Food and Drug Administration
Data to Measure the Timeliness of Reviews of Medical Device Applications Are Limited Gao ID: GAO-04-1022 August 30, 2004FDA reviews applications from manufacturers that wish to market medical devices in the United States. To ensure prompt approval of new devices and clearance of devices that are substantially equivalent to those legally on the market, the Congress passed the Medical Device User Fee and Modernization Act of 2002 (MDUFMA). The act authorizes FDA to collect user fees and, in return, requires FDA to meet performance goals that are tied to the agency's review process. The goals set actions FDA may take on applications and specify the time that FDA should take in certain phases of the review process. MDUFMA requires GAO to report on FDA's performance against the MDUFMA performance goals established for fiscal years 2003 and 2004 and to determine whether FDA is likely to meet the fiscal year 2005 performance goals. MDUFMA also requires GAO to report on the amounts FDA obligated in fiscal year 2002 for medical device compliance activities and inspections of manufacturers after their devices are marketed. GAO analyzed data provided by FDA that are based on actions taken on applications FDA received from October 1, 2002, through March 31, 2004. GAO also analyzed data on the amounts FDA obligated for medical device compliance and inspection activities for fiscal year 2002.
FDA had limited data that could be used to measure the agency's performance against most of the MDUFMA performance goals. Thus, it is uncertain whether FDA will meet the MDUFMA performance goals for fiscal years 2003, 2004, and 2005. For fiscal years 2003 and 2004, there were two performance goals in effect for each year. As of March 31, 2004, only one application was subject to the action tied to one of the two MDUFMA performance goals. On this application, FDA completed its review and made the decision to approve the application within the goal's established time frame. To determine the likelihood of meeting the 20 MDUFMA performance goals for fiscal year 2005, FDA is collecting data on its performance against these goals. GAO found that FDA had performance data for some, but not all, of the MDUFMA performance goals. From fiscal year 2003 applications, data were available to compare FDA's performance against 17 of the 20 fiscal year 2005 performance goals. FDA took actions tied to 14 of the 17 goals within the goals' established time frames. From fiscal year 2004 applications, data were available to compare FDA's performance against 11 of the 20 performance goals. FDA took actions tied to the 11 goals within the goals' established time frames. The results of FDA's performance against MDUFMA performance goals are preliminary, however, because 8 percent and 49 percent, respectively, of the applications FDA accepted in fiscal year 2003 and the first 6 months of fiscal year 2004 were awaiting action by FDA or responses from manufacturers. Because FDA's performance against the MDUFMA performance goals is based on the percentages of actions the agency takes within required review times, FDA's results could change as the agency completes its actions on all applications for which the goals apply. FDA obligated about $128 million for postmarket medical device compliance activities and inspections in fiscal year 2002. FDA obligated about $109 million for compliance activities for outreach coordination, such as guidance to field staff on reporting problems with medical devices, laboratory analyses, and research, such as the development of domestic and international standards to provide reasonable assurance that medical device products are safe and effective. FDA obligated about $19 million for inspections of device manufacturers' establishments, including routine surveillance inspections to determine compliance with medical device regulations and inspections resulting from device problem reporting or product recalls.
GAO-04-1022, Food and Drug Administration: Data to Measure the Timeliness of Reviews of Medical Device Applications Are Limited
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Report to Congressional Committees:
August 2004:
Food and Drug Administration:
Data to Measure the Timeliness of Reviews of Medical Device
Applications Are Limited:
[Hyperlink, http://www.gao.gov/cgi-bin/getrpt?GAO-04-1022]:
GAO Highlights:
Highlights of GAO-04-1022, a report to congressional committees
Why GAO Did This Study:
FDA reviews applications from manufacturers that wish to market medical
devices in the United States. To ensure prompt approval of new devices
and clearance of devices that are substantially equivalent to those
legally on the market, the Congress passed the Medical Device User Fee
and Modernization Act of 2002 (MDUFMA). The act authorizes FDA to
collect user fees and, in return, requires FDA to meet performance
goals that are tied to the agency‘s review process. The goals set
actions FDA may take on applications and specify the time that FDA
should take in certain phases of the review process.
MDUFMA requires GAO to report on FDA‘s performance against the MDUFMA
performance goals established for fiscal years 2003 and 2004 and to
determine whether FDA is likely to meet the fiscal year 2005
performance goals. MDUFMA also requires GAO to report on the amounts
FDA obligated in fiscal year 2002 for medical device compliance
activities and inspections of manufacturers after their devices are
marketed.
GAO analyzed data provided by FDA that are based on actions taken on
applications FDA received from October 1, 2002, through March 31, 2004.
GAO also analyzed data on the amounts FDA obligated for medical device
compliance and inspection activities for fiscal year 2002.
What GAO Found:
FDA had limited data that could be used to measure the agency‘s
performance against most of the MDUFMA performance goals. Thus, it is
uncertain whether FDA will meet the MDUFMA performance goals for fiscal
years 2003, 2004, and 2005. For fiscal years 2003 and 2004, there were
two performance goals in effect for each year. As of March 31, 2004,
only one application was subject to the action tied to one of the two
MDUFMA performance goals. On this application, FDA completed its review
and made the decision to approve the application within the goal‘s
established time frame. To determine the likelihood of meeting the 20
MDUFMA performance goals for fiscal year 2005, FDA is collecting data
on its performance against these goals. GAO found that FDA had
performance data for some, but not all, of the MDUFMA performance
goals. From fiscal year 2003 applications, data were available to
compare FDA‘s performance against 17 of the 20 fiscal year 2005
performance goals. FDA took actions tied to 14 of the 17 goals within
the goals‘ established time frames. From fiscal year 2004 applications,
data were available to compare FDA‘s performance against 11 of the 20
performance goals. FDA took actions tied to the 11 goals within the
goals‘ established time frames. The results of FDA‘s performance
against MDUFMA performance goals are preliminary, however, because 8
percent and 49 percent, respectively, of the applications FDA accepted
in fiscal year 2003 and the first 6 months of fiscal year 2004 were
awaiting action by FDA or responses from manufacturers. Because FDA‘s
performance against the MDUFMA performance goals is based on the
percentages of actions the agency takes within required review times,
FDA‘s results could change as the agency completes its actions on all
applications for which the goals apply.
FDA obligated about $128 million for postmarket medical device
compliance activities and inspections in fiscal year 2002. FDA
obligated about $109 million for compliance activities for outreach
coordination, such as guidance to field staff on reporting problems
with medical devices, laboratory analyses, and research, such as the
development of domestic and international standards to provide
reasonable assurance that medical device products are safe and
effective. FDA obligated about $19 million for inspections of device
manufacturers‘ establishments, including routine surveillance
inspections to determine compliance with medical device regulations
and inspections resulting from device problem reporting or product
recalls.
