[Federal Register: November 19, 2001 (Volume 66, Number 223)]
[Notices]
[Page 57967-57970]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr19no01-81]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. 01N-0450]
Prescription Drug User Fee Act (PDUFA); Public Meeting
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public meeting.
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SUMMARY: The Food and Drug Administration (FDA) is announcing a public
meeting on the Prescription Drug User Fee Act (PDUFA). The legislative
authority for PDUFA expires at the end of September 2002, and without
further legislation the fees and resources provided under PDUFA will
also expire. FDA is now evaluating the PDUFA provisions. The Federal
Food, Drug, and Cosmetic Act (the act) encourages FDA to consult with
stakeholders, as appropriate, in carrying out agency responsibilities.
Accordingly, FDA will convene a public meeting to hear stakeholder
views on this subject. FDA is proposing three specific questions, and
the agency is interested in responses to these questions and any other
pertinent information stakeholders would like to share.
Date and Time: The public meeting will be held on Friday, December
7, 2001, from 9 a.m. to 5 p.m. Registration to attend the meeting must
be received by November 30, 2001. Submit written or electronic comments
by January 25, 2002.
Location: The public meeting will be held at the Hyatt Regency
Bethesda, One Bethesda Metro Center, Bethesda, MD 20814.
For information regarding this notice contact: Patricia A.
Alexander, Office of Consumer Affairs, Office of Communications and
Constituent Relations (HFE-40), Food and Drug Administration, 5600
Fishers Lane, Rockville, MD 20857, 301-827-4391, FAX 301-827-3052, e-
mail: palexand@oc.fda.gov.
For registration information contact: Carole A. Williams, Office of
Consumer Affairs, Office of Communications and
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Constituent Relations (HFE-40), Food and Drug Administration,
Rockville, MD 20857, 301-827-4394, FAX 301-827-2866, e-mail:
pubmtg@oc.fda.gov. All registration materials should be sent to Carole
A. Williams. Electronic registration for this meeting is available at:
http://www.accessdata.fda.gov/scripts/oc/dockets/meetings/
meetingdockets.cfm. Registrations will be accepted on a first-come,
first-served basis. Individuals who register to make an oral
presentation will be notified of the scheduled time for their
presentation prior to the meeting. All participants are encouraged to
attend the entire day.
Registration and Requests for Oral Presentation: To register to
attend the meeting, submit your name, title, business affiliation,
address, telephone, fax number, and e-mail address. If you wish to make
an oral presentation during the open public comment period of the
meeting, you must specify on your registration you wish to make a
presentation. You must submit the following: (1) A written statement
for each question addressed, (2) the names and addresses of all who
plan to participate, (3) the approximate time requested to make your
presentation. Depending on the number of presentations, FDA may have to
limit the time allotted for each presentation. Presenters must submit
two copies of each presentation given. If you need special
accommodations due to a disability, please inform the registration
contact person when you register.
SUPPLEMENTARY INFORMATION:
I. Background
A. September 2000 Public Meeting
On September 15, 2000, FDA held a public meeting to discuss the
future of PDUFA and to listen to the views of all interested
constituents. This public meeting was held as the agency began to
prepare for new or amended authorizing legislation. At that meeting,
the agency learned more about the expectations and concerns of various
constituent groups and citizens regarding the PDUFA program. The
December 7, 2001, meeting will continue this dialogue.
B. PDUFA I and PDUFA II
In 1992, Congress passed PDUFA authorizing FDA to collect fees from
companies that produce certain human drug and biological products. The
original PDUFA (PDUFA I) had a 5-year sunset. In 1997, Congress passed
the Food and Drug Administration Modernization Act (FDAMA). Part of
FDAMA included an extension of PDUFA (PDUFA II) for an additional 5
years. PDUFA's original intent was to provide FDA with additional
revenue so it could hire more reviewers and support staff and upgrade
its information technology to speed up the application review process
for human drug and biological products without compromising review
quality.
C. Authority to Collect Fees
The revenues are provided by a set of three fees, with one-third of
the total annual revenue coming from each of the following fees: (1)
Application fees for the submission of certain human drug or biological
applications (in fiscal year (FY) 2001, $309,647 per application with
clinical data, and $154,823 per application without clinical data or
per supplemental application with clinical data); (2) annual
establishment fees paid for each establishment that manufactures
certain prescription drugs or biologicals (in FY 2001, $145,989 per
establishment); and (3) annual product fees assessed on certain
prescription drug and biological products (in FY 2001, $21,892 per
product). In the aggregate, these fees are expected to generate $135
million in FY 2002. (This is a downward adjustment-previously they had
been expected to generate about $162 million). No separate fees are
charged for investigational new drug applications (INDs). However,
since the review of investigational new drug applications is included
in the process for the review of human drug applications, as defined in
PDUFA, FDA uses some of the application, establishment, and product
fees collected for the review of INDs.
