[Federal Register: August 3, 2005 (Volume 70, Number 148)]
[Notices]
[Page 44660-44662]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr03au05-161]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. 2004N-0355]
Critical Path Initiative; Developing Prevention Therapies;
Planning of Workshop
AGENCY: Food and Drug Administration, HHS.
ACTION: Request for Comments.
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SUMMARY: The Food and Drug Administration (FDA) is planning a 2-day
workshop to explore approaches and potential obstacles to developing
drugs, disease biomarkers, medical devices, and vaccines to prevent or
reduce the risk of illness. The agency plans to hold the workshop as
part of its Critical Path Initiative. Speakers at the workshop will be
asked to discuss the challenges in developing chemoprevention therapies
(i.e., prevention therapies other than lifestyle changes, dietary
supplements, or dietary choices that could reduce the risk of certain
illnesses such as cancer, diabetes, and obesity). Because prevention of
illness is widely recognized to be an important goal and the possible
scope of this workshop is very broad, FDA welcomes comments related to
the scope of this workshop.
DATES: Submit written or electronic comments by November 1, 2005.
General comments are welcome at any time.
ADDRESSES: The FDA invites you to submit written comments on the
proposed scope of the workshop. Please submit comments to the Division
of Dockets Management (HFA-305), Food and Drug Administration, 5630
Fishers Lane, rm. 1061, Rockville, MD 20852. Submit electronic comments
to http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/dockets/ecomments.
FOR FURTHER INFORMATION CONTACT: Nancy Stanisic, Center for Drug
Evaluation and Research (HFD-05), Food and Drug Administration, 5600
Fishers Lane, Rockville, MD 20852, 301-827-1660, FAX: 301-443-9718, e-
mail: Stanisicn@cder.fda.gov.
SUPPLEMENTARY INFORMATION:
I. Background
The development of methods to prevent disease has been the single,
most effective advance in healthcare in the past century, particularly
in developed countries. The widespread ravages of smallpox, infantile
diarrhea, plague, cholera, typhoid, and polio are gone from the United
States.
The challenge that lies ahead is to prevent the diseases that still
ravage our population, including: Heart disease, cancer, diabetes,
Alzheimer's disease, and others. In recent decades, substantial effort
has been made in the chemoprevention or early intervention for some of
the top killers in the United States, notably cardiovascular disease
and some cancers. Examples of effective preventive interventions
include the aggressive treatment of hypertension to reduce the risk of
stroke, statins to lower cholesterol and decrease the risk of a
myocardial infarction, the use of low-dose aspirin and beta blockers to
prevent death in patients after a myocardial infarction, tamoxifen to
reduce the risk of recurrent breast
[[Page 44661]]
cancer, aggressive control of blood glucose to reduce the long-term
consequences of diabetes, and flu and pneumonia vaccination programs to
reduce morbidity and mortality.
Significant advances have also been made in the early
identification of healthy individuals at risk of developing disease.
Examples of predictors include genetic markers, such as BRCA 1 and 2
for malignancy; pap tests for identification of patients at risk for
cervical cancer; genetic alpha-1-antitrypsin deficiency for lung
disease; colonoscopy to identify polyps that predict an increased risk
of colon cancer; and family history, obesity, and ethnicity for type II
diabetes mellitus. Ongoing work in genomics and proteomics promises to
identify additional markers to predict specific health risks and
potential targets for intervention.
Although markers have been identified, candidate therapies require
prospective testing in clinical trials. The design and conduct of
chemoprevention trials offer substantial challenges. For example, in
the Women's Health Initiative, we learned that the epidemiologic study
results of the use of conjugated estrogens to prevent heart disease
could not be replicated in the randomized, double-blind clinical trial
setting. The Celebrex trial gives another example that prevention
studies, in this case polyp prevention trials, must be of sufficient
duration to ensure that the risks of long-term use of drugs are
captured. These risks may be unexpected and the Data Safety Monitoring
Boards need to pay careful attention as signals arise.
II. FDA Critical Path
On March 16, 2004, FDA published its Critical Path report,\1\ aimed
at identifying potential problems and solutions to ensure that
breakthroughs in medical science can be efficiently translated to safe,
effective, and available medical products. In the report, FDA
underscored the importance of FDA collaboration with academic
researchers, product developers, patient groups, and other stakeholders
to make the critical path more predictable and less costly. This
workshop and any activities that result from the workshop are part of
that broad effort.
