Women in Clinical Trials

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Women in Clinical Trials

  • Gender related differences in medical product performance
  • Women from diverse backgrounds still need to participate in clinical trials
  • Resource to assess whether a clinical trial is right for individual

LEARN


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FDA Voices: Perspectives From FDA Experts

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FDA Voices

Collection of perspectives into FDA work brought by FDA experts

  • thought-provoking insight on a variety of topics, issues and policies
  • areas of medical products, food, tobacco, policy, and consumer safety and enforcement

LEARN


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Bisimilars Learning Toolkit

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Biosimilar Development Process

Biological products that are demonstrated to be biosimilar to or interchangeable with an FDA-approved biological product

  • Abbreviated pathway to provide more treatment options, increase access, lower costs
  • Rigorous approval standards for  safety and effectiveness
  • Ten biosmilars approved to date

Learning toolkit to help promote understanding of biosimilars and interchangeable products

LEARN


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Digital Health Software Precertification (Pre-Cert) Program

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Digital Health Software Precertification (Pre-Cert) Program

GOALS

  • Develop tailored and pragmatic regulatory oversight
  • Trust organizations with demonstrated culture of quality and organizational excellence
  • Leverage transparency of organization’s excellence and product performance across entire lifecycle
  • Streamline premarket review to verify the continued safety, effectiveness, and performance

INTENTIONS 

  • Leverage information from all available sources to be more efficient and streamlined without compromising safety and effectiveness
  • Enable modern and tailored approach to allow timely software iterations and changes
  • Ensure high-quality by enabling companies to demonstrate their embedded culture of quality and organizational excellence
  • Learn, adapt, adjust key elements based on program effectiveness

WORKING MODEL

LEARN


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Spectrum of Disease Conditions

Spectrum of Disease Conditions

LEARN

Help FDA improve MedWatch System

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MedWatch is FDA gateway for clinically important safety information and reporting serious problems with human medical products

  • Physicians and Consumers can report unexpected side effects, adverse events, or other problems through MedWatch program
  • Report adverse events, product problems, errors with use,  evidence of therapeutic failure is suspected or identified

FDA is seeking comments to help improve adverse event information collection

  • Forms 3500, 3500A and 3500B

regulations.gov


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CDRH Device Evaluation Intern Program

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Device Evaluation Intern Program

Challenging and rewarding experience for individuals interested in pursuing careers in the fields of science, engineering, and/or medicine

  • Test educational interests in practical work environment
  • Gain professional “real work” experience
  • Work alongside Agency’s top healthcare authorities, establish professional contacts

LEARN


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Benefit-Risk Assessment in Drug Regulatory Decision-Making : 2018-2022

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Benefit-Risk Assessment in Drug Regulatory Decision-Making

Under FDARA 2017, FDA committed to furthering implementation of structured benefit-risk assessment into drug review

Commitments on Enhancing Benefit-Risk Assessment

  • Continue implementation of the Benefit-Risk Framework
  • Participate in stakeholder meetings
  • Draft guidance on benefit-risk assessment
  • Revise relevant MAPPs and SOPPs
  • Conduct second evaluation of Benefit-Risk Framework

Additional Opportunities to Enhance FDA’s Benefit-Risk Assessment

  • Improving accessibility for approved products
  • Use to support Advisory Committee meetings
  • Exploring additional tools to support assessment

LEARN


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Novel Materials and Manufacturing Research Program

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Medical device materials undergo physical and chemical changes during total product life cycle (TPLC). Some examples:

  • chemical reactions, separation/purification, temperature excursions
  • microstructure changes, molding/extrusion, weaving
  • 3D printing

Novel Materials and Manufacturing Research Program elucidates 

  • TPLC findings
  • Relationship to Safety and Effectiveness (S&E) of device
  • How seemingly minor manufacturing changes can lead to S&E changes

LEARN


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FDA Guidances: Product Recalls, Device Clinical Data from ex-US studies, 510(k) Refusal to File Policies

CapturePublic Warning and Notification of Recalls

Recall: Removal of marketed product that considered to be in violation of FDA laws; Agency would initiate legal action. NOT the same as market withdrawal

Recall Determination:  FDA assessment FDA that ongoing or completed removal or correction of a marketed violative product constitutes a recall

Recall Classification: I, II, or III, assigned by FDA on basis of the health hazard

  • Class I: Could cause serious health problems or death
  • Class II: Might cause a temporary health problem
  • Class III: Unlikely to cause any adverse health reaction, violate FDA labeling or manufacturing laws

Public Warning:  Alert public that product being recalled presents a serious health hazard; for Class I recalls

Public Notification of Recalls: Weekly FDA Enforcement Report

FDA 101: Product Recalls

READ


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Acceptance of Clinical Data to Support Medical Device Applications and Submissions

FDA regulations on data acceptance from clinical investigations conducted outside US

  • Conform with good clinical practices (GCP)
  • Applies to clinical data submitted to support IDE, 510(k), De Novo, PMA, PDP, HDE

Conformity with GCP

  • Statements regarding conduct of clinical investigations
  • Special considerations for in vitro diagnostic (IVD) device investigations using leftover, de-identified biospecimens

Supporting Information, Waivers, Records

READ


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Refuse to Accept Policy for 510(k)

Minimum threshold of acceptability and acceptance for substantive review of 510(k)  

  • Submission is administratively complete, includes information necessary reach determination regarding substantial equivalence
  • Same intended use as predicate device
  • Same technological characteristics as predicate device – or- different technological characteristics
  • Appropriate clinical or scientific data to demonstrate safety and effectiveness
  • Does not raise different questions of safety and effectiveness than the predicate
  • Pre-Submission Interaction encouraged

Refuse to Accept Principles

  • Not based on substantive review of information
  • Justification for any alternative approach
  • Consideration of device-specific and cross-cutting guidances

Checklist provided

READ


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FDA Soundcast: KYMRIAH, first CAR-T cell immunotherapy

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KYMRIAH, CAR-T cell immunotherapy- an entirely new treatment approach for patients with cancer FDA Approvals: KYMRIAH, MYLOTARG, VABOMERE, Benznidazole

  • Produced by taking patient’s blood sample, isolating T lymphocytes, and genetically engineering those T-cells to express a new chimeric receptor that recognizes CD19 on their leukemia and normal B-cells
  • Engineered T-cell should recognize and kill B-cells, including the malignant cells

LISTEN


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FDA Drug Databases

Drug Approvals and Databases

LEARN

 

Transparency at FDA

New platforms for analyzing and visualizing large, complex datasets

  • Harness data to advance the safe and efficient development of new therapies
  • Tailor clinical care to individual patient characteristics and preferences
  • Communicate information supporting regulatory decision-making to engender greater public confidence

Advance transparency without reducing incentives to innovate

  • FDA’s Data Dashboard: publicly available datasets, data visualization tools 
  • openFDA
    • APIs and full sets of downloadable FDA data files for adverse events, drug product labeling, recall enforcement reports
    • supports open source user community, sharing of source code and techniques for use through GitHub, Twitter, and StackExchange

New Steps to filter information and harness it into more usable knowledge

  • Greater transparency and efficiency in NDA and BLA reviews
  • Track product development in clinical trials posted on the ClinicalTrials.gov
  • Pilot (9 applications) to disclose key portions of Clinical Study Reports to decipher  key clinical evidence for approval decision
  • Add special identifier number to clinical trials registered through ClinicalTrials.gov  and link to FDA communications (e.g. labeling, advisory committee materials)
  • Explore possibility – under existing statutory authority- to release Complete Response Letters (CRLs) information directly relevant to patients

Technology, no matter how powerful, is always just a tool

  • Commitment to transparent, responsible, and science-based use to save and improve lives

FDA Commissioner Statement

Blueprint for FDA transparency


 

FDA Marketing Authorizations: FLUARIX, LYNPARZA, GILOTRIF, REMOVE System

Image result for fluarix logoFLUARIX QUADRIVALENT (Influenza Vaccine) Suspension for Intramuscular Injection

