PQA Releases Report to Define Quality Medication Use for Rare Diseases

The Pharmacy Quality Alliance (PQA) released a report defining quality medications for use in rare diseases, compiled from stakeholders. As part of the PQA Convenes: Quality Medication Use in Rare Disease event held on November 7, 2024, in Arlington, Virginia, the report highlights the perspectives of 4 key stakeholders in quality medication use: patients, caregivers, and patient advocates; specialty pharmacies and providers; biopharmaceutical industry and organizations; and public and private payers.¹

Pharmacy Quality Alliance (PQA) releases report on quality medications for rare diseases, compiled by stakeholder perspectives. | Image Credit: auremar | stock.adobe.com

Rare diseases are considered conditions that affect fewer than 200,000 patients in the United States. There are over 10,000 rare diseases, which are estimated to affect more than 30 million Americans, according to the Genetic and Rare Diseases Information Center.² PQA states that approximately 5% of rare diseases have medication approved by the FDA, but the development and approval of medications for these diseases has been growing, with more than half of the FDA novel drug approvals in 2022 being for rare diseases.^1,2

PQA states that quality medication use includes the care process and outcomes, which can include accessibility, affordability, appropriate use, safe use, effective use, adherence, persistence, and support. The report synthesized perspectives from approximately 20 speakers and moderators included in the event. Across all 4 panels, the topics of access, education, support, and data came up in every group.¹

The specialty pharmacy panel included 5 experts: Christopher R. Antypas, PharmD, president of Perigon Pharmacy 360; William Trombatt, PharmD, CSP, manager of clinical programs at Walgreens Specialty Pharmacy; Dustin Donald, PharmD, CSP, clinical pharmacy team lead for rare diseases at Vanderbilt Specialty Pharmacy, part of Vanderbilt University Medical Center; Michael Duteau, RPh, president of Nobel Health Services; and Chris Kotschevar, PharmD, CPHQ, director of stakeholder engagement at PQA, who served as the moderator.¹

Pharmacists provide many services for patients with rare diseases, and there are many pharmacists that have staff assigned to specific patients to optimize the coordination of care across providers and therapies, according to PQA. Additionally, pharmacists can be embedded into clinics, when possible, in order to assist patients in their therapy, especially being involved in conversations about adverse events and reminders about refills and adherence. To improve quality, the PQA panelists state that pharmacy access to broader patient health records and expanded access to medications can help pharmacists optimize the best care for patients.¹

Another big concern for the panelists is access to medication and care, especially cost and the burden of rare diseases. They state that pharmacists should acknowledge and look for barriers that patients face, and relationships should be built between pharmacists and other providers and the patient. They note that whole-person care is the most important aspect of high-quality care.¹

The panelists also identified expanding pharmacy services as a way to provide quality medication. They noted that payment models can be a barrier to services, but collaborating with biopharmaceutical industry partners and payers can help patients access expanded pharmacy services. Further, they noted that pharmacy information should be integrated into electronic health records as this could help close an information gap between care teams.¹

The panelists also offered opportunities for improvement, which included a decrease in time to treatment so that patients can access their therapy faster. They said that this can be achieved through partnerships between pharmacies and stakeholders. Barriers to distribution, expanded access to new medication, and accurate prescriptions can improve patient wait times and assist with medication therapy. Lastly, the panelists stated that collecting and sharing data on the pharmacy’s impact on turnaround times, hospitalization rates, adherence, and clinical outcomes are needed to determine the quality of care.¹

REFERENCES

1. PQA. PQA Convenes: quality medication use in rare disease. January 2025. Accessed January 23, 2025. https://www.pqaalliance.org/assets/docs/PQA_Convenes_Rare_Disease_Report.pdf

2. Genetic and Rare Diseases Information Center. Genetic and rare diseases information center. Accessed January 23, 2025. https://rarediseases.info.nih.gov/