for Patients and Caregivers |
& Unity |
Guidance for the NCI, the Industry, and the FDA
Research Funding and Insurance
we provide patient perspectives on a variety of issues, and invite you to E-mail
your comments, suggestions, and thoughts.
What is the Purpose of Research Advocacy?
Patients and caregivers endure their
diseases and sometimes the inadequacies of existing therapies. We
are the ones recruited for testing of the investigational therapies,
which, in some cases, will ultimately be utilized by many of us. It
is we who lose the most when clinical trials do not meet enrollment
goals and fail to answer the study question ... a very common and
Since we live with the disease and must
choose between therapeutic options, we can offer insights that will
not necessarily be recognized by medical professional such as:
How patients and treating physicians will
judge the protocol as a therapeutic decision,
Possible deficiencies in the informed consent process,
The significance of the events that are measured to estimate
Concerns with the enrollment criteria that may become unnecessary
obstacles to enrollment or that may call into question how well the
study findings will apply to the population with the disease.
I wish to participate because I feel that my
motivations, experiences, and training make me uniquely qualified to
contribute the patient perspective in a positive way.
I am a caregiver to my spouse; she is a
fifteen-year survivor of lymphoma. Together we have experienced the
uncertainties of treatment decisions, the disappointments and
successes - and have considered and participated in multiple
clinical trials and therefore have first-hand experience with the
clinical trial consent process.
I also serve the lymphoma patient community
on a daily basis, moderating support forums and updating our
non-profit website resource in response to patient questions, which
includes substantial information on clinical trials.
My experience includes providing support and
the review of studies for a broad range of lymphomas: low and high
risk, indolent and aggressive, including types that are managed
conservatively or that can have a rapid fatal clinical course if not
Perspectives on Clinical Trial Design
The need to make progress against cancers is
urgent and is dependent on the conduct and completion of
well-designed clinical trials. There is no other way.
Clinical trials must have the potential to
answer relevant clinical questions, while providing also a strong
therapeutic rational for participation. By this I mean that all
protocols within the study must have a potential to provide clinical
benefit roughly equivalent or superior to available standard
therapies, or best supportive care.
Failing to meet the first requirement there
is no purpose in doing the study. Failing to meet the second we
have a high probability that the study will not complete enrollment,
which means that some patients will have been exposed to risks for no benefit to society. Further, the conduct of poorly
designed trials dilutes the pool of available participants, making
it more challenging for better-designed studies to complete
I have a traditional
views about clinical trial design. I fully endorse the importance
of randomized controls in many cases, and sufficient sample size in order to
objectively assess efficacy and risks; and in the selection of
eligible participants by criteria that reflect the population the
therapy is ultimately intended to serve.
I am keenly interested in the discovery and
validation of biomarkers that may be used as surrogates for clinical
benefit to accelerate the assessment of future therapeutics that may
help to individualize therapeutic decisions - helping patients to
avoid unproductive toxicities, which can decrease the range of
effective therapies that may be used later by the same patient.
Developing the rationale for a clinical
study as a therapeutic decision requires insights and knowledge of
best practice and the natural history of the disease in the various
clinical settings. While this challenging task is best carried out
by experts in the field, it must be communicated in an intelligible
way to the would-be participants in order to be judged a reasonable
alternative to standard therapies and to achieve a high level of
informed consent. Thus, I believe that the rationale for
participation needs to be considered early and throughout the design
Finally, while I have received training in
the review of clinical trials from the FDA patient representatives
program and from independent work, I am aware that as a layperson I
will have gaps in my knowledge and therefore I will endeavor to
provide input mainly from the perspective of the patient – who has
the disease and is considering participation.
