CLC Comments on 21st
Century Cures Effort
(March 27,
2015)
March 27,
2015
The
Honorable Fred Upton
Chairman,
Committee on Energy & Commerce
United
States House of Representatives
Washington,
DC 20515
The
Honorable Diana DeGette
United
States House of Representatives
Washington,
DC 20515
Dear
Chairman Upton and Representative DeGette:
The
undersigned organizations represent cancer patients,
physicians, pharmacists, researchers,
and other health professionals who are engaged in
efforts to improve cancer
treatment and enhance the overall quality of cancer
care. We appreciate
the opportunity to comment on the January 2015
discussion draft, “21st
Century Cures Act.”
Our
comments will focus on the following objectives:
·
Balancing
the speed of regulatory
review against an assurance that new cancer drugs are
safe and effective;
·
Preparing
for review of precision
medicine drugs;
·
Ensuring
that “patient-focused
drug development efforts” are reflected in FDA programs
and regulatory
approaches;
·
Encouraging
the consideration of
patient-reported outcomes data in the review process;
·
Building
data-collection and
sharing efforts on a firm foundation of successful
clinical trials data
reporting;
·
Defining
the new roles of patient
advocacy and patient research foundations in the
therapeutic development
process; and
·
Ensuring
that new commissions,
panels, and reports serve the needs of patients, do not
duplicate existing
commissions and reporting requirements, and do not
create unreasonable burdens
for federal agencies.
Ensuring
a
Strong Regulatory Review Process
Cancer
patients, physicians, and other health care providers
have an interest in
eliminating any inefficiencies in the regulatory review
process and ensuring
patients access to safe and effective drugs at the
earliest possible
time. However, we want to be sure that those drugs
that are approved by
the Food and Drug Administration (FDA) are safe and
effective and will provide
clinical benefit to patients.
Cancer
patients and their health care teams have benefited
greatly from the efforts of
the Office of Hematology and Oncology Products to
improve the cancer drug
review process and expedite the review of cancer drugs
whenever possible.
The Office, within the Center for Drug Evaluation and
Research (CDER), has made
aggressive but appropriate use of the expedited programs
for serious
conditions, as defined by the Guidance for Industry
dated May 2014. These
expedited programs include fast track designation,
breakthrough therapy
designation, accelerated approval, and priority review
designation.
The 2014
review record of the Office of Hematology and Oncology
Products is
impressive. Drugs were approved for treatment of
advanced ovarian cancer,
acute lymphoblastic leukemia, three types of blood
cancer, metastatic non-small
cell lung cancer, and melanoma. This drug review
and approval record was
accomplished through use of the expedited development
and review pathways;
Prescription Drug User Fee Act (PDUFA) goals were met in
almost all 2014 drug
reviews and most drugs were approved on the first review
cycle.
In light
of the record of Office of Hematology and Oncology
Products, we offer cautions
about two proposals that are included in the
draft. First, we are not
persuaded that confirmatory trial requirements should be
eliminated for those
drugs that are subject to accelerated approval.
Those requirements should
remain in place for those drugs approved on the basis of
surrogate endpoints.
Second, we are concerned about a suggestion that
supplemental approvals might
be based on summaries of data, without a requirement of
submissions of the
underlying data. FDA has a proven track record for
efficient review of
cancer drugs, and changing the amount of data necessary
for an application is
neither necessary nor advisable.
Instead
of eliminating post-approval study requirements or
changing data requirements
for approval, we encourage evaluation and replication of
the work of the Office
of Hematology and Oncology Products. That effort
will identify effective
ways to utilize current expedited review
mechanisms.
Preparing
for
Review of Precision Medicine Drugs
Although
we are pleased with the performance of the cancer review
office to date and applaud
the willingness of the office staff to collaborate with
patient advocacy groups
and professional societies on issues ranging from
clinical trial design to
identification of surrogate endpoints, we see
significant challenges for the
office and for all of FDA in the future.
As we
move more completely into the age of precision medicine,
the office will need
assurance that all personnel possess the skills for
review of targeted
therapies. In addition, FDA reviewers need more
flexibility to attend and
participate in scientific and medical meetings.
These meetings are an
opportunity for continuing medical education and for
staying current on
developments related to precision medicines, and these
opportunities should be available
to review staff.
We note
that the committee has left in its discussion draft a
“placeholder” for FDA
personnel issues. We urge that this placeholder be
replaced by revisions
to FDA authority that will streamline hiring
processes. In addition,
travel and ethics rules should be addressed – if
necessary, in legislative
language – to guarantee FDA staff the ability to attend
important meetings in
their field.
Patient-Focused
Drug
Development Efforts
The Food
and Drug Administration Safety and Innovation Act
(FDASIA) included a number of
important patient-focused drug development
efforts. The patient-focused
drug development meetings have been of special interest
to patient advocates.
