March 3, 2025
By Dwight Barnes, OD, FIAOMC, IACMM
Photo Credit: Dreamstime Photos
Myopia management has grown substantially in recent years, from a relatively uncommon treatment provided by a small number of optometrists to being recognized by the World Council of Optometry in 2021 as the standard of care. With the drastic increase in the rate of juvenile-onset myopia worldwide and the clear data now available about the ocular health risks associated with higher myopia, it is our responsibility as primary eye care providers to educate parents and children regarding the myopia treatments available.
Since myopia management emerged as an eye care subspecialty, treatments have mainly fallen into the “off-label” category. However, this has begun to change over the past few years. Let’s explore the history of FDA approval related to myopia management options and how to best address parental concerns regarding the FDA approval status of these crucial treatments.
What Does “Off-Label” Mean, and Does it Really Matter?
The term “off-label” refers to prescribing a medication or medical device to treat a condition other than the one for which the FDA approved it. Once a drug or medical device has been approved for marketing by the FDA, it can legally be prescribed for other purposes. However, it is illegal for manufacturers or practitioners to advertise these unapproved uses.1 Off-label prescribing does not necessarily indicate that the treatment is unsafe, inappropriate, or ineffective. Medical professionals can use their professional judgment in prescribing, and in many cases, off-label prescribing represents the best treatment option. Off-label prescribing is very common, representing as much as one-third of all medication prescriptions in the United States.2
What is the FDA Approval Status of Our Current Myopia Management Options?Orthokeratology, multifocal soft contact lenses, and low-dose atropine are the most frequently utilized treatments for myopia management in the United States. While they are FDA approved, with a few specific exceptions, they lack a specific labeling indication for myopia progression control. Numerous randomized controlled trials demonstrate the safety and effectiveness of orthokeratology, atropine, and various soft lens options that are not explicitly indicated for myopia progression control.
The first orthokeratology lens approved by the FDA was Paragon CRT (now owned by CooperVision), which gained approval in 2002 for the temporary reduction of myopia in patients with up to -6.00D of myopia and -1.75D of corneal astigmatism. All other correction is considered off-label.
Most multifocal soft lenses available today are designed and approved to correct vision for patients with presbyopia. However, some of these products have demonstrated effectiveness in slowing myopia progression in randomized controlled clinical trials. Ophthalmic atropine has been available for a long time and was initially used for mydriasis and cycloplegia, but it has also proven effective in managing myopia progression. These treatments are FDA approved for a specific use, yet we prescribe them for a different but very appropriate application that falls outside the FDA’s labeling indication.
Myopia Control vs. Myopia Management
CooperVision’s MiSight 1 day lenses are the first — and only as of the date of publication — product in the United States approved with a labeling indication for myopia progression control. MiSight 1 day soft contact lenses received FDA approval in November 2019 for correcting myopic ametropia and for slowing the progression of myopia in children who, at the start of treatment, are between 8 and 12 years old and have a refraction of -0.75D to -4.00D (spherical equivalent) with less than or equal to 0.75D of astigmatism. All other corrections are considered off-label.
In 2021, Johnson & Johnson’s Acuvue Abiliti Overnight Therapeutic Lens for Myopia Management became the first orthokeratology lens to have its name approved to include “myopia management.” It’s important to note that the FDA does not view myopia management as a specific labeling indication, making it very different from myopia progression control.
In the next few years, we will likely see the addition of other products with a specific FDA labeling indication for myopia progression control. At some point in the next couple of years, we will see the approval and introduction of FDA-approved spectacle options in the United States, and we may also see approved commercially available, low-dose concentrations of atropine.
The presence or absence of an FDA labeling indication for myopia progression control does not necessarily indicate better or worse outcomes for your patients. In many instances, extensive studies of off-label products demonstrate a high level of effectiveness in slowing myopia progression. However, some patients and parents may have concerns regarding off-label use and may feel more comfortable using a product that has received an FDA labeling indication. Often, parents of patients enrolled in a myopia management program may be unaware of the FDA indications for the various treatments discussed. Doctors should be prepared to answer questions related to FDA approval and address the concerns of parents.
What is the Duty to Disclose to Patients When a Myopia Management Treatment is Off-Label?
The answer to this question relates to medical ethics. The FDA oversees the marketing and safety of drugs and medical devices, but it does not regulate the practice of medicine. Physicians are legally allowed to use these products for purposes other than those for which they are approved, provided that the use is backed by strong scientific rationale and medical evidence. Courts have been reluctant to conclude that special informed consent is required for off-label use. However, one could certainly argue that a patient should be informed when a product or medication is prescribed off-label if there is any perceived increase in risk, especially when alternative FDA-approved treatments are available.
