PQA members and health quality professionals gathered in Arlington, Va., on November 7 for PQA Convenes: Quality Medication Use in Rare Disease. This half-day event brought together health care quality experts, rare disease leaders and individuals with lived experience to discuss what constitutes high-quality rare disease medication use.  

PQA Chief Engagement Officer Richard Schmitz opened the event, addressing why PQA is focused on rare disease.

“The availability of medications to treat rare disease continues to grow, and drugs with orphan designations are now half of all drugs approved by the FDA. As an organization focused on the quality of medication use, PQA is in a unique position to help advance the conversation about what quality means for rare disease patients and those who care for them.”

- Richard Schmitz, Chief Engagement Officer

Attendees heard from patients, caregivers and patient advocates, specialty pharmacies, pharmaceutical industry organizations, and public and private payers.

Here are some of the top insights from these sessions.

• Care coordination among providers is critical for rare disease patients, as the burden often falls on the patient to find providers that address individual needs. Finding providers who will partner with and champion the patient is critical to quality outcomes.
• Health literacy is a barrier for patients. Education about the disease and the confidence to ask questions are essential, but the sense of community in rare disease is key. Having a rare disease can be isolating.
• The care process -- from the diagnostic odyssey to treatment -- for patients with rare disease is difficult. Patients need assistance to manage the process, insurance, and providers.
• Focus first on access to specialty pharmacies and then on ongoing engagement. High-quality care is individualizing the care of the patient. Specialty pharmacy is a high-touch model, but even it needs to go above and beyond for those with rare disease.
• Data insights are a challenge, but data collection is very important. Patient registries ensure long-term data and provide the ability to ask questions or follow up on outcomes. Collaborating with patient registries can help data insights. It's vital to break down data silos.
• It is important for payers to work holistically with patients, especially as it relates to health equity.

Attendees concluded the event by looking at a recently published survey report, Inequities in the Rare Disease Community, from the Rare Disease Diversity Coalition and National Organization for Rare Disease. The report includes insights on the factors are most likely to cause patients to delay or forego care.

PQA will publish a report from this convening event in the coming months to continue the conversation around quality medication use in rare disease.

Thank you to our sponsors!

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