Market Authorizations: ACUVUE OASYS Light Adaptive Contact Lens, IDx-DR retinal diagnostic software, OCS Lung System

Capture.JPGAcuvue Oasys Contact Lenses with Transitions Light Intelligent Technology 

Johnson & Johnson Vision care

USE: Soft Contact lenses that automatically darkens the lens when exposed to bright light. Indicated for daily use to correct the vision of people with non-diseased eyes who are nearsighted (myopia) or farsighted (hyperopia).

ADDRESSING UNMET NEED:  First contact lens to incorporate the same technology that is used in eyeglasses that automatically darken in the sun

DESCRIPTION:

  • Contains photochromic additive that adapts the amount of visible light filtered to the eye based on the amount of UV light to which they are exposed
  • Results in slightly darkened lenses in bright sunlight that automatically return to a regular tint when exposed to normal or dark lighting conditions. 

SAFETY AND EFFECTIVENESS:

  • Clinical study of 24 patients that evaluated daytime and nighttime driving performance while wearing the contact lenses
  • No evidence of concerns with either driving performance or vision while wearing the lenses
  • May cause inflammation or infection in or around the eye or eyelids 

REGULTORY PATHWAY: 510(k)

  • Classification: II
  • Regulation No. : 886.5925
  • Classification Product Code: LPL
  • Subsequent Product Code: MVN

REIMBURSEMENT

  • Medicare provides limited coverage for contact lenses under Medicare Vision Services
  • Acuvue Brand covered by private payors

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IDx-DR Retinal diagnostic software device

IDx LLC

INDICATION FOR USE: For use by health care providers to automatically detect more than mild diabetic retinopathy (mtmDR) in adults diagnosed with diabetes who have not been previously diagnosed with diabetic retinopathy. IDx-DR is indicated for use with the Topcon NW400.

ADDRESSING UNMET NEED:

  • Diabetic retinopathy is the most common cause of vision loss among the more than 30 million Americans living with diabetes
  • Leading cause of vision impairment and blindness among working-age adults
  • First medical device to use Artificial Intelligence (AI) to detect greater than a mild level of the eye disease diabetic retinopathy in adults who have diabetes

DESCRIPTION:

  • Software program using AI to analyze eye images taken with retinal camera, Topcon NW400
  • Doctor uploads digital images of retinas to cloud server with IDx-DR software
  • Software provides doctor with one of two results
    1. “more than mild diabetic retinopathy detected: refer to an eye care professional”
    2. “negative for more than mild diabetic retinopathy; rescreen in 12 months.”
  • If positive result – further diagnostic evaluation and possible treatment as soon as possible

GENERIC DEVICE TYPE: Retinal diagnostic software device.

Prescription software device that incorporates an adaptive algorithm to evaluate ophthalmic images for diagnostic screening to identify retinal diseases or conditions.

ACCURACY & PRECISION:

  • Clinical study of retinal images, n=900 patients with diabetes, 10 primary care sites
  • Correctly identify presence of more than mild diabetic retinopathy: 87.4%
  • Correctly identify patients who did not have more than mild diabetic retinopathy: 89.5%

IDENTIFIED RISK & MITIGATION MEASURE:

  • False positive results leading to additional unnecessary medical procedures (Diagnostic algorithm failure, Software failure): Clinical performance testing;
    Software verification, validation, and hazard analysis; Protocol for technical specification changes
  • False negative results leading to delay of further evaluation or treatment
    (Diagnostic algorithm failure, Software failure): Clinical performance testing
    Software verification, validation, and hazard analysis; Protocol for technical specification changes; Labeling
  • Operator failure to provide images that meet input quality specifications: Labeling,
    Training, Human factors validation testing

REGULATORY PATHWAY: De Novo request

  • Regulation No.: 21 CFR 886.1100
  • Regulation Name: Retinal diagnostic software device
  • Regulatory Class: Class II
  • Product Code: PIB

REIMBURSEMENT:

  • AI diagnostic approach could support CMS’ value-based reimbursement

ORDER


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Organ Care System (OCS) Lung System

TransMedics, Inc.

INDICATION FOR USE:  Portable organ perfusion, ventilation, and monitoring medical device indicated for the preservation of standard criteria donor lungs in a near physiologic, ventilated, and perfused state for double lung transplantation.

