Annie Ellis Patient Perspective from Ovarian Cancer Endpoints Workshop hosted by the Food and Drug Administration (FDA)
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The document discusses insights from an ovarian cancer workshop led by survivor Annie Ellis, emphasizing the need for more effective and patient-centered treatments. Key themes from a focus group indicate that patients prioritize quality of life and individualized care over traditional clinical endpoints like progression-free survival. Despite the challenges in defining treatment success, ongoing communication between patients and physicians is deemed essential for aligning treatment goals with patient values.
Annie Ellis Patient Perspective from Ovarian Cancer Endpoints Workshop hosted by the Food and Drug Administration (FDA)
- 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
Editor's Notes
- #2: Good afternoon & thank you’s
- #3: Here are some of the ways I serve the ovarian cancer community I’m just a foot soldier Start with a quote from my AACR SSP poster
- #4: Oops, we’re at the FDA, so…
- #5: That’s better! Ovarian cancer is complicated and we need so much
- #6: This is often what it feels like for patients to be involved in treatment selection, even up front. What are you going to get? Cure? Remission? Stable Disease? Might have an idea of S/Es, but won’t really know until we try Willing to try a lot of things to stay alive long enough for researchers to find the cure. Until more people are cured, recurrence will continue to be a reality; controlling disease has great value. It is important to determine who will benefit and avoid unnecessary toxicities NEED MORE PREDICATIVE BIOMARKERS for TX selection! COMMONLY MISSING in TX selection: Exploration of goals and priorities—what’s important to people living w/ovca?
- #7: Very happy to participate in this survey back in 2012. Attended 2013 SGO when this was presented —a buzz when through the room — Docs sitting behind me said, “This is good stuff.” Great surprise that doctors’ perceptions differed from patients’ regarding perceived outcome and toxicity thresholds Could this actually be a language disparity? Led to NYU/SHARE Collaboration
- #8: Study was published in the Journal of Gynecologic Oncology Objective: to explore the endpoints communication gap between physicians and patients Thank you to my colleagues at NYU, Stephanie Blank and Melissa Frey for the amazing opportunity to collaborate on this important work and for sharing the slides As well as my hard-working advocate colleagues at SHARE
- #9: Advocates invited women to participate in a physician-moderated focus group Distributed a questionnaire to collect demographics and preliminary information Session was brilliantly moderated
- #10: Here are some of the demographics.
- #11: Focus Group Themes: Without context, the term “Progression Free Survival” doesn’t mean much to patients. Especially with slight improvements in very small increments of time. When PFS question came up: dead silence Importance of QoL Yes, we all want more time, but what that time looks like is very important
- #12: Take a moment to discuss reasons for under-reporting side effects: Forgot to report b/c anxiety at appointments Fear of progression of disease Avoid additional tests Fear of dose reductions or that treatment would be stopped Do not want to be perceived as complaining Personal: “worthy of saving” AND Oliver Twist moment I perceived treatment to be working and I wanted to continue even when treatment was causing more harm than benefit Made me realize how important it is to accurately CAPTURE QoL/PRO measures during CLTRs Because it is equally important to know what else is being affected besides the cancer, especially with targeted therapies and combinations
- #13: Capturing QoL/PRO measures needs to be a PRIORITY Equal weight for qualitative measures
- #14: For today, important to reach out to the survivor community: Expanded and blended SGO/OCNA, NYU/SHARE surveys. TY to OCNA Inspire, SHARE email blast, FB Sisterhood, Yahoo Grp and everyone who reposted and shared w/others Survey open August 1-12 Over 400 site visits with 328 consented & completed surveys Any errors in survey design and analysis are my own Apologies: busy slides, short on time, will go through quickly. Here are some demographics: Not shown 93% Caucasian (Hispanic, Black, Asian, other) No surprise that most were dx at advanced stage 70% dx within last 4 years >20% Mutation carriers Other mutations (appear to be from BROCA Panel) included RAD51D, BRIP1, Chek2
- #15: Disease Status Almost half have experienced recurrence (44%) And quite a few were on active treatment (37%) Over two-thirds reported experiencing unresolved S/E’s (68%) 70% Off TX/NO RCR reported side effects (108/149) 50% of those reported neuropathy (74/149) Also fatigue and cognitive issues 46% reported multiple unresolved S/E’s (68/149) About 12% reported experiencing symptoms of advancing disease Over 70% w/symptoms also had unresolved side effects (27/38)
- #16: Treatment Expectations When selecting treatment, what is your expectation? Almost half chose Remission. Split between RCR (48%, 75/156) and NonRCR (52%, 81/156) Cure: Significantly more NonRCR (NonRCR: 82% , 93/115 vs RCR: 18%, 21/115) Stable: Significantly more RCR (RCR: 83%, 41/55 vs NonRCR: 17%, 9/55) Expectations about how long remission or stable disease would last was quite high.
