News & Views: Medical device reporting, Cannabidiol policy, Benefit-Risk framework for opioid analgesics, CDER modernization, New drug approval transparency, Heart Failure treatment, Complex Innovative Trial Design Program, Safety with paclitaxel-coated stents, Prostate tissue ablation devices

Capture.JPGCDRH efforts to increase transparency in medical device reporting 

CDRH is updating Medical Device Reporting (MDR) Program

  • End Alternative Summary Reporting (ASR) Program: Granted . exemption from individual medical device reports for well-known and well-established risks
  • Implement Voluntary Malfunction Summary Reporting (VMSR) Program   to efficiently detect potential safety signals
  • Modernize Manufacturer and User Facility Device (MAUDE) database
  • Actively engage in the National Evaluation System for health Technology (NEST)


Capture.JPG Sound, Science-based Policy on Cannabidiol

Apply  rigorous, science-based approach to products containing cannabis or cannabis-derived compounds, including cannabidiol (CBD)

  • Many unanswered questions about the science, safety, and quality of many of these products
  • If product being marketed to have a therapeutic effect – regulated as a drug subject to FDA review and authorization
  • If marketed as food, including dietary supplements- overarching goal of protecting consumers and  currently illegal

FDA Public Meeting on May 31st for stakeholders to share feedback and experiences. Remaining open questions:

  • Daily safe dose of CBD and variability depending on form
  • Drug interactions
  • Impacts to special populations – children, elderly, pregnant, lactating women
  • Long-term exposure risks


Capture.JPGBenefit-Risk framework for evaluating opioid analgesics

New draft guidance, “Opioid Analgesic Drugs: Considerations for Benefit-Risk Assessment Framework,”

  • Benefit-risk assessment framework to address opioid crisis with focus on  formal assessment of associated public health risks
  • Assess benefits and risks of proposed new opioid analgesics relative to already approved opioid and non-opioid analgesics
  • Provide information on mitigation of risks of overdose, abuse or addiction
  • Provide public health implications of product in terms of risks to non-patients, including members of the patient’s household, visiting relatives, friends, others
  • New REMS is safety profile different from already approved products


Capture.JPGModernizing FDA’s New Drugs Regulatory Program

Initiative to modernize the New Drugs Regulatory Program to better serve patients and  staff in their work to carry out center’s mission

  • Scientific Leadership: Grow scientific expertise;  clarify regulatory pathways 
  • Integrated Assessment: Whether applications meet legal & regulatory requirements
  • Benefit-Risk Monitoring: Monitor pre- and post- approval 
  • Managing Talent: Attract, develop, and retain outstanding people
  • Operational Excellence: Standardize workflow, business processes, roles, responsibilities
  • Knowledge Management: Facilitate identification, capture, distribution, effective use of information


Capture.JPGNew drug approval transparency efforts

Efforts to enhance transparency around our drug approval decisions

  • Focus on information to improve patient care and better inform health care professionals about the product
  • Posting portions of clinical study reports after a drug receives FDA approval
  • Requesting public comment about programs


Capture.JPGTreatment for hear failure: Endpoints for drug development

Guidance pertains primarily to treating both heart failure with reduced ejection fraction (HFrEF) and heart failure with preserved ejection fraction (HFpEF)

  • Mortality data: Primary efficacy and safety
  • Endpoints related to how patients feel and function: Evidence of effectiveness
  • Hospitalization and outpatient intervention: All-cause vs. cause-specific
  • Biomarkers and surrogate endpoints: For clinical benefit
  • Acute heart failure: Symptom relief
  • Heart failure with preserved ejection fraction
  • Pediatric population



Circulatory System Devices Panel recommendations on late mortality safety signal associated with paclitaxel-coated products

Panel met to discuss safety vs benefit of paclitaxel-coated balloons and paclitaxel-eluting stents used to treat peripheral arterial disease in the femoropopliteal arteries