DESCRIPTION:

  • Lung Console: Non-sterile, reusable, portable enclosure housing electronic display and non-sterile mechanical/electrical elements to warm, pump, ventilate, and manage gas content of perfusate
  • Lung Perfusion Set (LPS): Sterile, single-use perfusion module, organ chamber and circulatory system to perfuse and ventilate lung,  facilitate management of fluids
  • OCS™ Lung Solution: High oncotic solution used for ex-vivo flush and perfusion of donor lungs when combined with packed red blood cells (pRBCs)

EFFECTIVENESS AND SAFETY:

  • Randomized, controlled, multi-center, international, prospective
    clinical trial, OCS™ Lung System vs. current cold storage standard of care (SOC), n=407
  • Primary Graft Dysfunction (PGD) grading,  including reduced survival and
    increased incidence of Bronchiolitis Obliterans Syndrome (BOS)
  • Patient survival at day 30 post-transplantation and ISHLT PGD3 within 72 hours post-transplantation
  • No-inferiority vs. SOC, longer-term (2-year) survival and BOS rates comparable
  • Similar lung graft-related serious adverse events (LGRSAEs) through 30 days post-transplantation

REGULATORY PATHWAY: PMA

  • Class III, Product Code: QBA
  • Priority Review
  • Gastroenterology-Urology Devices Panel Meeting: Voted 11-2 that there is reasonable assurance the device is safe, 8-5 that there is reasonable assurance that the device is effective, and 9-4 that the benefits of the device do outweigh the risks
  • Post-approval studies : Long-term patient outcomes, OCS Lung Thoracic Organ Perfusion (TOP) PAS Registry

LABEL 


Image credit: J&J, IDX, TransMedic

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Model Informed Drug Development

Capture.JPGModel-Informed Drug Development Pilot Program

Pilot Program to facilitate the development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources

  • Quantitative methods to balance risks/benefits of drugs in development
  • Can improve clinical trial efficiency, increase probability of regulatory success, optimize drug dosing/therapeutic individualization without dedicated trials

Goals of the MIDD Pilot Program

  • Discuss application of MIDD approaches to development and regulatory evaluation of medical products
  • Provide advice about how particular MIDD approaches can be used in a specific drug development program

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FDA News: Medical device needs for rare diseases, Accelerating next generation sequencing-based tests, Essure sale retsriction

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Unmet medical device Needs for patients with rare diseases

FDA, National Center for Advancing Translational Sciences (NCATS)/Office of Rare Diseases Research (ORDR) at NIH sought to better understand medical device needs of patients with rare disease

  • Generate meaningful data to inform patients, practitioners, policymakers, and device developers
  • Needs, barriers, and incentives

Online survey of four clinician groups

  • satisfaction with current devices
  • unmet needs for specific rare diseases
  • impediments to medical device development

Survey Respondents: 1,342 clinicians

Findings 

  • Patients with rare diseases face numerous unmet needs
  • Device needs of pediatric patients – grow with child, be modified to smaller size, less invasive
  • Creating entirely new devices needed vs. modifying/repurposing existing devices
  • Limitations included lack of sensitivity/specificity, cumbersome and invasive
  • Costs of research, lack of profitability, challenges of conducting trials are challenges

READ


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FDA finalizes guidances to accelerate the development of reliable, beneficial next generation sequencing-based tests

Finalized two guidances for  efficient development of novel technology that scans DNA to diagnose genetic diseases-next generation sequencing (NGS)

  •  Recommendations for designing, developing, and validating tests; for continued advancement of individualized, genetic-based medicine
  • Modern and flexible framework

Use of Public Human Genetic Variant Databases to Support Clinical Validity for Genetic and Genomic-Based In Vitro Diagnostics

  • Reliance on clinical evidence from FDA-recognized public databases to support clinical claims e.g. ClinGen

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

  • Recommendations for designing, developing, validating tests to diagnose individuals with suspected genetic diseases

Based on extensive feedback from the public and stakeholders; continuation of creating regulatory efficiencies in the development and review of NGS tests

