Is FDA Approval Required for Legal Access to Specific Drugs?

General Reference (not clearly pro or con)
The US Food and Drug Administration (FDA) stated in the Apr. 20, 2006 press release "Inter-Agency Advisory Regarding Claims That Smoked Marijuana Is a Medicine":

"FDA is the sole Federal agency that approves drug products as safe and effective for intended indications. The Federal Food, Drug, and Cosmetic Act requires that new drugs be shown to be safe and effective for their intended use before being marketed in this country. FDA's drug approval process requires well-controlled clinical trials that provide the necessary scientific data upon which FDA makes its approval and labeling decisions."

Apr. 20, 2006 - US Food and Drug Administration (FDA) 

The US Food and Drug Administration (FDA) stated the following in a Jan. 1995 article titled "Benefit Vs. Risk: How FDA Approves New Drugs," posted on its website:

"Under current law, all new drugs need proof that they are effective, as well as safe, before they can be approved for marketing. But it's important to realize that no drug is absolutely safe. There is always some risk of an adverse reaction. It's when the benefits outweigh the risks that the Food and Drug Administration considers a drug safe enough to approve...

FDA has in a number of ways taken steps to make urgently needed drugs available sooner. These are drugs for treating serious or life-threatening diseases that have no good treatment.

Under the accelerated approval rule, the agency can rely as a basis for drug approval on a reasonable 'surrogate' endpoint -- that is, an effect of a drug on a marker of the disease, rather than an actual effect on survival or illness. In accelerated approval, FDA approves the drug on condition that the sponsor study the actual clinical benefit of the drug.

According to FDA Commissioner David A. Kessler, MD, 'We cannot wait for all the evidence when people are suffering and dying from a devastating disease. But, we must ensure that all the evidence we need eventually does get collected.'"

Jan. 1995 - US Food and Drug Administration (FDA) 

PRO (yes)

The Institute of Medicine published in its Mar. 1999 report titled "Marijuana and Medicine: Assessing the Science Base":

"Under the Federal Food, Drug, and Cosmetic Act, the FDA approves new drugs for entry into the marketplace after their safety and efficacy are established through controlled clinical trials conducted by the drugs' sponsors.

FDA approval of a drug is the culmination of a long, research intensive process of drug development, which often takes well over a decade....

The manufacturer submits an NDA [New Drug Application] to the FDA to gain approval for marketing when clinical testing is complete.... In 1996 the median time for FDA review of an NDA, from submission to approval, was 15.1 months...

With FDA approval of an NDA, the manufacturer is permitted to market the drug for the approved indication."

Mar. 1999 - Institute of Medicine 
"Marijuana and Medicine: Assessing the Science Base" (988 KB)  

The Merck Manual of Medical Information, printed in 1997, stated:

"By law, drugs are divided into two categories: prescription drugs and nonprescription drugs... In the United States, the Food and Drug Administration (FDA) is the government agency that decides which drugs require a prescription and which may be sold over the counter...

After many years of use under prescription regulation, drugs with excellent safety records may be approved by the FDA for over-the-counter sale...

Before the federal Food and Drug Administration (FDA) existed, just about anything could be be put in a bottle and sold as a sure-fire cure."

1997 - Merck Manual of Diagnosis and Therapy 

The US General Accountability Office (GAO) stated in its Aug. 2000 report to Congress titled Prescription Drugs: Many Factors Affected FDA's Approval of Selected "Pipeline" Drugs:

"Before marketing a new prescription drug in the United States, the pharmaceutical company sponsoring the drug must obtain approval from FDA.

To receive approval, the sponsor must demonstrate that the drug is both safe and effective for its intended use."

Aug. 2000 - US Government Accountability Office (GAO) 

The US Drug Enforcement Administration (DEA) stated the following in a May 9, 2005 article "Medical Marijuana: The Facts," posted on the DEA website:

"Since the passage of the 1906 Pure Food and Drug Act [the origins of the FDA's regulatory function], any drug that is marketed in the United States must undergo rigorous scientific testing. The approval process mandated by this act ensures that claims of safety and therapeutic value are supported by clinical evidence and keeps unsafe, ineffective and dangerous drugs off the market."

May 9, 2005 - US Drug Enforcement Administration (DEA) 

CON (no)

The US Food and Drug Administration (FDA) stated in its website section on "Access To Unapproved Drugs" (accessed Feb. 12, 2002):

"Access to unapproved drugs can occur through many legal mechanisms, including:

  1. enrolling in Clinical Trials,
  2. a Special Exception or
  3. Compassionate Exemption,
  4. Emergency Investigational New Drug (IND), and
  5. a Treatment IND.

One of the most common methods to have access to an unapproved drug is to enroll in a clinical trial. There are many sources of information about clinical trials.

Another alternative is for a physician to file a single patient or emergency IND directly with the FDA. This must be done with the cooperation and permission of the drug supplier."

Feb. 12, 2002 - US Food and Drug Administration (FDA) 

Daniel Klein, PhD, Associate Professor of Economics at Santa Clara University, wrote in his Sep. 2000 article "Economists Against the FDA," published in The Freeman: Ideas on Liberty:

"A drug's FDA-approved uses are called its 'on-label' uses. Once a drug is approved for any use, it may be used in any way doctors and users see fit.

Approved drugs are often found to have other benefits, and doctors learn to prescribe those drugs for such 'off-label' uses. Although off-label uses have absolutely no standing with or approval by the FDA, they are perfectly legal."

Sep. 2000 - Daniel B. Klein, PhD 

John V. Kelly, former New Jersey State Assemblyman (36th District), wrote the following in an Aug. 14, 2000 letter to Senator Robert Smith, Chairman of the Environment and Public Works Committee, and placed on the internet by the Fluoride Action Network:

"After filing a FOIA [with the FDA] and waiting six months, I was stunned when I was advised by the FDA that fluoride supplements were not approved by the FDA [underline in original]. Incredibly, in fifty years, no one has ever bothered submitting a petition to the FDA to have these products approved!

The water fluoridation debate has been raging for fifty years. The dose recommended for water fluoridation when adjusted for weight is the same dose recommended for prescription supplements. However, no clinical trials have been conducted and submitted to the FDA to demonstrate the effectiveness of ingesting fluoride.

So, the question remains, if the FDA does not recognize the safety and effectiveness of fluoride supplements, how can we even consider fluoridating water?"

Aug. 14, 2000 - John V. Kelly