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Authors: Shriji Patel, Stephen J. Kim, Paul Sternberg
Journal: Ophthalmology (2023)

We appreciate the thoughtful critique of our editorial by Dr. Maloney and wish to take this opportunity to address the important distinction between "minimally proven" and "investigational" treatments. Dr. Maloney provides insight into the nuances essential for health care providers to grasp when navigating the complex landscape of medical interventions. The observation regarding the potential confusion between these terms is well taken. Importantly, the distinction between "minimally proven" and "investigational" treatments is not always clear cut, and there is often overlap between the two categories. Dr. Maloney points out that "minimally proven" treatments are interventions that, while lacking robust scientific support, are frequently used in routine practice with the primary intention of benefiting the individual patient's well-being. In contrast, "investigational" treatments refer to interventions employed with the purpose of testing hypotheses, contributing to generalizable knowledge, and typically undergoing evaluation through formal research processes, such as clinical trials. Dr. Maloney argues that unproven (or minimally proven) treatments are not investigational if their sole purpose is to benefit the patient. We believe such a statement oversimplifies the multifaceted nature of this intricate topic. The core of our argument revolves around situations where practitioners opt for minimally proven treatments owing to a dearth of effective alternatives. In these instances, we can benefit our patients while simultaneously contributing to the overall fund of knowledge. Such opportunities not only inform our personal experience with a treatment, but also allow contributions to generalizable knowledge. We emphasize that practitioners must be diligent in assessing each unique clinical context and providing thorough informed consent. Although some instances may not strictly fit the criteria of investigational research, they still carry the ethical responsibility of transparency and due consideration of patient welfare. Dr. Maloney wrote, "Practitioners need to understand the distinction between minimally proven and investigational treatment, so they neither deny a needy patient an unproven treatment with a reasonable expectation of success or gather generalizable knowledge as part of routine practice without institutional review board approval." We fully acknowledge the importance of clear terminology in this context. By using precise language, health care providers can better communicate with colleagues, patients, and regulatory bodies, ensuring that the ethical considerations and implications of their decisions are well-understood. Our editorial does not contest this distinction; rather, it underscores the ethical considerations when health care providers choose to employ interventions not fully substantiated by scientific evidence or personal experience, yet hold potential benefits for the patient. Re: Patel et al.: Considerations when offering minimally proven investigational treatments (Ophthalmology. 2023;130:351–353)OphthalmologyVol. 131Issue 2PreviewPatel et al1 in their otherwise excellent editorial, make one significant error: they confuse "minimally proven" with "investigational." This confusion is evident even in the title. It is important that health care providers understand the difference between minimally proven treatments and investigational treatments. Full-Text PDF