GlaxoSmithKline

INDICATION: Active immunization for the prevention of disease caused by influenza A subtype viruses and type B viruses contained in the vaccine. Approved for use in persons aged 3 years and older

IMMUNOLOGICAL EVALUATION IN CHILDREN:

  • Randomized, double-blind, active-controlled, safety, immunogenicity, and noninferiority trial, FLUARIX QUADRIVALENT (n = 791) vs. comparator trivalent influenza vaccine (FLUARIX, TIV-1, n = 819 or TIV-2, 426 n = 801), age 3-17 years
  • Endpoint: % achieving seroconversion.  Non-Inferiority achieved

SAFETY:

  • Injection site adverse reactions:  pain, redness, swelling
  • Systemic adverse events: drowsiness, irritability, loss of appetite, fatigue, muscle aches, headache, arthralgia, gastrointestinal symptoms

REGULATORY PATHWAY: BLA

  • Initial approval in 2012
  • Extended to age range to include children 6 to 35 months of age

REIMBURSEMENT:  

  • Vaccine product codes as well as some common administration codes associated with immunization

LABEL


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LYNPARZA (olaparib) tablet

AstraZeneca Pharmaceuticals

SUPPLEMENTAL INDICATION: Indicated in patients with deleterious or suspected deleterious gBRCAm, HER2-negative metastatic breast cancer, who have been treated with chemotherapy in the neoadjuvant, adjuvant, or metastatic setting. Patients with hormone receptor (HR)-positive breast cancer should have been treated with a prior endocrine therapy or be considered inappropriate for endocrine therapy. Select patients for therapy based on an FDA-approved companion diagnostic

ADDRESSING UNMET NEED:

  • Approximately 20-25% patients with hereditary breast cancers and 5-10%  have a BRCA mutation
  • First treatment for breast cancer with a certain inherited gBRCAm HER2-negative metastatic breast cancer
  • Patients selected for treatment based on an FDA-approved genetic test – BRACAnalysis CDx.

MECHANISM OF ACTION:  Poly (ADP-ribose) polymerase (PARP) inhibitor

EFFICACY:

  • Open-label study (n=302) patients with gBRCAm HER2-negative metastatic breast cancer, LYNPARZA vs. healthcare provider’s choice of chemotherapy. Tested with the BRACAnalysis CDX to confirm deleterious or suspected deleterious gBRCAm status
  • Major efficacy outcome measure: Progression Free Survival (PFS)  assessed by blinded independent central review using RECIST version 1.1
  • PFS Number of events: 163 (80%) vs. 71 (73%), p=0.0009

SAFETY:

  • Common side effects: Anemia), neutropenia, leukopenia, nausea, fatigue, vomiting, nasopharyngitis, respiratory tract infection, influenza, diarrhea, arthralgia/myalgia, dysgeusia, headache, dyspepsia, decreased appetite, constipation and inflammation and sores in the mouth
  • Severe side effects:  Myelodysplastic syndrome/acute myeloid leukemia, pneumonitis
  • Cause harm to a developing fetus and newborn baby

REGULATORY PATHWAY: sNDA

  • Priority Review
  • First approved in 2014 to treat certain patients with ovarian cancer

REIMBURSEMENT:

  • No Medicare coverage for previously approved indication (specialty product); high copay

LABEL


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GILOTRIF (afatinib) tablets

Boehringer Ingelheim Pharmaceutical

SUPPLEMENTAL INDICATION:  First-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have non-resistant epidermal growth factor receptor (EGFR) mutations as detected by an FDA-approved test.
Limitation of Use: Safety and efficacy have not been established in patients whose tumors have resistant EGFR mutations

MECHANISM OF ACTION: Covalently binds to the kinase domains of EGFR (ErbB1), HER2 (ErbB2), and HER4 (ErbB4) and irreversibly inhibits tyrosine kinase autophosphorylation, resulting in downregulation of ErbB signaling

EFFICACY:

  • In NSCLC patients harboring non-resistant EGFR mutations (S768I, L861Q, and/or G719X) other than exon 19 deletions or exon 21 L858R substitutions enrolled in one of three clinical trials – pooled analysis
  • Non-resistant EGFR mutations identified using either Sanger sequencing or by  therascreen EGFR RGQ PCR Kit.
  • Confirmed overall response rate: 66% .
  • Among the 21 responders, 52% had response duration of ≥12 months and 33% had response duration  ≥18 months

SAFETY:

  • Most common adverse reactions: Diarrhea, rash/acneiform dermatitis, stomatitis, paronychia, dry skin, decreased appetite, nausea, vomiting, pruritis

REGULATORY PATHWAY: sNDA

  • Priority Review and Orphan Drug Designation
  • Initial approval in 2013 for treatment of patients with metastatic NSCLC whose tumors have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations
  • This approval broadens the patient population

REIMBURSEMENT: 

  • 100% of Medicare Part D and Medicare Advantage plan coverage; Tier 5 (non-preferred brand-name drugs)
  • Restrictions: Quantity limits, Prior Authorization

LABEL


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REMOVE System

Ovesco Endoscopy AG

INDICATION FOR USE: 

The remOVE System consists of the DC Impulse and the DC Cutter Set.
The remOVE DC Impulse is a medical electrical device for fragmentation of OTSC® (endoscopic device for effective treatment of hemorrhage and acute or chronic wall defects in the GI tract) and FTRD® (endoscopic device for full-thickness resection of colorectal wall lesions) clips made by Ovesco Endoscopy AG for the digestive tract.
The remOVE DC Cutter Set is a set of instruments for use in flexible endoscopy. It consists of a bipolar DC instrument for the fragmentation of OTSC (endoscopic device for effective treatment of hemorrhage and acute or chronic wall defects in the GI tract) and FTRD (endoscopic device for full thickness resection of colorectal wall lesion) clips from Ovesco Endoscopy AG, a pair of forceps and a cap for removal of these fragmented clips.

GENERIC DEVICE TYPE: Endoscopic electrosurgical clip cutting system

Prescription device that applies electrical energy to fragment metallic clips, which are devices placed in the digestive tract to close gastrointestinal perforations, hemorrhages, or perform resection.
The system includes instruments that are then used to remove the fragmented clips from the digestive tract.

RISKS AND MITIGATION MEASURES:

  • Unintended tissue damage (burns, perforations, bleeding):  Animal performance testing, Non-clinical performance testing, Electrical and thermal safety testing, Usability testing, Labeling
  • Electromagnetic interference / Electrical shock: Electromagnetic compatibility testing, Electrical safety testing, Labeling
  • Adverse tissue reaction: Biocompatibility evaluation
  • Infection: Sterilization validation, Shelf life testing, Labeling

REGULATORY PATHWAY: De Novo

  • Regulation Number: 21 CFR 876.4310
  • Regulation Name: Endoscopic electrosurgical clip cutting system
  • Regulatory Class: Class II
  • Product Code: QAG

REIMBURSEMENT: Pending

LABEL

CLASSIFICATION ORDER


Image credits: GSK, AstraZeneca, Boehringer Ingelheim, Ovesco

List of Off-Patent, Off-Exclusivity Drugs without an Approved Generic

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List of Off-Patent, Off-Exclusivity Drugs without an Approved Generic

  • To improve transparency and encourage the development and submission of  ANDAs in markets with limited competition
  • Part I of the list identifies those drug products for which FDA could immediately accept an ANDA without prior discussion
  • Part II identifies drug products for which ANDA development or approval may raise potential legal, regulatory, or scientific issues that should be addressed with the Agency prior to considering submission of an ANDA

LEARN


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Patent Certifications and Suitability Petitions

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Patent Certifications and Suitability Petitions

To continually improve its pre-ANDA interactions with applicants for generic drugs 

Paragraph IV Patent Certifications

  • Generic applicant provides “paragraph IV certification”  challenging brand product patent listed in FDA’s Orange Book
  • Must notify brand product sponsor/patent holder of ANDA submission and patent challenge
  • If approved, eligible for exclusive right to market generic drug for 180 days

