[Federal Register Volume 69, Number 157 (Monday, August 16, 2004)]
[Notices]
[Pages 50386-50388]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 04-18627]


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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planning to develop draft guidance on this topic during the coming 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to the docket before and after the workshop when developing the draft 
guidance.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: [email protected].

    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: [email protected], 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: [email protected].


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://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]
BILLING CODE 4160-01-S