Economic and Quality of Life Outcomes in Oncology: The Regulatory Perspective

Economic and Quality of Life Outcomes in Oncology: The Regulatory Perspective

ABSTRACT: The federal government's involvement in cost-effectiveness studies, outcomes measures, and practice guideline development is haphazard, with a number of agencies taking part in the process. The Health Care Financing Administration (responsible for Medicare and Medicaid) and other third-party government payers informally and unscientifically judge the cost-effectiveness of treatments, particularly new technologies, when making coverage decisions. FDA policy restricts pharmaceutical companies from disseminating information on cost-effectiveness by requiring that such data be part of the product labeling. The Agency for Health Care Policy and Research has produced only a small number of practice guidelines and at a much higher cost than the private sector. Medical societies and other private organizations will ultimately find it in their best interest to include cost-effectiveness and outcomes measures in study protocols and to develop treatment guidelines related to their specialties. [ONCOLOGY 9(Suppl):121-125, 1995]


Sensitivity to health care costs in general and the advent of
managed care in particular have greatly enhanced the perceived
value of outcomes measures of both cost-effectiveness and quality
of life. Since the late 1980s, the federal government has consciously
sought to exert a major influence on such activity, but, even
before that, federal agencies used these criteria to make treatment
and payment decisions. The medical profession is now seeking to
assume a leading role in defining practice parameters, but in
doing so it must be mindful of the regulatory environment in which
these decisions are made.

There are four US regulatory agencies with potential jurisdiction
over oncology outcomes measures: the Agency for Health Care Policy
and Research (AHCPR), Food and Drug Administration (FDA), Health
Care Financing Administration (HCFA), and Federal Trade Commission
(FTC). This article will explore the involvement of each of these
agencies in cost-effectiveness and quality of life outcomes and
how their policies affect the practice of oncology.

Agency for Health Care Policy and Research

In an effort to provide increased funding for and emphasis on
outcomes measures, Congress replaced the National Center for Health
Services Research with AHCPR in 1989. The agency's general purpose
is to conduct research to improve the quality, appropriateness,
and effectiveness of health care services. Congress hoped that
AHCPR's outcomes research would lead to the elimination of unnecessary
procedures and thus save a sizable amount of money.

The agency evaluates and disseminates information regarding the
cost-effectiveness and outcomes of health care services and procedures,
which may in some instances form the basis for practice guidelines.
In addition, AHCPR is specifically responsible for:

1. Funding Patient Outcomes Research Team (PORT) projects, 5-year
studies of the prevention and management of conditions such as
prostate disorders and diabetes.

2. Preparing the National Medical Expenditures Survey, which examines
national health spending every 10 years.

3. Evaluating the safety and effectiveness of specific medical
technologies at the request of the Medicare and CHAMPUS (Civilian
Health and Medical Program of the Uniformed Services) programs,
and recommending whether the federal government should pay for
them. [The Office of Health Technology Assessment (OHTA), a component
of AHCPR, performs these assessments.]

Track Record-Although its overall success may be open to
question, the AHCPR's achievements include the funding of a number
of PORTs and completion of 13 practice guidelines. A recent report
published by the Office of Technology Assessment (OTA) commended
the agency's PORTs-interdisciplinary research teams that study
medical conditions and the effectiveness of medical practices
to diagnose, treat, and manage them. According to OTA, the PORTs
have been particularly successful in developing outcomes measures
using patient assessments and in identifying the use and effectiveness
of medical practices in particular patient populations. Examples
of PORT projects include the Back Pain Outcome Assessment Team;
Assessing Therapies for Benign Prostatic Hypertrophy and Localized
Prostate Cancer; and Cure, Costs and Outcomes of Local Breast

The agency has also been praised for certain of its efforts in
guideline development, including the Cancer Pain Management Guidelines
released last year. Other practice guidelines produced or under
development include those relating to lower back problems, benign
prostatic hyperplasia, and colorectal cancer screening (this guideline
is being produced by the American Gastroenterological Association
under a contract with AHCPR).