We appreciate that the agency
recognizes the importance of involving patients in drug
development issues
consistent with FDASIA requirements. Although we are concerned
about adding
responsibilities to the portfolios of review teams,
which should be primarily
focused on new product review, we would like to see more
engagement of
reviewers in the planning and execution of the
patient-focused drug development
meetings. This is the most efficient means of
ensuring that the
patient-focused meetings undertaken by the agency are
integrated into the
operations and inform the thinking of the agency.
Patient-Reported
Outcomes
in the Regulatory Review Process
The
initial section of the discussion draft encourages the
use of patient
experience data to inform the risk-benefit
assessment. We are pleased
that the draft seems to encourage serious consideration
of patient-reported
outcomes in the regulatory process, but we recommend
more specific definitions
be included in this section of the bill. If
patient-reported outcome data
are to be utilized in a data-driven regulatory process,
the standards for those
data must be well-defined. It will not benefit
patients if the agency is
encouraged to consider patient anecdotes that do not
meet reasonable data
standards.
The
committee should consider setting goals for approval of
patient-reported
outcome tools by the agency and encouraging reference by
the agency to the
information provided through those validated
tools.
Building
Successful
Data-Collection and Data-Sharing Initiatives
We are
strong supporters of a movement toward “big data”
collection and sharing to
fuel strong cancer drug development and clinical care
improvement. In
fact, a number of our organizations have developed data
registries that track
the treatment and outcomes of our patients. We
urge that any federal
involvement in data collection and sharing efforts be
built on a strong
foundation. To that end, we encourage that recent
findings of limited
compliance with the reporting requirements of www.clinicaltrials.gov
be considered
by the committee. These findings should inform
efforts to strengthen
clinical trials reporting. In addition, a stronger
trials results
reporting system might serve as a foundation for other
data collection efforts.
Defining
the
Roles and Responsibilities of New Commissions and
Panels
A review
of the discussion draft raises some concerns related to
the number of new
commissions, consortia, and reporting requirements that
are authorized.
Our reservations are two. First, we are concerned
that some of the new
research and regulatory efforts and initiatives may be
redundant of existing
research and regulatory programs. For
example, has the National
Center for Advancing Translational Sciences been
evaluated to determine if
parallel clinical research programs are necessary?
Has the regulatory
science collaboration between the National Institutes of
Health (NIH) and FDA
been reviewed? What are the results of the
Critical Path Initiative?
Second,
we are concerned that the new consortia, commissions,
and reports will be
accompanied by significant costs that cannot easily be
absorbed by NIH and FDA
and that additional resources for these responsibilities
will not be available.
Understanding
the
Roles of Nonprofit Research Foundations
If the 21st
Century Cures Consortium and the Medical Products
Innovation Advisory
Commission are retained after the committee considers
any possible overlap with
existing programs and the cost associated with new
commissions, we recommend
that membership of both groups be redefined to include
more members drawn from
patient advocacy organizations and robust representation
from non-profit,
patient-driven research foundations. The Cures
Consortium would number 22
members, including 8 representatives of the
biopharmaceutical and medical
device industries and 9 who shall be “representatives of
academic researchers,
patients, health care providers, and health care plans
and insurers, to be
appointed by the Comptroller General of the United
States, after soliciting
nominations.” The Medical Products Innovation
Advisory Commission would
include 17 members, and the discussion draft does not
indicate that any will be
patient advocates or representatives of non-profit
research foundations.
We
believe that the membership categories for both of these
panels should be
redrafted to ensure strong representation of patient
advocates and inclusion of
individuals from non-profit research
foundations. Patients can
speak to unmet medical needs, and those representing
research foundations may
also bring extensive experience and expertise about
research and development of
new treatments. Over the last decade, there has
been nothing short of a
revolution in the operation of patient-driven research
foundations. These
groups have refined the manner in which they invest
their resources, expanding
beyond investigator-initiated grants to therapy
development programs. In
addition, many of them have been innovators in clinical
trial design and
recruitment and are pioneering data collection and
sharing efforts. Their
expertise must be reflected in the deliberations of
these commissions, and that
can be accomplished by guaranteeing robust membership
from their ranks.
Ensuring
Access
to New Therapies
We note
that the discussion draft focuses primarily on the
development and regulatory
review of new therapies, and we have confined our
comments to those
topics. However, it is critical that cancer care
delivery systems ensure
patients access to the treatments of the 21st
century. We are
actively involved in payment and delivery reform efforts
that will ensure
access to quality, affordable, and sustainable cancer
care.
Thank you
for the opportunity to participate in the process of
developing legislation to
encourage development of new treatments for the new
century.
Sincerely,
Cancer
Leadership Council
Association for Molecular
Pathology
CancerCare
Cancer Support Community
Fight Colorectal Cancer
Hematology/Oncology Pharmacy
Association
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
Us TOO
International Prostate Cancer Education and Support
Network
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