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Clinical and pharmacoeconomic impact of subgroup analysis in onco-hematological patients

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Abstract

Subgroup analysis evaluates a health intervention in subpopulations according to a characteristic or factor. It can be useful for generating new hypotheses or conducting new studies. However, subgroup analysis presents several limitations and it should be considered cautiously. The development of new onco-hematological drugs is accelerating in recent years and the impact of subgroup analysis on clinical decision-making is increasing. The interpretation of subgroup analyses can be controversial in some cases, negatively affecting patients and healthcare systems. This work is a review of the clinical and pharmacoeconomic impact of subgroup analysis in onco-hematological patients. The study describes some illustrative examples of inadequate interpretations about subset analysis: combination of pembrolizumab plus chemotherapy in lung cancer, inhibitors of cyclin-dependent kinases in breast cancer, daratumumab-based regimens in newly diagnosed multiple myeloma, combination of nivolumab with ipilimumab in melanoma and docetaxel in prostate cancer. Subgroup analysis can have a significant impact on the data selection for the development of studies; efficacy, safety, and convenience of treatments in onco-hematological patients; efficiency of therapies in health systems; and therapeutic positioning of antineoplastic drugs. There is a strong need to establish homogeneous criteria for the assessment of subgroup analysis and to develop new tools for its consideration.

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Acknowledgements

Emilio Jesús Alegre-del Rey has led the professional training of hospital pharmacists in evidence-based medicine for years. The development of this work would not have been possible without his active teaching and commitment to the healthcare system.

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Authors and Affiliations

  1. Pharmacy Department, Hospital Universitario Puerto Real, Puerto Real, Ctra. N-IV Km. 665, 11510, Cadiz, Spain

    Manuel David Gil-Sierra

  2. Department of Pharmacology, Facultad de Farmacia, Universidad de Sevilla, Calle Profesor García González 2, 41012, Seville, Spain

    Manuel David Gil-Sierra & Marina Sánchez-Hidalgo

  3. Pharmacy Department, Hospital Universitario Jerez de la Frontera, Carretera Trebujena, s/n, 11407, Jerez de la Frontera, Spain

    Maria del Pilar Briceño-Casado

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  1. Manuel David Gil-Sierra
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Contributions

Gil-Sierra Manuel David: conceptualization; data curation; formal analysis; investigation; methodology; project administration; resources; supervision; validation; visualization; roles/writing—original draft; writing—review and editing. Briceño-Casado Maria del Pilar: conceptualization; data curation; formal analysis; investigation; methodology; project administration; resources; supervision; validation; visualization; roles/writing—original draft; writing—review and editing. Sánchez-Hidalgo Marina: conceptualization; data curation; formal analysis; investigation; methodology; project administration; resources; supervision; validation; visualization; roles/writing—original draft; writing—review and editing. All authors have made substantial contributions to all of the following: Substantial contributions to the conception or design of the work; or the acquisition, analysis, or interpretation of data for the work; and drafting the work or revising it critically for important intellectual content; and final approval of the version to be published; and agreement to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

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Correspondence to Manuel David Gil-Sierra.

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Gil-Sierra Manuel David: membership of an advisory board (consultation fees), lecture for Janssen Pharmaceutica and Pfizer (reimbursement for attending symposia) of cancer drugs. The rest of the authors have no conflict of interest to declare.

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Gil-Sierra, M.D., del Pilar Briceño-Casado, M. & Sánchez-Hidalgo, M. Clinical and pharmacoeconomic impact of subgroup analysis in onco-hematological patients. Support Care Cancer 30, 3761–3772 (2022). https://doi.org/10.1007/s00520-022-06823-y

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