In commenting on a draft of this report, FDA generally agreed with its
findings.
www.gao.gov/cgi-bin/getrpt?GAO-04-1022.
To view the full product, including the scope and methodology, click
on the link above. For more information, contact Marcia Crosse at
(202) 512-7119.
[End of section]
Contents:
Letter:
Results in Brief:
Background:
Limited Data to Measure FDA's Performance against the MDUFMA
Performance Goals:
FDA Obligated over $128 Million for Postmarket Medical Device
Compliance Activities and Inspections in Fiscal Year 2002:
Agency Comments:
Appendix:
Appendix I: Comments from the Food and Drug Administration:
Tables:
Table 1: MDUFMA Performance Goals for Fiscal Years 2003 and 2004:
Table 2: MDUFMA Performance Goals for Fiscal Year 2005:
Table 3: Status of FDA's Performance for Fiscal Years 2003 and 2004
Compared to MDUFMA Performance Goals That Become Effective in Fiscal
Year 2005:
Table 4: Applications Pending within FDA's Review Process in the Fiscal
Year 2003 Cohort and the First 6 Months of the Fiscal Year 2004 Cohort:
Figures:
Figure 1: PMA Review Process:
Figure 2: 510(k) Review Process:
Abbreviations:
BLA: biologics license application:
CBER: Center for Biologics Evaluation and Research:
CDRH: Center for Devices and Radiological Health:
FDA: Food and Drug Administration:
FDCA: Federal Food, Drug, and Cosmetics Act:
GMP: good manufacturing practices:
HHS: Department of Health and Human Services:
MDUFMA: Medical Device User Fee and Modernization Act of 2002:
PMA: premarket approval:
Letter August 30, 2004:
The Honorable Judd Gregg:
Chairman:
The Honorable Edward M. Kennedy:
Ranking Minority Member:
Committee on Health, Education, Labor, and Pensions:
United States Senate:
The Honorable Joe Barton:
Chairman:
The Honorable John D. Dingell:
Ranking Minority Member:
Committee on Energy and Commerce:
House of Representatives:
The Food and Drug Administration (FDA) is responsible for regulating
medical devices--such as tongue depressors, pacemakers, and artificial
hearts--to provide reasonable assurance of their safety and
effectiveness for human use. As part of its regulatory
responsibilities, FDA reviews applications from manufacturers that wish
to have their new medical devices or devices substantially equivalent
to those already on the market approved for marketing in the United
States. When required, FDA inspects manufacturers' establishments prior
to approval. FDA is also responsible for implementing and enforcing
medical device regulations related to compliance activities, which
include the reporting of problems associated with the safety and
effectiveness of devices and inspections of manufacturers' device
establishments after devices reach the market.
Each year FDA receives approximately 10,000 medical device
applications. Members of the Congress, representatives of the medical
device industry, and others have expressed concern about the length of
time it takes FDA to review applications for marketing medical devices,
with the consequence of possibly delaying patients' access to useful,
and possibly life-saving, medical devices.
In October 2002, the Congress passed the Medical Device User Fee and
Modernization Act of 2002 (MDUFMA) to provide FDA with additional
resources to ensure prompt approval or clearance[Footnote 1] of
applications for marketing medical devices and licensing biological
products.[Footnote 2] MDUFMA authorized FDA to collect user fees from
manufacturers that submit several types of applications to FDA for
marketing medical devices. In return, MDUFMA requires FDA to meet
performance goals tied to the review process for medical device and
biological applications. MDUFMA also requires the Secretary of Health
and Human Services (HHS) to develop the specific goals FDA must meet.
The Secretary developed performance goals for fiscal years 2003 through
2007. These MDUFMA performance goals are set for certain actions FDA
may take during the application review process and specify lengths of
time for taking these actions. FDA's performance against the MDUFMA
performance goals is based on the percentages of actions the agency
takes on applications within required review times. To help FDA meet
the MDUFMA performance goals, the Secretary of HHS also identified
several goal-related activities for FDA to undertake, such as hiring
additional review staff.
The number of MDUFMA performance goals that FDA must meet increases
over time. For fiscal years 2003 and 2004, FDA must meet the same two
performance goals for each year. For example, one of the performance
goals requires FDA to review a manufacturer's response to the agency's
request for additional information on applications that are approvable
with minor corrections or clarifications,[Footnote 3] and then take the
next appropriate action, such as make a decision to approve the
application. To meet the performance goal, FDA must review and make the
decision within 30 days on 90 percent of the applications received for
each fiscal year. In fiscal year 2005, FDA must meet the two goals and
an additional 18 performance goals tied to medical device and
biological product applications, for a total of 20. The 20 performance
goals are tied to times for reviews, decisions, or a combination of
both.
MDUFMA requires us to report on FDA's performance as measured against
the fiscal years 2003 and 2004 MDUFMA performance goals and determine
whether the agency is likely to meet the fiscal year 2005 MDUFMA
performance goals. MDUFMA also requires us to report on the amounts FDA
obligated in fiscal year 2002 for medical device compliance activities
and inspections of manufacturers' device establishments after their
devices are marketed, excluding the amounts that were obligated for
inspections related to the review of medical device applications.
To examine FDA's performance, we analyzed actions taken by FDA on
applications that it received from fiscal year 2003 through the first 6
months of fiscal year 2004 (October 1, 2003, through March 31, 2004).
At the time of our review, performance data through March 31, 2004,
were the most current FDA data available. To assess FDA's performance
against the two MDUFMA performance goals that were established for
fiscal years 2003 and 2004, we analyzed performance data for
applications received in those years. To determine the likelihood of
FDA meeting its fiscal year 2005 MDUFMA performance goals, we compared
performance data that FDA is collecting on fiscal years 2003 and 2004
applications to the 20 MDUFMA performance goals that will be effective
in fiscal year 2005. We also reviewed relevant documents and
interviewed officials from FDA's Center for Devices and Radiological
Health (CDRH) and Center for Biologics Evaluation and Research (CBER)
about two key FDA medical device application review processes and the
agency's performance as measured against the MDUFMA performance goals.
These two processes are referred to as (1) the Premarket Approval (PMA)
review process, which is used to review an application for a new
medical device or when the risks associated with the device are
considerable, and (2) the 510(k) review process, which is used to
review an application for a type of device that may be substantially
equivalent to one already on the market. In addition, we reviewed
documentation of FDA's procedures for checking the reliability of
MDUFMA performance data and met with FDA officials to discuss their
efforts to verify the accuracy and consistency of their reported
performance data. We determined that the performance data were
sufficiently reliable for the purposes of this report.
