December
4, 2014
Margaret
Hamburg, M.D.
Commissioner
Food and
Drug Administration
10903 New
Hampshire Avenue
Silver
Spring, MD 20993-0002
Re:
Request
for Comments on FDA Activities for Patient Participation in
Medical
Product Discussions
Dear Dr.
Hamburg:
The
undersigned organizations of the Cancer Leadership Council (CLC)
represent
cancer patients, researchers, and health care
professionals. We
appreciate the opportunity to respond to the request for
comments regarding
patient participation in medical product reviews.
Current
Engagement of Patients in Advisory Committees
Patient
advocates affiliated with our organizations have been qualified
as special
government employees (SGEs) in order to serve as patient
representatives on
Food and Drug Administration (FDA) advisory committees.
Many others have
participated during public comment periods at advisory committee
meetings. These opportunities permit patient voices to be
heard at the
end of the cancer product review process. Although there
is value to
engagement late in medical product review, these opportunities
do not take full
advantage of the experience and expertise of patients and
patient
advocates. For example, patients have a valuable
perspective on unmet
medical need for those with their disease, insights about
risk-benefit
assessment and quality of life assessments, and advice about
clinical trial design
and the willingness of patients to enroll in trials of certain
design.
FDA and cancer
research and development entities would benefit from patient
advice on these
topics during early conversations about product development
plans.
Patient insights may influence trial design determinations and
may inform other
clinical development decisions.
Consultation
during Early Medical Product Discussions
We are
pleased that the agency has identified the potential for
increased patient
participation early in medical product development and has
clarified that such
participation would require a patient to be qualified as an
SGE. Patients
are willing to undergo this process in order to be engaged with
the agency and
medical product sponsors early in the regulatory process.
The
Office of Hematology and Oncology Products has been a pioneer in
the various
ways that it engages with patients on issues related to research
and
development and regulatory review of new cancer therapies.
The office has
collaborated with patient organizations, medical professional
societies, and
research foundations in meetings to consider a wide range of
topics that are
related to cancer product development and regulation.
These cooperative
meetings have considered surrogate endpoints, clinical trial
design, quality of
life measurement, risk-benefit analysis, and other research and
development
issues.
For
patient advocates, these meetings provide an opportunity to
share advice and
experience with the agency and drug developers early in the
development
process. In addition, the staffers of the office routinely
attend patient
meetings to explain and update the patient community regarding
the FDA
regulatory mission and the specific activities of the
office. The
philosophy of open and productive communication between FDA and
patients that
is embraced by the Office of Hematology and Oncology Products is
one that
should be adopted by other review offices at FDA.
Moreover, as FDA
considers additional options for patient participation in
medical product
discussions, it should protect the efforts that are already
working well.
We also
recommend that planning and coordination of the patient-focused
drug
development meetings be the responsibility of review
offices. We
appreciate that these meetings could represent a burden for
review staffers who
have significant review responsibilities and must meet
performance goals, and
that burden should be carefully considered. However, these
meetings could
be structured to provide relevant patient advice to review
staffers if those reviewers
were engaged in the design and implementation of the meetings.
Determination
of Conflict of Interest
We
observe that the determination of conflicts of interest, a
process defined in a
2008 guidance document that implements the Food and Drug
Administration
Amendments Act, seems to be resulting in the disqualification of
a significant
portion of advisory committee members from voting. This
raises questions
about the application of the conflict rules to patients and
patient advocates;
are the current rules so rigorous and the limits on the granting
of waivers so
severe that patients will be disqualified from medical product
discussions?
We urge a
discussion about the current standards for determining conflicts
of interest
and granting waivers. If the current process is depriving
the agency of
valuable expertise, the rules may need to be revisited.
Collaboration
with Patient Groups to Announce New Product Approvals
Cancer
patients carefully monitor the regulatory review process and the
consideration
of new drug applications, and the organizations that serve those
patients attempt
to communicate product approvals at the earliest possible
time. Patients
increasingly want detailed information about newly approved
drugs, including
the information that is included in product labeling. The
organizations
of the CLC see important opportunities for cooperation with FDA
to promptly
distribute information about product approvals. Although
such
collaboration is outside the scope of the medical product
discussions generally
defined in the request for comments, we believe these efforts
are a logical
outgrowth and complement to patient engagement in medical
product discussions.
We
appreciate the opportunity to offer advice about strategies for
patient
participation in FDA activities. We look forward to
continuing the
discussion with FDA on these issues, including the matter of
management of
conflicts of interest.
Sincerely,
CANCER LEADERSHIP
COUNCIL
CancerCare
Cancer
Support Community
International
Myeloma
Foundation
Kidney
Cancer Association
The
Leukemia & Lymphoma
Society
LIVESTRONG Foundation
Lymphoma
Research Foundation
Multiple
Myeloma Research
Foundation
National
Coalition for Cancer
Survivorship
National
Patient Advocate Foundation
Ovarian
Cancer National
Alliance
Pancreatic
Cancer Action
Network
Prevent
Cancer Foundation
Sarcoma
Foundation of America
Susan
G. Komen
Us TOO International Prostate Cancer Education and Support Network