FDA News and Views: Puerto Rico, Patient Representative Program, Patient Engagement, Opioid Abuse Deterrent Formulations – Drug and Device Digest

FDA BRIEF: Week of October 9, 2017

Hurricanes Irma and Jose

FDA’s continued assistance following the natural disaster in Puerto Rico

Pharmaceutical products and medical devices manufactured in Puerto Rico contribute substantially to medical products consumed by Americans

  • Facility closings and potential for shortages

FDA maintaining close watch on the most critical medical products

  • List of products for which shortage could have substantial impact on public health
  • Daily communication with the companies to stay on top of evolving challenges
  • Act quickly to prevent drug and device shortages
  • Help firms secure fuel to maintain production lines
  • Get clearance to move logistical support


FDA Patient Representative Program

Patient Representative Program is managed by the Office of Health and Constituent Affairs within Office of the Commissioner

  • Coordinates recruitment, training, and retention for over 200 FDA Patient Representatives, who are patients or primary caregivers to patients

Recruit Patient Representatives on an as-needed basis to 

  • Help advise us drugs, devices, and biologics being considered for approval
  • Early input in regulatory medical product development and review process

Looking for Patients or caregivers with advocacy experience who have been diagnosed or experience with

  • gastric cancer, hepatocellular carcinoma, retinitis pigmentosa, type II diabetes,  renal cell carcinoma,  bladder cancer,  male hypogonadism,  ALS (amyotrophic lateral sclerosis),  phenylketonuria, sarcoma, cochlear implants,  female hyposexual desire disorder,  retinal implants, naloxone use,  childhood cerebral adrenoleukodystrophy, neuroendocrine tumors,  glaucoma, metastatic melanoma,   merkel cell carcinoma,  keratoconus



Statement by FDA Commissioner Scott Gottlieb, M.D. on new steps by FDA to advance patient engagement in the agency’s regulatory work

First meeting of Patient Engagement Advisory Committee, PEAC, held on October 11th

  • Comprised solely of patients, care-partners, and those who represent their needs
  • Founded by CDRH to keep patients at the center of their work

Encourage inclusion of patient perspectives across the total medical device life cycle

  • Across device design and ideation, clinical trial process, postmarket evaluation
  • Inclusion in PMAs, HDE applications, de novo requests, and device labeling
  • Patient risk tolerance and benefit perspectives part of benefit-risk assessment

Integrate real world evidence into regulatory decision making process

  • Leveraging information in electronic health records, insurance claims databases, registries
  • Interactions with these patient-led registries
  • Use of smartphone platforms for collection of  real-world health information

Parameters for rigorous, systematically gathered patient preference information

  • Coordination of certain agency-wide and multi-center projects related to patient engagement
  • Facilitate cross-center policy making
  •  Facilitate development and use of patient-focused methods in regulatory activities
  • Develop and elevate common standards for how to integrate patient voice


CaptureMeasuring the Impact of Opioid Analgesic Formulations with Properties Designed to Deter Abuse in the Real World

Opioid formulation with “properties designed to deter abuse”

  • specifically formulated to make manipulation for abuse by specific routes, such as intranasal (snorting) or injection more difficult or less rewarding

Testing and approval of abuse deterrent formulations (ADF)

  • Category 1-3 studies to compare new product to an already approved product
  • Labeling based on these data

Challenges associated with ADF evaluation

  • Post-marketing requirements to evaluate whether product actually decreases abuse and related adverse outcomes in the real world
  • Challenges because existing data sources have substantial limitations

July 2017 workshop

  • Best practices in evaluating the real-world effectiveness of ADFs
  • Need for innovative and creative collaborations to address opioid crisis


Image credits: FDA


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