To determine the amounts FDA obligated in fiscal year 2002 for medical
device compliance activities and inspections of manufacturers' device
establishments after devices are marketed, we reviewed and analyzed
data from FDA's fiscal year 2004 budget justification and information
FDA provided related to these data. The fiscal year 2004 budget
justification contains FDA's fiscal year 2002 actual obligations for
medical device compliance activities and inspections. To assess the
reliability of these data, we reviewed supporting documentation that
FDA provided on the obligations reported in the agency's fiscal year
2004 budget justification related to compliance activities and
inspections for marketed devices. We also reviewed FDA's fiscal year
2003 audited financial statement report that contained information
about the reliability of fiscal year 2002 obligations data. The review
found no material weaknesses related to our work. Based on these
reviews, we determined that the obligations data were sufficiently
reliable for the purposes of this report. We conducted our work from
January 2004 through August 2004 in accordance with generally accepted
government auditing standards.
Results in Brief:
FDA had limited data that could be used to measure the agency's
performance against most of the MDUFMA performance goals. Thus, it is
uncertain whether FDA will meet the goals established for fiscal years
2003, 2004, and 2005. For example, for fiscal year 2003, the two
performance goals could be applied to 53 applications. However, as of
March 31, 2004, only one application was subject to a review and a
decision tied to one of the two MDUFMA performance goals. On that
application, FDA reviewed the manufacturer's complete response to FDA's
approvable letter and made the decision to approve the application
within the goal's established time frame. For fiscal year 2004, there
was no application subject to the review and decision tied to the two
fiscal year 2004 MDUFMA performance goals. FDA had performance data for
some, but not all, of the MDUFMA performance goals that will be
effective in fiscal year 2005. For applications received in fiscal year
2003, data were available to compare FDA's performance against 17 of
the 20 performance goals for fiscal year 2005. FDA took actions on
applications tied to 14 of the 17 performance goals within the
established time frames. For fiscal year 2004 applications, FDA had
performance data for 11 of the 20 performance goals. FDA took actions
on applications tied to the 11 goals within the established time
frames. The results of FDA's performance against MDUFMA goals are
preliminary, however, because many of the applications FDA received in
fiscal year 2003 and the first 6 months of fiscal year 2004 were
pending within the review process. Because FDA's performance against
the MDUFMA performance goals is based on the percentages of actions the
agency takes on applications within required review times, FDA's
performance results could change as the agency completes actions on all
applications for which the performance goals apply.
FDA obligated over $128 million for postmarket medical device
compliance activities and inspections in fiscal year 2002. FDA
obligated about $109 million for compliance activities for outreach
coordination, such as guidance to field staff on the reporting of
problems with medical devices; laboratory analyses; and research, such
as the development of domestic and international standards to help
provide reasonable assurance that medical devices are safe and
effective. FDA obligated about $19 million for inspections of
manufacturers' device establishments that included routine surveillance
inspections to determine compliance with medical device regulations and
inspections resulting from device problem reporting or product recalls.
We provided a draft of this report to FDA, which generally agreed with
its findings. FDA believes that it has made a good start in its
implementation of MDUFMA and believes its progress with the MDUFMA
performance goals is consistent with the extensive performance
improvements the agency is expected to achieve each year through 2007.
Background:
Under the Federal Food, Drug, and Cosmetic Act (FDCA),[Footnote 4] FDA
is responsible for ensuring that medical devices are reasonably safe
and effective before they go to market (premarket) and that marketed
device products remain safe (postmarket). Two FDA centers, CDRH and
CBER, are responsible for the PMA and clearance of medical device
applications.[Footnote 5] CDRH reviews applications for the majority of
devices marketed, such as artificial hearts, dialysis machines, and
radiological devices. CBER reviews applications for devices used in the
testing and manufacture of biological products, such as diagnostic
tests intended to screen blood donors (such as for the human
immunodeficiency virus), as well as devices used in cell and gene
therapies.
Each fiscal year, FDA obligates funds for postmarket compliance
activities and inspections related to medical devices and radiological
products. FDA provides field staff with guidance on inspecting
manufacturers' establishments after devices have been marketed, for
compliance with the good manufacturing practices (GMP) requirements,
monitoring manufacturers' corrections of problems with devices, and
removing unsafe devices from the market. FDA has 21 district offices
that support inspections of manufacturers' establishments. During
inspections for compliance with GMP requirements, FDA investigators
examine manufacturer facilities, records of manufacturing processes,
and corrective action programs.
Types of Applications Reviewed under MDUFMA Performance Goals:
MDUFMA identified eight types of applications for medical devices and
biological products that are subject to the performance goals developed
by the Secretary of HHS for fiscal years 2003, 2004, and 2005:
* Original PMA applications are generally required when the device is
new or when the risks associated with the device are considerable (as
would be the case if the device is implanted in the body for life-
supporting purposes).
* Premarket Notifications, or 510(k)s,[Footnote 6] are applications
used when the intent is to market a type of device that may be
considered substantially equivalent[Footnote 7] to one already on the
market and therefore, does not require PMA.
* Premarket Reports are applications required for high-risk devices
originally approved for a single use that a manufacturer has
reprocessed for additional uses.
* Panel-Track Supplements are applications used to supplement approved
PMAs or Premarket Reports. These supplements typically request approval
of a significant change in the design or performance of a device, or
for a new purpose for using the device.
* Expedited Original PMAs are used when a manufacturer seeks priority
status to market a medical device that is intended to treat or diagnose
a life-threatening or irreversibly debilitating disease or condition or
to address an unmet medical need.
* The 180-day PMA Supplements are used to supplement approved PMAs or
premarket reports. The application typically requests approval of a
significant change in aspects of a device, such as its design,
specifications, or labeling.
* Biologics license applications (BLAs)[Footnote 8] Resubmissions
(Class 1) are used to respond to information requested by FDA on a BLA
and may include matters related to product labeling or safety and other
minor clarifying information. BLA Supplement Resubmissions (Class 2)
are used to respond to an FDA request for information on a BLA
regarding the safety and effectiveness of products or a re-inspection
of the manufacturer's device establishment.
FDA's Medical Device Application Review Processes:
FDA primarily uses two medical device application review processes; the
PMA review process and the 510(k) review process.[Footnote 9] FDA
annually receives about 50 PMAs and about 4,000 510(k) applications for
review through these processes.
The PMA Review Process:
Under the PMA review process, FDA reviews applications for new devices
or those for which risks associated with the device are considerable.
