Peter L. Saltonstall
President and CEO, the National Organization for Rare Disorders
For many of the roughly 7,000 identified rare diseases, those who are diagnosed are one in a million. Health equity is needed to improve the lives of people with a rare disease.
In the United States, for a disease to qualify as rare, it must affect less than .06% of the population. Living with a rare disease is an isolating experience. But while the number of people impacted by an individual rare disease is often quite small, rare diseases do not discriminate. They affect people of all genders, ages, races, ethnicities, socioeconomic statuses, religions and sexual orientations.
Fighting for health equity in rare diseases
The theme for Rare Disease Day 2023 is health equity — which gives us a chance to highlight the challenges the world’s 300 million rare disease patients face and what we can do to help.
For those with rare diseases, health equity means that all patients and families would have a fair opportunity to achieve their best health and wellbeing, regardless of who they are, where they live and what disease they have. Unfortunately, rare disease patients face significant obstacles in achieving this ideal.
For the average patient, it takes between five to six years to get an accurate diagnosis.
Obstacles to achieving health equity
For many, the obstacles begin with getting an accurate and timely diagnosis of their condition. The journey to diagnosis is often long and costly. For the average patient, it takes between five to six years to get an accurate diagnosis — a process that often involves visits to multiple doctors and specialists and countless tests.
Once they receive a diagnosis, rare disease patients face more challenges — since fewer than 10% of rare diseases do not have approved treatments. For conditions that do have treatments, patients are often unable to access them due to cost or where they live.
How to help people with a rare disease
Health equity is possible by finding solutions to existing disparities and exploring ways to address the needs of every rare disease patient. Together, we can advocate for change by participating in campaigns and speaking up for policies in our respective countries that help rare disease patients.
Together, we can advance breakthroughs that discover new treatments and medicines. The rare disease journey is not easy. But because the worldwide rare disease community is stronger and better organised than ever before, health equity can be more attainable.
We hope everyone will learn about the realities of rare diseases and be moved to take action and help create a better future that eliminates disparities by advancing research, public policy and access to care.
