Monday, February 23, 2015

FDA Hindering Promising Therapies Derived From Patient's Own Cells

Interesting update from the Winter 2015 issue of City Journal: "Patient, Heal Thyself".

Their teaser line: "Treatments from our own cells could cure many diseases -- if Washington will only allow it to happen."

They discuss how the current FDA regulatory paradigm stifles this promising research:
Unlike conventional drugs, these cell therapies are created from scratch, one patient at a time, and many of the tools used to create them are simple, compact, and cheap enough to land in laboratories that serve hospitals, small clinics, and doctors in private practice. They have been landing there in growing numbers in the last decade, and Washington has been trying to keep pace.

The Food and Drug Administration (FDA) has taken the position—upheld in February 2014 by a federal appellate court—that a patient’s cells become a “drug” when extracted and manipulated in a laboratory, and may not be used to treat the patient without FDA approval. But it is far from clear how the agency should set about approving a custom-made drug that will be prescribed to only one patient, in whom its safety and efficacy will be largely determined by how the patient’s molecular biology interacts with itself....

[R]igidly scripted trials remain the norm at the FDA. Responding to the advent of drugs precisely designed to modulate specific molecular targets, the FDA has gradually come to accept that the drug-approval process must take into account the relevant patient-side molecular factors as well.

The FDA has, however, been slow to accept trial protocols that systematically investigate those factors and incorporate them into prescription protocols that increase the likelihood that the drug will be effective. With rare exceptions, the agency requires that the molecular factors that might affect a drug’s efficacy be identified by studying the disease before a clinical trial begins, or by analyzing the drug’s performance in short, early-phase trials that involve few patients—far too few to provide a full understanding of how variations in patient chemistry may affect a drug’s performance. This has already been recognized as a serious problem in the testing of certain categories of conventional drugs...
The scientific aspects are fascinating. And the regulatory aspects are infuriating.

For more details on both, see the full text of "Patient, Heal Thyself".

(Link via Instapundit.)

(Image from the article, captioned: "Emily Whitehead, a child given cell therapy for acute leukemia; her family says that she shows no sign of the cancer today. Ed Cunicelli, The Children's Hospital Of Philadelphia/AP photo.")