PFDD

Opening Remarks

Pamela Horn, MD Medical Officer Team Lead, DAAAP, CDER, FDA

  • Meeting addresses an unmet need for treatment that is finding patient-centered endpoints for clinical trials.
  • Hearing from patients will help steer drug development
  • FDA works to ensure safety, effectiveness, and quality of drugs

Overview of FDA’s Patient-Focused Drug Development Initiative

Sara Eggers, PhD OSP, CDER, FDA

  • Goal is to make more systematic way to gether patients inputs and perspectives
  • Originated from PDUFA V
  • This meeting is the 19th of 24 PFDD meetings for FY 2013-2017
  • Active patient involvement and participation encouraged

Background on Pain Neuropathies and Available Treatments

Steven Galati, MD, Medical Officer, DAAAP, CDER, FDA

  • Chronic Pain can be nociceptive (injury/inflammation) or neuropathic (pain without a stimulus)
  • Many different causes of neuropathy including diabetes and medical treatment
  • For the FDA to approve a neuropathy drug, there must be at least two successful trials
  • The FDA wants to know what symptoms bother patients the most so companies can target them in trials
  • Some challenges to drug development are that patients are still not satisfied with the drug and that one medication cannot cover multiple types of neuropathy

The Road from PFDD Meetings to Clinical Trial Endpoints

Nikunj Patel, PharmD Clinical Outcomes Assessments Staff, CDER, FDA

  • Patient recorded outcomes (such as answers to a questionnaire) will help create clinical trial endpoints
  • Stakeholders can listen to patients’ perspectives
  • PFDD meetings ensure that PRO’s accurately describe efficacy and drug safety
  • FDA encourages audience to engage others outside of PFDD meetings for more input
  • 3 pathways to generate outcome assessments
    • Input during investigational new drug submissions
    • Clinical outcome assessments (COA’s) to prove a treatment’s benefit during trials
    • Critical Path Innovation Meetings to advance drug efficacy using input from industry, academia, and patient-related groups

Panel #1 Discussion on Topic 1: Disease Symptoms and Daily Impacts

Adam Halper

    • Symptoms including soreness/muscle heaviness vary in intensity based on how much activity he does
    • Other symptoms including burning sensations are not “activity-dependent”
    • Limited mobility and cannot stand for more than 10-30 minutes
    • On worst days, can walk for only 5 minutes and cannot sleep comfortably
    • On best days, can walk about 3 miles
    • Intensity of symptoms fluctuates (no clear progression)

Susan Waldrop

    • Condition resulted from chemotherapy for colon cancer
    • Experiences burning sensations in limbs, electric shocks, and cannot walk, unpleasant numbness, increased sensitivity to cold
    • She cannot feel feet, causing problems with balance
    • Good days of normal activity are followed by bad nights

David Morrow (VP, Neuropathy Support Network)

    • Also resulted from chemotherapy for colon cancer
    • Slow progression
    • Experiences some foot and hand numbness, stabbing pains in feet, balance problems
    • Symptoms became less noticeable over time

Beth Lannon

    • Began as bilateral pain and slowly progressed
    • Sharp, constant pain (“like a hammer to my feet”), electrical shocks
    • Symptoms worsen with activity
      • Cannot sit for more than 15 minutes

Panel #2 Discussion on Topic #2: Current Approaches to Treatment

Linda Spinella

    • Pain because of multiple herniated disks
    • Was prescribed Lyrica, OTC pain relievers (incl. Tylenol), Prednisolone, opiods
    • Also went to the chiropractor and received epidurals for short-term relief
    • Cannot sleep without medication
    • Usually anxious, jittery, agitated

Cherie Pagett

    • Experiences numbness up to her knees
    • Takes Neurontin, Miradol, Lidocaine, opioids
    • When she took Lyrica, she gained 25 pounds in six weeks
    • Does not feel that her pain is under control because she will have intense flashes of pain

Louis Schmitt

    • 20 months of high dosage of IVIG to repair damaged nerves
    • Believes meditation managed anxiety/depression associated with his neuropathy
    • Prescribed 4000 mg neurontin but it did not work
    • Feels that 450 mg Lyrica is a “life saver” because it gives almost immediate relief
    • Tries to exercise as often as he can
    • Brain feels lagged (“mental fog”),
    • Experiences drowsiness, weight fluctuations, and swelling
    • If he were in a clinical trial, he would like to know of all side effects ahead of time and any estimate of effectiveness

Jackie Evangelista

    • Neuropathy caused by lyme disease
    • Slow progression of disease to the point that she cannot notice the worsening f some symptoms
    • Condition has not improved that much from medication
      • Feels treatment “only takes the edge off… does not stop advance”

Large-Group Facilitated Discussion: Topic 2

  • Trials should measure how much time one can do a specific physical task
  • Some patients feel that just pain relief is not the best indicator for drug effectiveness
  • Patients usually try a treatment for 3-4 weeks before deciding if it works
  • The issue of side effects is the most prevalent causing of switching medications/therapies
  • Some patients feel that support groups, meditation, and other activities to boost emotional well being help diminish the effect of pain

SLIDES

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