D. Review Performance Goals
In 1992, FDA agreed to meet a set of review performance goals that
became more stringent each year, if FDA also received sufficient fee
resources to enable goal achievement. These goals applied to the review
of original new human drug and biological applications, resubmissions
of original applications, and supplements to approved applications. FDA
met every PDUFA I performance goal.
Under PDUFA II, the review goals continue to shorten. By 2002, the
PDUFA II goals call for FDA to review and act on 90 percent of the
following: (1) Standard new drug and biological product applications
and efficacy supplements within 10 months; (2) priority new drug and
biological product applications and efficacy supplements (i.e., for
products providing significant therapeutic gains) within 6 months; (3)
manufacturing supplements within 6 months, and those requiring prior
approval within 4 months; (4) class 1 resubmissions within 2 months,
and class 2 resubmissions within 6 months.
In addition, PDUFA II added a new set of goals intended to improve
FDA's responsiveness to, and communication with, industry sponsors
during the early years of drug development. These goals specify
timeframes for activities such as scheduling meetings and responding to
various sponsor requests.
E. Impact on Drug Review Process
While PDUFA's original intent was to speed up the review process,
PDUFA II's intent is to speed up the entire drug development process.
By providing an influx of needed resources, PDUFA has had a dramatic
and undeniable impact on the drug review process. Total resources for
drug review activities have increased from $120 million in 1992, before
PDUFA was enacted, to an estimated $329 million in FY 2002, a little
more than half of which will come from fees paid by industry. These
resources allowed FDA to increase its drug and biological review staff
by almost 60 percent between 1993 and 1997, adding about 660 staff-
years to the program by 1997. By the end of PDUFA II in 2002, FDA
expects to have added another 340 staff-years of effort to this
program. These additional staff, and resources to support them, have
enabled FDA to respond more rapidly to new drug and biologic
applications without compromising review quality.
While it is important to note that PDUFA's goals specify decision
times, not approval times, both decision and approval times have
decreased dramatically. Total approval time, the time from the initial
submission of a marketing application to the issuance of an approval
letter, has dropped from a pre-PDUFA median of 23 months to an
estimated 15 months in 2001. Total approval time for priority
applications, those for products providing significant therapeutic
gains, has dropped from a median of over 12 months in the early PDUFA
years to 6 months. In addition, because FDA has put greater effort into
communicating what it expects applicants to submit, a higher percentage
of applications are being approved. Before PDUFA, only about 60 percent
of the applications submitted were ultimately approved. Now, about 80
percent are approved. For the consumer, this has meant more products
available more quickly.
F. Challenges
Notwithstanding these successes, the agency has encountered
challenges in trying to meet the PDUFA II goals.
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Assuring that enough appropriated funds are spent on the process for
the review of human drug applications to meet requirements of PDUFA,
and at the same time spending our resources in a way that best protects
the health and safety of the American people, is becoming increasingly
difficult. Each year, the amount that FDA must spend from
appropriations on the drug review process is increased by an inflation
factor. Yet, since 1992, FDA has not received increased appropriations
to cover the costs of the across-the-board pay increases that must be
given to all employees. The result is that our workforce and real
resources for most programs other than PDUFA have contracted each year
since 1992 while we struggle to ensure that enough funds are spent on
the drug review process to meet this PDUFA requirement. FDA will be
unable to continue to reduce staffing levels in FDA programs other than
drug review and still maintain those programs in a way that best
protects and promotes the public health and merits public confidence.
Another challenge we have faced in PDUFA II is that we
underestimated the resources we would need to meet the new, demanding
PDUFA II goals. In addition, the fees we have collected have been
significantly less than expected. Revenues have been lower than
projected due to the reduced number of fee-paying applications and the
increased number of fee-waived applications. This has also resulted in
lower than expected fee revenues from products and establishments. In
FY 2001, about 30 percent of applications received fee waivers. FDA
will need to spend all of the reserve funds available in order to try
to continue to meet PDUFA goals. FDA anticipates that by the end of
PDUFA II the agency will have depleted all fee reserves.
