On September 3, 2015, Ovarian cancer survivors and FDA Patient Representatives Peg Ford, Susan Leighton and Annie Ellis were invited to provide the patient perspective at the recent Ovarian Cancer Endpoints Workshop hosted by the Food and Drug Administration (FDA).  This meeting was co-sponsored by the Society of Gynecologic Oncology (SGO), the American Association for Cancer Research (AACR) and the American Society of Clinical Oncology (ASCO).   Many important topics to the ovarian cancer community were discussed, including novel clinical trial designs, biomarkers, and new classes of agents such as immunotherapies.

1. Ovarian Cancer Workshop
September 3, 2015
Annie Ellis
FDA Patient Representative
Ovarian Cancer Survivor/Research Advocate

2. Annie Ellis
 11-year Ovarian (2 recurrences) and 4-year Breast Cancer Survivor
 FDA Patient Representative
 CDMRP OCRP Integration Panel
 Ovarian Cancer National Alliance
◦ Research Advocate
◦ Formerly on Special Programs (Education) and Conference Committees
◦ Conference Speaker; Survivors Teaching Students®
 SHARE
◦ Helpline Peer
◦ Former Peer Support Group Facilitator
◦ “The Patient’s Perspective” presented at SGO 2008
 NY Presbyterian/Columbia Woman to Woman Peer
 OCRF: Clinical Trials Video; Symposium Speaker
 2014 AACR Scientist↔Survivor Program
 2013 Congressional Briefing on Ovarian Cancer for Society for
Women’s Health Research (SWHR)
 Roswell Park Ovarian Cancer SPORE Patient Representative

3. It is a truthuniversallyacknowledged,
that a womanin possessionof
ovariancancer
must bein want of more
effectivetreatments.
Adapted from Pride and Prejudice by Jane Austin.

4. It is a truthuniversallyacknowledged,
that a womanin possessionof
ovariancancer
must bein want of more
SAFE & EFFECTIVE treatments.
Adapted from Pride and Prejudice by Jane Austin.

5. Treatment for ovarian cancer is like…

6. Endpoints in clinical trials: What do our patients
consider important? A survey of the Ovarian
Cancer National Alliance (OCNA)
 Abstract presented at 2013 SGO Annual Meeting
 Revealed disparities: Perceived outcome
Toxicity threshold (including financial toxicity)
 Survey results incorporated into 2013 SGO White Paper

7. Journal of Gynecologic Oncology, November 2014, Volume 135, Issue 2, Pages 261–265

8. Methods
• SHARE - nonprofit organization of ovarian and breast cancer survivors
• Patient advocates identified women with ovarian, fallopian tube or primary
peritoneal cancer
• Pre-focus group questionnaire
– Demographics
• Age, ethnicity, profession
– Disease characteristics
– Treatment goals
– Quality of life priorities
• Focus group
– Physician moderated session
– Circular seating pattern
– Moderator guide utilized
– Session recorded, transcribed and coded
• Common themes identified and used to perform intra-case analysis by two
independent reviewers

9. Results
• 22 person focus group assembled
Pre-focus Group Questionnaire
Age (median, range)
65 years
(47-72)
Recurrent disease 13 (59%)
# of recurrences (median, range) 1 (1-9)
Currently receiving treatment 4 (18%)
Currently experiencing side
effects of treatment/disease
10 (45%)
Stage I
14%
Stage II
0%
Stage III
77%
Stage IV
9%
Stage of Disease

10. Common Themes from the Discussion Group
• PFS and OS hold minimal significance as treatment goals
– “I’m either sick or I’m not sick. The only reason I know of the terms
progression free and overall survival is because I’ve read studies.”
• Quality of life and minimizing side effects are most important
– “It’s about quality of life. I have to work everyday. The physical and
emotional are what hold importance.”
• Prefer an individualized approach to care focused on:
– Maintaining personal relationships
– Participating in life events

11. Common Themes from the Discussion Group
• Physicians should explore changing treatment goals over time
– “With each recurrence it became more clear that time is limited and
I started to prioritize differently.
– “My goals and expectations change as the disease progresses, I
have a new perspective on life.”
• Underreporting of treatment and disease related side effects
– 27% of women stated that they have underreported side effects
• Communication with physician is essential in treatment decisions
– “During a relapse there was a miscommunication between the
oncologist and the surgeon and I felt that tug and I didn’t know what
to do. I ended up going with my gut feeling and neither physician
truly understood my goals.”