READ 


CaptureFDA Restricts the Sale and Distribution of Essure

Order to restrict the sale and distribution of the Essure device

  • Ensure  all women provided with adequate risk information so that they can make informed decisions
  • Taking this step because some women were not being adequately informed of Essure’s risks before getting the device implanted
  •  Boxed Warning including perforation of the uterus and/or fallopian tubes, identification of inserts in the abdominal or pelvic cavity, persistent pain, and suspected allergic or hypersensitivity reactions
  • FDA closely evaluating new information on the use of Essure; requires additional, meaningful safeguards to ensure women are able to make informed decisions

New Essure labeling

  • Restricts sale and distribution to only health care providers and facilities that provide information to patients about the risks and benefits of this device
  • Review “Patient-Doctor Discussion Checklist – Acceptance of Risk and Informed Decision Acknowledgement”
  • Patient and physician required to sign
  • FDA will review and monitor Bayer’s plan to ensure compliance of restriction

READ


Image credit: FDA

Help FDA improve MedWatch System

CaptureHelp FDA Improve FDA MedWatch Forms

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


Image credit: FDA

2018 FDA Calendar

2018 FDA CALENDAR

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 New 


AUGUST

Aug 13-14: Public Meeting – Pediatric Medical Device Development. Identify strategies that enhance medical device ecosystem toward development and innovation of devices that serve the complex needs of children. INFORMATION


JUNE

Jun 19-20: FDA/IMSN Summit with international drug regulators to discuss medication safety issues. Discussion among international drug regulators on medication safety issues with regulated pharmaceutical products and how to minimize medication errors with labeling and packaging. INFORMATION

Jun 22: Clinical Outcome Assessments (COAs) in Cancer Clinical Trials. Discussion between academia, industry, international regulatory, HTA bodies, and patient groups to advance measurement of the patient experience in cancer clinical trials; characteristics of PRO measurement tools, standardize PRO data analysis, FDA approaches to PRO data review. INFORMATION

Jun 25-26: 2018 Center for Biologics Evaluation and Research Science Symposium. Discuss scientific topics related to the regulation of biologics and highlight science conducted at CBER by showcasing how scientific research informs regulatory decision making and to provide a forum for developing collaborations within FDA and with external organizations.


MAY

⊕ May 8: CBER Update: Office of Vaccine Research and Review Data Submission. Update on best practices INFORMATION

May 10: Endocrinologic and Metabolic Drugs Advisory Committee Meeting. Discuss NDA for volanesoren solution for subcutaneous injection, Akcea Therapeutics, Inc, for use as an adjunct to diet for the treatment of patients with familial chylomicronemia syndrome. INFORMATION

May 11: Medical Gas Workshop III. Provide opportunity for medical gas manufacturers and public to provide input on potential areas of Federal drug regulation that should be revised with respect to medical gases.  INFORMATION

May 15-16: FDA Regulatory Education for Industry (REdI). FDA-led forum that brings together the regulatory educators from FDA’s CDER and CDRH. Meeting Information

May 18: MDIC workshop on Patient-Centered Clinical Trial Design. Method for incorporating patient preference information to set significance levels in clinical trial design. Focus on Parkinson’s disease; may be generalizable to other diseases.  In collaboratorion with FDA (CDRH), MIT, RTI Health Solutions and Michael J. Fox Foundation. INFORMATION

May 22-23: Accreditation Scheme for Conformity Assessment (ASCA) of Medical Devices to Food and Drug Administration-Recognized Standards; Public Workshop.  Discuss draft design of pilot program, including goals and scope, framework, requirements, and streamline standards. INFORMATION

May 24: FY 2018 Generic Drug Research Public Workshop.  Provide overview of regulatory science initiatives for generic drugs and public input on research priorities INFORMATION


APRIL

Apr 3: Public workshop: CDER and You: Keys to Effective Engagement. Share information with stakeholders including patients, patient advocates, academic and medical researchers, health care professionals, drug developers. Share unique perspectives on drug development and safety. INFORMATION

Apr 6: US FDA and Health Canada: Joint Regional Consultation on the ICH. To provide information and receive comments on the current ICH activities as well as the upcoming meetings in Kobe, Japan INFORMATION

Apr 10: FDA Drug Topics: An Introduction to Drug Safety Surveillance and the FDA Adverse Event Reporting System. Introduce the many phases of drug safety surveillance from the earliest stages of drug development through post approval, and will focus on how FDA conducts pharmacovigilance, develops safety signals, and communicates our findings.INFORMATION

