Experimental Drugs, Public Health and a 4-Year-Old Girl With Cancer

Should desperately ill people be allowed to take experimental treatments that haven't yet been widely tested, much less approved?

It's a question with no black-and-white legal or ethical answer. The Wall Street Journal recently described one family's struggle to secure treatment for their daughter:

John London, a successful hedge-fund executive, is desperate to save his 4-year-old daughter, who suffers from a rare cancer. She clings to life at New York University Medical Center here, sucking her thumb and clutching her favorite teddy bear.

For the past month, Mr. London has been begging a small biotechnology firm to allow Penelope to be treated with an experimental cancer drug that might help. Mr. London has received high-powered support: Several legislators, including House Speaker Nancy Pelosi, have lobbied the company and its board to make the drug available. The Food and Drug Administration isn't blocking the way.

Neotropix Inc. of Malvern, Pa., says it would like to help, but the drug may not be safe for a child and dispensing it would be bad business. "For us to provide the drug to this child would be to put at significant risk a small company with limited financial resources," says P. Sherrill Neff, managing partner of Quaker BioVentures, a major investor in the firm, which is trying to tie up a vital round of financing of about $20 million. "You could delay the opportunity for lots of patients to get this drug if you sidetrack it for one patient," he says.

The journal characterizes the opposition to experimental access from a business perspective. Over at the American Journal of Bioethics Blog,
Alicia Oullette suggests that experimental access could undermine
America's drug development system:

It’s not just the risk to the company that is at stake. Allowing terminally ill patients to obtain investigational drugs outside of clinical trials will undermine the FDA’s ability to protect the public health. Current FDA restrictions are designed to move the drug industry toward better research, expanded clinical trials, and better drugs for all sick people. The phased and planned testing of drugs promotes good science, which ultimately serves the public by preventing charlatans from preying on vulnerable patients, and the unscrupulous from selling dangerous products. Allowing widespread access to experimental drugs will interfere with the process by removing the incentive for patients to participate in essential clinical trials, in which half of patients are typically assigned to receive a placebo instead of the drug.

Moreover, the decision to say no to Penelope may well have protected her. As the journal noted in passing, Neotropix cited safety concerns that buttressed its business decision. The company feared the unknown consequences of pumping massive amounts of the drug -- a virus -- into
Penelope. To be sure, Penelope chances of survival were not increased by company’s decision. But it is fiction to suggest she had nothing to lose had the decision gone her father’s way: the drug he sought could have caused her unspeakable suffering without providing her any benefit at all. Without clinical tests, there is no way to know what effect the drug would have had on the child.

Saying No to Penelope [Wall Street Journal]

Compassion, constitutional rights and Penelope London [American Journal of Bioethics Blog]
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Image: Leo Reynolds *

*Note: Approved drugs aren't always safe or fully understood either; in some cases, as this story shows, they are also experimental. *