Fight for Untested Treatment Spawns Debate

"The question is will you remove the ability to test your theories if everyone is allowed to jump over the promise of benefit?" asked Hadler.

In most cases academic researchers and, primarily, drug researchers who have a theory about a medical advance must do clinical trials to prove that the treatment works.

But in cases where drugs are approved for compassionate use, Hadler worries the public might latch onto treatments before the theories behind them are tested.

Hadler said the nation has seen this scenario before. In the late 1990s several patients brought lawsuits against insurance companies who denied coverage for experimental bone marrow transplant treatments for terminal breast cancer.

"There was a theory, and there were big lawsuits against HMOS and insurance companies that wanted to say 'wait a minute let's just see what this does,'" said Hadler.

Doctors thought they could use higher doses of chemotherapy safely if they also did bone marrow transplants to protect women from the toxic drugs, according to a 2002 review and timeline of the history in the British Medical Journal.

At the time, there "were few data" about the bone marrow transplants at all, but there was a lot of media attention given to the theory in national newspapers, according article by Gilbert Welch, Juliana Mogielnicki in the British Medical Journal.

Health insurance companies lost so many lawsuits from women wanting coverage that they actually paid for a clinical trial to determine if the bone marrow treatments were effective.

"It turned out to be more harmful than good," said Hadler.

"Where do we have the right to assume more benefit than risk and that the theory is right," he asked?

On the other hand, Dr. Scott Gottlieb, former deputy commissioner for medical and scientific affairs at the FDA, said the most recent examples of high profile battles for treatment in the news would not likely affect the way medical research in the nation as a whole.

Do Rare, High-Profiles Fights Even Matter?

Gottlieb said many of the latest high profile fights for untested treatments are for the diseases so rare "that the drug isn't being studied otherwise."

"The bigger problem is the way that we develop cancer drugs in this country," he said. "The conundrum is that we are forced to test drugs in situations where we only prolong life, not at the beginning of a disease where we could possibly have a better effect."

From that point of view, patients like Lopez and Hempel are actually on the front lines of research.

Hadler agreed and pointed out that just because a disease is rare, doesn't mean there isn't a way to study drugs to treat it, especially through the National Institutes of Health special programs for rare diseases and orphan drugs.

"You can still do systematic trials," he said. "It shouldn't even be held up to the same algorithm or roadmap as the much more common disease where there might be a commercial for a drug one day."

The NIH can't cover every orphan disease and every potential treatment, leaving people like Lopez fighting for insurance coverage and people like Hempel to do their own fundraising for hundreds of thousands of dollars.

Since her experience fighting for her daughters' treatment, Hempel started the Addi and Cassi Fund for other Niemann Pick Type C sufferers and to "bring attention to the drug development crisis and to push for change."

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