ABSTRACT
BACKGROUND: Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare disorders of heme biosynthesis characterized by severe cutaneous phototoxicity. Afamelanotide, an α-melanocyte-stimulating hormone analogue, is the only approved treatment for protoporphyria and leads to increased light tolerance and improved quality of life (QoL). However, published experience with afamelanotide in the US is limited. METHODS: Here, we report on all adults who received at least one dose of afamelanotide at the Massachusetts General Hospital Porphyria Center from 2021 to 2022. Changes in the time to phototoxic symptom onset, QoL, and laboratory parameters were assessed before and during treatment with afamelanotide. RESULTS: A total of 29 patients with protoporphyria were included, 26 of whom (72.2%) received ≥2 afamelanotide implants. Among the patients who received ≥2 implants, the median time to symptom onset following sunlight exposure was 12.5 min (IQR, 5-20) prior to the initiation of afamelanotide and 120 min (IQR, 60-240) after treatment (p < 0.001). Improvements in QoL during afamelanotide treatment were measured using two QoL tools, with good correlation observed between these two instruments. Finally, we found no improvements in the median levels of metal-free erythrocyte protoporphyrin, plasma protoporphyrin, or liver biochemistries during versus prior to the initiation of afamelanotide treatment. CONCLUSIONS: This study highlights a dramatic clinical benefit of afamelanotide in relation to light tolerance and QoL in protoporphyria, albeit without improvement in protoporphyrin levels or measures of liver function.
ABSTRACT
PURPOSE: Healthcare systems are challenged with implementing high-cost, high-impact medication therapies with increasing frequency. Pharmacy & therapeutics (P&T) committees may struggle to integrate requests for these medications into their existing formulary review process. This article describes one large academic medical center's experience with creating a new P&T subcommittee that addresses the clinical, operational, and financial challenges these medications present. SUMMARY: The High-Impact Medication Therapy Subcommittee was established to optimize the institution's process for reviewing formulary requests for medications that are defined as "high impact"-nononcology medications that are extremely expensive and/or have complex operational or clinical challenges. The multidisciplinary subcommittee has 3 chairs-a physician, a nurse, and a pharmacist-and includes representation from all areas of pharmacy practice (clinical pharmacy, operations, supply chain, finance, and informatics), as well as medical and nursing leadership, hospital finance, and patient access services. Additional relevant stakeholders are invited as needed. The first medication to be reviewed at this subcommittee was afamelanotide, a melanocortin receptor agonist indicated for treatment of erythropoietic protoporphyria. The subcommittee addressed cost-efficacy concerns and operational challenges, and the final recommendation was for formulary addition, with clearly defined restriction criteria and the creation of a new workflow to meet the unique operational considerations with this drug. CONCLUSION: As medication costs continue to rise at unprecedented rates and reimbursement requirements continue to increase in complexity, the High-Impact Medication Therapy Subcommittee provides a necessary venue for reviewing high-cost medications with complex clinical or operational considerations and proactively addressing implementation challenges.
