Taken together, rare diseases are not so uncommon. One in 10—nearly 30 million Americans—have a rare disease. Nearly two-thirds of these patients are children. This presents a major public health issue.

A silent killer

Many rare diseases, such as blood cancers, are chronic, life threatening and can develop seemingly out of nowhere and strike previously healthy individuals. For the 7,000 known rare diseases, fewer than 400 FDA-approved treatments exist. Patients are desperate for cures, despite a milestone year for the FDA, which approved more treatments for rare diseases in 2014 than ever before. Real action is needed now to raise awareness and develop safe, innovative treatment options.

One in 10—nearly 30 million Americans—have a rare disease. Nearly two-thirds of these patients are children.

Recent developments are providing patients and families hope for a better future. On Feb. 28, thousands of patients, health care advocates, legislators and pharmaceutical companies will come together for Rare Disease Day, an annual awareness event sponsored in the U.S. by the National Organization for Rare Disorders (NORE), which has grown to include more than 80 countries around the world.

A collaborative effort

In the first few weeks of 2015, Congress and the White House have released information on two key initiatives: 21st century Cures and the Precision Medicine Initiative (PMI). Both 21st Century Cures and PMI contain ideas on how to advance the development of new medical knowledge and therapies. My organization will continue to work closely with the committee and members of the House and Senate to advocate on behalf of patients as legislation moves forward.

With the potential for tailor-made medicine to become mainstream—for all, not just rare diseases—the future will likely look very different from the current medical landscape. In the not-too-distant future, all illness and disease may be treated with customized medicine tailor-made for an individual’s genetic makeup.

What is happening in the field of rare diseases is the microcosm for what is happening across health care. The futures of blood health, cancer and rare diseases are within our reach.