Apr 11-12: CDER Small Business and Industry Assistance Regulatory Education for Industry (REdI): Generic Drugs ForumInteract with FDA subject matter experts  on Generic Drug Review Program, program progress, current initiatives. INFORMATION

Apr 16: Evaluating Inclusion and Exclusion Criteria in Clinical Trials; Public Meeting. Discuss topics related to eligibility criteria in clinical trials and their potential impact on patient access to investigational drugs, and how to facilitate the enrollment of a diverse patient population. INFORMATION

⊕ Apr 16: Public Workshop: Study Design Considerations for Devices including Digital Health Technologies for Sleep Disordered Breathing (SDB). Appropriate design of clinical studies to evaluate devices including digital health technologies intended for the diagnosis, monitoring, or treatment of SDB INFORMATION

Apr 16-18: 2018 AAPS Workshop on Drug Transporters in ADME: From Bench to Bedside. Present next generation of transporters and transport mechanisms that may contribute to ADME properties of drugs in disease states considered in drug discovery and development. INFORMATION 

Apr 17: Public Meeting on Patient-Focused Drug Development for Opioid Use Disorder (OUD). To learn patients’ perspectives on OUD, effects on health and well-being, experience using prescription medical treatments and other treatments, challenges or barriers to accessing medical treatments for OUD. INFORMATION

Apr 19: Peripheral and Central Nervous System Drugs Advisory Committee Meeting Discuss NDA for cannabidiol oral solution, GW Pharmaceuticals, for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome in patients 2 years of age and older. INFORMATION

Apr 20: FDA/OCE Public Meeting on Relevant Molecular Targets in Pediatric Cancers: Applicability to Therapeutic Investigation FDARA 2017. Discuss provisional list of molecular targets for adult cancer indications but also relevant to cancer in children thereby providing a rationale for early pediatric evaluation. INFORMATION

Apr 21: Public Meeting on Electronic Submissions and Data Standards. Discuss current status of electronic submissions and data standards initiatives to improve predictability and consistency of electronic submissions process in support of human drug review program. INFORMATION

Apr 23: Arthritis Advisory Committee Meeting. Discuss NDA for baricitinib tablets, submitted by Eli Lilly and Company, for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate. INFORMATION

Apr 23-25: 12th Annual FDA/DIA Biostatistics Industry and Regulator Forum. Discuss relevant statistical issues associated with the development and review of therapeutic drugs and biologics. INFORMATION

Apr 30Public Workshop – Orthopaedic Sensing, Measuring, and Advanced Reporting Technology (SMART) Devices. Enhance engagement with stakeholders to facilitate device development and to discuss scientific and regulatory challenges associated with Orthopaedic SMART Devices.   INFORMATION


MARCH

Mar 1: Vaccines and Related Biological Products Advisory Committee Meeting. Hear  research program in the Laboratory of Mucosal Pathogens and Cellular Immunology (LMPCI), Division of Bacterial, Parasitic and Allergenic Products (DBPAP), Office of Vaccines Research and Review (OVRR), and discuss selection of strains to for vaccines for the 2018-2019 influenza season INFORMATION

Mar 1: 21st US-Japan Cellular and Gene Therapy Conference. Exchange ideas on cutting edge and diverse areas of biomedical research, and enhance opportunities for collaborations among scientists from the US and Japan.  INFORMATION

Mar 1: Neurological Devices Panel  Advisory Committee Meeting. Discuss safety and effectiveness of intracranial aneurysm treatment devices and factors affecting clinical outcomes such as aneurysm morphology, size, and location in the neurovasculature. INFORMATION

Mar 1-2: IASLC-FDA Lung Cancer Neoadjuvant Meeting.Discuss standardization and validation of endpoints in neoadjuvant lung cancer trials. INFORMATION

Mar 4-6: FDA-PhUSE Computational Science Symposium. Review progress on topics such as data standards, best-practices-driven analytical tool development, business processes for information systems, evaluation of current tools. INFORMATION

Mar 5: Risk Communication Advisory Committee Meeting. Committee will discuss  impact of pregnancy and lactation labeling information in prescription drug and biological products as modified under the Pregnancy and Lactation Labeling Rule.  INFORMATION

