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July 1998
Off-Label
Use of Drugs: A Sticky Situation
Introduction: Off-label uses and high-cost drugs: promotion and reimbursement
issues.
Shulman, SR. Drug Information Journal. 1998;32:363-5.
[ no abstract available ]
A shift in the regulatory approach.
Woodcock, J. Drug Information Journal. 1998;32:367-71.
[ see abstract below ]
The effect of Pharmacy Benefit Manager Clinical Programs and Services
on access to prescription drug benefit coverage.
Teagarden, JR. Drug Information Journal. 1998;32:373-7.
[ see abstract below ]
Implications of regulatory policies on off-label uses for industry competitiveness.
Ruggieri, NL. Drug Information Journal. 1998;32:379-81.
[ see abstract below ]
Questions to ponder concerning off-label uses.
Hansen, JC. Drug Information Journal. 1998;32:383-4.
[ see abstract below ]
Many of the drugs used in the practice of anesthesiology and perioperative
medicine fall subject to "off-label" applications. Off-label use can be
defined as any indication, dosage form, dose regimen, population, or other
parameter for use of the drug not mentioned in the approved labeling. In
essence, the term "off-label" refers to any deviation from the use defined
in the approved labeling. This series of short articles presented in the
Drug Information Journal (click
here to visit their web site) highlight some of the important issues
related to off-label uses of drugs in general; and with growing issues of
cost containment and evidenced-based medicine, all indications and uses
of virtually all drugs will be subject to increasing scrutiny in the future.
In her introduction to this series of articles, Dr. Sheila Shulman states:
"It is not unreasonable to expect that prescription drug benefits will
be the target of future restrictions as employers and third-party payers
experiment with more rigorous cost-containment interventions. Control of
drug expenditures has always posed a challenge for managed-care, and additional
pressures on this front can be expected with larger numbers of Medicare
enrollees integrated into HMOs. Closed or restricted formularies will become
a more attractive option. Payers increasingly will look for convincing evidence
that the drugs they agree to pay for actually deliver the promised clinical
benefit. Physicians can expect more aggressive monitoring of their prescribing
practices by third parties. Patients should not be surprised at the introduction
of more restrictive drug benefits or the expectation that they contribute
more out-of-pocket toward the cost of the drug therapy."
The four papers that follow Dr. Shulman's introduction touch on these issues
of access beyond the context of coverage or reimbursement for off-label
uses and more costly drugs. Dr. Janet Woodcock reviews the recent evolution
of the FDA's policies and procedures, suggesting a movement toward greater
flexibility on evidentiary standards for both new drugs and supplemental
applications. She also explains why the FDA may be reevaluating its approach
to off-label drug use. It would also appear that some areas of traditional
FDA control are being transferred to a more sophisticated marketplace which
will assume greater authority over the use of prescription drugs.
Mr. Nicholas Ruggieri addresses a contentious issue related to the dissemination
of materials discussing off-label uses. Recent legislative proposals have
included a relaxation of regulations governing this area. In his article,
Mr. John Hansen raises a number of important questions. One of these questions
reminds us that while drug companies are very interested in obtaining quick
FDA approval for new indications, they are also concerned about the cost
of getting a supplemental application approved by FDA. From a cost perspective,
the question, then, is when is it in a company's interest to actually pursue
FDA approval of a supplemental indication? At what point is balance achieved
between the interests of the FDA and the interests of the company? How quickly
can approval for a new indication make it worth the company's while to pursue
FDA approval for that indication?
These are just a few of the many issues that deserve attention during these
interesting times of competing cost-containment, quality, practice standards
and evidenced-based healthcare.
Return to the Current Literature
Review Front Page, or read the abstract:
ABSTRACT
Woodcock, J. A shift in the regulatory approach. Drug
Information Journal. 1998;32:367-71.
SUMMARY: This paper highlights the shifts in the Food and Drug Administration's
(FDA) approach to the off-label use of drugs, including definitions and
a discussion of current policies, reasons for the changes, and current initiatives
in off-label use. Off-label uses must be studied and effective uses should
be labeled. FDA is adopting new strategies to get more uses on the label.
Teagarden, JR. The effect of Pharmacy Benefit Manager Clinical Programs
and Services on access to prescription drug benefit coverage. Drug Information
Journal. 1998;32:373-7.
SUMMARY: This paper provides an overview of the effects of a pharmacy
benefit manager�s programs and services on access to prescription drug coverage.
Coverage is increasingly used by prescription drug benefit plans as a means
to reduce the potential for exposure to unnecessary costs of providing the
benefit. Certain fundamentals must be adhered to so as to avoid creating
unintended and inappropriate barriers to access and an appeals process must
be in place. Merck-Medco�s development and implementation of its clinical
programs and services that involve prescription drug coverage decisions
is highlighted.
Ruggieri, NL. Implications of regulatory policies on off-label uses for
industry competitiveness. Drug Information Journal. 1998;32:379-81.
SUMMARY: Regulatory policy with regard to off-label uses is discussed
from the perspective of a research and development-oriented pharmaceutical
manufacturer. Opening up the policy on dissemination of information on off-label
uses will affect incentives to go after orphan-specific labeling and poses
an issue for industry competitiveness because multiple sponsors for the
same drug with different indications can benefit from the work of the first
sponsor. When opening up dissemination, strict guidelines must be employed
to preserve incentives for research that will benefit patient populations
being targeted.
Hansen, JC. Questions to ponder concerning off-label uses. Drug Information
Journal. 1998;32:383-4.
SUMMARY: This paper provides an overview of the General Accounting
Office�s (GAO�s) role with respect to the Food and Drug Administration (FDA)
and the pharmaceutical industry. An independent, third-part study of off-label
drug uses conducted by the GAO is discussed. The perspectives on off-label
uses of FDA, the United States Congress, the pharmaceutical industry, and
pharmacy benefit managers are presented. Questions to be pondered are highlighted.
For more information visit the Drug Information
Journal
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