Current and former clinical trial participants discuss decision-making from a patient's perspective. What factors should you consider when choosing a clinical trial? What are the potential benefits of participating? What misconceptions might discourage people from seeking clinical trials? When is it best not to participate? Panelists include women living with metastatic breast and ovarian cancers.

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Maximizing Treatment Options:
What to Know When Considering a
Clinical Trial
November 18, 2015

2. Agenda
 Intro
 Myths
 Potential Risks
 Potential Benefits
 Factors to Consider
 Informed Consent Process and Consent Form
 Patient Experiences
 Q&A
 Final Thoughts
 Resources
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3. Annie Ellis
 2004 Ovarian Cancer (2 recurrences, currently NED)
 2010 Early Stage IDC Breast Cancer
 Ovarian Cancer Research Advocate
 CDMRP OCRP Integration Panel Consumer Member
 FDA Patient Representative
 Roswell Park Gynecologic Cancer SPORE Patient Advocate
 OCRF Clinical Trials Video and Symposium Speaker
 Journal of Gynecologic Oncology : A qualitative study of
ovarian cancer survivors' perceptions of endpoints and
goals of care (November 2014)
 Peer Support
 SHARE Helpline
 Woman to Woman
 Clinical Trial Participant
 Phase I Ovarian Cancer Remission Vaccine
 Imaging (Breast: Contrast Mammogram vs MRI)
 2 Genetic Clinical Trials
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4. • Clinical trials are a way to find out if new treatments are
safe, effective and better than or equal to the current
standard of care
• Recurrent or metastatic disease:
• Clinical trials can be used to expand treatment options
• Patients who have had fewer lines of chemotherapy
can qualify for more trials
• Not every clinical trial is right for every person
Clinical Trials: Intro

5. Questions Clinical Trials Can Answer
 Is a new treatment or combination safe? How much?
 Phase I
 Is a new treatment effective?
 Phase II
 Is a new treatment better than or equal to the current
standard treatment(s)?
 Phase III
 Will some patients benefit more than others?
 Post-trial subset analysis
 Biomarkers to stratify participants
 NCI Exceptional Responders Initiative and NCI Match

6. • Molecularly informed precision medicine:
The right drug for the right patient at the right time
• Umbrella trials: One tumor/histologic type is accrued
and then sorted into sub-studies
• Basket trials: Multiple tumor/histologic types are
grouped by genetic information
http://www.bhdsyndrome.org/http://www.bhdsyndrome.org/http://www.crwf.com/phase-i-ii-iii-or-iv/
Clinical Trials: New designs

7. Myth: Last Resort
 There are clinical trials in first-line treatment to see if
the current first-line standard of care can be improved
 If additional treatment is needed after the study, the
standard drugs will still be available
 Patients who have had fewer lines of chemotherapy
can qualify for more trials
 Some trials do not accept patients who have previously
received a drug being used in the trial
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FALSE!
Clinical Trials are only as a last resort when everything
else has failed and there are no other options available.
FALSE!

8. Myth 2: Guinea Pig
 Modern clinical trials are designed with protections
for participants
 Scientific oversight from Institutional Review
Boards (IRBs), Food and Drug Administration
(FDA) and others
 Patient rights
 Informed Consent Process and Consent Form
 Right to withdraw at any time, for any reason
 Test Pilot Analogy from NYT
(“Clinical Trials Need Cancer Patients” Stan Collender, June 2015)
http://www.nytimes.com/2015/06/19/opinion/clinical-trials-need-cancer-patients.html?_r=0
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FALSE!
Participants are treated like Guinea pigs or lab rats,
especially in phase 1 clinical trials to establish dosage.
FALSE!

9. Myth 3: Placebo
 In cancer clinical trials, everyone who needs
treatment will get treatment.
 The only time placebos are used in cancer clinical
trials are
1) if the current “standard of care” is to watch and wait,
such as a remission setting; or
2) when a new treatment is being added to an existing
treatment.
 For example: some participants receive standard therapy plus a
novel therapy and some participants receive standard therapy
plus a placebo—everyone gets treatment.
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FALSE! Patients will get a placebo instead of treatment. FALSE!

10. Myth 4: No Stopping or Going Back
 The participant has the right to withdraw at any time
for any reason
 Participants are protected: discontinuation for
disease progression or side effects
 Many trial participants are referred to cancer centers
for clinical trials by their home doctors
 Some participants are able to continue to see their
doctors during the trial and most return to their
home institution when the trial is over
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FALSE!
Once a clinical trial has started, there’s no stopping
or going back to one’s original doctor.
FALSE!

