Should the FDA Approve Experimental Treatment for Severe Diseases?

I’ve never had the pain and agony of having a kid who is truly sick.  Broken bones and minor surgeries don’t count.  Even one of my kid’s bout with malaria doesn’t rate, as this illness was easily cured.

Parents of kids with chronic illnesses would sacrifice anything to help their kids get better or to suffer less.  In the news recently is a conflict between families of kids with Duchenne muscular dystrophy and the Food and Drug Administration (FDA).  A very small study of an experimental drug called eteplirsen suggested some benefit.   Understandably, the families want the FDA to grant approval so that their kids and others could have access to this drug that will fight a dreadful disease that is fatal.  Families argue that these kids have nothing to lose and can’t wait another 5 years waiting for more definite evidence of efficacy to emerge. 

The FDA is legally required to approve drugs that are safe and effective.  Obviously, the definitions of safe and effective are subjective, but the agency requires that a reasonable threshold be crossed for both of these parameters.  Gray areas create agonizing conundrums for agency officials and patient advocates. 

Should Unproven Treatements be Approved?

The FDA commissioned committee, which will advise the agency proper, did not advocate drug approval, which caused great consternation among family members and their supporters.  These wrenching decisions must be guided by science and medical evidence, not hope and emotion.  Here are some potential objections to approving the medicine.

• The study was too small to have persuasive scientific validity.
• The study did not have a proper control group, which further weakened the conclusions.
• The drug may have unknown and serious side-effects.  Once the drug is approved, physicians can prescribe it lawfully for a variety of diseases and illnesses, some of which may not be life-threatening.
• Acceding to understandable family demands for premature FDA drug approval will prevent patients from entering larger clinical trials, preferably in a randomized study with a placebo control, where safety and efficacy can be more accurately verified.
• If the FDA weakens its standards for approval for a single drug to respond to a constituency, then be prepared for mission creep to erode standards across the board over time.  

It's the parents' job to everything they can do to protect their kid.  It's the FDA's job to protect all of us.  I wish we could protect and save everyone.