[Federal Register: August 16, 2004 (Volume 69, Number 157)]
[Notices]
[Page 50386-50388]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr16au04-73]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. 2004N-0355]
Scientific Considerations Related to Developing Follow-On Protein
Products
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop.
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SUMMARY: The Food and Drug Administration (FDA) is announcing a public
workshop on scientific and technical considerations related to the
development of follow-on protein pharmaceutical products. The agency is
planning to develop draft guidance on this topic during the coming
year. The purpose of this workshop is to obtain input from interested
persons on the topics outlined in this document related to developing
and approving follow-on protein pharmaceutical products. The agency
will consider presentations made at the workshop and comments submitted
to the docket before and after the workshop when developing the draft
guidance.
DATES: The public workshop will be held on Tuesday, September 14, 2004,
from 8:30 a.m. to 5 p.m. and Wednesday, September 15, 2004 from 8 a.m.
to 12 noon. Submit requests to make a presentation by September 7,
2004.
ADDRESSES: The public workshop will be held at the University of
Maryland--Shady Grove Conference Center, 9630 Gudelsky Dr., Rockville,
MD 20850.
Submit written comments on scientific topics related to follow-on
protein products to the Division of Dockets Management (HFA-305), Food
and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD
20852. Two copies of any comments are to be submitted, 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.
FOR FURTHER INFORMATION CONTACT:
To register to present: Marilyn Welschenbach, Center for Drug
Evaluation and Research (HFD-121), Food and Drug Administration, 5600
Fishers Lane, Rockville, MD 20852, 301-443-5089, FAX: 301-443-5245, e-
mail: Marilyn.Welschenbach@fda.gov.
With regard to the scientific topics outlined in this notice: Keith
Webber, Center for Drug Evaluation and Research, Food and Drug
Administration (HFD-121), 5600 Fishers Lane, Rockville, MD 20852, 301-
443-5089, FAX: 301-443-5234, e-mail: Keith.Webber@fda.gov, or Chris
Joneckis, Center for Biologics Evaluation and Research (HFM-1),
[[Page 50387]]
Food and Drug Administration, 1401 Rockville Pike, Rockville, MD 20892,
301-827-2000, e-mail: Christopher.Joneckis@fda.gov.
SUPPLEMENTARY INFORMATION:
I. General Information
During the past several years, FDA has received numerous inquiries
concerning how a sponsor may scientifically demonstrate that its
protein pharmaceutical product is similar enough to a product that FDA
has licensed under the Public Health Service (PHS) Act or approved
under the Federal Food, Drug, and Cosmetic Act to obtain licensure or
approval without conducting certain studies that would otherwise be
necessary. This public workshop is not intended to address legal or
regulatory issues. Because of the scientific complexity of protein
pharmaceutical products, FDA intends to conduct an extensive public
dialogue on the scientific issues relating to the development and
approval of such products. For the purposes of this workshop, we use
the term ``follow-on protein product'' to refer to a protein that is
intended to be a similar version or copy of an already approved or
licensed protein pharmaceutical product. Such proteins might be
produced through biotechnology or derived from natural sources. (This
public workshop is not intended to address ``second-generation protein
products'' which we have tentatively defined as products that are
similar to an already approved or licensed product but which have been
deliberately modified to change one or more of the product's
characteristics (e.g., to provide more favorable pharmacokinetic
parameters or to decrease immunogenicity)). This public workshop is
concerned only with scientific issues relating to follow-on protein
products.
On March 16, 2004, in its Critical Path report, available at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/oc/initiatives/criticalpath
, FDA announced an initiative
to identify the problems and some potential solutions to ensure that
breakthroughs in medical science can be 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. Consistent with the Critical
Path Initiative, FDA is seeking input from its broad stakeholder
community as it begins the process of exploring the scientific
framework for developing and approving follow-on protein products.
II. Information on the Public Workshop
A. Why Are We Holding This Public Workshop?
It is critical that the agency solicit the scientific and
technological perspectives of manufacturers, academia, and other
interested persons to determine the state of the science as it relates
to protein characterization, production, and assessment of similarity.
Such information will be critical to any guidance on follow-on protein
products.
B. Where Will This Public Workshop Be Held?
University of Maryland--Shady Grove Conference Center, 9630
Gudelsky Dr., Bldg. II, rm. 1422, Rockville, MD 20850.
