Deregulation Can Make Medications Cheaper

If the FDA policed only safety, not efficacy, the result would be more innovation at lower cost

By Charles L. Hooper and Solomon S. Steiner. Mr. Hooper is president of the life-science consultancy Objective Insights. Mr. Steiner is an emeritus professor of neuroscience at City University of New York and an adjunct professor of molecular pharmacology, physiology and biotechnology at Brown University Alpert Medical School. Excerpts:

"From 1938 through 1962, the Food and Drug Administration required proof of safety before drug approval but not proof of efficacy. The approach was abandoned due to a significant misunderstanding of the thalidomide tragedy—when thousands of babies outside the U.S. were born with severe birth defects."

"Congress required, through the Kefauver-Harris Amendments of 1962, proof of efficacy before granting marketing approval. The new rule addressed a problem that didn’t exist"

"bringing one successful drug to market costs about $9 billion on average."

"if a clinical trial shows that a drug works for 60% of patients, no one knows in advance if it will work for a particular patient." 

"Some patients respond well to [anticoagulants like] Pradaxa but not Xarelto, and vice versa. A drug might fail for one patient but be the right one for another. Fewer approved drugs means fewer chances for patients to find their match." 

"Drug companies would still run efficacy trials because it is in their interest to do so. Doctors, insurers and patients want assurances that a new drug actually works. But those trials would be designed to answer the medical community’s pressing questions, not to satisfy a bureaucratic checklist"