Paragraph IV Certifications List

  • To assist generic drug applicants, FDA publishes list of drug products for which ANDA received containing Paragraph IV patent certification

Suitability Petitions

  • Generic application may petition FDA for ANDA for drug product different from Orange Book listing (e.g. route of administration, dosage form, strength, or active ingredient)

LEARN


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CDRH FY 2018 Proposed Guidance Development & Retrospective Review

fda guidancesCDRH Fiscal Year 2018 (FY 2018) Proposed Guidance Development and Focused Retrospective Review of Final Guidance

Three lists:

  1. A-List:  fully intends to publish
  2.  B-List: intends to publish as resources permit
  3. Focused retrospective review: guidances issued in 2008, 1998, 1988, and 1978

A-List

Final Guidance Topics

  • Medical Device Accessories: Describing Accessories and Classification Pathway for New Accessory Types (revision)
  • Unique Device Identification: Policy Regarding Compliance Dates of Class I and Unclassified Devices
  • Appropriate Use of Voluntary Consensus Standards in Premarket Submissions for Medical Devices
  • Considerations for Design, Development, and Analytical Validation of Next Generation Sequencing (NGS)-Based In Vitro Diagnostics (IVDs) Intended to Aid in the Diagnosis of Suspected Germline Diseases
  • Use of Public Human Genetic Variant Databases to Support Clinical Validity for Genetic and Genomic-Based In Vitro Diagnostics

Draft Guidance Topics

  • Export Certificates
  • Multifunctional Device Products: Policy and Considerations
  • The Least Burdensome Provisions: Concept and Principles
  • Humanitarian Devices Exemption (HDE) Program
  • 510(k) Third Party Review Program
  • Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission Program
  • Expansion of the Abbreviated 510(k) Program: Demonstrating Substantial Equivalence through Performance Criteria
  • The Application of Acceptable Uncertainty to Support Marketing Authorization Decisions for Medical Devices
  • Principles and Procedures for the Recognition and/or Withdrawal of Voluntary Consensus Standards
  • Validation of Automated Process Equipment Software

LEARN


 

Updated Orange Book, Patent/Exclusivity FAQs

Orange Book 2.0  New Look, New Features

Orange Book

identifies drug products approved on the basis of safety and effectiveness by the FDA under the Federal Food, Drug, and Cosmetic Act (FD&C Act) and related patent and exclusivity information.  LEARN

Patent Submission Date

date on which FDA receives patent information from NDA holder (21 C.F.R. 314.53(d)(ii)(5) LEARN

Orange Book Update

search results and drug listings now show patent submission dates where available;  may help generic drug manufacturers determine earliest date for marketing LEARN

Patent vs. Exclusivity

Patent– granted by US Patent and Trademark Office anytime during drug development;  encompass a wide range of claims.

Exclusivity– granted per FD&C Act. delays and prohibitions on approval of competitor drugs; for NDA and ANDA holders;  promote balance between new drug innovation and  generic drug competition LEARN


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Influenza Virus Vaccine: 2017-2018 Season

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Influenza Virus Vaccine: 2017-2018 Season

  • Trivalent vaccines contain the following:
    • an A/Michigan/45/2015 (H1N1) pdm09-like virus
    • an A/Hong Kong /4801/2014 (H3N2)-like virus
    • a B/Brisbane/60/2008-like virus (B/Victoria lineage).
  •  Quadrivalent vaccines contain additional B/Phuket/3073/2013-like virus (B/Yamagata lineage)
  • Flu vaccine lots that have been released and available for distribution by manufacturers

LEARN


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Consortia-pedia

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  • A searchable catalogue of nearly 500 profiles on research consortia. Learn more about their work – including mission, structure, data sharing, partners, and more – and connect with them
  • framework report that presents a series of questions for those seeking to create new collaborative efforts, expand existing ones, or re-orient early-stage programs
  • 2013 landscape analysis in Science Translational Medicine analyzing characteristics of 369 consortia around the world
  • Highlighted by CDER-FDA  as a comprehensive public resource for researchers seeking partners with similar interests and objectives

LEARN


 

 

 

 

2017 FDA Science Forum

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2017 FDA Science Forum

  • Presentation of unique scientific research and collaborative efforts of 11,000 FDA scientists
  • Investigation and application of new science and emerging technologies to inform FDA’s regulatory decision-making and foster safe innovation
  • Concentrating on creating test methods and developing process knowledge to ensure safety and efficacy of products
  • FDA science is critical to product quality and safety

LEARN


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FDA CDER Presentations Library

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FDA CDER Presentations Library

  • CDER professionals participate in several meetings, conferences and workshops throughout the year
  • Topics can range from users fees, to drug advertising and marketing, to genomics, to over-the-counter products
  • Materials and overviews from some of those meetings are listed in the presentations library

LEARN


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NextGen Collaboration Portal

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NextGen Collaboration Portal

New technology platform to allow industry to request FDA pre-ANDA meetings for complex generic drug products

  • initiate requests by uploading meeting request package
  • submit supporting documents e.g. presentation materials, additional information responses, post-meeting comments
  • minimize manual data entry
  • support GDUFA II performance goals.

LEARN


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CLIA and Waiver by Application

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 CLIA  and Waiver by Application

CLIA: Clinical Laboratory Improvement Amendments

  • Regulate laboratory testing and require clinical laboratories certification prior to in vitro diagnostic (IVD) testing
  • Multiple types of CLIA certificates, based on  complexity of diagnostic tests
  • Responsible federal agencies: FDA, CMC, CDC

CLIA  IVD Categorization:

  • By degree of complexity: waived, moderate complexity, and high complexity

CLIA Waiver by Application:

  • For moderate complexity may test
  • Manufacturer may request categorization of the test as waived
  • Provides evidence to FDA that test meets CLIA statutory criteria for waiver

LEARN


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FDA Sentinel System

SentinelFDA Sentinel System

Sentinel is used by the FDA-CDER to assess the safety of prescription drugs, including biological therapeutics and generic drugs

  • Managed in CDER’s Office of Surveillance and Epidemiology
  • Important component of CDER’s postmarketing safety system
  • Used to proactively assess drug safety, under real-world conditions, that more closely reflect patient care
  • Sentinel assessments make use of the automated surveillance tools

LEARN


Image credit: Sentinel/FDA

 

FDA/CMS Hurricane Relief Efforts

Hurricanes Irma and Jose

Support of the Hurricane Relief Effort

Harvey, Irma, and Maria

FDA

Address safety and shortages

  • Ensuring safety of regulated products including medicines, medical devices, food, and the blood supply
  • Priority Actions : Recommendations on food and medical products handling, assess damage and impact to industry facilities, avoid food and crop loss, identify solutions to prevent shortages of life-saving therapies

LEARN

CMS

Gain access to health coverage immediately 

  • Medicare special enrollment period:  Enroll, dis-enroll or switch Medicare health or prescription drug plans
  • Federal Health Insurance Exchange special enrollment period: For individuals  unable to complete application, plan selection, and enrollment process

LEARN


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FDA Adverse Events Reporting System (FAERS) Public Dashboard

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FDA Adverse Events Reporting System (FAERS) Public Dashboard

Highly interactive web-based tool for FAERS data

  • Human adverse events reported to the FDA by the pharmaceutical industry, healthcare providers and consumers
  • Expand access of FAERS data to general public
  • Some limitations : Incomplete reports, uncertain causation, unverified information, unknown rates of occurrence

DASHBOARD

LEARN


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FDA Opioid Action Plan

Opioids and FDA Opioid Action Plan

To address  growing epidemic of opioid abuse, dependence and overdose in the US

  • Expand use of advisory committees
  • Develop warnings and safety information for immediate-release (IR) opioid labeling
  • Strengthen postmarket requirements
  • Update Risk Evaluation and Mitigation Strategy (REMS) Program
  • Expand access to abuse-deterrent formulations (ADFs) to discourage abuse
  • Support better treatment.
  • Reassess the risk-benefit approval framework for opioid use

LEARN


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Medical Device and Hurricane Emergencies

Hurricanes Irma and Jose

Medical Device and Hurricane Emergencies

During natural disasters medical devices may be exposed to fluctuating power, contaminants, or unusual levels of heat or humidity.

