The Need for Advocacy

Without treatment paid for by the National Institutes of Health, Lori’s son Nino might be unable to walk. Other than a small chest port under his shirt that allows doctors to administer his drugs, the nine-year-old’s body gives little indication of the rare and debilitating periodic fever syndrome (PFS) he suffers from. For now, Nino will continue receiving the off label drug that allows him to attend school every day and play sports. But the National Institutes of Health is suffering severe federal budget cuts and the Todaros’ health insurance has denied coverage for the treatment. The prospect of a backwards step in her son’s treatment is one that Lori Todaro isn’t willing to accept.

The most successful way to be a rare disease advocate is to identify and pursue opportunities to influence policy change to improve the situation for rare disease patients. Our top advocacy priority is Nino's Act (Senate Bill #2629 and House Companion Bill HR #5468), which has the potential to help thousands of children and families affected by a rare disease. The Legislation ensures that successful treatments for rare diseases received through studies at NIH are covered under Medicaid if the child’s insurance carrier does not recognize the off label treatment.” Currently Nino's Act sits in the Senate Finance Committee and in the House Committee on Energy and Commerce.

Fact: Children who suffer from a rare diseases and who enter into a drug trial are left without treatment funding once the successful drug trial has ended.

Fact: About 3,500 children with rare diseases are treated at the National Institute of Health each year.

Fact: In February 2008, Senators. Arlen Specter (R-Pa.) and Robert P. Casey Jr. (D-Pa.) introduced legislation, named for Nino Todaro, that seeks to provide continuous medical care to children who are transitioning out of research studies with the National Institutes of Health in Bethesda, Md.

Background Information

For children who suffer from rare-diseases, treatments can mean entering into a protocol/drug trial that is often times using orphan drugs or off label treatment options. Once a child responds to the treatments and he or she is stable enough to transition out of the
study, private insurers can deny the coverage for continuous treatments that have already proven effective. Nino’s Act would allow for these children transitioning out of the research trials with the National Institute of Health to gain access to continue treatments through Medicaid if their insurer does not recognize the treatments.

Nino’s Story

Nino Todaro is a 9-year-old boy who resides in Newville, Pa. with his parents and siblings. Unlike a typical 9-year-old, Nino suffers from a rare-disease, Undifferentiated Autoinflammatory Periodic Fever Syndrome, which causes him severe joint and bowel pains. Nino’s family nearly went bankrupt trying to pay for tests and treatments and therapies that were not stabilizing his condition. Nino would run extremely high fevers and be crippled with pain about every seven to 10 days. Then in 2003 he entered into the National Institute of Health’s treatment program. Nino’s condition stabilized upon receiving his treatments and in 2006 he was healthy enough to transition from the routine treatments that his protocol included into his home town medical facility for continued treatments.. Upon this request from his NIH team, the family’s insurance company denied coverage to Nino’s $10,000 monthly medical bills and Nino faced a reoccurrence of the disease without the continuation of the effective, although off label medications. Despite three appeals, the insurance company continued to deny the family coverage. Nino’s father, Tony, a Pennsylvania State Trooper, and his Mom, Lori, rallied support from the Pennsylvania State Troopers Association and Senator Arlen Specter(R-Pa.) and eventually the insurer reversed the decision and granted coverage for the year. However, this continued coverage must be renewed on an annual basis.

To help support Nino's Act 2008 please see our Tips to Be an Effective Nino's Act Advocate.

Take Action

Ready to take action?  Click here to learn more about how to be an effective rare disease advocate.