Transcript Off Label Use - Academy of Managed Care Pharmacy

Off-Label Use in Managed Care
Pharmacy
Presentation Developed for the
Academy of Managed Care Pharmacy
Updated: February 2016
Objectives
• Define the term off-label use
• Discuss the difference between off-label and
off-compendium
• Understand the issues regarding off-label use
and where it may be appropriate
Objectives
• Discuss the perspective of managed care
pharmacy on off-label use
• Off-label use and non-FDA approved uses
and Medicare Part D for Biologic and
Chemotherapeutic Regimens
Definition of Off-Label Use
The use of a drug for indications, dosage forms
or regimens, or other use parameter not stated
in the product labeling approved by the Food
and Drug Administration (FDA)
What are the issues?
• Promotion of off-label use would compromise
three key factors:
– Definitive clinical trial data
– Evidence-based medicine
– Safety and efficacy standards
• Off-label uses may be potentially harmful
and/or lead to ineffective results
Areas of High Off-Label Use of Drug Products
• Oncology
– Medical Necessity
– Compassionate Use
• Pediatrics
– Limited availability of trial data
– Many uses not listed in product labeling
Why is Off-Label Use Common?
• Rigorous and time-consuming FDA review
process
• Costs associated with conducting clinical trials
• Lack of incentive
– Some drugs already off-patent
– Reimbursement already ongoing
FDA Policies Regarding Off-Label Use
• The Guidance for Industry on Good Reprint
Practices http://www.fda.gov/oc/op/goodreprint.html
• Provides distribution of journal articles on
unapproved off-label uses of approved drugs
acceptable as long as the materials meet
certain criteria for objectivity.
FDA Policies Regarding Off Label Use
• Drug manufacturers cannot pro-actively
discuss or provide written materials on offlabel uses.
– The manufacturer can provide information if
asked directly
• No other parties are restricted from discussing
or distributing written materials on off-label
uses
FDA Policies Regarding Off Label Use
• Off-label use of drugs by prescribers is often
appropriate
• Prescribers and other parties may obtain,
when requested, information regarding offlabel uses from drug manufacturers (following
the policy on solicited information)
Academy of Managed Care Pharmacy Perspective
• In support of off-label use of FDA-approved
drugs when medically appropriate/necessary
• Opposed to government-mandated coverage
of specific pharmaceuticals
• Believes each drug should be used in
accordance with generally accepted medical
practices
Academy of Managed Care Pharmacy: http://www.amcp.org.
AMCP Perspective
• Recognizes rapidly changing nature of
pharmacy and that FDA-approval of new
indications may be delayed
• Supports the consideration of specific criteria
when determining coverage of drug products
for off-label uses
Academy of Managed Care Pharmacy: http://www.amcp.org.
Criteria Considerations for Drug Coverage
• Whether the drug has been proven effective and accepted for
the treatment of the specific medical condition for which it
has been prescribed according to:
–
–
–
–
–
American Hospital Formulary Service-Drug Information® (AHFS-DI®)
Truven Health Analytics Micromedex DRUGDEX®
Clinical Pharmacology
NCCN Drugs & Biologics Compendium™
Lexicomp
• Whether the drug is recommended for the particular
condition involved, and has been proven to be safe and
effective for that condition according to formal clinical
studies, the results of which have been published in peerreviewed professional medical journals.
Academy of Managed Care Pharmacy: http://www.amcp.org.
Solutions to Consider
• Adequate incentives for drug manufacturers
to conduct clinical trials investigating off-label
uses
• Adoption of new strategies to get more uses
on approved product labeling
• Practice of evidence-based medicine,
including extensive literature review, to
determine appropriateness of drug coverage
for off-label uses
Medicare Part D
• FDA-approved drugs used for indications other
than what is indicated on the official label may
be covered under Medicare if the carrier
determines the use to be medically accepted,
taking into consideration the major drug
compendia, authoritative medical literature
and/or accepted standards
Recent Developments in Medicare Coverage of Off-Label Cancer Therapies. J Oncol Pract. 2009 January; 5(1): 18–20.
