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Off-label prescription: developing a guideline and validating an instrument to measure physicians’ and clinical pharmacists’ knowledge and attitudes toward off-label medication use

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Abstract

Purpose

Off-label medications have been challenging in healthcare systems, and their significance is emphasized throughout pandemics. The study was designed to develop an ethics guideline for prescribing off-label medications and also aimed to develop a valid instrument for evaluating physicians’ and clinical pharmacists’ knowledge and attitudes regarding off-label medication use.

Methods

In 2021, the two-phase study was done. A gap analysis study and a review of relevant guidelines and peer-reviewed papers were conducted in the first phase. There were three hybrid expert panel discussions with nine specialists until a consensus was reached. In the second phase, a questionnaire was developed to assess physicians' and pharmacists' knowledge and attitudes toward off-label prescribing medications. An expert panel of nine ethicists, physicians, and pharmacists determined the face and content validity. To evaluate the reliability and construct validity of the instrument, 201 physicians and clinical pharmacists participated.

Results

This guideline provides 24 recommendations classified into seven themes to assist clinicians, pharmacists, and policymakers in managing off-label medication use. The preliminary questionnaire contained 72 items. Items were removed if their I-CVI and CVR were less than 0.79 and 0.78, respectively. The S-CVI/Average ratio was 0.937. The Cronbach α was 0.848. Ten factors were identified through exploratory factor analysis. These ten factors comprised 64.652% of the variance. There was no significant difference between general physicians, specialist physicians, and clinical pharmacists in one-way ANOVA [F = 0.584, P = 0.559].

Conclusion

We developed an ethical guideline for off-label medication use that can integrate ethical principles into related practice. Moreover, our valid and reliable questionnaire can evaluate the ethical adherence of physicians and pharmacists to scientific and ethical challenges of off-label medications in the country, especially during pandemics.

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Data availability

The data supporting this study's findings are available from the corresponding author, [Mahshad Noroozi], upon reasonable request.

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Funding

This research received no specific grant from any funding agency in the public, commercial, or not-for-profit sectors.

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Author notes

  1. Bagher Larijani

    Present address: Medical Ethics and History of Medicine Research Center, Tehran University of Medical Sciences, Tehran, Iran

Authors and Affiliations

  1. Medical Ethics and History of Medicine Research Center, Tehran University of Medical Sciences, Tehran, Iran

    Pooneh Salari

  2. Endocrinology and Metabolism Research Center, Endocrinology and Metabolism Clinical Sciences Institute, Tehran University of Medical Sciences, Tehran, Iran

    Bagher Larijani & Farzaneh Zahedi

  3. Department of Medical Ethics, School of Medicine, Iran University of Medical Sciences, Tehran, Iran

    Mahshad Noroozi

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Contributions

Conception and design: PS and BL. Data collection: PS, MN and FZ. Analysis and interpretation of data: MN and PS. Drafting of the manuscript: PS and MN. Critical comments and editorial review: BL and FZ.

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Correspondence to Mahshad Noroozi.

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The study was approved by the Ethics committee of Tehran University of Medical Sciences, Tehran, Iran (IR.TUMS.MEDICINE.REC.1399.437).

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Salari, P., Larijani, B., Zahedi, F. et al. Off-label prescription: developing a guideline and validating an instrument to measure physicians’ and clinical pharmacists’ knowledge and attitudes toward off-label medication use. J Diabetes Metab Disord 22, 1599–1608 (2023). https://doi.org/10.1007/s40200-023-01288-0

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