Following on from last week’s conversation, I left off with the issues surrounding the off-label prescriptive habits by physicians and the implicit role of the sponsors. The Academy of Managed Care Pharmacy (AMCP) supports off-label use of FDA-approved drugs when medically appropriate and necessary, but opposes government-mandated coverage of specific pharmaceuticals, whether for FDA-approved or off-label uses.
Mentioned last week was the original definition of off-label use as the use of a drug for clinical indications other than those stated in the labelling approved by the US Food and Drug Administration (FDA) and other regulators. An example would be the use of a drug for oncological treatment (e.g. pancreatic), where the drug would originally have been approved for treating other maladies of the same class (e.g. ovarian, bladder and genitourinary cancer).
To understand the off-label expansion, it is necessary to put physicians’ decisions in proper context. Cancer treatment is severe and life-threatening. Justifiably, physicians are asked to consider the best possible treatments at any given time. An extension of current therapies in related fields of use may, under circumstance, well justify administration of drugs which are not yet approved for the elected therapy. However life-threatening disorders need to be singled out from the discussion of off-label use, as these patients have acquired a special attention. Often, payors condone the off-label use in full understanding of the bill to be footed at the end of treatment.
The difficulty arises when drugs are being prescribed for a larger number of patients, therefore exposing patients more and more frequently to active pharmaceutical ingredients than originally tested. This use reopens the question of safety long-term (in the eyes of regulators), while the original marketing approval (MAA) had been granted on the basis of another data package altogether.
In contrast, AMCP believes each pharmaceutical agent on the market should be used only in accordance with generally accepted standards of medical practices. AMCP also recognises that pharmacotherapy is a rapidly changing field, and those FDA-approved indications for particular drug products do not always keep pace with changing clinical protocols and medical practices. Therefore, AMCP now suggests that managed care organisations and third party payors consider additional criteria before deciding whether to provide coverage of FDA-approved drugs for certain off-label uses. One thought is to include drugs which have been accepted In the current edition of American Hospital Formulary Service – Drug Information® (AHFS-DI®), Comprehensive Cancer Network (NCCN) Drug and Biologics Compendium™. Another consideration would be to focus on proven safety from formal clinical studies, which have already been published in peer-reviewed evidence-based medical literature. Here, it is necessary to add that randomised controlled trials are preferred over observational research or case studies.
Naturally, as the managing director of Clinuvel, I look for the relevance of these global changing regulatory and payors’ thought processes, and I continuously scan how this could affect our program and commercial plans. Caution is at place as I firmly hold the belief that new – meaning novel and first-in-class – drugs are subject to their own set of rules and regulations. To fully understand the implications of drug use, one should not lightly forget the mandate of these regulators: to act as guardian of the general population when reviewing food supplements, cosmetics and drugs.
Therefore, companies like Clinuvel ought to view the relationship with the regulators as long-term partners where shared responsibility becomes a modern issue when the drugs under review approach the market.
Next week, I shall take a closer look at Allergan’s actions against the FDA and potential impact on our industry.
See also AMCP’s Position Statements on Formularies; Prescription Drug Coverage; and Direct-to-Consumer Advertising.
- Philippe Wolgen
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