[Federal Register Volume 66, Number 223 (Monday, November 19, 2001)]
[Notices]
[Pages 57967-57970]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 01-29002]


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