We tend to focus a lot on rare genetic disorders, and with good reason as most of the 7,000 rare diseases are genetic disorders affecting children (75% of rare diseases affect children and 30% of rare disease patients die before the age of five).
However, I want to talk with you today about another type of rare disease because it came to my attention on Friday, and the experiences of the boy in this story are no less profound and poignant than the others we discuss here on these blogs:
Age: 25 years oldInsurance Status: UninsuredDiagnosis: Metastatic Testicular Cancer (Nonseminomatous germ cell tumor)
Testicular cancer was made “famous” by the great Lance Armstrong; here an incredible athlete who won the Tour de France a record seven consecutive times had testicular cancer that metastasized to his brain and lungs. Armstrong beat the cancer, and shortly thereafter founded the Lance Armstrong Foundation (remember all those yellow LIVESTRONG bracelets) AND returned to the Tour de France in 2009, after four years of retirement, taking third place. [Side note: The 2010 Tour de France ended today, July 25, and was won by Alberto Contador for the third straight time.]
Testicular cancers are incredibly rare, as they account for about 1% of all cancers in men, and less than 8,400 cases are reported annually in the United States; testicular cancer is listed as a “rare disease” by the Office of Rare Diseases (ORD) of the National Institutes of Health (NIH). Generally speaking, testicular cancer is one of the most curable forms of cancer. There are nearly 170,000 men who have survived testicular cancer in the United States.
However, the boy mentioned above has Stage III testicular cancer, and now needs more than just another round of chemotherapy for a glimmer of hope. He needs an experimental autologous cell bone marrow transplant. Transplant services are usually not covered under Medicaid or Emergency Medicaid. The Temporary High Risk Pools are not set up everywhere yet, and even so the patient may not be eligible due to immigration status.
There are only so many comprehensive cancer centers that are able to do such a procedure, much less one that would be willing to do it as “charity care.” I spoke with the National Marrow Donor Program and the Be the Match Registry to see if they could provide any resources, and they told me they mainly only work with patients in the leukemia and lymphoma society.
I share this story because it highlights the importance of the work that we all do, through alliances and partnerships of different patient Foundations, and the advocacy we facilitated towards passing health reform legislation. But our job is not yet finished. There is much more work to be done, and there are still patients who slip through the cracks. The system is not meeting the needs of patients with rare diseases and complex illnesses. What recommendations and solutions can you offer? Speak up, we need you
In the news:
You can read the testimony of Diane Edquist Dorman, Vice President for Public Policy of the National Organization for Rare Disorders (NORD), before the Senate HELP Committee on July 21 at their hearing on “Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures.”
A congressional caucus is a group of members of the United States Congress that meets to pursue common legislative objectives. A Rare and Neglected Diseases Caucus has been formed in the US House of Representatives by Congressmen Joseph Crowley (D-NY) and Fred Upton (R-MI). To find your representative’s phone numbers, you may visit https://www.house.gov, use the searchable online congressional directory at Congress.org, or call the U.S. Capitol Switchboard at (202)224-3121 and ask for your representative’s office. Remember that telephone calls are usually taken by a staff member, not the member of Congress. Ask to speak with the aide who handles health care issues, or the health legislative assistant. Nord has also provided a Dear Colleague Letter which you may be interested in sending.
RareArtist.org was created by the Kakkis EveryLife Foundation for artists affected by a rare disease. They received many exceptional works of art during their inaugural EveryLife Art Contest which inspired them to create a venue to display this art and invite others to upload and share their art. Cick here to check out the RareArtist Gallery.
MarbleRoad is planning a launch and fundraising event October 9, 2010 in Washington, DC. Save the date! The fundraising portion will feature an art auction of several professional artists, each who have a compelling story to share related to complex illness—from cystic fibrosis and organ donation to transverse myelitis and RSD/CRPS, and much, much more. Proceeds will be used to provide financial assistance and support services to patients with complex illness. Please stay tuned for additional information. If you have any questions please contact Howard Liebers.