Needs of People with Rare Diseases to be in the Spotlight with Leap Year Observance

2020 is a leap year, which brings an extra day to the calendar at the end of this month and a singular observance.  The National Organization for Rare Disorders (NORD), with national headquarters in Danbury, is the Official U.S. Sponsor of Rare Disease Day, which will be marked in Connecticut and around the country on and around Saturday, February 29.

NORD, established in 1983, is an independent, nonpartisan, nonprofit advocacy organization and the voice of the rare disease patient community. NORD addresses complex medical, research and public policy issues through programs and services shaped by a single guiding vision: to improve the lives of all Americans affected by rare diseases.

The Connecticut Rare Action Network will be on hand on Friday, February 28, 2020 at the Connecticut State Legislative Office Building, as patients, families, caregivers, medical professionals, industry representatives and state legislators gather to discuss the challenges rare disease patients face and to highlight how state residents can make a difference in the lives of rare disease patients and their families in Connecticut.

A rare disease is defined as any disease, disorder, illness or condition affecting fewer than 200,000 people in the United States. An estimated 25-30 million Americans, almost 1 in 10, have rare diseases. In Connecticut, an estimated 300,000 people have rare diseases. It is estimated that there are over 7,000 rare diseases, which are defined in the United States as diseases affecting 200,000 or fewer people. Today, over 90% of rare diseases still do not have a treatment specifically intended to treat the disease.

In a State of the States report issued late last month by NORD, seven categories were highlighted for their protections provided to individuals with rare diseases.  The annual report, first released in 2015, is designed to “serve as a tool for patients, advocates and policy makers as they strive to ensure that state policies best serve the needs of the rare disease community.”

In its latest state report card, evaluating the status of policy issues that are of significant importance to the rare disease community, Connecticut was one of only seven states to earn an A in prescription drug out-of-pocket cost sharing protections.  The state also earned an A in Medicaid eligibility, waivers, and home and community-based services; a B in newborn screening; a B in medical nutrition coverage; and a B in therapy patient protections,

The zebra is the official symbol of rare diseases in the United States and is noted for its black and white stripes, which are central to its uniqueness. As the NORD website explains, “everyone has his/her own stripes, those characteristics that make each individual distinct. While each of the more than 7,000 rare diseases are unique, there are many commonalities that unite the rare disease community. In the spirit of raising the profile of the rare disease community at large and celebrating Rare Disease Day, this year NORD will promote specific ways that individuals, organizations and groups can show their stripes.”

It is characterized by the uniqueness of Leap Year Day, on February 29, the last Saturday this month.