Applications reviewed under this process include Original PMAs,
Premarket Reports, Panel-Track Supplements, Expedited Original PMAs,
and 180-day PMA Supplements. After an initial screening of an
application and determination that the review should proceed, an FDA
multidisciplinary staff conducts a scientific review of the
application[Footnote 10] and determines whether it is complete, that
is, if it contains sufficient information to allow the review to
continue. (See fig. 1.):
Figure 1: PMA Review Process:
[See PDF for image]
[End of figure]
For complete applications, FDA may make one of five decisions. FDA may
(1) issue an order approving the application, which allows the
manufacturer to begin marketing the device; (2) send the manufacturer
an approvable letter pending a GMP inspection, which indicates that FDA
should be able to approve the device after the agency finds that the
manufacturer's device establishment is in compliance with GMP
requirements; (3) send the manufacturer an approvable letter indicating
that the agency should be able to approve the device if the
manufacturer can make minor corrections or clarifications to the
application; (4) issue a "not approvable" letter informing the
manufacturer that FDA does not believe that the application can be
approved because the data provided by the manufacturer do not
demonstrate that the device is reasonably safe and effective; or (5)
issue an order denying approval of the application, which informs the
manufacturer that the agency has completed its scientific review,
identified major safety and effectiveness problems, and decided not to
approve the application. FDA may refer complete applications to an
external advisory committee for evaluation when a device is the first
of its kind or when the agency believes it would be useful to have
independent expertise and technical assistance to properly evaluate the
safety and effectiveness of the device.[Footnote 11] For applications
referred to an advisory committee, the committee submits
recommendations to FDA on the safety and effectiveness of the devices.
Taking the committee's recommendations into consideration, FDA then
makes its decision.
For incomplete applications, that is, applications for which FDA
determines it needs significant additional information, FDA issues a
"major deficiency letter" to the manufacturer identifying the
information that is required to provide reasonable assurance of the
safety and effectiveness of the device. This request for significant
information is an action referred to as a cycle action. In general, FDA
takes cycle actions when it requests additional information from the
manufacturer, or when it evaluates additional information provided by
the manufacturer in response to an FDA request. The manufacturer
responds to FDA's request by submitting an amendment to the original
application. Cycle actions on an application can occur repeatedly until
FDA determines that the manufacturer has provided what the agency calls
a "complete response" to all of the agency's concerns. Then, FDA may
make a decision on the application.
Manufacturers that receive approvable letters pending minor corrections
or clarifications or not approvable letters can gain final approval by
submitting amendments with complete responses to FDA's concerns. For
both types of decisions, if FDA, as part of a cycle action, determines
that the manufacturer's amendment is a complete response, FDA issues an
order approving the application. If the amendment is not a complete
response, FDA issues another approvable letter pending minor
corrections or clarifications or a not approvable letter.
The 510(k) Review Process:
Under the 510(k) review process, FDA decides whether the application
contains sufficient information to determine whether the device is
substantially equivalent to one legally on the market (see fig. 2).
When a 510(k) application lacks information necessary for FDA to
complete its review and make a determination as to whether the device
is substantially equivalent, the agency issues a letter requesting
additional information from the manufacturer. This request for
additional information is a cycle action. The manufacturer may then
submit additional information responding to FDA's concerns. Once FDA
has obtained complete information from the manufacturer, FDA issues a
decision letter informing the manufacturer that the device is
substantially equivalent and therefore may be marketed or the device is
not substantially equivalent and may not be marketed.
Figure 2: 510(k) Review Process:
[See PDF for image]
[End of figure]
Measuring FDA's Performance under MDUFMA:
The MDUFMA performance goals specify a length of time for taking a
cycle action, making a decision, or a combination of both. The goals
designate a certain percentage of these actions that must occur within
the specified period in order for FDA to meet the performance goals. To
measure its performance against the MDUFMA performance goals, FDA
tracks the time, in calendar days, the agency takes to complete a cycle
action or make a decision--but not the time it takes a manufacturer to
respond to a request from FDA.[Footnote 12]
FDA measures its performance against a specific fiscal year's MDUFMA
performance goals using all the applications it received in that year-
-known as a cohort[Footnote 13]--regardless of when the final decision
is made for each of the applications in that year's cohort.The agency's
performance as measured against a fiscal year's MDUFMA performance
goals is preliminary until all applications in a cohort are completed -
-and this can take up to 3 or 4 fiscal years.
The two MDUFMA performance goals that FDA must meet for fiscal years
2003 and 2004 are tied to the same type of cycle action for different
types of applications. One performance goal applies to the cycle action
taken on PMAs, Panel-Track Supplements, and Premarket Reports, and the
other performance goal applies to Expedited PMAs. To meet each
performance goal, FDA must review the completeness of a manufacturer's
response to an approvable letter pending minor corrections or
clarifications and make a decision within 30 days of receiving the
manufacturer's amendment. FDA must meet this time frame on 90 percent
of the applications for which it takes a cycle action (see table 1).
Table 1: MDUFMA Performance Goals for Fiscal Years 2003 and 2004:
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
FDA cycle actions: Review whether an amendment is a complete response
to an approvable letter pending minor corrections or clarifications and
make a decision, such as approving the application;
Performance goal: Review time: 30 days;
Performance goal: Percentage of applications received in each fiscal
year required to meet review time: Fiscal year 2003 cohort: 90%;
Performance goal: Percentage of applications received in each fiscal
year required to meet review time: Fiscal year 2004 cohort: 90%.
Type of application: Expedited PMAs;
FDA cycle actions: Review whether an amendment is a complete response
to an approvable letter pending minor corrections or clarifications and
make a decision, such as approving the application;
Performance goal: Review time: 30 days;
Performance goal: Percentage of applications received in each fiscal
year required to meet review time: Fiscal year 2003 cohort: 90%;
Performance goal: Percentage of applications received in each fiscal
year required to meet review time: Fiscal year 2004 cohort: 90%.
Source: GAO analysis of HHS and FDA information.
[A] FDA groups these types of applications together when measuring
performance for this goal.
[End of table]
In fiscal year 2005, 20 MDUFMA performance goals will become effective,
including the 2 performance goals that have been effective since fiscal
year 2003 (see table 2). The 20 performance goals will apply to eight
types of applications identified under MDUFMA and will be tied to other
cycle actions and decisions FDA makes on the applications during the
review process.
Table 2: MDUFMA Performance Goals for Fiscal Year 2005:
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
FDA cycle actions and decisions: Cycle action: Issue first major
deficiency letter requesting significant information from the
manufacturer;
Performance goal: Review time: 150 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 75%.
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
FDA cycle actions and decisions: Decision: Issue decision letter
(approval, approvable pending GMP inspection, approvable pending minor
corrections or clarifications, not approvable, or denial) as a first
action on an application;
Performance goal: Review time: 180 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 75%.
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
FDA cycle actions and decisions: Cycle action: Issue second or
subsequent major deficiency letter requesting significant information
from the manufacturer;
Performance goal: Review time: 120 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 75%.
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
FDA cycle actions and decisions: Cycle action: Determine whether an
amendment contains a complete response to a major deficiency or not
approvable letter;
Performance goal: Review time: 180 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 75%.
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
FDA cycle actions and decisions: Cycle action: Review whether an
amendment contains a complete response to an approvable letter pending
minor corrections or clarifications and make a decision, such as
approving the application;
Performance goal: Review time: 30 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 90%.
