What Rare Disease Day Can Mean for the T1D Community

A T1D research analyst attends festivities to mark Rare Disease Day and thinks about how much these patient populations have in common with the T1D community.

February 28th is Rare Disease Day. To mark the day, I and a colleague from T1D Exchange took the short walk from the Exchange’s office over to the UMass Club in Downtown Boston to hear from patients, caregivers, and advocates for people with rare diseases at an event hosted by MassBio.

T1D may not fall into the classification of a rare or orphan disease, even though it is uncommon. The CDC’s 2017 National Diabetes Statistics Report estimates that type 1 diabetes makes up just five percent of the reported cases of diabetes, and people with type 1 diabetes represent less than one percent of the US population.

People with type 1 diabetes and their caregivers share many of the same concerns with patients with rare diseases and their caregivers. For example, both groups often must work hard to find a provider that is knowledgeable and compassionate about their condition. Both groups also often must endure initial misdiagnosis (about 25 percent of the Glu community was misdiagnosed with another condition before being diagnosed with T1D, and 30 percent were in DKA at the time of diagnosis). And both groups must fight for research dollars and lab space.

The hashtag for the day was #patientdriven, and it was fitting that the event overlooked the Golden Dome of the Massachusetts State House, as the Commonwealth has a strong track record of successful patient advocacy. Thanks to the tireless work of patient and caregiver advocates, legislators here have expanded Medicaid, mandated communication between doctors and patients about biosimilar therapies, and mandated that medical foods be covered by commercial health plans, among other policies. And the progress is continuing – The National Organization for Rare Disorders is currently meeting with Massachusetts legislators to ensure patients are protected against what is called step therapy – a “fail first” policy in which insurance denies coverage of more expensive medications until it is proven that cheaper therapies won’t work.

The T1D community has become a force for health care advocacy, but it is just one piece of a bigger puzzle. That’s why it is inspiring to see what disparate patient groups can accomplish together to shape health care policy for the common good. It is my hope that patient community groups that represent smaller populations of people can continue to band together to amplify our advocacy.


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