  • Unanimously agreed that the aggregate data showed a mortality signal
  • Could not conclude that the late mortality signal represented a class effect
  • Missing data (vital status, repeat interventional treatments, paclitaxel exposure)  hindered interpretation of analyses
  • Could not identify patient subgroups that might be at particularly high (or low) mortality risk
  • Observed rates of both cardiovascular and non-cardiovascular death were higher in patients treated with paclitaxel-coated devices vs. uncoated devices
  • Precluded determination of relationship between paclitaxel dose and mortality
  • Preclinical animal data do not indicate a potential biological mechanism for the late mortality signal
  • Short-term benefits of paclitaxel-coated devices continue to outweigh the risks, and  devices should not be removed from market; Additional comments on post-market surveillance and labeling


Capture.JPGComplex Innovative Trial Design (CID) Program

The CID Pilot Program is designed to:

  • Facilitate the use of CID approaches in late-stage drug development
  • Use of complex adaptive, Bayesian, and other novel clinical trial designs
  • Promote innovation by allowing FDA to publicly discuss the trial designs considered through the pilot program, including trial designs for medical products that have not yet been approved by FDA.


Capture.JPGClinical investigations for prostate tissue ablation devices

Draft guidance to provide recommendations for high intensity ultrasound systems for prostate tissue ablation:

  • Complying with clinical testing special control under 21 CFR 876.4340(b)(8) for 510(k)s)
  • Collecting clinical data to support marketing submissions for new types of prostatic tissue ablation devices

Marketing authorization for a general indication for ablation of prostatic tissue

  • High intensity therapeutic ultrasound energy into prostate to thermally ablate  defined, targeted volume of tissue
  • Achieve same clinical effect of ablating targeted tissue volumes using different sources of energy
  • Does NOT address intended uses for the treatment of a specific disease (e.g., prostate cancer or benign prostatic hyperplasia)


Image credit: FDA

FDA’s New History Exhibit


Forward Into the Past With FDA’s New History Exhibit

First permanent exhibit documenting the FDA’s history at the White Oak headquarters in Silver Spring, MD.

  • Includes many of the noteworthy achievements in the FDA’s long history
  • FDA continues to understand and learn from its history, to inform its work
  • Experience the new exhibit to gain better understanding of how FDA has evolved over the years and how its history continues to pave the way for the future



Image credit: FDA on Flickr




Webpage: Patient-Reported Outcomes (PROs) in Medical Device Decision Making


Patient-Reported Outcomes (PROs) in Medical Device Decision Making

This web page focuses on the use of patient-reported outcomes (PRO) as one type of patient input.

  • What Are Patient-Reported Outcomes
  • How CDRH Uses PROs in Regulatory Decision Making
  • CDRH Collaborations, Studies, Articles, and Workshops on Patient-Reported Outcomes
  • How to Incorporate Patient-Reported Outcome Measures (PROMs) in a Regulatory Submission


Image credit: FDA

News & Views: Measles vaccine, Interchangeable biosimilars, Warning for vaping social influencers, Project Facilitate for expanded access, Enhancing diversity of clinical trials, Fixed-quantity packaging for opioids, Safety throughout drug’s lifecycle, Participation in Pre-Cert program, Better prescription drug advertising, Global drug quality

CaptureVaccination Is the Best Protection Against Measles

Measles, Mumps, and Rubella Virus Vaccine Live (M-M-R II, commonly called MMR)

  • For people ages 12 months and older
  • Two doses of the vaccine is about 97% effective at preventing measles

Measles, Mumps, Rubella and Varicella Virus Vaccine Live (ProQuad, commonly called MMRV)

  • For children ages 1 through 12 years.
  • Also prevents chickenpox


CapturePolicy advancements to help bring interchangeable biosimilars to market

Pathway for “interchangeable” biologics

  • Substituted without the involvement of the prescriber
  • Similar to how generic drugs are routinely substituted for brand name drugs

 Final guidance on the pathway for “interchangeable” biologics

  • Overview of important scientific considerations in demonstrating interchangeability
  • Biological product is biosimilar to reference product and produces same clinical result
  • Risk of safety or diminished efficacy of alternating or switching will not be greater than the risk of using the reference product without such alternation or switch


Capture.JPGActions for violations by online posts on flavored e-liquids by social media influencers

FDA and FTC  warning letters to four firms that manufacture, sell, advertise flavored e-liquid products for social media influencers