FDA provides information on using medical devices during and following emergency situations

  • Re-Use of Devices
  • Disposal of Contaminated Devices
  • Reopening Dialysis Clinics
  • Medical Devices Requiring Refrigeration
  • Medical Devices Exposed to Heat and Humidity
  • Blood Glucose Meters
  • Mammography Facilities

LEARN


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Digital Health Entrepreneur-in-Residence Program

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Entrepreneur-in-Residence in CDRH Digital Health team 

  • To support and help develop our Software Precertification (PreCert) Pilot Program —a critical piece of CDRH’s Digital Health Innovation Action Plan
  • Need to have experience in digital health and passionate about developing innovative solutions to complex challenges
  • Help FDA establish the most appropriate criteria for standing up a firm-based precertification program

LEARN


 

FDARA & User Fees

FDARA

FDARA and FDA User Fee Programs

President signed into law the Food and Drug Administration Reauthorization Act (FDARA)

  • Revises and extends FDA’s user-fee programs for prescription drugs (PDUFA), medical devices (MDUFA), generic drugs (GDUFA) and biosimilar (BsUFA) products
  • Provides FDA resources for increasing regulatory efficiency and expedite availability of innovative, safe and effective medical products
  • Read how FDARA is making a difference

Drug User Fees

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Medical Device User Fees

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LEARN


 

 

Expanded Access Navigator

Expanded Access Navigator launched by Reagan-Udall Foundation in collaboration with patient advocacy groups, pharmaceutical industry, FDA, and Federal government

  • online tool to guide patients, caregiver, physicians, through process for requesting single-patient expanded access for unapproved investigational drugs
  • for patient who has serious or immediately life-threatening disease or condition for which there is no FDA-approved treatment
  • permits the product’s manufacturer, with FDA authorization, to provide investigational drug for patient
  • physician submits Single Patient Expanded Access request

LEARN


Image Credit: Reagan-Udall Foundation for the FDA 

Digital Health Innovation Action Plan

FDA announced the Digital Health Innovation Action Plan 

  • integrated approach to digital health technology and the implementation of the 21st Century Cures Act
  • recognize the unique characteristics of digital health technology and the marketplace
  • promote innovation of high-quality, safe, and effective digital health devices
  • Software Pre-Cert Pilot Program a key component of this plan

Additional information can be found on the Digital Health Webpage

LEARN


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Digital Health Software Precertification (PreCert) Program

FDA reimagining oversight of digital health technology

 “Software Precertification (PreCert) Pilot Program.” – new pilot program

  • looking first at the software developer and/or digital health technology developer, rather than primarily at the product

Goals 

  • modern approach to software iterations and changes
  • high quality medical product software throughout the life of the product
  • adjust key elements and measure based on program effectiveness

LEARN


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Purple Book Updates

The Purple Book - med

Purple Book Updates

“Purple Book” lists biological products, including any biosimilar and interchangeable biological products

LEARN


 

Cures Web Page

images of FDA-regulated innovative products

Cures Web Page

The 21st Century Cures Act (Cures Act) signed into law on December 13, 2016

  • Help accelerate medical product development
  • Bring new innovations to patients faster and more efficiently
  • Incorporate patient perspectives into the development of medical products
  • Modernize clinical trial designs and clinical outcome assessments

Newly created webpage details FDA plans and progress 

LEARN


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Humanitarian Device Exemption Updates

Humanitarian Use Device (HUD) 

Humanitarian Device Exemption (HDE)

  • Marketing Application for an HUD;  exempt from the effectiveness requirements and is subject to certain profit and use restriction

Learn about

  • Definitions: HUD & HDE
  • Prohibition on Profit
  • Annual Distribution Number

LEARN

Class of 2017 Commissioner’s Fellowship Program

Commissioner's Fellows

FDA Accepting Applications for Class of 2017 Commissioner’s Fellowship Program 

For health care professionals, scientists, and engineers

For regulatory science training, conduct cutting edge research on targeted scientific, policy, or regulatory issues under FDA senior scientist mentorship

Application deadline:  July 7, 5 pm EST
LEARN


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ORA Program Alignment

ORA: Aligning for the Future

  • FDA Program Alignment: Modernize and strengthen workforce to keep pace with acceleration of scientific innovation, global expansion, new programmatic mandates
  • Office of Regulatory Affairs (ORA): Implement program-based management to align staff by FDA-regulated product – enhance communication effectiveness, processes, ability to keep pace with scientific innovation and protect public health

LEARN

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National Drug Code (NDC) App

NDC Express mobile application

Section 510 of the Federal Food, Drug, and Cosmetic Act (Act) (21 U.S.C. (1972)

  • Requires registered drug establishments to list of  drugs manufactured, prepared, propagated, compounded, or processed for commercial distribution
  • Drug products have unique, three-segment number- National Drug Code (NDC)
  • Published in NDC Directory which is updated daily

New Mobile NDC App for quick directory searches 

LEARN


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Drug, Cosmetic or Both?

 

Product’s Intended Use determines regulation as Drug, Cosmetic, or Both 

  • Drugs:  Diagnosis, cure, mitigation, treatment, or prevention of disease and affect structure/function of body
  • Cosmetics: Rubbed, poured, sprinkled, sprayed on, introduced into, applied to human body for cleansing, beautifying, promoting attractiveness, or altering the appearance
  • Drug + Cosmetic:  Example – antidandruff shampoo
  • FDA does not recognize the term “cosmeceutical”

LEARN

FDA Research on Health Disparities

Health Disparities refer to differences in the health status of different groups of people

  • May affect disease frequency, severity, mortality among certain groups
  • Exist for conditions, including HIV/AIDS, hepatitis, diabetes, cancer, heart disease
  • Racial and ethnic minorities may be more likely to have disparities

Office of Minority Health (OMH) Research and Collaboration program strengthening the FDA’s ability to identify and address these disparities

  • Fund scientific research in academia
  • Advance student fellowships on understanding of health
  • Focus on consumer communication, FDA-academia collaboration on how to improve the health literacy and cultural competence

LEARN

VIBERZI Drug Safety Podcast

FDA Logo, hands holding pills

Viberzi (eluxadoline) Safety Podcast

FDA review shows increased risk of serious pancreatitis in patients without a gallbladder

  • Prescribed to treat irritable bowel syndrome with diarrhea (IBS-D)
  • Patients, without a gallbladder, have increased risk of developing serious pancreatitis that could result in hospitalization or death
  • Pancreatitis caused by spasm of certain digestive system muscle in the small intestine
  • FDA working with manufacturer, Allergan, to address safety concerns

LISTEN & LEARN


 

FDA Approvals & Classification: ZEJULA, DUPIXENT, OCREVUS, BRAIN SENTINEL

FDA BRIEF: Week of March 27, 2017

FDA approved


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ZEJULA (niraparib) capsules

Tesaro, Waltham, MA, USA 

INDICATION: Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy

ADDRESSING UNMET NEED:

  • >2,000 women will be diagnosed in 2017;  >14,000 will die
  • Maintenance therapy important for patients with positive response
  • ZEJULA offers new treatment option to delay growth regardless of genetic mutation

REG PATHWAY:  NDA

  • Fast Track, Priority Review and Breakthrough Therapy designation
  • Orphan Drug designation for treating recurrent epithelial ovarian cancer.
  • Application approved approximately three months ahead of the goal date
  • Postmarketing Requirement:  PK trial in patients with moderate hepatic impairment

MECHANISM OF ACTION: Inhibitor of poly(ADP-ribose) polymerase (PARP) enzymes, PARP-1 and PARP-2, which play a role in DNA repair

EFFICACY:

  • Single double-blind, placebo-controlled trial, n=553, patients with platinumsensitive recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. Patients with deleterious or suspected deleterious germline BRCA mutations (gBRCAm) were assigned to the germline BRCA mutated (gBRCAmut) cohort (n=203), and those without germline BRCA mutations were assigned to the non-gBRCAmut cohort (n=350)., ZEJULA vs. placebo
  • Primary efficacy outcome: PFS (progression-free survival)  determined by RECIST
  • gBRCAmut cohort: 21 mo. vs  5.5 mo. (p<0.0001)
  •  non-gBRCAmut cohort: 9.3 mo. vs 3.9 mo. (p<0.0001)

SAFETY:

  • Common adverse reactions:  Thrombocytopenia, anemia, neutropenia, leukopenia, palpitations, nausea, constipation, vomiting, abdominal pain/distention, mucositis/stomatitis, diarrhea, dyspepsia, dry mouth, fatigue, decreased appetite, urinary tract infection, AST/ALT elevation, myalgia, back pain, arthralgia, headache, dizziness, dysgeusia, insomnia, anxiety, nasopharyngitis, dyspnea, cough, rash, and hypertension.