11. Potential Risks/Unknowns
Although novel therapies may be described as
“exciting” or “promising” because of preliminary
evidence, it is important to remember:
 It is not known if the study drug will work and for
how long
 It is not known what specific side effects (short-
term and long-term) will be experienced
 There is no guarantee that the novel drug being
studied will be better than standard drugs
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12. Potential Benefits of Clinical Trials
 Contribute to medical knowledge and bring better
treatments to future patients
 Access to new drugs and treatments not yet available
outside of studies
 Increase treatment options
 Close monitoring, perhaps at a different medical
institution with an additional medical team
 Gain information about one’s condition, especially if
genomic sequencing is offered
 Potential for personal benefit
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13. Information to get started
Copies of current tests, scans and pathology
Location and volume
Sub-Type
Breast: Luminal A/B, Triple Negative, Her2 status
Ovarian: High/Low Grade Serous, endometrioid, clear cell, etc.
BRCA mutation status; other hereditary mutations
Genetic counseling is an important part of the testing process
Genetic testing is recommended for everyone with a diagnosis
of ovarian cancer
Genomic information about the tumor(s)
 If already available: to participate in trials based on specific
mutations instead of organ site
If not already available: discuss with medical team
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14. Clinical Trials: Factors to Consider
 Finding a Trial
 Doctor recommendation
 Clinical Trial Matching Service
 Second opinion
 Word of mouth
 Eligibility Criteria
 First-line or recurrence
 Measurable disease; Rising markers
 Prior chemotherapy
 Current status: blood counts, kidney and liver function, etc.
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15. Clinical Trials: Factors to Consider
 Scans/Tests
 Additional tests and scans may be required to determine
eligibility
 Extra tests and scans may be required during the trial to monitor
effectiveness
 Time
 Administration of the trial medication can take longer than
traditional chemotherapy
 More frequent visits to the clinic may be required
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16. Clinical Trials: Factors to Consider
 Travel
 How far are you willing to travel if a specific clinical trial is not
offered at your institution?
 Are transportation and housing assistance provided?
 Costs
 Insurance coverage
 Additional scans and tests
 Check and double check with investigating team and insurance
company
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17. Clinical Trials: Factors to Consider
 Randomization
 A way to distribute participants between trial arms
 Experimental arm receives novel therapy
 Control arm receives standard of care
 Some trials allow participants to “crossover” to the experimental
arm if disease progression occurs while on the control arm
 Blinding to avoid bias
 Blind: The participant does not know which arm has been
assigned
 Double Blind: Both the participant and investigator do not
know which arm has been assigned
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18. Clinical Trials: Informed Consent
Informed consent is a process for the participant’s protection
 Information
Provides written and verbal information about the trial for the patient
to make an informed decision whether or not to participate
 Questions and Discussion
To gain understanding, the patient has the right to ask questions about
any aspect of the clinical trial and have adequate time to think about
and discuss the research protocol with others
 Consent Form
The patient signs the consent form to show understanding of the
information presented and voluntary participation
 On-going
As the clinical investigation progresses, new information is provided as
the situation requires
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19. Clinical Trials: Informed Consent
 Consent form includes the following information:
 Experimental nature of the trial
 Purpose of the trial
 Procedures and duration
 Possible benefits and risks
 Other treatments that might be considered
 Protect confidentiality
 The right to withdraw at any time for any reason
 Contact information in case the participants have questions or
experience side effects or problems during the trial
 and more…
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20. Reporting Side Effects
 Important to know the type and severity of side effects
experienced by participants
 Adverse Events are reported by the medical team
 Patient Reported Outcomes (PROs) are reported
directly by patients (without interpretation of
the patient's response by a clinician or anyone else)
 Collection Tool: Patient-Reported Outcomes version
of the Common Terminology Criteria for Adverse
Events (PRO-CTCAE)
http://healthcaredelivery.cancer.gov/pro-ctcae/
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21. Jane
 Breast Cancer Survivor
 Diagnosed 2011
High grade clear cell uterine cancer
 2014: Local Recurrence of uterine
cancer which progressed to
Metastatic Recurrence
 Participated in early phase
immunotherapy Clinical Trial
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22. Jane
 Did not want to repeat first-line
therapy after disease progression
 Learned more about her specific
cell type
 Did not qualify for the first two
clinical trials she was interested in
 Needed new biopsy to participate
in trial
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23. Jane
 Clinical trial team very responsive
and advocated for appointments
with different specialists when
necessary
 Trial discontinued due to side
effects
 Received extraordinary care while
on a clinical trial
 Feels safer when watched closely
on a clinical trial
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24. Jessica Safran
 Diagnosed May 2011
Stage IIIC Ovarian Cancer
 September 2013
Recurrence
 Participated in 1 Clinical Trial
 Randomized Phase II
 Considers Clinical Trial
Participation at Every Decision
Point
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25. Jessica Safran
 Participated in Surgical Clinical
Trial for Recurrence
 HIPEC: hyperthermic (heated)
intraperitoneal chemotherapy
 Became comfortable with
randomization
 Had successful debulking surgery
with excellent surgeon
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26. Jessica Safran
 Sought clinical trials for
recurrence locally and at NCI
 Did not qualify: not enough
measureable disease
 Comfortable proceeding with
standard chemotherapy
 Planning ahead and “shopping”
for clinical trials if needed in the
future
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27. Eliza Adams
 Diagnosed 2005
Stage IIB Invasive Breast Cancer
 May 2013
Metastatic Breast Cancer
 Participated in 5 Clinical Trials
 2 Observational
 3 Interventional
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28. Eliza Adams
 Front-line observational trial:
biopsies performed throughout
treatment
 Double-blind randomized phase 3
trial
 Genetic clinical trial: received
genetic counseling and genetic
information about herself and her
tumor
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29. Eliza Adams
 Phase II Clinical Trial
 Received intervention to boost
blood counts while on trial
 Disease progression prevented
continuation on trial
 Phase I immunotherapy trial
 Participates in trials to support
research, diversify treatment
options, and delay standard
chemotherapy
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30. Karen Durham
 Diagnosed 1989
Stage II Invasive Breast Cancer
 January 2009
Metastatic Breast Cancer
 Participated in 2 Clinical Trials
 Randomized Phase II
 Randomized, Double-Blind Phase 11
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31. Karen Durham
 Exceptional Responder in a
Negative Clinical Trial
 Continued to receive novel
therapy after trial ended
(nearly 6 years total)
 Did not realize the impact of so
many trips to cancer center for
treatment, scans and tests
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32. Karen Durham
 Participated in Randomized,
Double-Blind Phase II Clinical
Trial
 Un-blinded after disease
progression, was on standard
therapy (control arm)
 Cross-over to novel therapy
(experimental arm)
 Recommends clinical trial
participation to others
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33. Q&A
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34. Final Thoughts
• Clinical trials are a way to find out if new therapies are
safe, effective and better than or equal to the current
standard of care
• Clinical trials can be used to expand treatment options
• Patients who have had fewer lines of chemotherapy
can qualify for more trials
• Not every clinical trial is right for every person
• Informed Consent / Understanding
• Comfort level
• Be your own advocate: Due diligence
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35. SHARE’s Clinical Trial Matching Service
http://www.sharecancersupport.org/share-new/clinicaltrial_1/clinicaltrial/

