Olivia Egge is a high school senior in Arlington.
“Now we sit and wait to see if the cancer comes back,” my doctor said as she unhooked the hanging bag of chemotherapy. That moment marked the end of my nine months of treatment for osteosarcoma, a rare bone cancer disproportionately afflicting children and young adults like me.
There have been no therapeutic advances in treating osteosarcoma in the United States since the advent of chemotherapy nearly 40 years ago. The cancer affects fewer than 1,000 Americans annually, and it has a five-year survival rate of 60 to 80 percent if the cancer remains localized, according to the American Cancer Society; if the cancer has spread, the survival rate drops to 15 to 30 percent.
The prospect of waiting for a verdict on the cancer’s possible return was frustrating, but I knew that I was more fortunate than most Americans with osteosarcoma.
After I received the diagnosis last year, my family searched for treatments beyond chemotherapy that might help. Doctors in the United States don’t generally discuss drugs that haven’t been approved by the Food and Drug Administration, so we weren’t told about an immunotherapy treatment for osteosarcoma that has been in use in the European Union for more than nine years. The chemical name of the treatment is muramyl tripeptide phosphatidyl ethanolamine; patients and their families call it MTP, or its commercial name, Mepact.
MTP reduces deaths from osteosarcoma by 28 percent — a significant achievement. My father discovered the use of MTP in Europe by researching Journal of Clinical Oncology articles online and talking to pediatric oncologists in Spain, Switzerland and Britain. Why isn’t it available in the United States? Because in 2007, an FDA panel rejected it based on a clinical study that showed the drug’s efficacy but also had a design flaw that raised questions about whether the efficacy was because of MTP or the presence of a chemotherapy drug (which has since been shown not to improve outcomes).
Given the expense of clinical trials and the limited potential market for the drug, that was the end of the story for MTP in the United States. Meanwhile, the European Medicines Agency, the FDA of the European Union, reached the opposite conclusion and in 2009 approved the drug for use in the E.U.
The FDA works hard to ensure the safety and efficacy of drugs sold in the United States. The agency’s “orphan drug” policy encourages pharmaceutical companies to produce drugs that might not be profitable but will help patients with rare diseases. The “right to try” law passed by Congress last year helps terminally ill patients obtain access to experimental therapies not yet approved by the FDA.
But I worry that my experience suggests a gap in FDA policy that warrants the agency’s attention, not just for patients with osteosarcoma, but for anyone with a rare disease that might be treatable with drugs that have been proved safe and effective by respected authorities outside the United States.
As my family found, gaining permission to use a non-FDA-approved drug in the United States is a time-consuming and torturous process. Patients must obtain an individual license, a relevant doctor’s approval, institutional review board approval, approval from the hospital where the treatment will be administered and more. Along with requiring countless phone calls, for us the process entailed paperwork that took weeks to fill out — under the intense stress of a cancer diagnosis, when speed matters.
Then there is the expense. MTP cost $100,000 for 48 rounds of treatment. I was fortunate that my family was able to afford it, but many other patients can’t, and insurance companies generally won’t cover non-FDA-approved drugs. It is wrenching to think of a child with osteosarcoma whose family, even after going through the arduous process of obtaining permission to use MTP, doesn’t have enough money to pay for it. Many aspects of health-care policy are frequently under review; I would advocate for insurance companies to consider covering non-FDA-approved drugs that are regarded by an institution such as the European Medicines Agency as safe and effective in treating rare diseases.
Patients without other options should not have to cross oceans, waste precious time or expend resources that few families have in the hope of obtaining safe, effective and potentially life-saving drugs. Patients now struggling, and survivors like me, are desperate for the FDA to approve new treatments and for drug researchers to have the incentives to develop them.
In cases where drugs used overseas show years of clinical evidence demonstrating safety and efficacy, the FDA should consider reviewing the existing data instead of requiring new trials in the United States. “Wait and see” is bad enough for any cancer patient to hear, but children with rare cancers, and their parents, should at least be confident that they have been given the best chance of survival.