Mar 8: FDA Grand Rounds:  Stem cell-based cellular therapies. Use of stem cell-based products is new and characterizing the product still faces hurdles.  FDA conducting research into identifying cell therapy product characteristics that will predict the reliably of the performance of cell-based therapies in humans. INFORMATION

 Mar 8: Gastrointestinal Drugs Advisory Committee Meeting. Discuss sNDA for XELJANZ (tofacitinib) proposed for the treatment of adult patients with moderately to severely active ulcerative colitis. INFORMATION

Mar 8: Public Workshop: Safety Assessment for Investigational New Drug Safety Reporting. Engage external stakeholders in discussions related to finalizing the draft guidance entitled “Safety Assessment for IND Safety Reporting.” INFORMATION

Mar 15: Oncology Center of Excellence Listening Session; Solicit comments regarding what stakeholders desire of the OCE in terms of structure, function, regulatory purview, and activity. INFORMATION

Mar 20: Promoting the Use of Complex Innovative Designs (CID) in Clinical Trials. March 20, 2018. Discuss use of CID in drug development and regulatory decision making, CID pilot program. INFORMATION

Mar 21-22: Joint Meeting of the Blood Products Advisory Committee and the Microbiology Devices Panel. Discuss reclassification from Class III to Class II of nucleic acid and serology-based point-of-care and laboratory-based in vitro diagnostic devices indicated for use as aids in the diagnosis of human immunodeficiency virus (HIV) and hepatitis C virus (HCV) infection.⊕ INFORMATION

Mar 22: Webinar – Duodenoscope Sampling and Culturing. FDA, CDC, ASM and other endoscope culturing experts will review the voluntary duodenoscope surveillance sampling and culturing protocols to monitor quality of reprocessing procedures.  INFORMATION

Mar 22:  Patient Engagement in the National Evaluation System for health Technology (NEST): Lessons Learned and Best Practices Workshop.  Gather lessons learned and best practices for patient engagement in evidence generation (planning, collection of data and information, analysis, and dissemination). INFORMATION

Mar 22: Pediatric Advisory Committee and Endocrinologic and Metabolic Drugs Advisory Committee Meeting. To discuss major objectives of Phase 3 drug development program for treatment of children with achondroplasia (ACH) submitted by BioMarin Pharmaceutical Inc. INFORMATION

Mar 23: Advisory Committee Meeting: Pediatric. Discuss the following products for CDER – BANZEL, INTUNIV, LEXAPRO and CDRH – FLOURISH, ACTIVA, LIPOSORBER, IMPELLA RP SYSTEM. INFORMATION

Mar 26-28: Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics for Alcoholic Hepatitis and Alcohol Associated Liver Disease and Pediatric Irritable Bowel Syndrome and Pediatric Functional Constipation Workshop, Facilitate dialogue among industry, academia, and other stakeholders on common data elements needed to be included in clinical trials, clinical trial designs, potential surrogate and clinical benefit endpoints, and practical issues with managing clinical trials.  INFORMATION

Mar 27: Meeting of the Psychopharmacologic Drugs Advisory Committee. Discuss  NDA for  lofexidine hydrochloride, US WorldMeds, LLC, for mitigation of symptoms associated with opioid withdrawal and facilitation of completion of opioid discontinuation treatment. INFORMATION

Mar 28: Promoting the Use of Complex Innovative Designs in Clinical Trials
Inform development of guidance document and CID pilot program. INFORMATION


FEBRUARY

Feb 1: FDA-ISoP Public Workshop: Model Informed Drug Development (MIDD) for Oncology Products. Discuss integrating human pharmacokinetic, pharmacodynamic, efficacy, safety data into models, use of  novel imaging techniques, diagnostic, predictive biomarkers, shift from traditional endpoints,  regulatory implications.  INFORMATION

Feb 7-8: 10th Annual Sentinel Initiative Public Workshop.  Bring stakeholder community together to discuss a variety of topics on active medical product surveillance. INFORMATION

Feb 14-15:  Joint Drugs Advisory Committee Meeting: Anesthetic and Analgesic Products and Drug Safety and Risk Management. Discuss NDA for HYDEXOR, for the short-term management of acute pain severe enough to require an opioid analgesic while preventing and reducing opioid-induced nausea and vomiting. Also discuss sNDA  for EXPAREL (bupivacaine liposomal injectable suspension) to produce local analgesia and as a nerve block to produce regional analgesia. INFORMATION

