compassion, constitutional rights and Penelope London
The question of whether terminally ill patients have the constitutional right to access experimental drugs is the subject of hot scholarly debate. As the ten judges of the United States Court of Appeals for the District of Columbia Circuit are reconsidering the original panel opinion in the Abigail Alliance case, which held in favor of such a right, scholars duke it out here, here, and here.
Today’s Wall Street Journal features an article entitled Saying No to Penelope that personalizes the human drama behind the lofty constitutional issues raised in the Abigail Alliance case. Indeed, the heartbreaking story about four-year old Penelope London may present the strongest argument in favor of individual access to unapproved drugs outside a clinical trial: the moral imperative to respect an individual family’s decision to do whatever it takes to save the life of their dying child.
The article details father John London’s heroic effort to obtain experimental cancer drugs for his four- year-old daughter, who is dying from a recurring and aggressive neuroblastoma. Having exhausted all traditional forms of therapy including radiation, chemotherapy, a bone marrow transplant, and surgery, London turned to experimental treatments for his daughter. When the cancer returned after three different experimental treatments, Mr. London thought he was out of options for Penelope. Then he heard about a drug being developed by Neotropix Inc. of Malvern Pa. The drug is a virus that strikes pigs. In early test-tube and mouse experiments, the virus appears to attack certain cancer cells. The risks of injecting the virus into a human being are unknown, however; it has been tested in just six adult human beings.
With the support of several legislators, including House Speaker Nancy Pelosi, London prevailed on the company to sell him the drug for use by his daughter. The FDA has said it will not stand in the way (presumably invoking its “compassionate use” exception to its ban on the use of unapproved drugs). The company refused the request.
The article describes the company’s refusal as principally a business decision: the company fears that if it provides the drug and Penelope dies, its ongoing clinical trials will be stopped cold. The company’s CEO reported that the FDA had put Neotropix’s trial on hold for four months after one of the adult patients in an early trial. “You could delay the opportunity for lots of patients to get this drug if you sidetrack it for one patient,” an investor backing the firm told the Journal. The CEO said, “in a small company with limited financial resources and a high risk profile, you really have to reduce the risks to drug development.”
Couching the story in terms of a seeking the appropriate balance between creating a risk to a business against sure death for Penelope, the Wall Street Journal article makes a strong case against the company’s refusal to give the drug to the child and her family.
The issues are not that simple, however. 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.
With the national media spotlight on Penelope’s case, Neotropix might very well revisit its decision. In the meantime – and regardless of the ultimate decision about the contested medication – Penelope London and her family must carry on. My thoughts and best wishes are with them.
-Alicia Ouellette