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\1\ For the complete report, see http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/oc/initiatives/criticalpath
.
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III. Topics Related to Planning the Public Workshop
Because the range of potential topics that could be discussed at
such a workshop is so wide, we are seeking the public's input on what
key topics should be addressed at this initial meeting.
Although the prefix ``chemo-'' is often used in relation to
treatments for cancer, we are using the term ``chemoprevention'' in
this notice to describe prevention therapies other than lifestyle
changes, dietary supplements, or dietary choices that could reduce the
risk of certain illnesses. We welcome comments on the use of the term
``chemoprevention.''
What follows is a list of topics and questions we have identified
for possible discussion at the workshop. We welcome comment on whether
these topics and questions are appropriate for discussion at a workshop
on chemoprevention therapies? Are there other related issues that
should be discussed at the workshop? What are they? Currently, we
envision a 2-day workshop, with the first day devoted to identifying
hurdles and challenges in designing and implementing chemoprevention
studies from a broad perspective. The second day may consist of
breakout sessions devoted to specific diseases or disease categories.
We welcome input on the format for the 2-day workshop.
Does the following list of questions reflect the kinds of questions
we should try to answer at a 2-day workshop on chemoprevention
therapies? What questions would you be interested in having answered?
In addition to the following topics, what other topics should be
included in the scope of the meeting?
1. What have our successes been so far, and what lessons have we
learned from past experience with regard to the development of the
following preventive therapies:
a. Vaccines
b. Cardiovascular disease
c. Cancer
i Breast
ii Colon polyps
2. Which diseases are the most promising with regard to development
of chemoprevention therapies?
3. What options are available now for identifying populations at
risk for those diseases?
a. Screening
b. Genomics
c. Other
4. What techniques are available for assessing the risks and
benefits of new therapies in prevention?
5. How much risk from the candidate therapy is acceptable?
6. Are there specific regulatory concerns in developing
chemopreventions (e.g., Long trials, safety and efficacy issues,
registries)? And what steps can FDA take to facilitate development in
this area, such as the following?
a. Mechanisms to streamline the regulatory process
b. Mechanisms to facilitate the scientific process and clinical
trials
i. To better and more efficiently answer questions regarding
product efficacy
ii. To better and more efficiently answer questions regarding
product safety
7. What are some of the obstacles facing manufacturers who wish to
develop new or existing compounds for chemoprevention? For example, are
there specific industry perspectives that need to be considered?
8. What patient perspectives are important to consider?
We have proposed the following topics and questions for discussion
on the second day during breakout sessions. Are these appropriate? What
other issues would you be interested in discussing at these breakout
sessions?
1. Cancer prevention issues
a. What characteristics of particular cancers make prevention
promising?
b. What characteristics from epidemiologic, early trials, or other
models make particular drugs promising?
c. What trial design issues should be addressed (e.g., endpoints,
surrogates, population, adverse event data collection)?
d. Are there obstacles to marketing prevention drugs?
2. Cardiovascular prevention issues
a. What characteristics of cardiovascular disease make prevention
promising?
b. What characteristics from epidemiologic, early trials, or other
models make particular drugs promising?
c. What trial design issues should be addressed (e.g., endpoints,
surrogates, population, adverse event data collection)?
d. Are there obstacles to marketing prevention drugs?
3. Cerebrovascular prevention issues
a. What characteristics of cerebrovascular disease make prevention
promising?
b. What characteristics from epidemiologic, early trials, or other
models make particular drugs promising?
c. What trial design issues should be addressed (e.g., endpoints,
surrogates, population, adverse event data collection)?
d. Are there obstacles to marketing prevention drugs?
4. What other conditions should be discussed?
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IV. Submission of Comments
Interested persons may submit written or electronic comments to the
Division of Dockets Management (see ADDRESSES). Submit a single copy of
electronic comments or two paper copies of any mailed comments, except
that individuals may submit one copy. Comments are to be identified
with the docket number found in brackets in the heading of this
document. Received comments may be seen in the Division of Dockets
Management between 9 a.m. and 4 p.m., Monday through Friday. You can
also view received comments on the Internet at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/ohrms/dockets/dockets/dockets.htm
Dated: July 28, 2005.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 05-15282 Filed 8-2-05; 8:45 am]
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