Feb 22-23: FDA-AACR-ASTRO Regulatory Science and Policy Workshop – Clinical Development of Drug-radiotherapy Combinations. Address the lack of drug development for products intended specifically for use with radiation therapy.   INFORMATION

Feb 27:  Webinar – Custom Device Annual Reporting. Custom Device Exemption allows manufacturers to market medical devices designed to treat a unique pathology or physiological condition without premarket approval. Webinar to discuss custom device annual report requirement. INFORMATION

Feb 28: Public Meeting: Enhanced Drug Distribution Security under the Drug Supply Chain Security Act.Provide members of the drug distribution supply chain and other interested stakeholders an opportunity to discuss strategies and issues related to the enhanced drug distribution security provisions of the Act. INFORMATION


JANUARY

Jan 8: CLIA Waiver Applications Draft Guidance Documents. Discuss draft guidances on CLIA waiver applications and Dual 510(k) and CLIA waivers INFORMATION

Jan 9:  Webinar – Pediatric Information for X-ray Imaging Device Premarket Notifications:  Discuss final guidance on radiation safety of pediatric populations in the design of X-ray imaging devices. INFORMATION

Jan 10:  Webinar – Technical Considerations for Additive Manufactured Medical Devices. Technical aspects associated with AM processes, recommendations for device design, manufacturing, testing considerations. INFORMATION

Jan 11: Public Workshop – Self-Collection Devices for Pap Test. Obtain feedback on feasibility, benefits, risks for self-collection cervical sampling devices for cervical cancer screening by Pap testing INFORMATION

Jan 11:  Safety Assessment for IND Safety Reporting.Convened by the Duke-Robert J. Margolis Center for Health Policy at Duke University and FDA; to bring stakeholder community together to discuss IND safety topics INFORMATION

Jan 11: FDA Grand Rounds. Marker of brain injury increased in African Americans with Alzheimer’sBetter understanding of ethnicity and gender differences involved in the cause and progression of Alzheimer’s Disease could contribute to better drugs–and other types of interventions–to slow Alzheimer’s progression INFORMATION

Jan 16:  Webinar – FDA Categorization of Investigational Device Exemption (IDE) Devices to Assist the Centers for Medicare and Medicaid Services (CMS) with Coverage Decisions. Discuss final guidance on FDA categorization of IDE devices that is used by CMS to determine whether an IDE device, and certain related services, may be covered by CMS. INFORMATION

Jan 26: Evaluating Nicotine Replacement Therapies. Public comments on FDA’s approach to evaluating the safety and efficacy of nicotine replacement therapy (NRT) products, including how they should be used and labeled. INFORMATION

Jan 29: Weighing the Evidence: Variant Classification and Interpretation in Precision Oncology. To discuss how genetic sequencing data is best implemented in patient management to advance innovative regulatory strategies to support development of safe and effective precision-based drugs and devices. INFORMATION

Jan 30: Opioid Policy Steering CommitteePrescribing Information  Receive stakeholder input on how FDA REMS authority, might improve the safe use of opioid analgesics by curbing over-prescribing to decrease the occurrence of new addictions and limit misuse and abuse INFORMATION

Jan 30-31: Fostering Digital Health Innovation. Developing the Software Precertification Program. Discuss progress of pilot precertification program and seek input on ongoing development of the Software Precertification Program. INFORMATION


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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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Market Authorizations: DEXCOM iCGM, BLINCYTO, Lifeline/ReviveR Defibrillator

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Dexcom G6 Integrated Continuous Glucose Monitoring System (iCGM)

Dexcom, Inc.

INDICATION FOR USE: Real time, continuous glucose monitoring device indicated for the management of diabetes in persons age 2 years and older.

The Dexcom G6 System is intended to replace fingerstick blood glucose testing for diabetes treatment decisions. Interpretation of the Dexcom G6 System results should be based on the glucose trends and several sequential readings over time. The Dexcom G6 System also aids in the detection of episodes of hyperglycemia and hypoglycemia, facilitating both acute and long-term therapy adjustments.