12. A qualitative study of ovarian cancer survivors’
perceptions of endpoints and goals of care
 RESULTS: The main themes identified were barriers to communication,
importance of frequent communication between patient and physician
regarding goals, and expectations of treatment changing with position along the
treatment continuum. One hundred percent of participants identified
communication with their physician as an essential element in determining
treatment course. However, only 14% reported having a discussion about
goals, values and perceptions with their physician preceding treatment
decisions. Participants reported that the terms progression free and overall
survival held minimal significance for them and instead they preferred an
individualized approach to care focusing on quality of life. Many women
underreported side effects with reasons ranging from fear of dose reductions
and additional tests to forgetting about symptoms due to anxiety.
 CONCLUSIONS: An objective measure of treatment success meaningful to
survivors, physicians and regulators is, at present, elusive and may not exist.
Ideally, future trial design would place equal weight on quantitative and
qualitative measures and include information about goals of treatment.

13. Ovarian Cancer Survivorship Survey (August 2015)
Demographics

14. Ovarian Cancer Survivorship Survey (August 2015)
Status

15. Ovarian Cancer Survivorship Survey (August 2015)
Expectations
For those who expect Remission:
•64% expect it to last over 2 years (100/156)
•90% expect it to last 1 year or more (140/156)
For those who expect Stable Disease:
•80% expect it to last 1 year or more (44/55)

16. Ovarian Cancer Survivorship Survey (August 2015)
MINIMUM
Extension of PFS
Filtered by Expectation
Remission
Mode: 6 months
(36/156; 23%)
Second: 1-2 years
(29/156; 19 %)
60%: 1 or more years
(94/156)
Stable
Mode: 6 months
(17/55; 31%)
42%: 1 or more years
(23/55)

17. Ovarian Cancer Survivorship Survey (August 2015)
MINIMUM
Extension of OS
Filtered by Expectation
Remission
Mode: 1-2 years
(30/156; 19 %)
Second: 6 months
(28/156; 18 %)
71%: 1 or more years
(111/156)
Stable
Mode
Tie: 6 months/1-2 years
(9/55; 16%)
56%: 1 or more years
(31/55)

18. Ovarian Cancer Survivorship Survey (August 2015)

19. Ovarian Cancer Survivorship Survey (August 2015)
Cure
“Anything that has less than a 20% chance
of killing me immediately.”
“I would accept any side effects for a CURE!!”
“These were difficult to answer, since
there is virtually no chance of me having a cure…
I would not be willing to risk blindness or deafness.”
“I have experienced most of these already”
“If these were temporary I could probably tolerate all of them.”
“But not all at the same time… I expected that I might suffer
any of these …but if it were constant and severe
I would tolerate none. All things must pass!”
“Within reason. Quality of life and not exposing
my loved ones to watching a harsh death means more to me
than survival. I know where I am going.”

20. Ovarian Cancer Survivorship Survey (August 2015)
Remission
“None”
“Hard to check off here because
the remission time is not stated.”
“Again, I've had all these except two already.”
“I would tolerate these side effects if they were temporary.”
“…would depend on the expected severity and duration or
frequency. I would only tolerate minimal vision loss.”
“Whatever it takes to give me 5 yrs,
my daughter is 14 and I am a single mother”
“I'd rather not tolerate any side effects.”
“If remission was lengthy.”
“They can all be managed? right?”

21. Ovarian Cancer Survivorship Survey (August 2015)
Stable disease
“Obviously, I just want to live!”
“All of the above!”
“Basically already endure these...”
“As long as these are temporary
side effects and not permanent.”
“Depends on severity and QOL tradeoffs. This is a much
bigger issue if "stable disease" is the best you can get.
Facing this now, and learning that my tolerance for
side effects is pretty low under these conditions”
“Almost anything if it leads to a year or more.
Maybe a cure will come .”