Patient Advocacy Background and
Invited participant in
NCI Progress Review Group for Blood Cancers in 2001,
NCI Biospecimen Best Practices workshops, and the
NCI Technical Evaluation Panel: “Development of a Common Biospecimen
Coordination System and Informatics Infrastructure for NCI Prostate
Faculty member in a workshop sponsored by FDA, NCI, ASCO, and Duke
“Accelerating Anticancer Agent Development and Validation Workshop”
AACR/ASCO: “Methods in Clinical Cancer Research,” 2011 and 2012
Provided patient perspectives on clinical trial topics to various
stakeholders as an invited speaker, such as
“The Demand for Innovation vs. Safety”,
Leigh Thompson Renaissance Conference;
“Announcing The Cancer Genome Atlas
Project,” sponsored by NCI at the National Press Club; and
“Evaluating Online Medical & Support
Information, ” sponsored by the Lance Armstrong Foundation, and
Leukemia and Lymphoma Society.
"Why Patients Participate in Clinical
Trials" CARE subcommittee, CALGB
Our study on “Interest, Attitudes and
Participation in Clinical Trials among Patients with Lymphoma” was
published by ASCO in 2009 (e19514).
Presently, serve as a patient representative
and consultant to the Food and Drug Administration (since 2002) and
have participated in four FDA advisory committee deliberations.
Also serve as a patient advocate on the
Alliance cooperative group (formerly CALGB).
Vaccines and Autism link:
It's easy to
confuse correlation with causation -
Tony L. Hines
Does Progression Free Survival
predict better survival in Follicular Lymphoma?
Letter to CMS on Radioimmunotherapy
Perspectives on Drug
Reviews, Vaccine misinformation, Level of Evidence
Open Letter to SEER:
Regarding capturing statistics for lymphoma subtypes PAL
The SEER website has improved, but the utility of the
statistics for non-Hodgkin's lymphomas continue to suffer because
subtypes of the disease are not yet provided.
UPDATE FROM SEER:
Biobank Best Practices: Patient Perspectives on the
issue of transfer of tissue for clinical use –
particularly for use in translational clinical research - PDF
"we may not have fully considered that requests for
transfer of tissue for clinical use could many times be in
harmony with the ultimate objective of achieving “personalized
medicine,” and the oft-stated principle of “partnering
with patients” – particularly when used to determine
eligibility for clinical trials."
For Patients and Caregivers:
Guidance: How to evaluate medical claims and data
Is this strong or weak information? Who is saying it?
It is reproducible? Is it free of bias? ...
What NOT To Say To A Cancer Survivor
Whether you are a newly diagnosed survivor or someone who loves
him or her, cancer rocks your world. We survivors have our own
issues to face, but those of you who care for us can help by
learning what not to say. We know you mean well, but please avoid
the following platitudes ....
URGENCY & UNITY
For the NCI, the Industry, and the FDA
To help accelerate the development and evaluation
of investigational therapies for lymphomas.
attitudes towards trials: Report from our Lymphoma Patient Survey regarding
interest and participation in clinical
Adaptive Design Conference, July 2006, Washington
Goals with Meeting the Clinical Needs of the Participants
with narrative) PDF
Harmonizing Research Goals with
Meeting the Clinical Needs
of the Participants - Apr 2006 -
Presentation at Favrille Investigators Conference
Study Proposal: Combine molecular
profiling research with evaluating first-line use of
patient-specific idiotype cancer vaccines -
Update: No longer feasible given the disappointing outcomes from
vaccine phase III trials.
Advocating for Coordinated Immunotherapy Research
Eliminating Suffering and Death Due to Cancer by 2015: The Future of Cancer Research
Let's not forget Immunotherapy! - A letter to Dr. Von Eschenbach
"The patient is waiting"
- patient perspectives
on aspects of toxicity and the need for innovation -
an appeal for NBN in a
brochure format PDF
Ablation for lymphoma? PAL
trial design and participation - We consult with
Patients, the FDA, Drug Sponsors, the NCI, investigators, and
physicians. Our goal is to increase participation in order
to accelerate progress against the disease. How else?
Ablation for lymphoma? PAL
WHEN? Rationale for the National Biospecimen Network
There's an urgent need for new approaches and tools to accelerate
clinical research, such as the National Biospecimen Network.
One reason that even well-controlled studies fail to provide
definitive answers, it seems, is they are often unable to account
for underlying biological differences in the tumors and patients.
AND INSURANCE COVERAGE