CONTROVERSIES ABOUT CONTACT LENSES are ongoing, and the leadership of the American Optometric Association Contact Lens and Cornea Section is once again excited to provide a new series on this topic in our annual article for Contact Lens Spectrum. These controversies include: 1) On-label versus off-label treatment for myopia management; 2) Are we overprescribing scleral lenses?; and 3) Should all contact lens patients be fit into a daily disposable contact lens?
CONTROVERSY 1
ON-LABEL VERSUS OFF-LABEL TREATMENT FOR MYOPIA MANAGEMENT
Myopia prevalence is increasing worldwide. In the absence of intervention, the global prevalence is projected to reach 50% by the year 2050.1 A concomitant increase in the prevalence of high myopia will likely lead to a rise in consequent conditions that threaten sight. This has stimulated interest in methods that slow myopia progression.
Validated therapies available in the United States include low-dose atropine, daytime soft multifocal and dual focus contact lens wear, and overnight orthokeratology. Currently, some of the aforementioned therapies are approved by the U.S. Food and Drug Administration (FDA) while others are prescribed as off-label protocols for myopia management. With this background in mind, arguments that favor either FDA-approved or off-label treatments can be made. For the didactic purposes of better understanding the benefits of both FDA-approved and off-label treatments, and for making evidence-based clinical decisions, below are arguments for using one treatment over the other.
FDA approval is important, and on-label therapies should be highly preferred over off-label. The FDA does not regulate the practice of medicine, and off-label use of therapies is common. There may not be an approved therapy available or dosage may differ from that on the label. Until recently, approved therapies for myopia were lacking. The FDA employs a rigorous evidence-based process to approve therapies that are both safe and efficacious. In the practice of medicine, favoring FDA-approved therapies maximizes benefit while minimizing risks, and such therapies often become the standard of care. Due to a general lack of studies, off-label use in pediatric populations is especially common. It is therefore fortunate that approved myopia therapies are available for use in children.
Safety is a major consideration when prescribing for children. Children may not react the same as adults and they have a long time to live with unwanted outcomes. Thus, even if there are robust data and approved therapies for adults, one may not necessarily predict with confidence the response of children. When approved therapies exist for children, they may be favored over off-label treatments. Not only is safety and efficacy evaluated up front by the FDA, but ongoing post market surveillance is also integrated into the process to uncover any rare and/or serious deleterious outcomes. This alone justifies using FDA-approved therapies over off-label uses.
When prescribing off-label treatments, the practitioner assumes a great burden of responsibility. Motivated and experienced clinicians who are familiar with the known risks and benefits of an off-label therapy are best positioned to employ such treatments effectively and safely. This requires critical evaluation of all the available data as well as an understanding of the known mechanisms of action. This knowledge, combined with anecdotal clinical experience, enables clinicians to consider the possible outcomes and weigh the risks and rewards. Off-label use is, therefore, best left to those who are specialists in a particular field.
FDA-approved treatments are also more accessible to patients. Additionally, approved pharmaceuticals and optical devices are often covered by insurance, which increases access to care. For myopia management, atropine drops may soon be available at all pharmacies, not just specialized compounding facilities. In addition, the recent approval of soft contact lenses for myopia control, and the assignment of a medical code for such usage, portends eventual insurance coverage.
Off-label therapies are appropriate and should not be devalued compared to on-label options.Although FDA approval is valued among both practitioners and patients when choosing a myopia management option for a child, some of our most effective options are considered off-label. Currently, the only FDA-approved options for myopia management are contact lenses. If practitioners confine patients to an FDA-approved option, they are severely limiting the number of patients who could potentially benefit from myopia management.
Off-label use of ophthalmic products in myopia management has broadened the scope of practice and allows for advancements in both practitioners’ knowledge and treatment options for patients. Thus, there are several arguments in favor of also incorporating off-label options into the arsenal of tools to combat progressive myopia.
First, most off-label options have been used far longer than the current FDA-approved option, and several robust clinical studies back their efficacy. The Bifocal Lenses In Nearsighted Kids (BLINK) study has validated that high-add center-distance multifocal contact lenses are effective in reducing myopic progression and slowing axial elongation in children.2 The Low Concentration Atropine for Myopia Progression (LAMP) study has shown that 0.05% is the most effective dosage of atropine and has minimal side effects and rebound effect.3 Finally, a number of studies demonstrate the effectiveness of orthokeratology in slowing myopia progression, on average, approximately 50%.4
The second argument is that off-label contact lens options include widened prescription parameters for both myopia and astigmatism, which significantly increases the number of patients who could benefit from myopia management. The currently approved options are limited to –6.00DS and –1.50DC. Thus, parents of patients who have highly myopic or astigmatic prescriptions must seek off-label alternatives to correct their children’s vision and manage their myopia progression.
Third, the cost of a product is often impacted by FDA approval, whereas a “generic” or off-label option typically costs less. Having a more cost-effective option will allow even the most budget-conscious family to have access to an affordable treatment. This is especially beneficial to families who have more than one child in need of myopia control.
Lastly, despite certain lenses and low-dose atropine being used in an off-label manner, the base products themselves are FDA-approved for ophthalmic use. Atropine 1% has been used for cycloplegia in young children, as well as in patients struggling with ocular pain and inflammation, for decades. Overnight orthokeratology designs have been approved for temporarily minimizing refractive error for 20 years. Similarly, soft center-distance multifocal lenses are FDA-approved modalities for vision correction. Thus, the safety of all of these modalities has been well established.
