Understanding on- and off-label prescribing
In this article:
On- and off-label prescribing means different things to eye care professionals in different countries. In the context of myopia management, these designations apply to medications like topical atropine, as well as medical devices such as spectacles or contact lenses. What does this mean in terms of clinical practice? Does this restrict prescribing and influence informed consent processes?
On-label and off-label prescribing of medications
In terms of topical atropine, off-label prescribing applies because the medication is commonly compounded, as there are very few commercially manufactured options for the low concentrations prescribed for myopia control. Compounding medications is necessary when a commercially manufactured drug is not available which meets the unique needs of an individual patient.1 This could be changing the delivery of the medication type (eg from a tablet to a liquid) or changing its concentration - in the case of atropine, from the commercially manufactured 1% down to the low 0.01% to 0.05% concentrations typically prescribed for myopia control.
On-label and off-label prescribing of medical devices
Most eye care professionals have heard of 'FDA approval' of medications or medical devices, which applies within the USA: indicating an approval given by the US Food and Drug Administration (FDA) that a drug or device has been verified as safe and effective for particular conditions in particular patient demographics. Prescribing this same approved drug for unapproved uses - for patients who don't fit these particular conditions or demographics - is considered off-label.
When it comes to myopia management, an on-label treatment has been tested and regulatory approved specifically for slowing myopia progression. Off-label treatments may very well be evidence-based, but these products have not passed the regulatory hurdle to confirm that they have met standards of safety, efficacy and quality in the target population, being children with myopia.
The International Myopia Institute Industry Guidelines and Ethical Considerations for Myopia Control Report4 states that "eye care professionals (ECPs) are encouraged to rigorously apply informed consent processes whenever prescribing or recommending off-label treatments for myopia control." Employing shared decision making and "disclosing the nature, risks, benefits, and alternatives for each myopia control treatment is vitally important."4
Is off-label prescribing safe and legal?
Off-label prescribing is not necessarily unsafe, nor is it illegal. In medicine, off-label prescribing to children is common due to a lack of pediatric-specific data regarding the dosing, efficacy and safety of medications regularly prescribed to children.5
Informed consent can take written and verbal forms, and involve a variety of resources including hard copy and digital forms. It involves a patient (and/or guardian, in the case of children) with decision-making capacity, who freely authorizes a treatment plan aimed at a mutually acknowledged treatment goal. Consent is 'informed' when the eye care professional has disclosed and the patient / guardian understands the diagnosis, relevant treatment options (including no treatment) and any respective risks and benefits. This process should be thoroughly documented.6
Meet the Authors:
About Kate Gifford
Dr Kate Gifford is a clinical optometrist, researcher, peer educator and professional leader from Brisbane, Australia, and a co-founder of Myopia Profile.
About Cassandra Haines
Cassandra Haines is a clinical optometrist, researcher and writer with a background in policy and advocacy from Adelaide, Australia. She has a keen interest in children's vision and myopia control.
This content is brought to you thanks to unrestricted educational grant from
- Donovan G, Parkin L, Brierley-Jones L, Wilkes S. Unlicensed medicines use: a UK guideline analysis using AGREE II. Int J Pharm Pract. 2018 Dec;26(6):515-525. doi: 10.1111/ijpp.12436. Epub 2018 Jan 30. (link)
- Gifford KL, Richdale K, Kang P, Aller TA, Lam CS, Liu YM, Michaud L, Mulder J, Orr JB, Rose KA, Saunders KJ, Seidel D, Tideman JWL, Sankaridurg P. IMI - Clinical Management Guidelines Report. Invest Ophthalmol Vis Sci. 2019 Feb 28;60(3):M184-M203. (link)
- Menicon Bloom White Paper, 'Why CE Approval Matters', May 2019 (link)
- Jones L, Drobe B, Gonzalez-Meijome JM, Gray L, Kratzer T, Newman S, et al. IMI - Industry guidelines and ethical considerations for myopia control report. Invest Ophthalmol Vis Sci. 2019;60(3):M161-M83. (link)
- Allen HC, Garbe MC, Lees J, Aziz N, Chaaban H, Miller JL, Johnson P, DeLeon S. Off-Label Medication use in Children, More Common than We Think: A Systematic Review of the Literature. J Okla State Med Assoc. 2018 Oct;111(8):776-783. (link)
- Hall DE, Prochazka AV, Fink AS. Informed consent for clinical treatment. CMAJ. 2012;184(5):533-540. (link)
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