Joseph R. Anticaglia MD
Medical Advisory Board
In the late 1950/s and early 1960’s, pregnant women worldwide used a “wonder drug” promoted to combat a variety of conditions including morning sickness, insomnia and depression. Thousands of women who took the drug were horrified when they gave birth to babies with misshapen hands, feet, arms or legs.
These children were labeled “Thalidomide Babies.” Critics said, “Why didn’t the government or pharmaceutical companies do more research to protect these women and children before unleashing such a harmful drug on the public?”
During the early 1980s in the U.S., there was no successful treatment for the victims of the AIDS epidemic. The number of cases each year was increasing by the thousands due to this retrovirus. Unless treated, the virus assaults its victims’ immune system causing them to waste away suffering a miserable, hopeless, prolonged death.
Larry Kramer, a playwright, author, and one of the leaders of “Act Up,” chided the FDA: “There are eight drugs the FDA is sitting on. We cannot wait ten years for drugs to be processed through the FDA (clinical trials). AIDS patients don’t have enough time.”
The Federal Drug Administration seems to be caught in the middle. On the one hand, the FDA is criticized for being overly cautious and taking too long before it gives approval for potential life-saving drugs and treatments. On the other, it is lambasted for rushing drugs, treatments and devices to the public without sufficient clinical trials to insure their safety and effectiveness…
Clinical Trials
Clinical trials are research projects designed to test drugs, treatments and devices on people to establish whether they are safe and effective. The researchers also want to know whether they are superior to the ones that may already be in use.
Trials ordinarily proceed in a stepwise fashion involving four phases after animal experiments demonstrate that the drugs are safe and effective.
Snapshot of the Four Phases
Phase 1: SAFETY
Healthy volunteers (20-89)
Determine if the drug is safe
Decide whether or not it’s reasonable to proceed to Phase 2
Phase 2: EFFICACY
Patient participants (100-300)
Determine the drug’s efficacy
Note side effects
Phase 3: EFFECTIVENESS
Patient participants (300-3000)
In depth study of drug’s effectiveness and safety
Monitor side effects
Phase 4: FOLLOW-UP
FDA has approved the drug for public use
Long term follow-up of drug’s effectiveness
Report side effects to appropriate agencies
The FDA years ago initiated a program to speed up the clinical trial approval process to get drugs and treatments to patients quickly (Accelerated Approval Program). The program is open to controversy, but there are many breakthrough therapies which have benefitted people and saved lives.
Clinical research is the linchpin for the development of new drugs and treatments. It has made people healthier and live longer. The challenge is to bring new medications to the public as quickly as possible, while guaranteeing their effectiveness and safety as well as safeguarding the health of the volunteers.
References
Learn About Clinical Studies; Clinical trials.gov; January 2017
FDA; Fast Tract, Breakthrough Therapy, Accelerated Approval, Priority Review; September14, 2015
NIH; What Are Clinical Trials; June 27, 2016
This article is intended solely as a learning experience. Please consult your physician for diagnostic and treatment options.