tools (BEST) Resource

FDA BEST Resource

  • FDA-NIH Joint Leadership Council harmonized of terms used in translational science and medical product development
  • BEST developed to  promote consistent use of biomarker terms and concepts
  • Clarifies terminology and uses of biomarkers and endpoints as they pertain to the progression from basic biomedical research to medical product development to clinical care


Using Symbols to Convey Information in Medical Device Labeling


Antoinette (Tosia) Hazlett, MSN, RN, Senior Policy Analyst, CDRH

Scott Colburn CAPT, USPHS, Director, CDRH Health Standards Program

Tosia Hazlett

Scott Colburn

Symbols on medical devices need to be understood by users

Use of Symbols in Labeling final rule in 06/2016

  • not to use symbols
  • use symbols with adjacent explanatory text
  • use stand-alone symbols that have been established

 Stand-alone symbols

  • reduce design costs
  • consistent with EU and other foreign market labeling
  • replace small and difficult-to-read text
  • more user-friendly and understandable

Symbols Glossary : help user familiarity

Symbol Statement “Rx Only” or “℞ only” : For prescription devices



FDA Webinar Debrief : Neurological Devices




Key Takehomes
– New Division
– Design studies based on Indication for Use and Label claims
– Time studies based on your product roadmap
–  Keep documentation and assess impact of product design changes
– IRBs can be a helpful resource for IDE waivers
– Early Feasibility Studies (EFS) encouraged for streamlined development
– Several options of getting in touch directly with Division