LABEL


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DUPIXENT (dupilumab) injection

Regeneron Pharmaceuticals,  Tarrytown, NY, USA

INDICATION:  Treatment of adult patients with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. DUPIXENT can be used with or without topical corticosteroids

ADDRESSING UNMET NEED:

  • Atopic dermatitis most common of the many types of eczema; onset in childhood and can last through adulthood
  • New and innovative therapy for skin disease not controlled by topical therapies

REG PATHWAY: BLA

  • Priority Review, Breakthrough Therapy designation
  • Postmarketing requirements: Pediatric studies, Registry-based observational study on maternal, fetal, and infant outcomes,  Retrospective cohort study on pregnancy outcomes

MECHANISM OF ACTION: Human monoclonal IgG4 antibody, inhibits interleukin-4 (IL-4) and interleukin-13 (IL-13), inhibits cytokine-induced responses, including the release of proinflammatory cytokines, chemokines and IgE

EFFICACY:

  • 3 randomized, double-blind, placebo-controlled trials, n=2,119 , patients with moderate-to-severe atopic dermatitis, DUPIXENT or placebo for 16 weeks
  • Primary endpoint: Change from baseline and at least a 2-point improvement
  • Other endpoints: Eczema Area and Severity Index, itch reduction,  improvement in pruritus
  • Greater response of clear or almost clear skin,  itch reduction
SAFETY:
  • Serious allergic reactions and eye problems- conjunctivitis, keratitis
  • Most common side effects: Injection site reactions; cold sores, eye and eyelid inflammation

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OCREVUS (ocrelizumab) intravenous infusion

Genentech, Member of Roche Group, San Francisco, CA, USA

INDICATION: Treatment of adult patients with relapsing or primary progressive forms of multiple sclerosis (PPMS)

ADDRESSING UNMET NEED:

  • MS most common causes of neurological disability in young adults and occurs more frequently in women than men
  • Approximately 15 percent of patients with MS have PPMS
  • OCREVUS first approved drug for PPMS

REG PATHWAY: BLA

  •  Breakthrough Therapy Designation, Fast Track Designation, Priority Review
  • Postmarketing requirements: Pediatric study, Longitudinal observational study in adult patients for breast cancer incidence,  prospective pregnancy exposure registry, pre-and postnatal development study in primates

MECHANISM OF ACTION:  Binding to CD20, resulting in antibody-dependent cellular cytolysis and complement-mediated lysis

EFFICACY:

  • 2 studies, n= 1,656, treated for 96 weeks; OCREVUS vs. Rebif (interferon beta-1a)
  • Primary outcome: Annualized relapse rate (ARR)
  • Additional outcomes: Confirmed disability progression, new gadolinium (Gd)-enhancing lesions,  new or enlarging MRI T2 hyperintense lesions
  • Significantly lowered ARR and tdisability progression
  • 1 randomized, double-blind, placebo-controlled clinical trial in PPMS,n=488
  •  Primary outcome: Onset of disability progression
  • Onset of disability progression significantly longer for OCREVUS vs. placebo
SAFETY:
  • Should not be used in patients with hepatitis B infection
  • Must be dispensed with patient Medication Guide
  • Can cause serious  infusion-related reactions, which can be serious
  • May increase the risk for malignancies, particularly breast cancer
  • Most common side effect:  Upper respiratory tract infection, skin infection, and lower respiratory tract infection

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Brain Sentinel Monitoring and Alerting System

Brain Sentinel, San Antonio, TX, USA

INDICATION FOR USE: Adjunct to seizure monitoring in adults in the home or healthcare facilities during periods of rest.

The device is to be used on the belly of the biceps muscle to analyze surface electromyographs (sEMG) signals that may be associated with generalized tonic-clonic (GTC) seizures and to provide an alarm to alert caregivers of unilateral, appendicular, tonic extension that could be associated with a GTC seizure.

The System records and stores sEMG data for subsequent review by a trained healthcare professional

REG PATHWAY: De Novo request

  • Regulation Number: 21 CFR 882.1580
  • Regulation Name: Non-EEG physiological signal based seizure monitoring system
  • Regulatory Classification: Class II
  • Product Code: POS

GENERIC DEVICE DESCRIPTION: Non-invasive prescription device that collects physiological signals other than EEG to identify physiological signals that may be associated with a seizure

RISKS & MITIGATIONS:

  • Adverse tissue reaction: Biocompatibility evaluation
  • Equipment malfunction leading to injury to users (shock, burn): Electrical safety, thermal, and mechanical testing, Electromagnetic compatibility testing, Labeling
  • Interference with or from other electrical devices: Electromagnetic compatibility testing
  • Incorrect alerts, including missing a seizure or false alarm: Clinical performance testing, Non-clinical performance testing, Software verification, validation and hazard analysis, Labeling, Training

CLASSIFICATION ORDER


 

CDER Oncology Fellowships

Students participating in the Commissioner's Fellowship Program

Oncology Genomics Fellowship – CDER

Oncology Bioinformatics Fellowship – CDER

Join a multi-disciplinary team to pursue next generation sequencing and molecular biology translational research in order to understand complex regulatory and scientific questions in oncology. Multiple projects collaborating with outside academic or government institutions will be pursued. As many of the projects will be performed using next generation sequencing technology, experience in this technique will be key.

Oncology Genomics Fellowship – CDER

Oncology Bioinformatics Fellowship – CDER

FDA – Office of Translational Sciences

Whats new in regulatory science

Office of Translational Sciences (OTS) – CDER

Promotes and Protects Public Health by:

  • scientific collaboration and innovation in drug regulatory review
  • validity of clinical trial design and analysis
  • quantitative and statistical approaches to decision making
  • alignment of CDER research with CDER goals
  • establishing technology transfer agreements
  • knowledge management databases
  • bioequivalence inspections

Offices:

Office of Biostatistics

Office of Computational Science

Office of Clinical Pharmacology

Office of Study and Integrity and Surveillance

LEARN

FDA Patient Representative Program

FDA Patient Representative Programpatient

Managed by Office of Health and Constituent Affairs, Office of Commissioner

FDA Patient Representatives

  • knowledgeable and experienced in over 300 diseases and conditions
  • participate in FDA Advisory Committees and panels, review division meetings
  • provide direct input to inform decision-making for drugs, biologics, and devices

 

Role of the FDA Patient RepresentativeCriteria for Becoming a FDA Patient RepresentativeConflict of InterestFrequently Asked Questions

LEARN


 

Biomarkers Used as Outcomes

biomarker

Biomarkers In Development of FDA-Approved Therapeutics

October 2007- December 2015

BIOMARKERS

  • used as clinical trial outcome
  • accepted as basis for drug and biologic approvals
  • consult with FDA review division as early as possible in drug development regarding the use of a particular biomarker in clinical trials
  • examples of approved biomarkers below

