Only about five percent of rare diseases have treatments that are approved by the FDA, but the situation is improving. Today, drugs designed to treat rare diseases make up half of new drug approvals each year.
This is important progress, as there are more than 7,000 rare diseases. Together, they affect more than 30 million Americans.
With the growth of rare disease medications, there is a need and opportunity to consider what constitutes high-quality medication use.
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.
To help address this, PQA convened last November nearly two dozen individuals with lived experience and professional expertise. They shared insights on what constitutes quality in rare disease medication use and how to improve it from the perspectives of:
- patients, caregivers and patient advocates
- specialty pharmacies and providers
- biopharmaceutical industry organizations, and
- public and private payers
Today, PQA issued a report from that event, PQA Convenes: Quality Medication Use in Rare Disease.
Here is a key quality insight from each panel:
- Care coordination is critical for organizing, managing and supporting a patient’s complex needs across multiple care providers and specialties.
- Access to patient health records enables specialty pharmacies to help patients navigate medication approval processes, coordinate care and provide needed services.
- Ongoing collection of rich data that captures the full patient experience is critical to establishing the quality and value of rare disease medications.
- Care management is the backbone of quality.
These factors and the report’s other insights may represent an opportunity for cross-sector collaboration to improve the quality of rare disease medication use. We hope the report is a useful addition to the conversation on rare disease and quality, and PQA looks forward to engaging the industry as it advances work in this area of care.
Thank you to the more than 100 individuals who attended this PQA Convenes event, all of our speakers and moderators, and the organizations whose generous support made the event and report possible: Pfizer, Novo Nordisk and Jazz Pharmaceuticals.