Olivia Egge is an osteosarcoma cancer survivor and an advocate for all those living with rare conditions like her own. The senior in high school living in Arlington, Virginia recently wrote an article published in The Herald News advocating for a change in FDA policy. She thinks the United States regulatory agency needs to reevaluate how they utilize rare disease research which is not completed within the country. As part of her plea, she shares her own story.
Olivia Egge’s Cancer Journey
Last year, Olivia was diagnosed with a rare bone cancer called osteosarcoma. This disease disproportionately affects children/young adults. Approximately 1,000 people in the U.S. are diagnosed each year. 60-80% of patients live five years past their diagnosis if the cancer hasn’t spread. If it has, only 15-30% of patients live that long. In the U.S., there are no FDA approved treatments for the condition besides chemotherapy. Considering the fact that chemotherapy emerged 40 years ago, one might think the scientific community would have made more progress by now. As it turns out, in terms of research for osteosarcoma, things weren’t as cut and dry as the FDA made it seem.
After her diagnosis, Olivia was treated for nine months with chemotherapy as that was the only conceivable option presented to her. Following her last treatment, she was told by her doctor that it was time to just “wait and see” if the cancer was going to return.
Unwilling to accept this as her fate, Olivia and her family set about doing their own research. After talking to cancer researchers in Britain, Spain, and Switzerland, and reading many journal articles, her father found something. It turns out another treatment for osteosarcoma did exist in the world, it just wasn’t approved in the U.S. As a result, it wasn’t mentioned to their family.
The Treatment
This treatment was an immunotherapy which had been approved and used in the European Union for over nine years. It’s called muramyl tripeptide phosphatidyl ethanolamine, otherwise known as MTP or Mepact.
This treatment had been investigated in the U.S. but after a trial in 2007 proved inconclusive, it was never investigated again. Meanwhile, in the European Union, a successful 2009 trial led to the treatment’s approval by the European Medicines Agency. The drug has been shown to reduce death in osteosarcoma patients by 28%.
Obviously, MTP was intriguing for Olivia and her family as a potential treatment option. But was there any way for her to obtain it in the U.S.? There is a process. But it’s long, complicated, and expensive.
Accessing the Treatment
Below are just some of the things you must obtain in order to gain access to a drug in the United States which has not been approved by the FDA-
- An individual license
- Your doctor’s approval
- Approval from the institutional review board
- Approval from the hospital which will administer the treatment
Paperwork for these approvals can take weeks to fill out, numerous phone calls, and ultimately undue stress on patients and their families. It’s not a fast process, and for rare disease patients, there often isn’t time to just wait around.
Thankfully, Olivia was able to receive MTP, but only because her family was able to afford it. For 48 rounds of the therapy, MTP costs 100,000 dollars. This is because treatments like MTP which aren’t FDA approved usually won’t be covered by U.S. insurance agencies. That means accessing treatments approved abroad is even more burdensome for patients. For many people, it’s a completely unrealistic endeavor.
Olivia is extremely grateful to have gained access to this therapy, and she’s now advocating to help others do the same. She makes it clear this fight is not just for fellow osteosarcoma patients but for all those living with a rare disease.
Olivia’s Message
The FDA has various policies in place to help rare disease patients access treatment in the U.S.— FDA approved or not. But Olivia contests there’s a gap in their policies concerning patient’s ability to access life-saving treatments which are not FDA approved but have proved their safety and efficacy abroad.
Regulatory agencies such as the European Medicines Agency who approved MTP are respected authorities in the field. Is this not enough to allow patients access to the treatments these agencies approve?
To insurers— It’s time to start considering covering treatments which are not FDA approved, but approved by other respected authorities.
To the FDA— It is time to start collaborating across country lines and utilizing research completed and data found from institutions outside of the US. This would minimize the amount of clinical trials we need to conduct, reduce time from trials to approval, and increase the range of treatments accessible to U.S. rare disease patients.
“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.”
You can read more about Olivia’s story and her thoughts on this issue here.