LEARN

Anesthesiology T1*; magnitude of T4/T1* ratio by acceleromyography
Cardiology Blood pressure
Serum low-density lipoprotein (LDL-C)
Hematology Hemoglobin
Platelet count
Ecarin clotting time; activated partial thromboplastin time; thrombin time; activated clotting time; plasma diluted thrombin time
Serum ferritin
Infectious Disease Hepatitis C virus (HCV) RNA*
Human immunodeficiency virus (HIV)-1 RNA
Sputum culture conversion to negative
Parasite count resolution
Inborn Errors of Metabolism White blood cell count; neutrophil count; red blood cell count; mean corpuscular volume
Growth in height or weight
Serum LDL-C
Blood phenylalanine
Forced vital capacity (FVC)
Hemoglobin
Plasma ammonia; plasma glutamine; and plasma citrulline
Splenic volume by magnetic resonance imaging (MRI)
Metabolism and Endocrinology Body weight
Bone mineral density by DEXA* scan
Hemoglobin A1c*
Serum calcium; oral calcium supplements; oral vitamin D supplements
Serum LDL-C
Urinary free cortisol (UFC)
Vertebral fractures by X-ray
Visceral adipose tissue (VAT) by computed tomography (CT) scan
Nephrology Hemoglobin
Serum sodium
Oncology CD34 positive cell count
Complete blood count (e.g., absolute neutrophil count)
Tumor burden by Bcr-Abl* (Philadelphia chromosome)
Tumor burden by Philadelphia chromosome positive cells
Plasma methotrexate
Splenic volume
Serum asparaginase
Serum testosterone
Tumor burden by imaging (using criteria such as RECIST* or EBMT*)
Ophthalmology Anterior chamber cells
Intraocular pressure (IOP)
Vitreomacular adhesion (VMA) by optical coherency tomography
Pulmonology Forced expiratory volume in one second (FEV1)
FVC
Respiratory distress syndrome (RDS) by chest X-ray and fraction of inspired oxygen (FiO2)
Rheumatology Joint angle
Uric acid
Transplant Biopsy-proven acute rejection (BPAR)

Medical Imaging Qualitative assessment of cerebral distribution of radioactive signal
Qualitative assessment of radioactive uptake (such as lymph node or tumor detection)
Qualitative regional assessment of localized radiographic signal intensity
Quantitation of arterial narrowing
Radioactive uptake in myocardial segments
Semi-quantitative lesion characteristics (such as internal morphology; contrast enhancement; border delineation)
Ultrasonographic signal intensity

 

Office of Manufacturing Quality Presentations

omq

LEARN

RAT (Regenerative Advanced Therapy Designation) & OTAT (Office of Tissues and Advanced Therapies)

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WHAT: Resource for seeking designation as Regenerative Advanced Therapy – RAT

  • Cell therapy, therapeutic tissue engineering product, human cell and tissue product, any combination

WHY: Described in Section 3033 of the 21st Century Cures Act

WHEN: Either concurrently with IND submission or as an IND amendment

WHO: CBER, Office of Tissues and Advanced Therapies (OTAT)

LEARN

Is It Really ‘FDA Approved?’

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Clarification of   “FDA approved!” claim

DOES approve:

  • New Drugs and Biologics – by determining e benefits of the product outweigh the known risks for the intended use
  • Medical Devices – use risk-based, tiered approach
  • Human Cells and Tissues – use risk-based approach
  • Food and Color additives – for safety

DOES NOT approve :

  • Companies – health care facilities, laboratories, or manufacturers; can inspect
  • Compounded drugs
  • Tobacco products – regulated based on public health standard
  • Cosmetics – except color additives
  • Medical foods – for dietary management
  • Infant formula
  • Dietary Supplements
  • Food Label, including the Nutrition Facts panel
  • Structure-Function claims on dietary supplements and other foods

Misuse of FDA’s logo may violate federal law

LEARN

EvGen- Evidence Generation

evgen

WHAT:  National Medical Evidence Generation Collaborative (EvGen) for high quality scientific evidence to support medical product development and decision making
WHY: Leverage previously isolated data systems to utilize available information collected during healthcare-related activities (e.g., medical research, medical product development, clinical care)
HOW: Combining insights, expertise, and technologies from across the spectrum of federal and private health sectors

LEARN

22 FDA Case Studies with Divergent Phase 2/Phase 3 Results

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22 Case Studies with Divergent Phase 2/Phase 3 Results

22 case studies of drugs, vaccines and medical devices since 1999 – No confirmation of:

  • 14: effectiveness
  • 1 : safety
  • 7: safety and effectiveness

Why?

  • Unexpected Results with Large Randomized Controlled Studies
  • Presumed Mechanism of Action Does Not Automatically Predict Clinical Effects
  • Biomarkers Do Not Reliably Predict Clinical Outcomes

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Drug Safety Priorities, 2015-2016

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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

  • 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

LEARN

2016 Novel Drug Summary

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22 NOVEL DRUGS

Innovative products that serve unmet medical needs/significantly advance patient care. Have chemical structures that have never been approved before.

Adlyxin,  Anthim,  Axumin,  Briviact,  Cinqair, Defitelio, Epclusa, Eucrisa, Exondys 51, Lartruvo, Netspot, Nuplazid, Ocaliva, Rubraca, Spinraza, Taltz, Tecentriq, Venclexta, Xiidra, Zepatier, Zinbryta, Zinplava

SUMMARY

  • Impact: Public health, First-in-class,  Rare diseases
  • Innovation: Breakthrough, Fast Track, Pirority Review, Accelerated Approva
  • Predictability: Met PDUFA goal date
  • Access: First cycle approval, First approval in US

LEARN

Laboratory Developed Tests

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WHAT: Laboratory developed test (LDT) is in vitro diagnostic test designed, manufactured and used within a single laboratory

WHY: To measure or detect analytes in sample taken from a human body

WHO:  Individual labs. However, diagnostic devices not considered to to be LDTs if they are designed or manufactured completely, or partly, outside of the laboratory that offers and uses them.

HOW: FDA does not enforce premarket review as tests are relatively simple. However, FDA concern with some high-risk LDTs (e.g. cancer detection)  with unsubstantiated claims, erroneous results, data falsification. Discussion paper issued in Jan 2017

LEARN

 

Medical Device Recalls, Database & OpenFDA API

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Medical Device Recalls
WHAT: Manufacturer takes a correction or removal action for medical device problem. FDA reviews strategy, assesses health hazard, violatiion of law, assigns classification
  • Class I: Reasonable chance to cause serious health problems or death
  • Class II: May cause temporary/reversible health problem
  • Class III: Not likely to cause health problem or injury
WHY:

  • Inspecting for problems
  • Repairing device
  • Adjusting settings
  • Re-labeling
  • Destroying
  • Notifying patients of problem
  • Monitoring patients for health issues

WHO: Company (voluntarily) or by FDA

HOW: FDA notifies public
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Searchable Database

  • Recalls since Nov 2002
  • Beginning Jan 2017, includes correction or removal actions prior to recall classification

LINK


api
Downloads [/device/recall]
  • OpenFDA is Elasticsearch-based API that serves public FDA data
  • Zipped JSON files
  • Records in same format as API calls to endpoint

LINK


 

FDA -CDRH FY2017 Proposed Guidances

Image result for CDRH guidances

 

WHAT: Guidance documents that CDRH intends to publish in FY2017 & previously-issued final guidances for which CDRH is interested in receiving external feedback regarding whether these final guidances should be revised or withdrawn.

WHY: Per MDUFA III negotiations, to help get safe and effective medical devices to market more quickly

HOW:  3 lists

  • A List: To be published
  • B List: To be published  as resources permit
  • Retrospective review of 2007, 1997, 1987, 1977 documents

LEARN

 

Patient-Reported Outcomes with LASIK (PROWL)

Patient-Reported Outcomes with LASIK (PROWL)

Image result for patient reported outcomes with LASIK

WHAT:  Patient experiences, not health care provider, to measure impact symptoms directly had on performing usual activities.

