Rare Disease Day and a Focus on Quality

Today is Rare Disease Day. It’s an opportunity to raise awareness of the impact of rare diseases.

There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments. In the United States, rare disease affects about one in 10 people. The burden and cost of treating rare disease is high for patients, our care system and society.  

The economic burden of rare disease reached nearly $1 trillion in 2019, and the average drug approved under the Orphan Drug Act costs nearly $120,000 per year.

Ensuring the quality of medication use is critically important for rare disease patients, payers, clinicians, specialty pharmacies and everyone with a role in the care process. Like all patients, individuals with rare disease want medications that are accessible, safe, effective and help them achieve the best outcomes possible.

PQA is the national quality organization dedicated to improving medication safety, adherence, appropriate use and medication management services. High-quality medication use is an essential part of preventing disease, managing chronic conditions and supporting overall long-term health, well-being and positive patient outcomes.

Medication use quality improvement – which depends significantly on standardization – is incredibly challenging for rare disease due to the number of conditions and the great variation in disease presentation, progression and treatment.

Despite these obstacles, PQA is eager to provide a forum to discuss quality issues in rare disease medication use and an opportunity to elevate the needs of rare disease patients and those who care for them.

We are in the early stages of planning an event, PQA Convenes: Quality Medication Use in Rare Disease, which will bring our members and the rare disease community together to explore quality in medication use. This one-day event will take place on Thursday, November 7, 2024, in Arlington, Va., prior to the PQA Leadership Summit.

There is a broad range of largely internal quality indicators used by health care organizations to evaluate rare disease care today. They represent the many opportunities to improve quality, but there is enormous variation in rare disease and its treatment – and many perspectives on what constitutes quality and how to improve it. As a result, establishing a consensus on what constitutes high-quality rare disease medication use is paramount.

This PQA Convenes event is an opportunity to:

  • highlight the importance of quality in rare disease medication use,
  • explore areas for consensus among patients and the diverse payers, providers, clinicians and others who care for them, and
  • identify opportunities for research or other projects that could advance quality improvement of rare disease medication use.

Through expert presentations and interactive panel discussions, PQA will provide a platform for the perspectives of:

  • Patients, caregivers and patient advocates
  • Specialty pharmacy
  • Pharmaceutical industry organizations
  • Public and private payers

To create an informed and responsive event, PQA will convene a diverse, multi-stakeholder planning committee to guide the focus of our sessions. PQA will broadly share the results of the event to further elevate the importance of medication use quality for individuals with rare disease and those who care for them.

We look forward to sharing more about this event in the coming months. If you have questions or would like to be involved, email us at [email protected].


About PQA and PQA Convenes 

PQA is a national quality organization dedicated to improving medication safety, adherence and appropriate use. A measure developer, researcher, educator and convener, PQA’s quality initiatives support better medication use and high-quality care. PQA was established in 2006 as a public-private partnership with the Centers for Medicare & Medicaid Services. PQA was created because prescription drug programs were a major area of health care where there was no organization or national program focused on quality improvement. Today, PQA is an independent, non-profit organization with nearly 240 diverse members across health care.

PQA Convenes brings together national leaders in medication use quality to build consensus and develop plans of action to promote innovative and timely opportunities for improving patient care and outcomes. A gathering of diverse thought leaders and decision makers, PQA Convenes is designed to:

  • Explore how medication use quality and pharmacist-provided care can improve care delivery, patient and provider experiences, and patient outcomes.
  • Clarify unmet market needs, gaps in care, or interventions that can be realized through research, education, and collaboration.
  • Stimulate calls to action, which can include (a) white papers or consensus statements; (b) follow-up or expanded convenings; and (c) communications and engagement strategies to build broader awareness.
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