Since its inception, AHCPR has produced only 13 guidelines. The
agency is currently writing nine additional guidelines and plans
to announce new guideline topics this spring. The 13 guidelines
written by AHCPR represent a very small portion of the total of
approximately 1,500 practice guidelines produced to date. The
vast majority of practice guidelines are produced by more than
45 private organizations, including many medical specialty societies.
The cost to the agency of approximately $1 million per guideline
is dramatically more than the amount medical societies pay, which
in most cases is $100,000 or less.

Shortcomings-While OTA's recent report acknowledged AHCPR's
contributions, it did note certain weaknesses as well. Because
AHCPR is not the only federal agency responsible for drafting
practice guidelines, the government lacks a coordinated approach
to evaluating outcomes measures and producing practice guidelines.
Other agencies responsible for writing guidelines include the
Centers for Disease Control and Prevention (CDC), and the National
Institutes of Health (NIH).

In response to this problem, OTA recommended that Congress designate
a single lead agency to coordinate guideline activities. The OTA
report sets out a number of options for creating this centralized
system: (1) Fold AHCPR into a "new, larger agency with a
broader mandate and more resources," (2) designate AHCPR
the lead agency for coordinating guideline efforts, or (3) encourage
or require the various existing agencies to act collaboratively
through "administrative mechanisms."

According to OTA, a central failure of AHCPR and the entire federal
effectiveness effort has been the inadequate use of prospective
comparative studies, particularly randomized clinical trials,
to evaluate the comparative effectiveness of alternative treatments.

The OTA also reported that while AHCPR's efforts in effectiveness
research will probably improve the quality of health care, they
"will not necessarily reduce health care costs significantly."
This finding countered Congress' intent that AHCPR's activities
would lead to health care savings. The report stated that effectiveness
research should be considered a good "buy" if it improves
health care and pays for its own research-related costs.

The AHCPR's "passive dissemination of guidelines" contributes
to the failure to sufficiently transfer the guideline findings
into clinical practice, the OTA report said. Providers also often
do not readily adopt the recommendations in guidelines.

Future-Last year, during the health care reform debate,
many of the Democratic bills included provisions that would have
increased the role of AHCPR. Sponsors of such provisions believed
that outcomes measures conducted by AHCPR and others were necessary
to combat rising health care costs and help ensure the affordability
of the expansive health care system they envisioned.

Now, with Congress controlled by the Republicans, AHCPR may become
a prime target for budget cutting. While in the past the agency
has had bipartisan support in Congress, this year it may have
to ward off efforts to trim its activities, if not eliminate it
completely. Given the high cost of producing practice guidelines
and the expanding, more cost-effective private sector efforts
in this field, Congress may choose to limit the federal role in
guideline development.

Food and Drug Administration

Through its regulation of information concerning products under
its jurisdiction, FDA exerts a significant impact on dissemination
of cost-effectiveness and outcomes data. The agency approves not
only drugs, devices, and biologicals, but also the labeling that
accompanies them. Moreover, it takes a very expansive view of
what constitutes "labeling," with the result that pharmaceutical
companies and other product sponsors are barred from disseminating
any information not completely consistent with the product's approved

In addition, FDA policy seeks to regulate presentations by physicians
or others when the program is funded by pharmaceutical money.
In the past, FDA has denounced physicians presenting on off-label
uses at pharmaceutical-sponsored seminars as the "paid agents"
of the sponsor and included them in enforcement actions. Consequently,
FDA often finds itself in the ironic position of asserting that
products are "misbranded" and therefore subject to enforcement
action because the sponsor has been found to be involved in dissemination
of entirely truthful and accurate information about product uses
beyond those on the approved label.

