For the majority of the population, behavioral changes lead to better health.  There are an endless number of scientific studies that show, for many of us, making lifestyle changes will lead to lower health care costs and increased quality of life. But not everyone is able to improve their health by eating better or exercising more. For some people, the road to a healthier life is controlled by someone else.  No matter their lifestyle, they are unable to access the necessary treatment. This is often the case for people with rare diseases.

A rare disease is classified as such if there are less than 200,000 people in the US with the condition. There are nearly 7,000 rare diseases.  When looking at health from a population standpoint, 200,000 is just a drop in the bucket.  Especially when compared to the whopping 8% of the US population that has diabetes. But sometimes it is important to remove our ‘population lenses’ and realize that those 200,000, however small, represent 200,000 mothers, daughters, brothers, fathers, friends, and loved ones. When you give a face to each of those individuals you realize that their pain, despite its rarity, is just as real as anyone else’s.

IVIG is used to treat autoimmune diseases, primary immune deficiencies, and neurological disorders (as well as some chronic diseases) by replacing necessary antibodies (immunoglobulin). The individuals with one of these conditions, and the doctors who treat it, often call IVIG a miracle drug.  There are few options for those in need of immunoglobulin, and this is one of the rare treatments that will allow them to live and dramatically reduce their symptoms.  But since 2005, many of the people who need this treatment are denied access. 

The Medicare Modernization Act of 2003 cut reimbursement under Medicare and significantly reduced coverage for IVIG.   Providers of IVIG are now difficult to come by, and doctors are not able to guarantee treatment coverage of IVIG.  In 2009, a bill was brought forth to provide easier access to IVIG coverage for those patients in need.  This is a population who may be small, but whose voices are loud and deserve to be heard. Rare diseases may only directly impact a small population, but the volume in a handful of voices is unimaginable.

Recently, a group of patients with Fabry’s Disease petitioned to break a patent on the only product available to treat their inherited disorder.  An overturning of the patent would allow additional manufacturers to produce this drug which is being rationed due to a current shortage. Patients of rare diseases need not be alone in the fight to improve access to necessary healthcare and medications. While the majority of mainstream attention focuses on more common conditions, it is important for us to remember the people who are not always in the spotlight.  Because, after all, if we all listen a little more closely, they may not have to scream quite so loud.

Tags: Behavioral Insight, Policy, Rare Disease