36. Resources
 American Cancer Society: ACT/About Clinical Trials
http://www.learnaboutclinicaltrials.org/
 American Cancer Society: Informed Consent
http://www.cancer.org/acs/groups/cid/documents/webcontent/003014-pdf.pdf
 National Cancer Institute: Questions to ask your doctor
http://www.cancer.gov/about-cancer/treatment/clinical-trials/questions
 NCI Designated Cancer Centers:
http://www.cancer.gov/researchandfunding/extramural/cancercenters
 NCI Match: http://www.cancer.gov/about-cancer/treatment/clinical-trials/nci-supported/nci-match
 NCI Exceptional Responders: http://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2015-C-0146.html
 Cancer.Net (ASCO): Patient Safety and Informed Consent
http://www.cancer.net/navigating-cancer-care/how-cancer-treated/clinical-trials/patient-
safety-and-informed-consent
 FDA: Clinical Trials: What Patients Need to Know
http://www.fda.gov/ForPatients/ClinicalTrials/default.htm
 Reporting Complaints Related to FDA-Regulated Clinical Trials
http://www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTrials/Complaintsrelati
ngtoClinicaltrials/default.htm
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37. Resources: Questions to Ask (from ClincalTrials.gov)
Anyone interested in participating in a clinical study should know as much as possible about the study and feel comfortable
asking the research team questions about the study, the related procedures, and any expenses. The following questions might be
helpful during such a discussion. Answers to some of these questions are provided in the informed consent document.
 What is being studied?
 Why do researchers believe the intervention being tested might be effective? Why might it not be effective? Has it
been tested before?
 What are the possible interventions that I might receive during the trial?
 How will it be determined which interventions I receive (for example, by chance)?
 Who will know which intervention I receive during the trial? Will I know? Will members of the research team
know?
 How do the possible risks, side effects, and benefits of this trial compare with those of my current treatment?
 What will I have to do?
 What tests and procedures are involved?
 How often will I have to visit the hospital or clinic?
 Will hospitalization be required?
 How long will the study last?
 Who will pay for my participation?
 Will I be reimbursed for other expenses?
 What type of long-term follow-up care is part of this trial?
 If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends?
 Will results of the study be provided to me?
 Who will oversee my medical care while I am participating in the trial?
 What are my options if I am injured during the study?
https://www.clinicaltrials.gov/ct2/about-studies/learn#HowAreParticipants
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