On the subject of the FDA carefully testing drugs for both safety & efficacy… when some people propose cutting corners because 'It's an emergency, what have you got to loose?', I see red. The FDA very carefully tests both new drugs and already approved drugs if they will be used in a different way than originally approved. Why? I quote (see the indented text) from Wikipedia at length on the history of two disasters involving medications that were not tested carefully enough.
In the early 1960s there was an infamous case of a drug,
Thalidomide, (also see
Thalidomide scandal) which was was first marketed in West Germany in 1957, where it was sold over-the-counter. When first released, it was promoted for anxiety, trouble sleeping, "tension", and morning sickness in pregnant women. While initially deemed to be safe in pregnancy, concerns regarding birth defects were noted in 1961 and the medication was removed from the market in Europe that year. The total number of people affected by use during pregnancy is estimated at 10,000, of which about 40% died around the time of birth. Those who survived had limb, eye, urinary tract, and heart problems. Its initial entry into the US market was prevented by Frances Kelsey at the FDA. The birth defects of thalidomide led to the development of greater drug regulation and monitoring in many countries.
This disaster prompted many countries to introduce tougher rules for the testing and licensing of drugs, such as the Kefauver Harris Amendment (US), Directive 65/65/EEC1 (E.U.), and the Medicines Act 1968 (UK). In the US, the new regulations strengthened the FDA, among other ways, by requiring applicants to prove efficacy and to disclose all side effects encountered in testing. The FDA subsequently initiated the Drug Efficacy Study Implementation to reclassify drugs already on the market.
Theralizumab
In 2006 there was another example, Theralizumab, aka TGN1412.
It was withdrawn from development after inducing severe inflammatory reactions in the first-in-human study by PAREXEL in London in March 2006. The developing company, TeGenero Immuno Therapeutics, went bankrupt later that year.
In its first human clinical trials, it caused catastrophic systemic organ failures in the subjects, despite being administered at a supposed sub-clinical dose of 0.1 mg per kg; some 500 times lower than the dose found safe in animals. Six volunteers were hospitalized on 13 March 2006, at least four of these suffering from multiple organ dysfunction. Tentative opinions from an as-yet uncompleted inquiry suggest that the problems resulted from "unforeseen biological action in humans", rather than breach of trial protocols, and the case therefore has had important ramifications for future trials of potentially powerful clinical agents.
The Theralizumab story goes on much longer in that Wikipedia link. Feel free to read it, and if you understand all that immunology, please fill me in about it. It's not an easy subject. The long term result of the Theralizumab story is that the FDA now can require non-human primate testing of drugs before they are tested clinically. The use of non-human primates is controversial… monkey testing.
The bottom line is that these laws and regulations are still in effect today, and I see no reason to ignore or by-pass them.
