“What is your thinking about the risk/benefit profile? How much risk are you willing to tolerate? No drug is completely safe.  You all recognize that, but there is a scale there, and we need to hear from you how much risk you’re willing to tolerate,” said Director Dr. Bob Rappaport.

“Dr. Rappaport touched on a number of topics that are critical for our decision making at CDER. This is why we have patient-focused drug discussions. You talk about the severity of the condition and provide an analysis of current treatment options. That information is very important in setting the context in which we make our decisions. Our decisions are not made in a vacuum … We did not have a meeting mechanism that could allow us to systematically get at the patient perspective, which we should get as good of an understanding as we can about the patient’s perspective on the disease and current treatment options,” observed Patrick Thomas of the FDA. FDA professional panel dignitaries included:

• Bob Rappaport, MD, Director, Division of Anesthesia, Analgesia, and Addiction Products (DAAAP), CDER, FDA made the meeting’s opening remarks. • Patrick Frey, MPP, Director, Office of Programs and Strategic Analysis, CDER, FDA, gave an overview of the FDA’s Patient-Focused Drug Development Initiative • Elizabeth Kilgore, MD, Medical Officer of DAAAP, CDER, FDA • Sharon Herz, MD, Deputy Director, DAAAP, CDER, FDA tied together testimony and comments made by the fibromyalgia panels and audience members in her closing remarks.

The Fibromyalgia Patient Panel #1 was made up of five fibromyalgia (FM) members, including one who utilized the conference call-in number. Each panel member discussed their personal experiences concerning Topic 1: Disease symptoms and daily impacts that matter most to patients. After the panel members’ discussion of Topic 1, a large-group facilitated discussion by audience patients and patient representatives ensued. After a short break, Panel #2, made-up of five other people with FM presented information in line with Topic 2: Patients’ perspectives on current approaches to treating fibromyalgia. This was followed by another large-group facilitated discussion with audience members. The last part of the meeting was for open public comment by people from the audience who had registered to speak about their perspectives. The FDA panel members also asked questions and made comments based on the FM patients’ testimonies.

It was evident from comments made by Panel #1 FM patients that besides pain, people suffer with a variety of symptoms, including fatigue, sleeplessness, fibro fog, headaches, including migraines, bowel problems and a variety of overlapping chronic pain conditions. People with FM taking part in this FDA meeting were able to electronically vote on the single symptom besides pain that affects their quality of life the most. After pain, fatigue was the number one issue followed by fibro fog and bowel distress that seems to impact a person with FM’s quality of life the most.

Jan Chambers, President of the National Fibromyalgia & Chronic Pain Association, was part of FM patient Panel #2. She gave a detailed account of her personal journey with FM, including information about a successful chiropractic treatment that helped alleviate problems with a lack of lordotic curve of her cervical spine. This therapy decreased compression of her cervical spinal cord, which reduced many of her most severe FM symptoms.

Other types of treatments discussed by the panel included regimens incorporating medications, exercise and other alternative therapies such as hot baths and showers. Of the people with fibromyalgia who were in the audience and participating through the webcast, many stated that none of the three FDA approved fibromyalgia medications, Lyrica, Cymbalta or Savella, have helped alleviate their pain and other symptoms. But those people in the audience or webcast who have been helped by these prescription medications touted their benefits and the positive changes the medications have made in their lives. Side effects, including weight gain and daytime sleepiness, along with a variety of other issues were voiced by many who had tried these specific medications. An important point was made that the original research study data for these FDA approved FM prescription medications had revealed the medications helped 30% of the FM patients who tried them with about 50% efficacy. For those people the success of these FDA approved prescription drugs has been a blessing.

Several people in the audience and a couple of members on Panel #2 said that they take opioid prescription medications. All of these people felt their lives were much better now that they have access to medications that help them live a better quality of life with less pain. One of the audience members shared his story of difficulty getting his opioid medications paid for by his insurance company. Another issue shared by these people was the stricter regulations connected with getting their medications and some of the problems they have personally encountered.

At the end of the meeting, comments from registered audience members was invited. During her allotted three-minute presentation time, Rae Marie Gleason, Medical Education & Research Director for the NFMCPA discussed the issue of the ongoing problems for people acquiring an FM diagnosis. She stated that it still takes up to 5 years for an FM diagnosis to be made. In the mean time thousands of dollars are spent by people searching for answers to their illness and pain along with billions more in lost productivity and other medical costs. She pointed out that until an accurate diagnosis is made it doesn’t make any difference what treatment options are available for FM symptoms. She then stated the confusion regarding up to four FM diagnostic criteria that is now offset by the availability of the FM/a blood test that was researched at the University of Illinois. This simple procedure for both people who suspect they have FM and the doctors who treat them is a major scientific breakthrough that is poised to change the course of FM recognition and treatment.

You can learn more about this FDA Fibromyalgia Patient Meeting by accessing the recordings of the conference at:
• Part 1 - https://collaboration.fda.gov/p3045ldlvgc
• Part 2 - You are encouraged to submit your comments on the impact of fibromyalgia symptoms (except for pain) on your life before May 26, 2014, click here.  Easy step-by-step instructions are posted there.

In concert with the Prescription Drug User Fee Act V (PDUFA V), FDA is committed to a new initiative called Patient-Focused Drug Development with the goal of obtaining the patient perspective on certain disease areas during the five-year period of PDUFA V. Assessment of a product’s benefits and risks involves an analysis of the severity of the condition treated and the current treatment options available for the given disease. This information is a critical aspect of FDA’s decision-making as it establishes the context in which the regulatory decision is made. FDA believes that drug development and FDA’s review process could benefit from a more systematic and expansive approach to obtaining the patient perspective on disease severity and current available options in a therapeutic area. For more information about the Patient-Focused Drug Development initiative visit: http://www.fda.gov/default.htm During the next five years FDA will hold four patient centric meetings each year for a total of 20 meetings with a possibility of several disease additions along the way. On March 26, 2014, FDA held a meeting focused on people with fibromyalgia (FM) at its Silver Spring campus buildings from 1-5 pm. It was well attended by about 150 people (approximately 35 people with fibromyalgia (FM)). Another 300+ people with FM participated through the meeting’s webcast format. FDA Panel Member, Sara Eggers, PhD, from the Office of strategic Programs, Center for Drug Evaluation and Research (CDER), FDA, facilitated the meeting and welcomed the FM patient panel members, and audience participants affected by FM.

March 26, 2014, Silver Spring, Maryland