It’s long been a tradition in medical practice to use FDA approved therapies in new, “off-label” ways. Is this safe? Botox, a treatment so prevalent as to have become synonymous with cosmetic medicine, is approved to treat four problems: muscular eye disorders, muscular neck disorders, excessive sweating (hyperhydrosis), and frown lines or wrinkles between the eyebrows. Yet, it is common practice for clinicians to use Botox in a myriad of other ways. Botox, a butulinum toxin isolated from bacteria, works by locally paralyzing the region where it is injected. A new version of this toxin, Dysport, has recently been approved as well. With botulinum toxins, doctors have had success treating anal fissures, pelvic muscle spasms, chewing problems, migraine headaches, and in the world of anti-aging therapies, ridding the aging face of crow’s feet and wrinkles around the mouth.
Is it reasonable to adopt novel uses of these drugs before rigorous study under governmental supervision establishes the appropriateness of such therapy? Doctors are entitled to employ FDA approved therapies in unapproved ways based upon their experience with their use. For patients and clinicians alike, it seems difficult to discern whether off-label use of these treatments is a reasonable therapeutic leap based upon the safety and practice guidelines already established or if it’s just experimentation on the unsuspecting public.
I recommend that anyone considering off-label treatments of an approved medical device or medication consider the following:
1) Ascertain why the treatment has not been approved for the proposed use. Silicone gel breast implants were reapproved by the FDA in October, 2006. However, they are not approved for women under 22 years of age. This is clearly not due to a lack of device testing or understanding of gel implant safety. A gel implant would not be less safe in a 21 year old as compared to a 22 year old. The imposed age restriction on approved usage of these devices implies that the FDA is suggesting an age-related degree of maturity necessary to make this particular decision. If I meet a mature, self-aware, 21 year old who has well thought out her desire to have silicone gel implants, this may be a reasonable off-label use of these devices.
2) Determine if the clinician has an extensive history of using the treatment. In the example of Botox, a clinician who has injected many people for brow furrows would have a particular aptitude for safely determining Botox usage in other areas of the face.
3) Ask if there’s a substantive, theoretical basis behind the proposed therapy: it’s efficacy and safety. We know that Botox paralyzes muscles locally. There is voluminous evidence that Botox rarely effects areas distant from the treatment site unless used in extremely large doses. Because of it’s mechanism of action, the present data on the local therapeutic affect, and the miniscule side effect profile, it is theoretically reasonable to treat local disorders related to muscle tightening or spasm at appropriate doses.
4) If the therapy is a medication, ask if it’s off-label use proposed is in similar doses to it’s approved use.
The debate over off-label device and drug usage will not be solved any time soon. The FDA restricts companies who make medical treatments from promoting their non-approved applications. Allergan, the manufacturer of Botox, recently filed a law suit against the federal government claiming that this restriction violates their First Ammendment rights to speek freely with doctors regarding the promotion of their drug. We’ll see how this plays out. Meanwhile, clinicians can and will continue to use Botox in non-approved ways: ways that are in great demand and improving the quality of life for many people. While this should not trivialize the FDA’s role in safely regulating medical treatments, patients can continue to benefit from off-label drug applications as long as they are involved in the process of determining the appropriateness of such usage.