By Nikki Blocker
September 29, 2013
Notes on FDA Public
Meeting on Patient-Focused Drug Development
September 24, 2013
I would like to
familiarize anyone reading this with a little background before
writing about the actual meeting. My fiancé and I had driven about
fifteen hours and only had about four hours of sleep when we attended
the meeting. The meeting room was almost full; therefore we were
forced to sit in the back and I’m a bit hard of hearing. A
transcript of the meeting will supposedly be available on the FDA
website. Not only were there two panels of people chosen to speak,
there were results included from those watching the webcast, and
people were able to phone in.
We parked in
the designated area and immediately met three other people attending
the meeting. One was an immaculately dressed lady named Nancy. She
and I immediately discussed our symptoms and our medications. We are
both on Xyrem, and we began having bizarre symptoms while in college.
She had typed a report using
her receipts to show how the cost of Xyrem had increased over
time, and had also included how Jazz Pharmaceuticals is listed as
number one on the list of Fortune 500 companies. Based in Dublin,
Ireland, a three year average ranks Jazz number one with revenue
growth of 68%, profit growth of 279%, and a total return of 106%.
Jazz reported revenues ($ millions): 680, and net income ($
millions): 304.3.i
During the meeting Nancy raised her hand several times but was never
handed the microphone.
Seating was
scarce because so many people had attended. As we were seated near
the back of the room, a lady with the FDA was speaking about the
public meetings they had decided to hold and how there had been such
a massive response from the narcolepsy community. Patients
were given a clicker to answer questions. 58% of those
attending were from out of town, 37% were age 41 to 50, and 37% were
recently diagnosed. She explained to us narcolepsy is considered a
rare disease because fewer than 200,000 people have it and fewer than
50,000 have been diagnosed. Some facts from the FDA:
- Symptoms can begin as young as age 7.
- Cataplexy causes slurred speech.
- Restless Leg Syndrome is a symptom of narcolepsy.
- Auto Behavior includes sleep walking.
- Symptoms can improve with lifestyle and diet changes.
A few minutes
later the first set of panelists took their seats at the front of the
room. Names of the panelists will probably be available on the
official transcript. Notes are limited to important details.
First Panel
Panelist I
- Severe symptom onset began in college.
- Was on the Dean’s list but is now unable to maintain good grades.
- Ran 5k everyday. No longer exercises because exercise makes symptoms worse.
- Said diet improved symptoms but didn’t elaborate
Panelist II
- 15 yrs old
- Ran and exercised daily. No longer exercises because exercise makes symptoms worse.
- Was an honor’s student but is now unable to maintain good grades.
- Hallucinations include the inability to tell whether or not something was a dream, or if it really happened.
- Auto behavior occurs while mowing the lawn.
- Is now on gluten free diet and symptoms are improving.
Panelist III
- Sleep paralysis began at age 29
- Hallucinations included speaking to the spirit of her dead father. She believed this until she began seeing the spirit of her sister who is still alive.
- Developed cataplexy at age 30.
- Perimenopause made symptoms worse.
- Was diagnosed while working as a pharmaceutical representative. She visited a neurologist and happened to read a pamphlet about narcolepsy. She asked to be tested and was positive.
- Has fallen asleep while driving.
- Symptom onset began while in college.
- Always late for class and noticed she was “losing time”. Would arrive at a location with no recollection of how she got there.
- Hallucinations include dark shadowy figures.
- Carbohydrates and sugar make symptoms worse.
- Cataplexy is caused by anger.
- Diagnosed at age 39.
- Sleeps 12 to 18 hours per day.
- Has sleep attacks while out in public.
- Has “Brain Fog”. (At the mention of this term there was a collective epiphany throughout the room because so many people had brain fog but did not know there was an actual term for it.)
- Exercised daily but is no longer able to.
- Has lost most of his teeth.
After the first
panel finished comments were taken from the audience. One woman has
a ten year old son with narcolepsy who passed every neurological
exam. Physicians did not believe the child had narcolepsy until his
parents filmed him having an attack of cataplexy. The audience was
asked if there was anything unusual to alert them to an on coming
attack of cataplexy. Many people with cataplexy have a “popping
feeling” when it is about to happen. Someone with the FDA stated
children have been known to mask their emotions to avoid a cataplexy
attack. For example, a child may keep a straight face instead of
laughing.
Another audience member stood and was handed the microphone. She
told of how she allocates a certain amount of time for each task
throughout the day. Using some kind of timer with an alarm, she will
allow ten minutes for showering, breakfast, getting dressed, etc.
She began using the timer and alarm system to avoid spending an hour
on her hair or makeup thus making her late for work. She also uses a
GPS everywhere she goes to help her track where she is and how to get
home.
One of the FDA
physicians brought up the subject of auto-behavior. He asked if
anything unusual alerted anyone to impending auto behavior. It took
a while, and a few attempts at clarification, for anyone to
understand his question. Finally a woman spoke up and told him it
only occurred when she was extremely tired. Another woman described
it as a “conscious sedation” and said it occurs when she cooks.