The Dexcom G6 System is also intended to autonomously communicate with digitally connected devices, including automated insulin dosing (AID) systems. The Dexcom G6 System can be used alone or in conjunction with these digitally connected medical devices for the purpose of managing diabetes

ADDRESSING UNMET NEED: 

  • First type of CGM to be used as part of an integrated system with other compatible medical devices and electronic interfaces, which may include automated insulin dosing systems, insulin pumps, blood glucose meters or other electronic devices used for diabetes management
  • Enables developers of future iCGM systems to bring their products to market in the least burdensome manner possible.

DESCRIPTION:

  • Patch device applied to the skin of the abdomen and contains a small sensor that continuously measures the amount of glucose in body fluid
  • Device transmits real-time glucose readings every five minutes to a compatible display device such as a mobile medical app on a cell phone
  • Will trigger an alarm when a patient’s blood sugar enters a danger zone soaring too high or dropping too low
  • If integrated with an automated insulin dosing system, a rise in blood sugar would trigger the release of insulin from the pump
  • Patch device should be replaced every 10 days

GENERIC DEVICE TYPE: Integrated continuous glucose monitoring system.
Intended to automatically measure glucose in bodily fluids continuously or frequently for a specified period of time. iCGM systems are designed to reliably and securely transmit glucose measurement data to digitally connected devices, including automated insulin dosing systems, and are intended to be used alone or in conjunction with these digitally connected medical devices for the purpose of managing a disease or condition related to glycemic control.

EFFECTIVENESS AND SAFETY: 

  • Two clinical studies, n=324 adults and children aged 2 years and older with diabetes, multiple clinical visits within a 10-day period where system readings were compared to a laboratory test method that measures blood glucose values. No serious adverse events were reported during the studies.
  • Risks may include hypoglycemia (low blood sugar) or hyperglycemia (high blood sugar),  skin irritation or redness around the device’s adhesive patch

IDENTIFIED RISKS AND MITIGATIONS: 

  • Clinical action based on falsely high or falsely low inaccurate glucose values or inaccurate alerts may lead to inappropriate treatment decisions: General and Special Controls
  • Clinical action in pediatric patients based on falsely high or falsely low inaccurate values or inaccurate alerts due to poorer or different performance in pediatric populations. General and Special Controls
  • The inability to make appropriate treatment decisions when glucose values are unavailable due to sensor signal drop-out or loss of communication with digitally connected devices. General and Special Controls
  • Patient harm due to insecure transmission of data. General and Special Controls
  • Use of an iCGM as part of another digitally connected medical device system, such as an AID system, when the iCGM has inadequate analytical or clinical performance to support the intended use of the digitally connected device. General and Special Controls

REGULATORY PATHWAY: De Novo request

  • An earlier generation Dexcom G5 system, received FDA approval in 2016 but was not designed as an integrated system to be used with compatible devices
  • Regulation Number: 21 CFR 862.1355
  • Classification: III
  • Product Code: QBJ

REIMBURSEMENT: 

  • Earlier generation Dexcom G5 system, covered by Medicare

ORDER


Capture.JPGBLINCYTO (blinatumomab) for injection, for intravenous use

Amgen Inc.

SUPPLEMENTAL INDICATION: Treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1% in adults and children.

ADDRESSING UNMET NEED:

  • First FDA-approved treatment for patients with MRD-positive ALL
  • Approximately 5,960 people in US will be diagnosed with ALL this year and approximately 1,470 will die from the disease

EFFICACY:

  • Single-arm clinical trial, n=86, in first or second complete remission who had detectable MRD in at least 1 out of 1,000 cells
  • Efficacy: Achievement of undetectable MRD in assay that could detect at least one cancer cell in 10,000 cells after one cycle of Blincyto treatment, length of time that the patients remained alive and in remission (hematological relapse-free survival)
  • MRD was achieved by 70 patients, >50% patients remained alive and in remission for at least 22.3 months

SAFETY:

  • Adverse Effects: Consistent with other uses. Infections, pyrexia, headache, infusion related reactions, neutropenia, anemia, febrile neutropenia,  thrombocytopenia
  • Boxed warning:  Cytokine release syndrome, Encephalopathy
  • Serious risks: Effects on the ability to drive and use machines,pancreatitis, and preparation and administration errors 

REGULATORY PATHWAY: sBLA

  • First approval in 2014  for treatment of Philadelphia chromosome (Ph)-negative relapsed or refractory positive B-cell precursor ALL
  • This application granted  Priority Review, Orphan Drug Designation and Accelerated Approval
    • Based on MRD response rate and hematological relapse-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
  • Accelerated Approval requirements:  Randomized trial to verify clinical benefit in adult and pediatric patients,
  • Modified REMS

REIMBURSEMENT:

  •  Eligible for inclusion in the Durable Medical Equipment (DME) External Infusion Pump Local Coverage Determination (LCD).