22. “Questions don't really address the combo effects. I would be willing to
endure some severe effects on a temporary basis and some milder side
effects on a permanent basis…”
“I already have gone into bankruptcy and debt. I'm not willing to risk
more debt, as it is my husband who would be stuck with that debt. I could
tolerate moderate side effects if there was a good chance of achieving
remission”
“maybe...probably....for how long, how severe, how will it affect my life
and ability to live it? impossible to answer this honestly....fighting cancer is a
weekly, sometimes daily, wheel of fortune of issues that change....and you
make decisions and choices in that moment”
“I find these distinctions of cure vs. remission vs. stable disease not really
relevant from the patient perspective if a good QUALITY OF LIFE may be
maintained. If I could live for a significant period with stable disease AS IF I
am in remission, or AS IF I am disease free, then that would be really great.
You cannot tell from the outside and from patient behavior where patients are
in disease progression if they have a high quality of life - could you? From afar,
I don’t look sick at all….”
Ovarian Cancer Survivorship Survey (August 2015)
Side Effects Comments

23. Ovarian Cancer Survivorship Survey (August 2015)
“I've been in 5. I learned about 4 from my
doctors.”
“Access to clinical trial became available due
to testing of tumor. More of this needs to
happen. Totally changed the game for me, in
a life or death way.”
“to feel as if I did every single thing I could
to get a cure or very long remission”

24. Ovarian Cancer Survivorship Survey (August 2015)
“First trial they did not take my
insurance. The second time I had just
finished 6 months of chemo. Too
exhausted to enter into trial.”
“After careful study of the particular
trial, I preferred the standard of care
available. I did not want to risk one of
the severe possible side effects.”
“Trial was not available when I needed
treatment.”
“A trial that I would qualify for had just
been filled.”
“None suitable keeping a look out but
also needs to be an attainable
distance!”
“I'm still going through regular chemos
available”

25. Ovarian Cancer Survivorship Survey (August 2015)
Final Comments
“These questions are great, the trouble
is, when starting a new treatment, the
doctors don't know what side effects
the patient will experience, nor do they
know if the patient will become stable,
achieve remission or a cure. We can
tolerate a lot; particularly if we know
the outcome will be worth it. Trouble
is, there is no way of knowing that at
this time.”
“While I'm willing to risk a lot to save
my life, it only goes so far, and for so
long. As long as a treatment is doing
what it's supposed to, and I can
maintain some quality of life, I'm
willing to endure...”
“I am interested in specific long term
consequences of the disease and side
effects of treatment.”
“Quality of life is very important to me.
I would not take a medication that
would cause severe side effects and
only prolong life for a couple of
months…As a society, we need to look
at how we accept the inevitability of
death.”
“I would endure anything to be cured
of ovarian cancer I would do anything
to be in remission for ovarian cancer. I
would do anything to stabilize the
disease and not get worse.”

26. “It is our nature to survive. Most of us will find the strength to do
whatever it takes to insure our survival. But, chemo feels like a
slow killing. How does one heal something they are trying to kill?”
“I found it very difficult to answer the series on side effects I would
endure for cure, remission, stabilization, because my side effects
were not too terrible and my outlook is good enough that the
tradeoffs don't seem real to me right now.”
“I would do just about anything to hear the words, "You do not
have to worry about recurrence ever again. We have found a cure
for ovarian cancer.“
“I want to get well”
Ovarian Cancer Survivorship Survey (August 2015)
Final Comments

27. Keeping Options in Play
Staying in the game

28. Summary: Until the cure…
 Until molecularly-informed precision medicine and
more effective treatment options are widely
available, too many women will continue to
experience multiple recurrences, chronic disease,
end stage and death
► Predicative biomarkers
► QoL/PRO measures in clinical trials
► Patient-centered decision aids and tools for
informed, shared decision-making along the
entire treatment continuum

29. The work is not over until everyone has a chance
to have a lasting and meaningful Dance with NED.
No Evidence of Disease