HOW: Pilot, PROWL-1 and PROWL-2 studies

  • Evaluate new scales to measure visual symptoms, patient satisfaction, and expectations for and following LASIK surgery

RESULTS:

  • Difficulty driving at night, severely impacted a patient’s daily living
  • Debilitating vision symptoms and severe dry eye
  • <1% experienced difficulty performing their usual activities following LASIK surgery

LEARN

MATx App for Opioid Use Disorder

WHAT: Free App to  support practitioners who provide medication-assisted treatment (MAT), as well as those who plan to do so in the future.

WHY: 80% of Americans with opioid use disorder do not receive treatment

HOW: 

  • Information on FDA approved treatment approaches and medications
  • Buprenorphine prescribing guide
  • Clinical support tools – treatment guidelines, ICD-10 coding, working with special populations
  • Access to critical helplines and SAMHSA’s treatment locators.

LEARN

LASIK Quality of Life Collaboration Project

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WHAT:  LASIK Quality of Life Collaboration Project (LQOLCP) to understand potential risk of severe problems from LASIK

WHY: Develop tool to assess patients with difficulties with usual activities and identify predictors

WHO:  FDA, National Eye Institute and Department of Defense

HOW: Patient-Reported Outcomes with LASIK (PROWL) studies

  • visual symptoms before and after their LASIK surgery
  • impact of symptoms on performing usual activities

LEARN

 

FDA News: Orphan Disease R&D, Mouse Model for Zika, FDA-CMS Shared Responsibility

FDA BRIEF: Week of Nov. 14, 2016

FDA Voice


Orphan Disease R & D Has a Home at FDA

John J. Whyte, MD, MPH, Director, Professional Affairs and Stakeholder Engagement, FDA

Gayatri Rao, MD, JD, Director, Office of Orphan Products Development, FDA

LISTEN


FDA Research on Zika Virus Vaccines and Therapeutics

Neonatal mouse model provides a new platform for Zika virus research

  • New mouse model developed by FDA for exploring activity of Zika vaccines/therapeutics
  • C57BL/6 mouse strain susceptible to Zika, develops neurological symptoms and eventually recover from disease

PLoS Publication


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  • Acknowledges ‘ fragmentation’: FDA approval/clearance vs. approval for coverage and payment
  • States shared sources of evidence for both Agencies while still applying the most appropriate criteria to their decision making
  • Clarified differing FDA and CMS standards:
    • FDA: Product approval or clearance, based on  “substantial evidence”
    • CMS: Coverage approval based on  “reasonable and necessary”
  • Recommends early involvement of health systems and payers to help to understand and address the kinds of evidence needed to incorporate the new product into practice
  • Highlights several FDA-CMS initiatives to facilitate gathering adequate evidence for both Agencies
  • Provides perspectives on ‘personalized medicine’

 

 

Regulatory Pharmaceutical Fellowship

Image result for Regulatory Pharmaceutical Fellowship clipart

WHAT: 2-year fellowship – Rotations with FDA’s CDER and Industry

WHY: Train selected candidates in one of three tracks focused on the medical and regulatory aspects of drug information dissemination, drug advertising and promotion, or medication safety

WHO: Jointly Sponsored by: FDA, Purdue University, Eli Lilly and Company, Janssen Scientific Affairs, LLC, and Johnson and Johnson

WHEN: Available Positions for 2017 – 2019

LEARN

Reporting Allegations of Regulatory Misconduct

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WHAT:  FDA reporting  of allegation of regulatory misconduct of medical device manufacturer or individuals marketing medical devices

WHY: Help FDA identify potential risks to patients, determine whether further investigation is warranted, and identify steps needed to address or correct violation

WHO: Anyone may file a complaint; identity or contact information will not be shared publicly

 

HOW: Allegations of Regulatory Misconduct Form, by email, or by regular mail.

LEARN

 

 

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

LEARN


 

Dealing with ADHD

Dealing with ADHD: What You Need to Know

boy struggling in class (600x400)

Diagnosis 

  • 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

LEARN

Medical Device Misconnections

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Reducing Risks Associated with Medical Device Misconnections


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. 

LEARN

 

Bioresearch Monitoring Information System (BMIS)

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WHAT: Bioresearch monitoring web site that makes clinical research information available to the public.

WHO: CDER

WHY:  To foster transparency and encourage information sharing within the clinical research community. Contains information on clinical investigators, contract research organizations, and institutional review boards involved in the conduct of IND studies with human investigational drugs and therapeutic biologics.

HOW: Extracted from IND-related documents  for whom FDA has received Form FDA 1572

LINK

FDA National Evaluation System for health Technology (NEST)

nest

WHAT:  Foundation to efficiently generate evidence for medical device evaluation and regulatory decision-making

WHY: National evaluation system would generate evidence across the total product lifecycle (TPLC), by leveraging real-world evidence (RWE)and applying advanced analytics

WHO:  Link and synthesize data across the medical device landscape – clinical registries, electronic health records and medical billing claims

HOW: High quality RWE  for health care providers and patients to make better informed treatment decisions; strike right balance between safety vs innovation and patient access

LEARN

 

 

FDA Division of Biology, Chemistry, and Materials Science

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Stent

A scientist working with test tubes.

WHAT: Part of  Office of Science and Engineering Laboratories (OSEL), CDRH

WHY:

  • Determine effects of implanted devices, potential device adverse effects,  source and impact of device degradation
  • Develop experimental data, test methods, and protocols
  • Develop measurements, methods, and analytical procedures to support CDRH pre-market and post-market activities.

WHO: Experts in biology, biomaterials science, biomedical and chemical engineering, chemical physics, materials science, pharmacology, physical chemistry, polymer science, and toxicology

HOW: Focus on

  • analytical chemistry and electrochemistry
  • biocompatibility, toxicology, and biological risk assessment
  • biosensors and biomarkers
  • extractables, leachables, and chemical contamination
  • genomic and genetic technologies
  • immune and cellular responses
  • infection control, sterility, and biofilms
  • materials characterization, processing, and materials degradation
  • modeling of physiological processes
  • multicomponent mass transfer and reaction kinetics
  • nanotechnology

FDA CERSI Lecture Series, MDSAP Pilot

CERSI Lecture Series

Science and Research at FDA

WHAT: Lecture series to present current research and advancements in regulatory science related to pre-clinical evaluation of safety and efficacy of medical products, emerging technologies, and assessing diverse data sets through information sciences to improve health outcomes.

WHO:  Centers of Excellence in Regulatory Science and Innovation

WHEN: CERSI Workshops & Meetings

HOW: Presented by scientists from the CERSI academic institutions or collaborators of CERSI investigators.

LEARN


 

Medical Device Single Audit Program (MDSAP)

Slide 1

 

WHAT : Invitation to manufacturers to participate in innovative regulatory single audit program.

WHO: MDSAP Regulatory Authority Council

HOW: Learning modules in CDRH Learn – see links below

Inspections – Global Harmonization  (New modules 9/8/16)

Primer – International Medical Device Regulators Forum (IMDRF) Medical Device Single Audit Program (MDSAP) Pilot
Presentation Closed Captioned   Printable Slides   Transcript

1. Introduction to MDSAP
Presentation

2. MDSAP Management
Presentation

3. MDSAP Device Marketing Authorization and Facility Registration
Presentation

4. MDSAP Measurement, Analysis and Improvement
Presentation

5. MDSAP Medical Device Adverse Events and Advisory Notices Reporting
Presentation

6. MDSAP Design and Development
Presentation

7. MDSAP Production and Service Controls, part 1
Presentation

8. MDSAP Production and Service Controls, part 2
Presentation

9. MDSAP Production and Service Controls, part 3
Presentation

10. MDSAP Purchasing
Presentation


 

Drug Safety Labeling Changes

 

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WHAT: Drug Safety Labeling Changes (SLC) database provides approved safety labeling changes

WHEN: Data from from January 2016 forward. Prior information at MedWatch website.

WHO: CDER’s Office of Communications – OCOMM

WHY:To more efficiently gather, organize and distribute information related to drug safety label changes. Prompt access to  health care providers and consumers to better promote patient health.

HOW: Visit site, Sign up for updates

LINK

FDA-CDC Free Webinars

Heater-Cooler Device

WHAT: Free Continuing Education Webinars from CDC for Healthcare Providers. CE is available! There are no fees for CE.