LABEL 


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Lifeline/ReviveR ECG and DDU Automated Defibrillators

Defibtech, LLC

INDICATION FOR USE:  For use on victims of sudden cardiac arrest (SCA) who are:1) Unconscious and unresponsive; and 2) Not breathing or not breathing normally.

DESCRIPTION: 

  • 2 multifunction defibrillation electrodes, placed on the patient’s chest, to acquire a patient’s electrocardiogram (ECG)
  • If abnormal heartbeat detected, it may advise the user that a high-energy shock is necessary
  • User interface will provide text/icon prompts and voice instructions to guide the user through the rescue process including cardiopulmonary resuscitation (CPR)
  • Will provide an audible rhythmic beeping sound to help the user deliver the correct rate of compressions while giving CPR
  • If defibrillation shock is required, the device will prompt user to deliver an electrical shock, through the electrodes.

REGULATORY PATHWAY: PMA

  • Product Code: MKJ
  • Classification: III
  • Regulation No. : 870.5310
  • Description: Non-wearable prescription use only AED

PMA Order


Image credits: Dexcom, Amgen, Defibtech

FDA News: Blood Supply Model, Nanotechnology Programs, Patient Perspectives in Drug Development

Blood Supply and Demand Simulation Model Could Help Nation Prepare for Emergencies

FDA developed blood supply model to estimate amount of blood available in system during national emergencies

  • Estimates how much blood is available during pandemic influenza outbreak and  mass casualty event caused by nuclear device detonation
  • Could threaten blood supply by reducing number of people to donate or by increasing the amount of blood needed
  • Allows calculation of supply—and potential for shortages—based on how long RBCs (short half life) have been in storage

Based on real-world sources of data

  • National blood supply produced by America’s Blood Centers
  • Center for Medicare and Medicaid Center for Medicare and Medicaid Services database
  • National Blood Collection and Utilization Survey
  • Centers for Disease Control and Prevention

Ran simulation on how pandemic could affect inter-regional blood supplies

  • Estimated 541,000 RBC units were lost overall; South had highest percentage of blood lost (15.5%), while the East had the lowest lost (13.8%)

Based on current levels of blood collection, use, and other factors, the U.S. blood supply and demand system is flexible and reliable enough to respond to emergencies

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Nanotechnology Programs at FDA

Build regulatory science knowledge, facilitate collaborations and partnerships to establish a flexible, product-focused, science-based approach to regulation

  • Nanotechnology Task Force
  • Nanotechnology Task Force Report
  • Nanotechnology Guidance Documents
  • Nanotechnology Partnerships at FDA

National and International Activities 

  • Member agency in the National Nanotechnology Initiative
  • World-class R&D program, transfer of new technologies,  educational resources,  infrastructure and tools to advance and responsible development of nanotechnology
  • International Organization for Standardization (ISO)
  • Organisation for Economic Co-operation and Development (OECD)

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FDA’s efforts to enhance the patient perspective and experience in drug development and review

Patients are becoming driving force of medical research enterprise

  • Capturing patient experience may be quantitative or qualitative
  • Transforming nearly every aspect of medical product development
  • Informing about benefits that matter most and risks they are most concerned

Benefit-Risk Assessment in Drug Regulatory Decision-Making

  • Enhance benefit-risk assessment in human drug review and FDA’s decision-making process
  • Outline how patient experience data and related information can be used to inform benefit-risk assessment

Incorporating Patient Voice into Benefit-Risk Assessments

  • Inform clinical context and provide insights to frame assessment of benefits and risk
  • Provide a direct source of evidence regarding the benefits and risks based on  methodologically-sound data collection tools
  • Hosting patient-focused drug development public meetings
  • Encouraging patient stakeholders to conduct externally-led, patient-focused drug development meetings
  • Providing patients, caregivers, advocates  more channels to provide meaningful input
  • Launching pilot programs to foster design of clinical trials that place less burden on patients

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Image credits: FDA