The Clinical Pharmacist's Role in Curative Gene Therapies: A Patient Journey Framework for Access, Safe Use, and Long-Term Monitoring - A Narrative Review

Abstract Background: The approval of curative gene therapies for haemophilia A and B, sickle cell disease, and spinal muscular atrophy has introduced a category of clinical intervention with no precedent in conventional pharmaceutical care. These therapies demand a level of pharmacist expertise spanning genomics, complex logistics, financial navigation, and decades-long safety surveillance that the current pharmacy workforce is not consistently prepared to provide.

Objective

To develop a comprehensive, patient journey framework mapping clinical pharmacist responsibilities across all phases of curative gene therapy delivery, and to define the core competency domains required for effective pharmacist integration in this setting.

Methods

A narrative review was conducted searching MEDLINE/PubMed, Scopus, Web of Science, Embase, and Google Scholar from January 2015 through December 2025, supplemented by regulatory documents, REMS communications, and professional society repositories. Evidence from clinical trials, cohort studies, regulatory guidance, and consensus statements was synthesized narratively across five sequential phases: candidate identification, access navigation, preparation, administration, and long-term monitoring.