- #17: Next, participants were asked to choose MINIMUM amount of PFS that would be considered meaningful or “worth it” NOTE: For OCNA/SGO survey, choices 1, 2, 3, 4, or 5+ months (Discussion to expand to find “sweet spot”) Again, the bar appears to be set pretty high Since those expecting cure are looking for “forever,” I filtered by those expecting Remission and Stable Disease
- #18: Then what was MINIMUM amount of OS that would be considered meaningful or “worth it” Also filtered by expectation of Remission or Stable Disease
- #19: Then, Which of these is most meaningful to you? Only one answer could be chosen: Total amount of time Easily managed S/E’s or Amount of time off treatment Ability to engage in daily activities or attend a single event “Total amount of time” was selected slightly more frequently than “Ability to engage in daily activities” But combining QoL answers revealed QoL preference NOTE: “Others” were all QoL related. INTERESTING: When filtered by status (RCR, S/E's, Symptoms) “Total amount of time” remained in 38-44% range, but differences were noted when filtered by expectation (cure 53%, remission 31%, stable 34%,)
- #20: Next came a series of questions about the tolerability of S/E’s, which I handled differently than the 2012 OCNA/SGO survey First, type of side effects considered tolerable if the goal is cure Respondents could choose more than one answer Manageable, Mild, Moderate, Severe? Temporary or Permanent? Financial toxicity All/none And then respondents were asked to “sign up” for specific S/E's from a long—but not comprehensive—list The comments were quite powerful. I wish there was time to share them all. “20% chance”; “blindness/deafness”; “not all at the same time”; “Within reason”
- #21: Then same questions about S/E's if the goal is remission With equally impressive comments “temporary”; “depend on the severity/duration/frequency”; “If remission was lengthy”; “They can all be managed? right?”
- #22: Here are the responses if the goal is to maintain stable disease Needs further analysis All three (Cure, Remission, Stable disease) showed: Preference for S/E's that can be managed with meds Same Top 3 and Bottom 3 S/E's (Top 3: Fatigue, Hair Loss, Weight change) (Bottom 3: Bowel obstruction, Breathing issues, Infection) Observation: As this series of questions progressed, more and more respondents chose to not answer (8, 21, 31). Possibly indicating discomfort with the subject, maybe survey fatigue, or not realizing that these were different questions.
- #23: And here are a few particularly insightful and eloquent comments about side effects. I added bold to highlight some compelling phrases “wheel of fortune” of changing issues “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…”
- #26: At this point, I’d like to express my deepest gratitude to the many women living with and beyond ovarian cancer who took the time to take this survey I am so thankful for their trust and for the privilege of sharing all of this here at this meeting I always leave a place for comments at the end of my surveys Deeply moved by what is expressed in this space There were also requests to study rare subtypes.
- #27: Here are more final comments IMPORTANT: “my outlook is good enough that the tradeoffs don't seem real to me right now.” This last comment pretty much sums it up: We all just want to get well.
- #28: Before I conclude, poker game analogy to explain to peers Cards: Tx options; Chips: costs to our bodies Expand options w/clinical trials and maybe return to something that has worked in the past The more options and chips we can hold on to, the longer we can stay in the game We lack adequate decision aids NEED PATIENT-CENTERED tools for informed decision making Time Trade Offs in tiny increments (month) are really difficult, especially up front when cure is on the table Put PATIENT first: examine current status & life priorities Use Algorithms to calculate risk & potential impact of therapy Develop one tool to use throughout entire TX continuum It can be done! NEED to include QoL/PRO measures in CLTRs
- #29: Thank you for your attention and continued commitment to improve the treatment landscape for women w/ovarian cancer. Innovative Clinical Trial Design is coming up after the break The most valuable resource is the clinical trial participant. Make their participation count no matter what and capture QoL/PRO measures! Thrilled when everyone works together with regulatory agencies to avoid poor trial design. Consider having an advocate at the table to provide valuable insight (like it's not cool to randomize a participant twice within the same trial)
- #30: Finally, survivors don’t use the word “Cure” a lot, but it’s what we continue to hope for Even “Cancer-Free” can feel misleading Our community likes to use “No Evidence of Disease” or “Dancing with NED” Picture: OCNA conference survivor party July 2015 So much recent progress--It feels like we are SO CLOSE!! Let’s get everyone a lasting and meaningful Dance with NED. We are counting on you to lead the way!