Medicare Benefit Policy Manual. Chapter 15: 50.4.2
Approved CMS Compendiums and Resources
• American Hospital Formulary Service Drug
Information (AHFS-DI)
• Truven Health Analytics Micromedex DrugDex
• Elsevier/Gold Standard Clinical Pharmacology
• National Comprehensive Cancer Network
(NCCN) Drugs & Biologics Compendium
Medicare Benefit Policy Manual. Chapter 15: 50.4.5
Compendium Criteria
• In general, a use is identified by a compendium as medically
accepted if the:
• indication is a Category 1 or 2A in NCCN, or Class I, Class IIa,or
Class IIb in DrugDex; or,
• narrative text in AHFS-DI or Clinical Pharmacology is supportive.
• A use is not medically accepted by a compendium if the:
• indication is a Category 3 in NCCN or a ClassIII in DrugDex; or,
• narrative text in AHFS or Clinical Pharmacology is “not
supportive.”
Medicare Benefit Policy Manual Ch. 15 50.4.5
Peer-Reviewed Medical Literature
• Peer-reviewed medical literature may appear in scientific,
medical, and pharmaceutical publications in which original
manuscripts are published, only after having been critically
reviewed for scientific accuracy, validity, and reliability by
unbiased, independent experts prior to publication.
• In-house publications of entities whose business relates to the
manufacture, sale, or distribution of pharmaceutical products
are excluded from consideration.
• Abstracts (including meeting abstracts) are excluded from
consideration.
Medicare Benefit Policy Manual Ch. 16 50.4.5.C
Biologics and Chemotherapeutic Regimen Summary
• FDA-approved drugs and biologics may also be considered for
use in the determination of medically accepted indications for
off-label use if determined by the Medicare contractor to be
reasonable and necessary.
• If a use is identified as not indicated by the Centers for
Medicare and Medicaid Services (CMS) or the FDA, or if a use
is specifically identified as not indicated in one or more of the
compendia listed, or if the contractor determines, based on
peer-reviewed medical literature, that a particular use of a
drug is not safe and effective, the off-label usage is not
supported and, therefore, the drug is not covered.
Medicare Benefit Policy Manual Ch. 16 50.4.5.D
Summary
• Off-label use refers to the use of a drug
product for indications not stated in the FDAapproved labeling
• Off-label use is common practice and may be
appropriate in many cases (Pediatrics and
Oncology)
• Extensive review of off-label uses, including
clinical trials, is necessary to determine
appropriateness of drug coverage
Ustekinumab (Stelara)
• 2009: Ustekinumab (Stelara) is FDA-approved for the treatment
of adult patients with moderate to severe plaque psoriasis who
are candidates for phototherapy or systemic therapy
• 2013: Ustekinumab (Stelara) is FDA-approved for the treatment
of adult patients with active psoriatic arthritis
• 2015: Ustekinumab (Stelara) is considered investigational in
Crohn’s disease
– Phase III data (UNITI-2) showed treatment induced clinical response and
clinical remission in patients with moderate to severe Crohn’s disease who
had previously failed conventional therapy
Phase 3 data show Stelara induced clinical response and remission in the treatment of patients with moderate to severe Crohn’s disease
[press release on the Internet]. Honolulu (HI): Janssen. 2015 Oct 19 [cited Dec 31 2015]. Available from: http://www.janssen.com/phase3-data-show-stelara-induced-clinical-response-and-remission-treatment-patients-moderate-severe.
Example: Ustekinumab (Stelara) Request
• A request for Stelara was received for a 50 year
old female with moderate to severe Crohn’s
disease. The patient had failed trials with
steroids and immunosuppressive therapy and
had an adverse reaction (rash) to adalimumab
(Humira).
– Is this request considered “off-label”?
– Would you approve or deny the request?
Thank you to AMCP member
Tasmina Hydery for updating
this presentation for 2016.