Type of application: Expedited PMAs;
FDA cycle actions and decisions: Decision: Issue decision letter
(approval, approvable pending GMP inspection, approvable pending minor
corrections or clarifications, not approvable, or denial) after taking
a cycle action on an application;
Performance goal: Review time: 300 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 70%.
Type of application: Expedited PMAs;
FDA cycle actions and decisions: Cycle action: Issue first major
deficiency letter requesting significant information from the
manufacturer;
Performance goal: Review time: 120 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 70%.
Type of application: Expedited PMAs;
FDA cycle actions and decisions: Decision: Issue decision letter
(approval, approvable pending GMP inspection, not approvable,
approvable pending minor corrections or clarifications, or denial) as
a first action on an application;
Performance goal: Review time: 170 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 70%.
Type of application: Expedited PMAs;
FDA cycle actions and decisions: Cycle action: Issue a second or
subsequent major deficiency letter requesting significant information
from the manufacturer;
Performance goal: Review time: 100 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 70%.
Type of application: Expedited PMAs;
FDA cycle actions and decisions: Cycle action: Determine whether an
amendment contains a complete response to a major deficiency or not
approvable letter;
Performance goal: Review time: 170 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 70%.
Type of application: Expedited PMAs;
FDA cycle actions and decisions: Cycle action: Review whether an
amendment contains a complete response to an approvable letter pending
minor corrections or clarifications and make a decision, such as
approving the application;
Performance goal: Review time: 30 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 90%.
Type of application: 180-day PMA Supplements;
FDA cycle actions and decisions: Decision: Issue decision letter
(approval, approvable pending GMP inspection, approvable pending minor
corrections or clarifications, not approvable, or denial) after taking
a cycle action on an application;
Performance goal: Review time: 180 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 80%.
Type of application: 180-day PMA Supplements;
FDA cycle actions and decisions: Decision: Issue a not approvable
letter as a first action on an application;
Performance goal: Review time: 120 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 80%.
Type of application: 180-day PMA Supplements;
FDA cycle actions and decisions: Decision: Issue decision letter
(approval, approvable pending GMP inspection, approvable pending minor
corrections or clarifications, or denial) as a first action on an
application;
Performance goal: Review time: 180 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 80%.
Type of application: 180-day PMA Supplements;
FDA cycle actions and decisions: Cycle action: Determine whether an
amendment contains a complete response to a not approvable letter;
Performance goal: Review time: 160 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 80%.
Type of application: Premarket Notifications (510(k)s);
FDA cycle actions and decisions: Decision: Issue decision letter (for
example, a letter for a device that may be marketed because it is
substantially equivalent to one already on the market, or that may not
marketed because it is not substantially equivalent);
Performance goal: Review time: 90 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 75%.
Type of application: Premarket Notifications (510(k)s);
FDA cycle actions and decisions: Cycle action: Issue first additional
information letter;
Performance goal: Review time: 75 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 70%.
Type of application: Premarket Notifications (510(k)s);
FDA cycle actions and decisions: Cycle action: Issue second or
subsequent additional information letter;
Performance goal: Review time: 60 days;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 70%.
Type of application: BLA Resubmissions and BLA Supplement
Resubmissions;
FDA cycle actions and decisions: Cycle action: Review and act on a
Class 1 resubmission to an original BLA or BLA supplement (for example,
issue a letter requesting limited labeling or safety information from
the manufacturer);
Performance goal: Review time: 2 months;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 75%.
Type of application: BLA Resubmissions and BLA Supplement
Resubmissions;
FDA cycle actions and decisions: Cycle action: Review and act on a
Class 2 resubmission to an original BLA or BLA supplement (for example,
issue a letter requesting data on product safety and effectiveness
from the manufacturer);
Performance goal: Review time: 6 months;
Performance goal: Percentage of applications received in fiscal year
2005 cohort required to meet review time: 75%.
Source: GAO analysis of HHS and FDA information as of March 31, 2004.
[A] FDA groups these types of applications together when measuring
performance for this goal.
[End of table]
Limited Data to Measure FDA's Performance against the MDUFMA
Performance Goals:
FDA had limited data that could be used to measure the agency's
performance against most of the MDUFMA performance goals. Thus, it is
uncertain whether FDA will meet the MDUFMA performance goals for fiscal
years 2003, 2004, and 2005. For fiscal year 2003, the two performance
goals could be applied to 53 applications. However, as of March 31,
2004, only one application was subject to a cycle action tied to one of
the two MDUFMA performance goals. In its review of that application,
FDA determined that the manufacturer provided a complete response to an
approvable letter pending minor corrections or clarifications and made
the decision to approve the application within the goal's time frame.
For fiscal year 2004, there were no applications subject to the cycle
action tied to the two fiscal year 2004 MDUFMA performance goals. In
addition, the likelihood of FDA meeting the fiscal year 2005 MDUFMA
performance goals is uncertain because FDA had performance data for
some, but not all, of the MDUFMA performance goals that will be
effective in fiscal year 2005. FDA's performance results could change
as the agency completes its actions on all applications for which the
performance goals apply.
Limited Data to Measure FDA's Performance against the MDUFMA
Performance Goals Established for Fiscal Years 2003 and 2004:
Limited data exist to measure FDA's performance against the two MDUFMA
performance goals established for fiscal years 2003 and 2004. Our
analysis shows that FDA received 43 PMAs, 7 Panel-Track Supplements,
and 3 Expedited PMAs in the fiscal year 2003 cohort.[Footnote 14] As of
March 31, 2004, one application--an Expedited PMA--had been subject to
the cycle action tied to one of the two MDUFMA performance goals for
fiscal year 2003.[Footnote 15] In its review of this application FDA
determined that the manufacturer submitted a complete response to an
approvable letter pending minor corrections or clarifications, and
approved the application within 30 days. For PMAs and Panel-Track
Supplements, the same MDUFMA performance goal applied for fiscal year
2003. However, FDA did not have any applications that required the
issuance of an approvable letter. Similarly, none of the 15 PMAs, 1
Panel-Track Supplement, and 8 Expedited PMA applications from the
fiscal year 2004 cohort required issuance of approvable letters.
Many of the applications from the fiscal years 2003 and 2004 cohorts
were pending within the review process for FDA review actions or
manufacturers' responses to FDA. Therefore, FDA's performance results
are preliminary. In the fiscal year 2003 cohort, 21 of the 50 PMAs and
Panel-Track Supplements and 1 of the 3 Expedited PMAs FDA received were
pending as of March 31, 2004. Similarly, in the fiscal year 2004
cohort, 13 of the 15 PMAs, 1 Panel-Track Supplement, and all 8 of the
Expedited PMAs FDA received were pending action. Because FDA's
performance against the MDUFMA performance goals is based on the
percentages of actions the agency takes on applications within required
review times, FDA's performance results could change as the agency
completes actions on all applications for which the performance goals
apply. Data were not readily available from FDA on the status of all
pending applications within the review process. FDA anticipates having
complete data by the end of fiscal year 2004.