WHEN: August 29, 2016 : Invasive Nontuberculous Mycobacterium Infections Associated with Exposure to Heater-Cooler Units during Cardiac Surgery

WHY: Recent reports of suggest that heater-cooler units used in cardiopulmonary bypass procedures, are putting patients at risk for invasive nontuberculous mycobacterium (NTM) infections. CDC offering expert panel to discuss

  • Evidence
  • Clinical manifestations, diagnosis, and treatment strategies
  • Risk identification and mitigation strategies

LINK

Web-based Orange book, HIPAA @ 20

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WHAT: Web-based version of the Orange Book, which identifies drug products approved on the basis of safety and effectiveness by the FDA, first appeared as a published list in October 1980.

WHY: Updated design with new, user-friendly search options that help users customize their research experience.

HOW :  Redesigned web page conveniently collects search and browse options onto the home page to improve access of these commonly used features.

LINK


HIPAA

WHEN: August 21, 1996, a bipartisan legislation called the Health Insurance Portability and Accountability Act or HIPAA

WHAT:  More than medical privacy and security law.  Allows transfer and continue health insurance after change or job loss – under Consolidated Omnibus Budget Reconciliation Act (COBRA). Further developed to Affordable Care Act (ACA) amendment and expansion of original HIPAA consumer protections.

LINK

MedSun

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WHAT: Work collaboratively  with a network of 250 hospitals, nursing homes and home health facilities in US

WHO: CDRH

WHY: To assist in detecting, understanding, and sharing information on safety of devices

WHEN: Launched in 2002

HOW: Secure, on-line system for reporting problems with the use of medical devices.

LINK

 

 

Medical Device Interoperability

IOT

WHAT:  FDA position and guidance on medical device interoperability  – ability to safely, securely, and effectively exchange and use information among one or more devices, products, technologies, or systems.

WHY:  Interoperable devices with the ability to share information across systems and platforms can:

  • Improve patient care,
  • Reduce errors and adverse events
  • Encourage innovation.

WHO: CDRH – along with  hospitals, health care providers, manufacturers, standards development organizations, and other interested parties to promote medical device interoperability.

LINK

FDA on LinkedIn, REMS@FDA Tutorial

SBIA.JPG

WHAT:  Stay up to date with SBIA by receiving updates directly to your LinkedIn feed

WHY:  Will post items such as:

  • Conferences and workshops
  • Webinars
  • SBIA Chronicles e-newsletters and Podcasts
  • SBIA Education Series
  • Guidance documents
  • New meeting announcements
  • FDA Job Announcements, as applicable
  • and more!

HOW: 

https://www.linkedin.com/company/cder-small-business-and-industry-assistance

FDA LinkedIn page group.


 

REMS.JPG

REMS@FDA

WHAT: FDA may require a Risk Evaluation and Mitigation Strategy (REMS) to  ensure that the benefits of certain drugs outweigh their risks. FDA has a new  and improved  REMS@FDA website

WHY: FDA pharmacists show how to navigate FDA’s user-friendly REMS website. The new website makes it easier  what is required to prescribe or dispense a drug with a REMS.

LEARN

 

 

OPDP

OPDP.JPG

WHAT:  Office of Prescription Drug Promotion

WHY: To protect the public health by ensuring that prescription drug information is truthful, balanced, and accurately communicated.

HOW: Comprehensive surveillance, enforcement, and education program, and by fostering better communication of labeling and promotional information to both healthcare professionals and consumers

WHO: CDER

WHAT ELSE: Bad Ad Program –  Truthful Prescription Drug Advertising and Promotion
The Prescriber’s Role – Recognize and Report

LEARN1

LEARN2

522 Postmarket Surveillance Studies

522

 

WHAT:  Design, Tracking, Oversight, Review of studies mandated under section 522, Federal Food, Drug and Cosmetic Act

WHY: To ensure well-designed 522 postmarket surveillance studies, conducted effectively and efficiently in the least burdensome manner

WHEN: Since 2008

WHO: Division of Epidemiology , Office of Surveillance and Biometrics, CDRH

HOW: CDRH Internal Tracking System, PUBLIC WEBPAGE

LINK

FDA 3D Printing Webpage

 

3D web

3D Printing of Medical Devices

 WHAT: New webpage on 3D printed medical devices (e.g. orthopedic and cranial implants, surgical instruments, dental restorations such as crowns, external prosthetics). FDA has cleared more than 85 3D printed medical devices!

WHO:  CDRH

HOW: FDA standards discussed in presentations, workshops and a new guidance posted in this blog

LINK

CDEReCATS, CDRH Emergency Use Authorizations

CDEReCATS

WHAT: New electronic system to request a Certificate of Pharmaceutical Product (CPP) online as an alternative to a paper submission

WHO: CDER

WHY:  Possible reduction in certificate processing time, a guided step-by-step application process, real-time validation of data, and e-mail notifications of status updates

HOW: Electronic submission procedures guide

LINK


Emergency use

 

WHAT: Listing of  current and terminated Emergency Use Authorizations that make available diagnostic and therapeutic medical devices

WHO : CDRH

WHY: To diagnose and respond to public health emergencies

LINK


 

FDA Biomarker Qualification Program

Biomarker qual

WHO: CDER

WHAT:  To facilitate drug development process by  supporting  regulatory qualification of  biomarker for a particular context of use

WHY: 

  • Provide framework for development and regulatory acceptance
  • Facilitate integration into regulatory review
  • Encourage identification of new and emerging biomarkers
  • Support outreach to stakeholders

HOW: Submit biomarker for qualification

LINK

CDRH National Evaluation System, Mobile Health Apps Interactive Tool

 

NES

WHAT:  To generate evidence across total product lifecycle by leveraging real-world evidence for medical device evaluation and regulatory decision-making

WHY:  Quality real-world evidence for health care providers and patients to make informed treatment decisions and balance safety vs device innovation and patient access

WHEN: Initial report in 2012. Included in CDRH 2016-2017 priorities

LEARN


 

 

tool2

WHAT: Web-based tool for developers of health-related mobile apps

WHO: Federal Trade Commission (FTC), Dept of  Health and Human Services, FDA

WHY: Help developers understand what federal laws and regulations  might apply to their apps – FTC Act, HIPAA, FD&C Act

LEARN

 

Medical Device Bans

ban

glove

WHAT: Total prohibition on the current and future sales, distribution, and manufacturing of a medical device

WHY:  Analyzing and weighing the risks and benefits the device poses to individuals. Can ban a device without actual proof of illness or injury, and only needs to find that a device has the potential to present the required degree of risk based on all available data and information

WHEN: Acts on this authority very rarely. Until 2016, the FDA banned only one other medical device, prosthetic hair fibers. In 2016, FDA proposed ban on powdered gloves on the unreasonable and substantial risk of illness or injury to exposed individuals

HOW: Notice of proposed rulemaking is published in the Federal Register

LINK


 

CDRH Experiential Learning Program

Experiential learning

WHAT: CDRH 2016 Experiential Learning Program (ELP)  to invite medical device industry, academia, and health care facilities to  participate in training program for FDA’s medical device review staff

WHY: CDRH staff training to

  •  understand policies, laboratory practices, and challenges with device development life cycle
  • enhance communication and facilitate the premarket review process.
  • understand current industry practices, innovative technologies, regulatory impacts, and regulatory needs

NOT a  compliance inspection

WHO: Medical device industry, academia, and health care facilities  can participate

HOW:  Submit electronic or written request for participation

LINK

FDA Medical Devices Research Programs

 

research

WHAT: Medical Devices Research Program aim to ensure patients have access to high quality, safe and effective medical devices.

WHY: Advance regulatory science of developing new tools, standards and approaches to assess the safety, efficacy, quality, and performance of medical devices and radiation-emitting products.

HOW: Laboratory and field research in the areas of physical, life, and engineering sciences as well as epidemiological research in postmarket device safety. Collaborations with academia, healthcare providers, other government agencies and industry.

LINK