  • Violations related to online posts by social media influencers on each company’s behalf, including failure to include the required nicotine warning statement
  • Enforcement action to address increased popularity of e-cigarettes among youth leading to health concerns, nicotine addiction, and more likely to transition to conventional cigarettes


Capture.JPGProject Facilitate to assist physicians seeking access to unapproved therapies for patients with cancer

New pilot program to assist oncology health care professionals in requesting access to unapproved therapies for patients with cancer

  • New call center called Project Facilitate as a single point of contact
  • Help submit Expanded Access request for an individual patient, including follow-up of patient outcomes
  • Expanded Access is pathway for treatment by investigational treatment outside of clinical trials for immediately life-threatening or serious disease or condition
  • Expanded Access requests help weigh whether potential benefit of investigational treatment justifies the potential risks



Draft Guidance for Industry on Enhancing the Diversity of Clinical Trial Populations

  • More inclusive trial practices, trial designs, and methodological approaches to broaden eligibility criteria
  • Increase enrollment of more diverse populations in clinical trials
  • Maximizes generalizability of trial results
  • Better understand therapy’s benefit-risk profile across the patient population likely to use the drug in clinical practice, without jeopardizing patient safety
  • Reflects FDA policy encouraging inclusion in clinical trials of participants representative of the broad population of patients including minorities


CaptureFixed-quantity blister packaging for certain opioid pain medicines to help decrease unnecessary exposure to opioids

Misuse of prescription opioids by getting them from a friend or family member


  • >191 million opioid prescriptions dispensed to ~ 60 million patients, either as first-time prescriptions or refills; ~ 90% patients reported not finishing what was prescribed to them

Special opioid packaging for reducing unused doses available for misuse, abuse, inappropriate access, accidental poisoning or overdose- considertions

  • Actual opioid use compared to prescribed amounts for common surgical procedures and acute pain conditions

  • 5-, 10-, and 15-Count Blister Packages of Certain IR Opioid and IR Opioid/Acetaminophen Products

  • New Packaging/Disposal REMS Element

  • Safety-Enhancing Benefits of Fixed-Quantity Blister Packaging for Opioid Analgesics for Treatment of Acute Pain

  • National Academies of Sciences, Engineering, and Medicine (NASEM) to help advance guideline development

Remove the Risk” campaign


Capture.JPGTracking and Acting on Safety Data Throughout a Drug’s Lifecycle

Agency tracks, analyzes and acts on the adverse event data collected over time to discover and communicate new risks associated with approved drugs 

  • Reports from patients, health care professionals and manufacturers about adverse events in  FDA Adverse Event Reporting System (FAERS)
  • Monitor FAERS database to identify safety signals, analyze and interpret data, combine with other scientific information for decision-making
  • If new risk confirmed, require addition to product’s label and may require a Risk Evaluation Mitigation Strategy (REMS) to ensure benefits outweigh risks
  • From 2002 through 2014, new safety issues discovered and added to labels for 278 drugs


CaptureParticipation in 2019 Test Plan for Digital Health Software Precertification (Pre-Cert) Program

Agency seeking test cases from software organizations planning to submit a De Novo Request or 510(k) submission for software as a medical device (SaMD)  to meet the goals of the Test Plan

  • Small and large software development firms
  • Companies that develop a range of products, including both low- and high-risk SaMD
  • Companies that are not considered to be traditional medical device manufacturers but who intend to make SaMD


Capture.JPGHelping to Better Communicate the Risks and Benefits in Prescription Drug Advertising

Direct-to-consumer advertising influence awareness of prescription drug treatments, influence patients and doctors

  • Using eye-tracking technology, researchers are examining elements of these ads that can affect the viewer’s ability to understand risks and benefits
  • With high-distraction ad – less able to recall or recognize risks described, spent less time on written statement of risks
  • Objective to help drug companies develop ads that better inform public


CaptureAssuring Drug Quality Around the Globe

Programs to address the challenges to drug quality posed by globalization to protect patients and consumers

  • Tools to assure continued safety, effectiveness, quality post-approval
  • Same quality for both domestic and foreign manufactured products
  • Addressing quality inspections and issues


Image credit: F DA