Limited Performance Data to Determine the Likelihood of FDA Meeting the
Fiscal Year 2005 MDUFMA Performance Goals:
The likelihood of FDA meeting the MDUFMA performance goals for fiscal
year 2005 is uncertain because data to measure the agency's performance
are limited. Specifically, FDA had data that allowed us to compare its
performance against some, but not all, of the 20 MDUFMA performance
goals that will be effective in fiscal year 2005. For example, from the
fiscal year 2003 cohort, data were available to compare FDA's
performance against 17 of the 20 performance goals for fiscal year
2005. FDA took actions on applications tied to 14 of the 17 performance
goals within the established goal time frames. For 7 of the 14
performance goals, FDA's performance was based on one or two actions on
applications. FDA did not take actions within the established goal time
frames on applications tied to 3 of the 17 performance goals.
Similarly, from the fiscal year 2004 cohort, FDA had data for 11 of the
20 MDUFMA performance goals. We found that FDA took actions on
applications tied to each of the 11 performance goals within the
established goal time frames. For 4 of the 11 performance goals, FDA's
performance was based on no more than three actions on applications.
(See table 3.):
Table 3: Status of FDA's Performance for Fiscal Years 2003 and 2004 Compared to MDUFMA Performance Goals That Become Effective in Fiscal Year 2005:
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
Cycle actions and decisions: Cycle action: Issue first major deficiency
letter requesting significant information from the manufacturer;
Performance goal: Review time: 150 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 75;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 85%; (22 of 26 cycle actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 100%; (2 of 2 cycle actions).
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
Cycle actions and decisions: Decision: Issue decision letter (approval,
approvable pending GMP inspection, approvable pending minor corrections
or clarifications, not approvable, or denial) as a first action on an
application;
Performance goal: Review time: 180 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 75;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 96%; (22 of 23 decisions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 100%; (3 of 3 decisions).
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
Cycle actions and decisions: Cycle action: Issue second or subsequent
major deficiency letter requesting significant information from the
manufacturer;
Performance goal: Review time: 120 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 75;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (1 of 1 cycle action);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
Cycle actions and decisions: Cycle action: Determine whether an
amendment contains a complete response to a major deficiency or not
approvable letter;
Performance goal: Review time: 180 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 75;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 89%; (8 of 9 cycle actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: PMAs, Panel-Track Supplements, and Premarket
Reports[A];
Cycle actions and decisions: Cycle action: Review whether an amendment
contains a complete response to an approvable letter pending minor
corrections or clarifications and make a decision, such as approving
the application;
Performance goal: Review time: 30 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 90;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: [B];
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: Expedited PMAs;
Cycle actions and decisions: Decision: Issue decision letter (approval,
approvable pending GMP inspection, approvable pending minor corrections
or clarifications, not approvable, or denial) after taking a cycle
action on an application;
Performance goal: Review time: 300 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 70;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (2 of 2 decisions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: Expedited PMAs;
Cycle actions and decisions: Cycle action: Issue first major deficiency
letter requesting significant information from the manufacturer;
Performance goal: Review time: 120 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 70;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (2 of 2 cycle;
actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 100%; (4 of 4 cycle actions).
Type of application: Expedited PMAs;
Cycle actions and decisions: Decision: Issue decision letter (approval,
approvable pending GMP inspection, approvable pending minor corrections
or clarifications, not approvable, or denial) as a first action on an
application;
Performance goal: Review time: 170 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 70;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (1 of 1 decision);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 100%; (1 of 1 decision).
Type of application: Expedited PMAs;
Cycle actions and decisions: Cycle action: Issue second or subsequent
major deficiency letter requesting additional information from the
manufacturer;
Performance goal: Review time: 100 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 70;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: [B];
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: Expedited PMAs;
Cycle actions and decisions: Cycle action: Determine whether an
amendment contains a complete response to a major deficiency or not
approvable letter;
Performance goal: Review time: 170 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 70;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (1 of 1 cycle action);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: Expedited PMAs;
Cycle actions and decisions: Cycle action: Review whether an amendment
contains a complete response to an approvable letter pending minor
corrections or clarifications and make a decision, such as approving
the application;
Performance goal: Review time: 30 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 90;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (1 of 1 cycle action);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: 180-day PMA Supplements;
Cycle actions and decisions: Decision: Issue decision letter (approval,
approvable pending GMP inspection, approvable pending minor corrections
or clarifications, not approvable, or denial) after taking a cycle
action on an application;
Performance goal: Review time: 180 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 80;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 95%; (194 of 205 decisions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 100%; (15 of 15 decisions).
Type of application: 180-day PMA Supplements;
Cycle actions and decisions: Decision: Issue a not approvable letter as
a first action on an application;
Performance goal: Review time: 120 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 80;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 19%; (6 of 32 cycle actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 83%; (5 of 6 cycle actions).
Type of application: 180-day PMA Supplements;
Cycle actions and decisions: Decision: Issue decision letter (approval,
approvable pending GMP inspection, approvable pending minor corrections
or clarifications, or denial) as a first action on an application;
Performance goal: Review time: 180 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 80;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 96%; (166 of 173 decisions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 100%; (9 of 9;
decisions).
Type of application: 180-day PMA Supplements;
Cycle actions and decisions: Cycle action: Determine whether an
amendment contains a complete response to a not approvable letter;
Performance goal: Review time: 160 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 80;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (15 of 15 cycle actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 100%; (1 of 1 cycle actions).
Type of application: 510(k)s;
Cycle actions and decisions: Decision: Issue decision letter (for
example, a letter for a device that may be marketed because it is
substantially equivalent to one already on the market, or that may not
be marketed because it is not substantially equivalent);
Performance goal: Review time: 90 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 75;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 80%; (2,869 of 3,598 decisions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 96%; (865 of 904 decisions).
Type of application: 510(k)s;
Cycle actions and decisions: Cycle action: Issue first additional
information letter;
Performance goal: Review time: 75 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 70;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 58%; (1,004 of 1,718 cycle
actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 74%; (375 of 504 cycle actions).
Type of application: 510(k)s;
Cycle actions and decisions: Cycle action: Issue second or subsequent
additional information letter;
Performance goal: Review time: 60 days;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 70;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 53%; (283 of 530 cycle actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: 96%; (54 of 56 cycle actions).