Despite this fluctuation in revenues, our workload under PDUFA II
continued to rise. Many of the activities covered by PDUFA II
performance goals do not, themselves, generate fees, yet the workload
in these areas has been substantial. For example, the numbers of
commercial INDs, efficacy supplements, and manufacturing supplements
are up, and the number of meetings, responses to clinical holds and
special protocol assessments, all of which have specific PDUFA II
performance goals, have been higher than anticipated. The new pediatric
and fast track provisions of FDAMA, none of which received specific
additional funding, also have contributed significantly to this
increased workload.
FDA is also concerned about the safety of new drugs and biologics
following approval and marketing. FDA's postmarket monitoring
activities are not currently funded by PDUFA. More rigorous safety
monitoring of newly approved drugs in the first few years after a
product is on the market could help to detect unanticipated problems
earlier. The current system for detecting adverse drug and biologics
events does not provide sufficient data on the actual incidence of
problems. Another concern is the growth in prescription drug
advertising. Current PDUFA funding does not cover the agency's cost of
reviewing promotional materials (over 37,000 pieces in 2000).
Although FDA has been able to meet most of its performance goals
despite these challenges, we do not believe this will continue in the
future. We do not foresee increasing or even maintaining performance
levels until resources are available to meet the increased workload.
These resources can be provided either from appropriated dollars or
from user fees. However, to date we have not seen increases in
appropriated dollars needed to meet the shortfalls we have experienced.
We may, in fact, be seeing that our efforts to meet the new PDUFA
II goals have led to an unintended consequence regarding approval times
of standard new drug and biologics applications. These approval times
have begun to increase because more applications require multiple
review cycles to reach approval. We believe this may be due to the fact
that reviewers, pressed to meet the new PDUFA II goals for drug
development (e.g., meetings, special protocol assessments, and
responses to clinical holds), have had less time to devote to resolving
last minute problems with these standard applications in time to meet
the action goal date. As a result, the application must undergo an
additional review cycle with its attendant timeframes and goals. Our
statistics on this trend are preliminary and we are watching it
closely. However, if our user fee program is to continue, it must be on
a sound financial footing and based on reliable estimates of workload
and resources.
II. Scope of Discussion
The legislative authority for PDUFA II expires at the end of
September 2002. Without further legislation the fees and resources it
has provided will also expire. Public input is important at this time
as final preparations are being made to propose reauthorization.
Section 903(b) of the act (21 U.S.C. 393(b)) encourages FDA to consult
with stakeholders, as appropriate, in carrying out agency
responsibilities. Accordingly, FDA will convene a public meeting on
December 7, 2001. Interested persons are invited to attend and present
their views. A list of questions that we are asking interested parties
to address at this meeting follows:
1. Has PDUFA supported FDA's mission to protect and promote public
health? What should be retained or changed to enhance the program?
2. Should PDUFA allow the use of user fee funding to monitor safety
after new drug or biologic approval?
3. How can FDA ensure that PDUFA goals are met if there continues
to be a funding shortfall? If the funding shortfall persists, should
FDA, in order to best protect and promote the public health, set review
priorities and, if so, how? Should there be flexibility in setting user
fees to cover the increased cost of the program?
III. Comments
Interested persons may submit to the Dockets Management Branch
(HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061,
Rockville, MD 20852, written comments on or before January 25, 2002.
Submit electronic comments to fdadockets@oc.fda.gov or http://
www.accessdata.fda.gov/scripts/oc/dockets/edockethome.cfm. You should
annotate and organize your comments to identify the specific questions
to which they refer. (See above.) You must submit two copies of
comments, identified with the docket number found in brackets in the
heading of this document. You may review received comments
approximately 15 days after the meeting in the Dockets Management
Branch, Monday through Friday between 9 a.m. and 4 p.m. or on the
Internet at http://www.fda.gov/oc/pdufa/meeting2001/.
IV. Transcripts
You may request a copy of the transcript in writing from the
Freedom of Information Office (HFI-35), Food and Drug Administration,
5600 Fishers Lane, rm. 12A-16, Rockville, MD 20857, approximately 15
days after the meeting at a cost of 10 cents per page. You may also
examine the transcript Monday through Friday between 9 a.m. and 4 p.m.
in the Dockets Management Branch or on the Internet at http://
www.fda.gov/oc/pdufa/meeting2001/.
V. Electronic Access
Persons with access to the Internet may obtain more information
about PDUFA at http://www.fda.gov/oc/pdufa/default.htm.
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Dated: November 14, 2001.
Margaret M. Dotzel,
Associate Commissioner for Policy.
[FR Doc. 01-29002 Filed 11-15-01; 4:39 pm]
BILLING CODE 4160-01-S