Another FDA
physician asked if anyone had symptoms that improved at certain times
and then became worse. Several people volunteered information. Many
women find symptoms worsen during menstruation. Summertime makes
symptoms worse for some patients; however a larger number of people
said symptoms became severe with the lack of sunlight that
accompanies autumn and winter. Some patients use a Phillips GoLite
BLU Energy Light as therapy. It produces a 470nm blue light that is
not supposed to be looked at directly. Phillips states that by
shining the light on the side of your face for 15 to 30 minutes per
day it will activate hormones to naturally boost your mood. It is
endorsed by the Mayo Clinic.ii
At this time a
call was taken from someone watching the webcast. The called stated
she was perfectly normal until she contracted a virus and had a high
fever. She has had narcolepsy ever since.
The meeting
stopped for a brief break during which we could pay for coffee along
with a carbohydrate and sugar loaded snack. A woman struck up a
conversation with me about how the emotions had exhausted her.
I started to ask about diet, however she began telling me about her
organization and handed me her business card. She was with
Narcolepsy Network. Narcolepsy Network had several members attending
the meeting and they seemed dissatisfied with the FDA.
After the break
the second set of panelists took their seats at the front of the
room. It should be noted that many of the people in each panel were
reading word for word scripts of what they had turned in. Panelist V
did not stick to his script.
Second Panel
Panelist I
- Had to have four sleep tests.
- Used to be a runner, no longer can exercise.
- Gluten sensitive and is now on gluten free diet showing improvement.
Panelist II
- Her 9 year old daughter is on Xyrem and two other drugs.
- She is worried about her daughter’s liver.
Panelist III
- EDS began in high school.
- Developed a tremor at age 21.
- Has major depression and is unable to lose weight.
- Says Xyrem is a miracle drug and her cataplexy is almost completely gone.
- To manage side effects of Xyrem she takes magnesium and potassium supplements.
- Symptoms become worse in fall and winter.
Panelist IV
- Diagnosed at age 42
- Also has asthma, allergies, and fibromyalgia
- Had seizures in her sleep when she took Xyrem. No longer takes it.
- Never eats processed food but still eats whole grains.
- Takes magnesium, B vitamin complex, DHA, & probiotics.
- Has auditory hallucinations.
Panelist V
- Symptoms vary with seasons.
- Has a problem with tolerance.
- Believes results would be greater with a holistic approach.
- Wants screening for early detection.
- Said patients should be divided into three groups: Narcolepsy, Narcolepsy with Cataplexy, Narcolepsy with Cataplexy and multiple problems
- At age 9 he had an IQ of 153.
- Night terrors and onset of narcolepsy began at age 11. Grades fell as a result.
- Diagnosed at age 22
- Said the FDA and physicians need to stop treating us with a “blanket approach”.
At the
conclusion of the second panel mumbling from the audience could be
heard. Several members seemed to become a bit angry. People began
to wonder why the FDA had never thought of these things.
Another set of
questions were posed to the audience and answered with our clickers.
One question involved dental problems. Many members of the audience
had lost teeth. Biotene, a line of products to prevent a condition
known as “dry mouth”, was mentioned by an audience member. The
FDA changed the subject.
The results of
the next question angered most members of the audience. Those with
clickers were asked what therapies were used to improve our symptoms.
Several therapies were listed on the giant screen visible at the
front of the room, and more than one could be chosen. Those listed
were naps, diet, exercise, and a few other things. 91% said naps
helped, 66% picked diet, 66% picked exercise, and percentages were
negligible for the other choices. The subject of diet was never
covered. Everyone seemed to want to tell their sad story or talk
about how Jazz Pharmaceuticals was a monopoly. People began swerving
away from the topic with long stories, often losing their train of
thought. The audience began getting aggravated and stopped paying
attention to the people talking until a lady stood up and began
telling the story of her son. She started the organization Wake Up
Narcolepsy and told the people with the FDA she didn’t care if she
went over two minutes. She began reading her own list of statistics
she had compiled and basically told the FDA they weren’t doing
anything to help us.
The next
speaker is only known to me as The Man in the Hat. He said it was
all about Orexin and stated he wanted a drug to replace Orexin.
Nobody knew what Orexin was and a lot of mumbling was heard. He said
without it homeostasis was flawed and became harder as people become
older. He angrily stated napping is not a therapy and how dare the
FDA list it as one. He told the FDA all they provided for us were
expensive band aids. The audience had another epiphany as everyone
realized the idea of napping as a therapy was ignorant.
By the time the
man in the hat was done everyone was angry about something and nobody
wanted to listen to a long drawn out dialogue. That is when I was
picked to speak and handed the microphone. I looked straight at the
man in the hat and said, “The man in the hat is correct. It is
all about Orexin.” I turned to face the FDA physicians head on and
stated, “I am on a gluten free and sugar free diet, and it has
dramatically improved my symptoms.”
A few parting
comments ended the meeting and we were instructed to turn in our
comment cards. I’ll spare the reader most of what was written on
mine. I will state that I complained about being charged for
horrible tasting coffee.
In conclusion,
the Public Meeting on Narcolepsy ended on a negative note. People
who began the meeting with smiles left the meeting in tears. If the
other public meetings went as poorly for the FDA as this meeting,
surely they will do something in the future to steer the meetings
toward more positive topics.
____________________________
i
Green, Shannon. Jazz Pharmaceuticals – Fastest-Growing Companies
2013- Fortune Magazine