Type of application: BLA Resubmissions and BLA Supplement
Resubmissions;
Cycle actions and decisions: Cycle action: Review and act on a Class 1
resubmission to an original BLA or BLA supplement (for example, issue
a letter requesting limited labeling or safety information from the
manufacturer);
Performance goal: Review time: 2 months;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 75;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: [B];
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Type of application: BLA Resubmissions and BLA Supplement
Resubmissions;
Cycle actions and decisions: Cycle action: Review and act on a Class 2
resubmission to an original BLA or BLA supplement (for example, issue
a letter requesting data on product safety and effectiveness from the
manufacturer);
Performance goal: Review time: 6 months;
Performance goal: Percentage of actions required to meet the goal for
the
fiscal year 2005 cohort: 75;
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2003 cohort: 100%; (2 of 2 cycle actions);
Percentage of actions taken on applications within goal‘s established
time frame: Fiscal year 2004 cohort: [B].
Source: GAO analysis of FDA data as of March 31, 2004.
[A] FDA groups these types of applications when measuring performance
for this goal. In both the fiscal year 2003 and 2004 cohorts, FDA did
not receive any Premarket Report applications.
[B] FDA did not have any applications that required the agency to take
the action tied to the performance goal.
[End of table]
As previously mentioned, many of the applications from the fiscal years
2003 and 2004 cohorts are awaiting actions by FDA or responses from
manufacturers. Our analysis of the applications pending within the
review process shows that of the 4,175 applications received in the
fiscal year 2003 cohort, 339, or 8 percent, were pending further action
by FDA as of March 31, 2004. Of the 1,781 applications received in the
fiscal year 2004 cohort, 859, or 48 percent, were also pending as of
March 31, 2004. The number of applications pending varied by
application type. (See table 4.) For example, in the fiscal year 2003
cohort, 21--or 42 percent--of the 50 PMAs and Panel-Track Supplements
were pending, while 316--or 8 percent--of the 3,914 510(k)s were
pending further action by FDA. As FDA completes its actions on more
applications, its performance results could change.
Table 4: Applications Pending within FDA's Review Process in the Fiscal
Year 2003 Cohort and the First 6 Months of the Fiscal Year 2004 Cohort:
Type of application: PMA and Panel-Track Supplements;
Fiscal year 2003: Total number of applications: 50;
Fiscal year 2003: Number (percentage) pending: 21; (42%);
Fiscal year 2004: Total number of applications: 16;
Fiscal year 2004: Number (percentage) pending: 13; (88%).
Type of application: Expedited PMAs;
Fiscal year 2003: Total number of applications: 3;
Fiscal year 2003: Number (percentage) pending: 1; (33%);
Fiscal year 2004: Total number of applications: 8;
Fiscal year 2004: Number (percentage) pending: 8; (100%).
Type of application: 180-Day PMA Supplements;
Fiscal year 2003: Total number of applications: 206;
Fiscal year 2003: Number (percentage) pending: 1; (1%);
Fiscal year 2004: Total number of applications: 42;
Fiscal year 2004: Number (percentage) pending: 27; (64%).
Type of application: 510(k)s;
Fiscal year 2003: Total number of applications: 3,914;
Fiscal year 2003: Number (percentage) pending: 316; (8%);
Fiscal year 2004: Total number of applications: 1,714;
Fiscal year 2004: Number (percentage) pending: 810; (47%).
Type of application: BLA Resubmissions and BLA Supplement
Resubmissions;
Fiscal year 2003: Total number of applications: 2;
Fiscal year 2003: Number (percentage) pending: 0; (0%);
Fiscal year 2004: Total number of applications: 1;
Fiscal year 2004: Number (percentage) pending: 1; (100%).
Total;
Fiscal year 2003: Total number of applications: 4,175;
Fiscal year 2003: Number (percentage) pending: 339; (8%);
Fiscal year 2004: Total number of applications: 1,781;
Fiscal year 2004: Number (percentage) pending: 859; (48%).
Source: GAO analysis of FDA data.
[End of table]
According to FDA officials, in fiscal year 2003, FDA began implementing
activities that are intended to enhance its ability to meet MDUFMA
performance goals that become effective in fiscal year 2005. These
activities, identified in the Secretary of HHS's November 2002 letter
establishing the MDUFMA performance goals, include hiring additional
staff, consulting with experts outside the agency more frequently, and
holding meetings with manufacturers to ensure high-quality applications
are submitted to FDA. For example, in fiscal year 2003, CDRH hired
staff for 67 new positions, such as medical officers, scientists, and
engineers, to improve the timeliness of its device reviews. As of April
2004, CDRH filled 23 of the 65 positions it plans to fill in fiscal
year 2004. CBER also filled each of the 11 full-time equivalent
positions that it received in fiscal year 2003 and planned to fill 9
full-time equivalent positions during fiscal year 2004 to improve the
timeliness of device reviews as well as other activities.[Footnote 16]
In addition, to help FDA meet its performance goals, FDA held about
100 meetings with manufacturers to discuss ways to improve the quality
of applications prior to their submission.
FDA Obligated over $128 Million for Postmarket Medical Device
Compliance Activities and Inspections in Fiscal Year 2002:
In fiscal year 2002, FDA obligated about $128 million for postmarket
medical device compliance activities, which include inspections of
device manufacturers' establishments. FDA obligated about $109 million
for compliance activities and about $19 million for inspections. Of the
approximately $109 million, FDA obligated about $99 million for
postmarket compliance activities that encompass outreach coordination,
applied research, and laboratory analyses. Outreach coordination
included funds for guidance to field staff on matters such as the
reporting of problems with medical devices, epidemiology studies,
device recalls, and other activities. Obligations for applied research
included funding for activities such as the development of domestic and
international standards to help provide reasonable assurance that
medical devices are safe and effective. Obligations for laboratory
analyses included funding for activities such as FDA laboratory-based
investigations and scientific training for FDA inspection staff. FDA
also obligated an estimated $10 million in rental expenses for office
space for FDA staff who conduct compliance activities.[Footnote 17]
In fiscal year 2002, of the $19 million obligated for inspections, FDA
obligated about $17 million for inspections of domestic and foreign
establishments and about $2 million in estimated office space rental
expenses for FDA staff who conduct inspections.[Footnote 18] These
inspections included routine surveillance inspections to determine
compliance with medical device regulations, inspections resulting from
the reporting problems with devices or product recalls, and compliance
inspections to collect evidence for pending enforcement actions.
Agency Comments:
In commenting on a draft of this report, FDA generally agreed with our
findings. FDA stated that it believes the agency has made a good start
in its implementation of MDUFMA and FDA believes that its progress in
meeting the MDUFMA performance goals is consistent with the extensive
performance improvements the agency is expect to achieve each year
through 2007. FDA provided technical comments primarily clarifying
aspects of the medical device review process, which we incorporated as
appropriate. FDA's comments are reprinted in appendix I.
