INFOGRAPHIC: How Rare are Rare Diseases?
Rare diseases are serious, life-altering and often catastrophic conditions for patients and their families – and they’re more common than you might think. While each rare disease is unique, there are over 7,000 known rare diseases impacting 10% of Americans, a staggering 30 million people in our country today.
From early mortality rates to critical emergencies, these specialty patients face alarming realities: Patients with cystic fibrosis have an average life expectancy of 42 years. Minor head bumps and abdominal injuries can lead to fatalities for patients with hemophilia. Prior to 2008, patients with hereditary angioedema (HAE), had zero FDA approved, targeted therapies available to treat their condition, leaving them relying on less effective treatments in order to manage through their debilitating swelling attacks. Due in part to the genetic component involved in rare diseases, more than half of these patients are children - many of which who unfortunately won’t live to see their 5th birthday.
But, quality and length of life aren’t the only challenges in this space. Rare diseases remain both difficult to research and diagnose. In fact, on average patients wait nearly five years for an accurate rare disease diagnosis and see an average of seven physicians during that time. Access to treatments from both a cost and availability standpoint is another. While 35% of specialty drugs in the pipeline are orphan indicated, today only 5% of known rare diseases have an FDA approved treatment available. Those with access to an approved therapy struggle with the high costs associated. The top 10 most expensive drugs in the world are orphan drugs which cost between $450,000 and $700,000 a year. In 2016, the average cost per patient per year for an orphan drug was $140,443 compared to $27,756 for non-orphan drugs. Affordable access to these medications is often quite literally a matter of life and death.
Many of these drugs are life-changing, even curative for patients with certain rare diseases, and it’s our responsibility as advocates, payers and providers to ensure we’re supporting these patients from both a cost and care perspective. We can help manage the unique complexities of patients with a rare disease by matching big leaps in drug development for patients with applicable management strategies and best-in-class clinical care.
Talk to your account representative to learn more about our care model and identify which management solutions might be right for you. And, if you’d like to experience the personalized care patients with a rare disease receive at Accredo first-hand, we invite you to join us at our Rare Disease Therapeutic Resource Center in Memphis.