Given the substantial amount of clinical evidence related to the safety and efficacy of the current myopia management treatments, one could certainly argue that there is no ethical obligation to inform patients about the approval status. However, with the recent availability of a treatment option that has received an FDA labeling indication for myopia progressional control, some eye care professionals may choose to share this information with potential patients. For example, consider an orthokeratology product used for both vision correction and myopia management. The vision correction aspect of the treatment is clearly “on-label,” while its use for slowing myopia progression is typically regarded as “off-label.” In this situation, the choice may depend on how the doctor presents this treatment to their patients.
It is worth noting, however, that most eye care professionals prescribe off-label medication regularly. For example, Moxeza (moxifloxacin, Alcon) is FDA-approved for the treatment of bacterial conjunctivitis. Would any of us hesitate to use this medication for the treatment of microbial keratitis? Would we consider disclosing this clearly off-label use to the patient? What about using an FDA-approved orthokeratology product for a level of myopia slightly outside its approved Rx range?
Patient and Parent Communication
Ultimately, each practitioner must determine when off-label prescribing requires disclosure to the patient and how to communicate this effectively. If an eye care professional decides to inform their patients about the FDA labeling indication, this can be included in the informed consent paperwork that the patient signs before beginning treatment, or it may be conveyed verbally. Because the average patient is not familiar with the FDA approval process and the prevalence of off-label prescribing, it is crucial to feel confident discussing how off-label prescribing works and its rationale with the parents of children.
It may be useful to create a brief document explaining the FDA approval status of available myopia management treatments in simple terms and print copies to provide to patients to facilitate discussion. Given the importance of offering this valuable service to patients, we should utilize all available treatments to slow myopia progression. This list of options includes on-label and off-label therapies, and it is each doctor’s responsibility to evaluate which treatments offer the best safety and efficacy for each patient.
Even with the recent introduction of a product that has received FDA labeling specifically for myopia progression control, off-label prescribing is likely to remain a significant aspect of myopia management. When prescribing off-label treatments for myopia, most parents will probably understand and trust your clinical judgment about which treatment is most suitable for their child. If the parent prefers to use a product with an FDA-approved indication specifically for myopia progression control, you should make every effort to follow their wishes. Using peer-reviewed studies to demonstrate the effectiveness of common off-label treatments can be helpful in showcasing their value. Effectively and confidently communicating with parents about these treatment options helps parents of young children make informed decisions regarding their child’s care.
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Dr. Dwight Barnes attended Southern College of Optometry, where he graduated Magna Cum Laude, in 2002. After graduation, Dr. Barnes completed a residency in primary care optometry and ocular disease at the Department of Veterans Affairs in Boston. He is co-owner of Cary Family Eye Care in Cary, NC, with his wife Kelly. In addition to routine care, Dr. Barnes has a passion for myopia control and orthokeratology, and he established a Myopia Control Clinic within Cary Family Eye Care to focus on helping slow progression of myopia in young patients. He is a Fellow and Board Member of the International Academy of Orthokeratology and Myopia Control (FIAOMC) and also has the designation International Academy Certification in Myopia Management (IACMM). He has lectured and written articles on various aspects of practice management and myopia management, and he provides consulting services to doctors adding/expanding myopia management services. He is currently the co-host of “The Corrected View,” which is the AAOMC’s myopia management podcast. |
References
1 Norman G. Off-label use vs off-label marketing of drugs. JACC: Basic to Translational Science. 2023
2 Brauner JV, Johansen LM, Roesbjerg T, Pagsbert AK. Off-label prescription of psychopharmacological drugs in child and adolescentvpsychiatry. J Clin Psychopharmacol. 2016;36:500–507.
3 Syed SA, Dixson BA, Constantino E, Regan J. The law and practice of off-label prescribing and physician promotion. J Amer Acad Psych Law. 2021;49:53–59.
Some products covered in Review of Myopia Management are prescribed off-label in the United States because they are not FDA-approved for slowing the progression of myopia in children.
MiSight 1 Day soft contact lenses are FDA-approved for the correction of myopic ametropia and for slowing the progression of myopia in children with non-diseased eyes who, at the initiation of treatment, are 8-12 years of age and have a refraction of -0.75 to -4.00 diopters (spherical equivalent) with less than or equal to 0.75 diopters of astigmatism.
