Off-label use is a prescription of a drug in a manner different from that approved by the FDA. This use of a drug is legal and common, and it can be done without adequate supporting data from clinical trials.
The company cannot promote the off-label use of drugs (with commercials). This use of approved drugs can be based on its mechanism of action ... or on outcomes from studies for a similar indication.
The key limiting factor can be insurance reimbursement. The prescribing doctor might have to provide the scientific rationale and medical necessity for this use of the drug and provide supporting data for similar indications.
NOTE: Off-label use should not be confused with Compassionate Use (expanded access), which is dependent on the company's willingness to provide a drug that's investigational. This use of an investigational drug is basically a single-patient study requiring also IRB approval from the treating center. Under new rules for expanded access the company can receive compensation for providing the experimental drug for compassionate use.
For off-label use the company approval is not the issue. The main issue is reimbursement by the patient's insurance provider. We may see reimbursement contested for expensive oral drugs, which can have also very high out-of-pocket costs. For example, ibrutinib, which was approved recently for relapsed Mantle Cell Lymphoma is a very expensive oral drug.
Medicare must cover off-label uses of anticancer chemotherapeutic regimens if they are supported by a citation in at least one of the following compendia:
American Hospital Formulary Service Drug Information (AHFS DI),
United States Pharmacopoeia Drug Information (USP DI) or
American Medical Association Drug Evaluations.3
Source: http://bit.ly/1nkq84m
There are good and bad aspects of off-label use, nicely described in the following article.
NEJM: Perspective: Regulating Off-Label Drug Use — Rethinking the Role of the FDA Randall S. Stafford, M.D., Ph.D.” http://bit.ly/1dZJ7Ld
THE GOOD SIDE: Physicians' freedom to prescribe drugs off-label carries important advantages. It permits innovation in clinical practice, particularly when approved treatments have failed. It offers patients and physicians earlier access to potentially valuable medications and allows physicians to adopt new practices based on emerging evidence. And it can provide the only available treatments for “orphan” conditions.
THE NEGATIVE SIDE: At the same time, off-label use has potentially negative consequences. It undercuts expectations that drug safety and efficacy have been fully evaluated. When newer, more expensive drugs are used off-label, it increases health care costs. It undermines the incentives for manufacturers to perform rigorous studies — and instead subtly encourages them to game the system by seeking approval for secondary indications for which clinical trials are less complicated and less expensive. And off-label use may discourage evidence-based practice.
.gif) |
Off-Label Drug Use in Cancer Treatment - National Cancer Institute http://bit.ly/2iAhNwd
|
|
Off-label Use: The Fine Line Between Illegal Promotion and Useful Information |BioWorld http://bit.ly/2iAmCpv
|
|
FDA Questions Need for Looser Off-Label Promotion Restrictions | RAPS http://bit.ly/2hD1T5O
|
|
NEJM: Perspective: Regulating Off-Label Drug Use — Rethinking the Role of the FDA Randall S. Stafford, M.D., Ph.D.” http://bit.ly/1dZJ7Ld
|
|
2013, Medscape:
Significant Off-Label Chemo Use in Elderly Cancer Patients http://bit.ly/1bfpP7W
|
|
Pharmacyclics, Inc. (PCYC): Long-time Pharmacyclics bull says near-term off-
label ibrutinib use may be curtailed - Seeking Alpha http://bit.ly/JSzYeC (may not be available anymore)
SNIP: "While subsequent compendium listings ... in RR CLL/SLL may lead to a change to this policy, we expect the absence of support of about $8900/patient contribution under Medicare Part D payment to be a significant barrier to the near-term use of the drug in the larger off-label settings," Wade says, adding that "there's a good amount of off-label expectations baked into some of the Street's expectations."
Comment: supporting my previous understanding that reimbursement is the main obstacle to off-label use (not rule) ... and that oral drugs makes it more challenging because of the very high out-of-pocket costs to patients
|