We are sending copies of this report to the Secretary of HHS and the
Commissioner of FDA, appropriate congressional committees, and other
interested parties. We will also make copies available to others on
request. In addition, the report is available at no charge on the GAO
Web site at [Hyperlink, http://www.gao.gov]. If you or your staffs have
questions about this report, please contact me at (202) 512-7119 or
James McClyde at (202) 512-7152. Darryl Joyce, Donna Bulvin, and
Krister Friday also made key contributions to this report.
Signed by:
Marcia Crosse:
Director, Health Care--Public Health and Military Health Care Issues:
[End of section]
Appendixes:
Appendix I: Comments from the Food and Drug Administration:
DEPARTMENT OF HEALTH & HUMAN SERVICES:
Food and Drug Administration:
Rockville MD 20857:
August 18, 2004:
Marcia G. Grosse, Ph.D.
Director, Health Care - Public Health and Military Health Care Issues:
United States Government Accountability Office:
441 G Street, N.W., Room 5A14:
Washington, DC 20548:
Dear Dr. Crosse:
FDA appreciates the opportunity to review and provide comments on GAO's
draft report, Data to Measure the Timeliness of Reviews of Medical
Device Applications Are Limited. The draft is thorough, provides a good
overview of the complex and challenging performance goals FDA is
working to achieve under the Medical Device User Fee and Modernization
Act of 2002 (MDUFMA), and we generally agree with GAO's findings. We
are providing brief technical comments to improve the accuracy and
completeness of GAO's description of FDA's medical device review
processes and to provide additional suggestions where we believe minor
clarifications would be helpful.
I believe FDA has made a good beginning in our implementation of
MDUFMA. The performance goals that are the primary focus of your report
are a key feature of MDUFMA, but only one of the challenges we are
working to meet. We are also working to meet expectations for third-
party establishment inspections, the new regulatory requirements for
reprocessed single-use devices, and new provisions for pediatric
devices. While we work to make these and other improvements, we're also
working to ensure that we maintain our performance in areas that don't
have specific MDUFMA performance goals.
Although only two of MDUFMA's measurable performance goals were in
effect for FY 2003 and FY 2004, our progress to date is, we believe,
consistent with the extensive performance improvements we are expected
to achieve each year through FY 2007. We will face some significant
challenges as we work to meet all of MDUFMA's goals-this is
particularly true for MDUFMA's cycle goals - but we believe we are now
laying the foundation for future progress, even on the most difficult
goals. We are successfully recruiting essential scientific and medical
experts and we are expanding our consultation with experts outside the
agency; these new resources strengthen our review processes and help us
conduct quality reviews in less time. We're working to improve and
expand our guidance to industry, to consult with stakeholders as we
make policy and program improvements, and to provide additional
opportunities for direct communications with applicants. We are mindful
of the need to be responsible custodians of the user fees and
additional appropriations MDUFMA is providing, and we continue to
invest these new resources where they will do the most good for the
process for the review of device applications. We're also working hard
to modernize our IT infrastructure to enable rapid, efficient reviews,
and to position the agency for future electronic submissions and
reviews. We look forward to continued cooperative efforts with all
interested parties to ensure the MDUFMA legislation is recognized as an
unqualified success.
Along with the entire FDA team, I am strongly committed to meeting
MDUFMA's challenging performance goals and to making the other process
improvements necessary to provide a fair, transparent, and predictable
process to rapidly bring new medical devices to patients and the health
care community.
If you have any questions concerning our comments, or need additional
information to complete your final report, please feel free to contact
me or Linda Kahan, Deputy Director, CDRH. Ms. Kahan may be reached at
(301) 827-7975, or by e-mail at linda.kahan@fda.gov. Thank you again
for allowing us to review and comment on your report.
Sincerely,
Signed for:
Lester M. Crawford, D.V.M., Ph.D.
Acting Commissioner of Food and Drugs:
[End of section]
(290344):
FOOTNOTES
[1] The term "approval" is generally used for applications for new
devices, while the term "clearance" is used for devices that are
substantially equivalent to those legally on the market.
[2] Pub. L. No. 107-250, 116 Stat. 1588.
[3] FDA's request for additional information on such applications is
known as an "approvable" letter.
[4] Ch. 675, 52 Stat. 1040 (1938) (codified as amended at 21 U.S.C. §§
301 et seq. (2000)).
[5] In general, medical device applications for PMA that are reviewed
by FDA include information on the device and its components; proposed
labeling for the device; and when applicable, clinical and nonclinical
studies that provide reasonable assurance of the device's safety and
effectiveness.
[6] FDA uses the term 510(k) to refer to a premarket notification
submission.
[7] Substantial equivalence means that a device has (1) the same
intended use and same technological characteristics as a marketed
device or (2) the same intended use and different technological
characteristics but is as safe and effective as the marketed device and
does not raise new questions of safety and effectiveness.
[8] BLAs are used to request permission to introduce and license
biological products into interstate commerce.
[9] According to FDA officials, BLA applications go through a review
process that is similar to the PMA review process.
[10] The scientific review can include reviews of results from clinical
investigations of the device that involve human subjects. FDA also
reviews nonclinical studies of the device, studies that may include
microbiological, toxicological, and engineering tests.
[11] According to FDA, approximately 13 percent of the PMAs, Panel-
Track Supplements, and Expedited PMA applications are referred to
external advisory committees.
[12] Except for BLA Resubmissions and BLA Supplement Resubmissions,
there are also limits on the length of time manufacturers have to
respond to the agency's requests for additional information. For
example, as required by FDA regulation, manufacturers submitting
amendments to PMAs have 180 days to respond to major deficiency
letters. Manufacturers submitting amendments to PMAs can also apply for
extensions of up to 180 days beyond the required response time.
Manufacturers submitting amendments to 510(k)s have 30 days to respond
to first or subsequent letters requesting additional information. For
amendments to 510(k)s, manufacturers can apply for extensions of up to
180 days from the date of the first or subsequent letters.
[13] FDA refers to cohorts as "receipt cohorts."
[14] FDA received other types of applications in the fiscal year 2003
and 2004 cohorts, but these types are not connected to the MDUFMA
performance goals established for fiscal years 2003 and 2004. FDA did
not receive any Premarket Report applications, the fourth type of
application tied to the fiscal year 2003 and 2004 MDUFMA performance
goals.
[15] The Expedited PMA is tied to one of the two MDUFMA performance
goals.
[16] CBER did not hire additional staff to work exclusively on device
applications because its staff works with both devices and biologics.
However, biologics account for the majority of CBER's resources.
[17] FDA does not include rental amounts for each center in its annual
budget justification. Rental expenses are reported as a single amount
for all centers. Therefore, the amounts obligated for rental expenses
are estimated.
[18] These amounts exclude obligations for inspections of mammography
facilities under the Mammography Quality Standards Act of 1992.
Mammography inspections are not postmarket device establishment
inspections as described in section 704(g)(10)(B) of FDCA.
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