Off-Label Uses in Oncology-Many, if not most, anticancer
drugs are broadly accepted in the medical community for additional
"off-label" uses. Cancer patients rely heavily on off-label
uses, since most chemotherapy regimens involve one or more such
use. Furthermore, the pace of change in oncology therapy is rapid.
Results from the many ongoing clinical trials are quickly incorporated
into practice, and oncology drug labels are quickly superseded
by new information. FDA has no efficient mechanism for updating
drug labeling, thus making labels meaningful for the practitioner.

Variations in use of anticancer chemotherapy drugs can have significant
impact on both therapeutic outcome and treatment toxicity. Patients
and their physicians need ready access to reliable information
about these products. FDA has been urged to use previously published
peer-reviewed clinical research data as the basis for approving
supplemental new drug applications (SNDAs) for new uses of an
already approved product. While FDA accepts this approach in theory,
its practice has been not to approve SNDAs for new indications
without full-blown clinical trials with all the supporting background

Quality of Life and Cost-effectiveness Claims-In addition
to restricting information about new off-label uses of approved
products, FDA increasingly is paying attention to and regulating
claims made by pharmaceutical companies regarding their products'
cost-effectiveness or quality of life benefits. The agency is
in the process of formulating its policies concerning this subject,
and guidance in this area often is not clear.

Underlying this issue is a debate regarding the type of data necessary
to make quality of life and cost-effectiveness claims. FDA representatives
have suggested that data should come from controlled clinical
studies. The agency is recommending that pharmaceutical companies
design protocols to show cost-effectiveness and quality of life
advantages over competing products. Companies would have to show
that their products have fewer side effects, work faster, and
require less follow-up care than competing drugs. The pharmaceutical
industry, on the other hand, contends that valuable cost data
can emerge from after-the-fact analysis of patient records.

Legal Challenge to FDA-While FDA must prevent manufacturers
from making false claims regarding the cost-effectiveness of their
products, the agency now finds itself in the position of restricting
the free flow of truthful information. A conservative think tank,
the Washington Legal Foundation, has submitted a Citizen's Petition
and filed a lawsuit challenging FDA's policy on promotion of off-label
uses on the grounds that it exceeds the agency's statutory authority
and violates the First Amendment.

The Foundation argues that FDA should not seek to restrict the
dissemination of truthful information about products, even if
such dissemination is facilitated by the sponsoring company. FDA
sought public comments regarding the Foundation's challenge in
a Federal Register notice of November 18, 1994 (59 Fed Reg 59820).

Proposed Legislation-During the last Congress, Rep. Ron
Wyden (D-OR) proposed legislation that would have encouraged pharmaceutical
companies to conduct trials to demonstrate cost-effectiveness
in comparison to other therapies. Such legislation would address
one of the weaknesses identified in OTA's report on AHCPR-the
inadequate number of trials comparing the effectiveness of alternative

Under various versions of this bill, which was never introduced,
companies that conducted cost-effectiveness trials would have
received certain incentives, including additional limited periods
of exclusivity, expedited approval of supplemental new drug applications,
the ability to charge for products involved in such studies, a
requirement that the Secretary of Health and Human Services review
Medicare and Medicaid reimbursement and coverage barriers that
limit access to products deemed superior under an approved study
and remove any such barriers, and a provision that FDA's ability
to regulate marketing cost-effectiveness claims be limited to
requiring disclosure of the assumptions underlying such cost claims.

This proposed legislation went nowhere, in great part because
the exclusivity provisions were deleted. Industry maintains that
additional exclusivity is necessary to provide companies with
the resources needed to conduct research on cost-effectiveness.
Similar legislation including the exclusivity incentive could
receive a better reception in the new Republican majority Congress.

Legislation like that initially proposed by Rep. Wyden, including
the exclusivity incentive, would encourage pharmaceutical companies
to continue and augment their trend toward incorporating cost-effectiveness
claims into clinical trials. While companies have been slower
to include quality of life in their trials, there is a clear and
growing trend to conduct cost-effectiveness analysis in clinical
trials of new treatments. In today's marketplace, companies recognize
that both cost-effectiveness and quality of life claims can provide
them with a competitive advantage. Managed care plans often require
such analyses.


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