C. When Will This Public Workshop Be Held?
The public workshop will be held on September 14, 2004, from 8:30
a.m. to 5 p.m. and September 15, 2004, from 8 a.m. to 12 noon.
D. How Will the Public Workshop Be Organized?
The agency is seeking input on a series of scientific topics (see
section III of this document) and is asking interested persons to make
presentations on these and other pertinent scientific topics. A panel
of agency experts will listen to the presentations organized by the
categories listed in section III of this document, after which they may
ask followup questions of the presenters.
E. How Can I Participate?
1. In Person
Persons who wish to make a presentation during the public workshop
must file an electronic, written, or facsimile notice of participation
with Marilyn Welschenbach by September 7, 2004 (see FOR FURTHER
INFORMATION CONTACT). The notice of participation shall contain the
speaker's following information:
Name
Title
Business affiliation, if any
Address
Telephone number
Fax number
A brief summary of the presentation
Designate topic categories A through F (see section III of
this document) for the presentation
Approximate amount of time requested for the presentation
(presentations should be limited to 10 minutes in duration).
We recommend that individuals and organizations with common interests
consolidate or coordinate their presentations and request time for a
joint presentation. After registration has closed, FDA will inform
participants of the amount of time available for their presentations
based on the final agenda and on which day they will be scheduled to
present. Persons requiring a sign language interpreter or other special
accommodations should notify Marilyn Welschenbach by September 1, 2004.
2. In Writing
FDA has established a public docket for comments. Comments can be
submitted until November 12, 2004. It is important that comments
submitted to the docket be identified with the docket number found in
brackets in the heading of this document. Submit written comments to
the Division of Dockets Management (see ADDRESSES).
F. Is There a Registration Fee for This Public Workshop?
There is no registration fee for this public workshop.
G. What if I Have Scientific or Logistical Questions?
If you have any logistical questions about the public workshop,
please contact Marilyn Welschenbach; scientific questions may be
addressed to Keith Webber or Chris Joneckis. Contact information is
listed under the FOR FURTHER INFORMATION CONTACT section of this
document.
H. Can I Get a Transcript of This Public Workshop?
A transcript of the public workshop will be available from the
Division of Dockets Management, approximately 15 business days after
the workshop at a cost of 10 cents per page. The transcript of the
workshop will also be available for public examination at the Division
of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday.
III. Background Information
FDA seeks comment on the following topics and other scientific
issues related to follow-on protein products:
A. Manufacturing Issues
1. What aspects of the manufacturing process determine the
characteristics of a protein product whether produced through
biotechnology or derived from natural sources?
2. What parts of the manufacturing process should the agency focus
on when assessing similarity between products?
B. Characterization
1. What is the capability of current analytical technology to
adequately characterize protein products?
[[Page 50388]]
2. Are there new technologies that hold promise for helping to
characterize proteins?
3. What factors, including quality attributes, impurity profiles,
and changes in the manufacturing process, should be considered when
assessing similarity of different protein products?
4. Is it possible to accurately predict safety and efficacy from
analytical studies?
C. Immunogenicity
1. How, and to what extent, should immunogenicity be evaluated for
a follow-on protein product?
2. Under what circumstances should comparative immunogenicity
studies be conducted?
D. Preclinical and Clinical
1. When and how would it be appropriate to streamline or eliminate
certain animal or human studies during development of a follow-on
protein product?
E. Potency and Surrogates for Efficacy and Safety
1. What factors should be considered regarding bioactivity and
potency assays used for comparing two products?
2. What is the role of in vitro and in vivo assays for use as
surrogates in establishing safety and efficacy?
F. Terminology
1. Please comment on the appropriateness of this notice's working
definition of ``follow-on protein'' as a protein that is intended to be
a similar version or copy of an already approved or licensed protein
pharmaceutical product.
2. Please comment on this notice's working definition of a
``second-generation protein product'' as a product similar to an
already approved or licensed product but which has been deliberately
modified to change one or more of the product's characteristics (e.g.,
to provide more favorable pharmacokinetic parameters or to decrease
immunogenicity).
Dated: August 10, 2004.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-18627 Filed 8-11-04; 11:15 am]
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