June 2019 – Drug and Device Digest

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)

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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

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New draft guidance, “Opioid Analgesic Drugs: Considerations for Benefit-Risk Assessment Framework,”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

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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

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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

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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

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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

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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.

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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)

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Image credit: FDA

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