Rare disease day: A closer look at rare diseases
When we hear the word “rare,” we might think of something uncommon or unlikely to happen. But in the world of healthcare, a rare disease is a serious medical condition that affects a small percentage of the population.
A rare disease is “a disease or condition that affects less than 200,000 people in the United States,” according to the Food and Drug Administration. Over 30 million Americans are affected by rare diseases, and 95% of these diseases have no approved treatment.
There are over 7,000 known rare diseases, many of which are genetic and and three-fourths of them affect children. Some well-known rare diseases include Cystic Fibrosis, Huntington’s disease, and Duchenne Muscular Dystrophy.
Because these diseases are uncommon, they often receive less research funding, fewer treatment options, and limited awareness, making diagnosis and care more challenging for patients and their families.
Rare Disease Day
Rare Disease Day is observed annually on the last day of February—typically February 28, but on leap years, it falls on February 29, the rarest day of the calendar. The day was first established in 2008 by the organization EURORDIS (Rare Diseases Europe) and has since grown into a global movement.
The purpose of Rare Disease Day is to:
- Push for Policy Changes – Influence governments and policymakers to improve healthcare access and resources.
- Raise Awareness – Shine a light on the challenges that patients, caregivers, and healthcare professionals face.
- Advocate for Research – Encourage funding and scientific advancements to develop new treatments.
- Support Patients & Families – Give a voice to those living with rare conditions and build a stronger support network.
Focus on the patient
At Maxor, we recognize the importance of individualized care—something essential for rare disease patients.
Maxor Specialty Pharmacy has over 35 years of experience with limited distribution drugs (LDDs), which treat rare and orphan diseases. Our experts specialize in rare diseases including cystic fibrosis and non-tuberculous mycobacteria.
Maxor Specialty Pharmacy is a URAC Rare Disease Center of Excellence. URAC gives this designation to entities that “rise to the challenge of providing the high level of care necessary to service patients with rare diseases.”
We provide enhanced care with a high-touch customer service model, putting patients first with robust clinical programs and dedicated support. Our patient management programs are designed to maintain patient engagement, prevent gaps in therapy, and lessen the risk of suboptimal outcomes. We develop disease-specific care plans, use proactive refill reminders and refill assessments, and perform yearly clinical reviews. Our purpose is to improve outcomes for patients with rare and orphan diseases.
Further reading
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