Decoding gene therapy: Current impact and future considerations for health-system and specialty pharmacy practice.

PURPOSE: To provide health systems with baseline knowledge on existing and pipeline gene therapy treatments, including considerations that health-system pharmacies and specialty pharmacy programs may reference when evaluating and implementing services around gene therapies. SUMMARY: Advancements in research and biotechnology have recently led to the development and launch of the first commercially available gene therapy treatments in the United States. These treatments have the ability to significantly alter and even effectively cure diseases. Alongside these significant advances and clinical benefits, these therapies present unique challenges due to their cost and complexity. Given the large number of additional gene therapy treatments that are currently in late-stage clinical development, stakeholders across the healthcare industry must increasingly adapt and ready themselves to meet these challenges. The diagnosis and treatment of patients with diseases being targeted by gene therapies largely occurs within health systems, and judging by the gene therapy pipeline, this trend is likely to continue. To prepare for these novel treatments, health systems must understand and consider the methods in which gene therapies are developed, procured, reimbursed, administered, and monitored. CONCLUSION: The future of health-system pharmacy practice must include comprehensive gene therapy services and stakeholder engagement strategies to ensure patients have access to these life-changing treatments.

Navigating the Wild West of Medication Adherence Reporting in Specialty Pharmacy.

Estimating medication adherence through the use of pharmacy claims-based adherence calculations such as medication possession ratio (MPR) and proportion of days covered (PDC) plays a significant role in specialty pharmacy practice. Although MPR and PDC are frequently used in clinical practice, calculation methodologies vary, making meaningful comparisons of adherence rates difficult. In addition, MPR and PDC are increasingly used by insurance companies, pharmacies, accrediting bodies, and drug manufacturers to demonstrate quality differences or clinical benefit across the specialty pharmacy industry. Therefore, recognizing the source and effect of calculation variability is necessary to fully understand reported adherence results. This article highlights the challenges in standardizing adherence methodologies, minimum methodology considerations that should be reported with MPR and PDC results, and key elements to consider when interpreting and applying adherence results. Further, recommendations are provided to promote a more consistent description of calculation methods and to aid pharmacies in adherence measure analysis, interpretation, and application to practice, with a focus on specialty pharmacy programs. A detailed description of methodology as outlined in this article must be provided to ensure reproducibility, external validation, and scientific rigor. In the absence of standardization, specialty pharmacies should be prudent in their use of adherence calculations as a clinical benchmarking tool or comparative quality indicator with outside organizations. Furthermore, specialty pharmacies should consider using current adherence measure calculations to identify and provide targeted interventions to patients with potential adherence problems and strive to better demonstrate ties between adherence measures and direct clinical and cost outcomes. DISCLOSURES: No outside funding supported the writing of this article. Anguiano is a speaker and research consultant for United Therapeutics. The other authors have nothing to disclose.