[Federal Register: April 22, 2004 (Volume 69, Number 78)]
[Page 21839-21840]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]



Food and Drug Administration

[Docket No. 2004-N-0181]

Critical Path Initiative; Establishment of Docket

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.


SUMMARY: The Food and Drug Administration (FDA) is establishing a 
public docket to obtain input on activities that could reduce existing 
hurdles in medical product design and development. As described in a 
recently released Report, ``Innovation/Stagnation: Challenge and 
Opportunity on the Critical Path to New Medical Products,'' there is an 
urgent need to modernize the product development toolkit, to make the 
development process more predictable and less costly. FDA is seeking 
input in identifying and prioritizing the most pressing medical product 
development problems, and the areas that provide the greatest 
opportunities for rapid improvement and public health benefits. To this 
end, we are establishing this open docket to obtain input from 
industry, patients, academics investors, and all interested parties.

DATES: Submit written or electronic comments through July 30, 2004.

ADDRESSES: Submit written comments concerning this document 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: Lisa Rovin, Office of the Commissioner 
(HFP-1), Food and Drug Administration, 5600 Fishers Lane, Rockville, MD 
20857-0001, 301-827-1443.


I. Background

    On March 16, 2004, FDA released a report, ``Innovation/Stagnation: 
Challenge and Opportunity on the Critical Path to New Medical 
Products.'' (The full report is available at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/oc/initiatives/criticalpath/whitepaper.pdf.
) The report notes the recent 

slowdown in new medical products submitted for approval to FDA, and 
describes ways in which the product development process, the ``critical 
path,'' could be modernized to make product development more 
predictable and less costly. According to Acting FDA Commissioner 
Lester Crawford, ``A new focus on updating the tools currently used to 
assess the safety and efficacy of new medical products will very likely 
bring tremendous public health benefits.''
    Recent investments in basic medical research and translational 
research are intended to promote scientific discoveries and move some 
of them into medical testing. At that point, however, a potential 
medical product's journey from concept to commercialization is far from 
complete. To produce a commercial medical product, developers must 
successfully negotiate a ``critical path'' to ascertain whether the 
potential drug, device, or biologic is effective and sufficiently safe 
for use, and how it can be safely and reliably manufactured. Each of 
the three dimensions of the critical path--assessment of safety 
testing, proof of efficacy, and industrialization--presents its own set 
of scientific and technologic challenges, often unrelated to the 
science behind the mechanism of action of the product.
     The ethics of human testing required that there 
be a reasonable assurance of safety before people are exposed in 
clinical trials. The tools used to predict preclinical safety (e.g., 
animal toxicology) are time consuming and cumbersome. In some cases, 
particularly for assessment of products based on recent innovative 
science, entirely new tools must be developed. There is an urgent need 
for new biomarkers for evaluating safety during human trials.
     Demonstrating the medical effectiveness of a 
product is one of the most difficult challenges in product development. 
Even identifying the best way to assess whether a product is effective 
(what symptoms or physiologic indicators should be followed, and for 
how long) can present significant unknowns.
     Product development companies must figure out 
how to manufacture large amounts of the product reliably. Turning a 
laboratory prototype into a mass-produced medical product requires 
solutions to problems in physical design, characterization, 
manufacturing scaleup and quality control. These problems can be rate-
limiting for new technologies, which are frequently more complex than 
traditional products.
    Because of its unique vantage point, FDA can work with outside 
experts in companies and the academic community to coordinate, develop, 
and/or disseminate solutions to critical path problems, to improve the 
efficiency of product development industrywide.
    The first step is to identify and prioritize the most pressing 
medical product development problems, and the areas that provide the 
greatest opportunities for rapid improvement and public health 
benefits. It is critical that we enlist all relevant stakeholders in 
this effort. Such a national ``Critical Path Opportunities List'' is 
intended to bring concrete focus to tasks (whether best undertaken by 
industry, academia, FDA, by others, or jointly) that can modernize the 
critical path.
    For additional information, you may visit FDA's critical path home 
page at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/oc/initiatives/criticalpath.

[[Page 21840]]

II. Request for Comments

    We are seeking input on identification of the most pressing 
scientific and/or technical hurdles causing major delays and other 
problems in the drug, device, and/or biologic development process, as 
well as proposed approaches to their solution. For each critical path 
hurdle, we are particularly interested in receiving the following 
information. Please note that all material submitted to this docket 
will be publicly available.
    1. Hurdle Identification. Please describe the product development 
issue, the nature of the evaluation tool that is out-of-date or absent, 
how this problem hinders product development, and how a solution would 
improve the product development process. Please be as specific as 
    2. Please rank each hurdle identified in Question 1, above, in 
priority order according to which hurdles create the most severe 
product development problems. That is, which problems present the 
greatest opportunity for improving product development processes? Our 
goal is to identify those aspects of product development that would 
most benefit from new evaluation tools.
    3. For each problem identified, please indicate the type of drug, 
biologic, or device to which the hurdle applies.
    4. For each problem identified, if a solution would facilitate the 
development of drugs, biologics, and/or devices for a particular 
disease or categories of disease, please indicate which diseases would 
be affected?
    5. Nature of the Solution. For each problem identified, please 
describe the evaluation tool that would solve the problem and the work 
necessary to create and implement the tool/solution. For example, would 
a solution come from scientific research to develop a new assay or 
validate a new endpoint? If the solution involves biomedical research, 
please specify the necessary research project or program. Would a tool 
be developed through data mining or computer modeling? Would the right 
tool be a new FDA guidance or industry standard? If work on a solution 
is underway, what steps remain? Are there other innovative solutions 
that could be explored?
    6. For each solution identified, please indicate which could be 
accomplished quickly, in less than 24 months, and which require a long-
term approach?
    7. For each problem identified, what role should FDA play and what 
role should be played by others? Should FDA play a convening role, 
bringing the relevant parties together to discuss an approach or 
solution? If so, who else should participate? Should FDA coordinate 
scientific research, the results of which would be publicly available? 
We are seeking input on ways to target FDA scientific and collaborative 
activities to help industry bring more safe and effective medical 
products to us for review.
    8. What factors should guide FDA in setting priorities among the 
hurdles and solutions identified?

III. 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: April 16, 2004.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-9147 Filed 4-21-04; 8:45 am]