Animal Efficacy Rule

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The animal efficacy rule ( English :. Efficacy = effectiveness) is one of the American US Food and Drug Administration (FDA) in 2002 established approval process for certain drugs . These medicinal products must protect or at least alleviate serious or life-threatening illnesses caused by exposure to noxious agents, although clinical studies to check their effectiveness in humans cannot be carried out for ethical or practical reasons. In these cases, it is sufficient to demonstrate the effectiveness of model organisms in animal experiments .

description

For vaccines or therapeutics to be approved , extensive human clinical trials are usually required. The aim of these studies is to prove the effectiveness and tolerability. The therapeutic success that can be achieved through effectiveness must outweigh possible damage caused by side effects. For example, the significant side effects of chemotherapy for cancer are tolerated to a certain extent if efficacy is proven. The benefit / risk ratio is of central importance. Before the Animal Efficacy Rule was passed , it was not possible to approve drugs, for example against bioterrorist attacks or sudden epidemics. For ethical reasons it is forbidden to infect healthy people with spores of the anthrax pathogen Bacillus anthracis in order to prove the effectiveness of a previously administered vaccine or a therapeutic agent administered afterwards. Since the Animal Efficacy Rule was passed in July 2002, evidence of the effectiveness of laboratory animals ( animal efficacy ) has been sufficient . In such cases, efficacy studies on humans are no longer necessary. Clinical studies on healthy volunteers are still required to determine the safety profile and to determine the dose. In the example of Bacillus anthracis , tests on two different suitable animal species would suffice to demonstrate the effectiveness. The safety profile would be established with healthy volunteers given the later scheduled dose.

The studies with the model organisms must be as close as possible to the human clinical picture and the desired effectiveness must be transferable to humans as far as possible. Good Laboratory Practice and animal welfare ( animal welfare act ) must be complied with. In the event that the measure developed under the Animal Efficacy Rule is actually used in humans (for example in the event of a nuclear accident or a bioterrorist attack), the FDA reserves the right to conduct efficacy studies on humans. The approval of a drug or a measure according to the Animal Efficacy Rule accordingly means that the developing companies have planned studies to prove the effectiveness of humans in the event of a crisis.

The Animal Efficacy Rule was laid down in Code of Federal Regulations 21 (Foods and Drugs) Part 314, Subpart I.

Objectives of the Animal Efficacy Rule

The Animal Efficacy Rule is intended to enable the development of medical measures for the prevention or treatment of disease states that are caused by chemical, biological, radiological or nuclear effects (especially weapons of mass destruction ). The Animal Efficacy Rule excludes products or measures that can be approved with other standards, such as surrogate markers or other clinical endpoints as survival rate or irreversible morbidity.

Drug examples

The first active ingredient approved under the Animal Efficacy Rule was pyridostigmine bromide in 2003 . It has been cleared by the FDA for treatment after exposure to the nerve agent soman . A different dosage of pyridostigmine had previously been approved for the treatment of myasthenia gravis . The second active ingredient, hydroxocobalamin , has been approved for the treatment of cyanide and smoke poisoning .

further reading

Individual evidence

  1. ↑ a b Guidance for Industry. Internal Radioactive Contamination - Development of Decorporation Agents. (PDF; 181 kB) US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), March 2006
  2. ↑ D. Hart: The benefit / risk balance in pharmaceutical law. An element of the Health Technology Assessment. In: Bundesgesundheitsblatt Volume 48, 2005, pp. 204-214. doi : 10.1007 / s00103-004-0977-2 PMID 15726462
  3. ↑ A. Kloesel, W. Cyran: AMG § 5 Note. P. 31ff.
  4. ↑ a b c G. K. Gronvall, D. Trent, L. Borio, R. Brey, L. Nagao: The FDA animal efficacy rule and biodefense. In: Nature Biotechnology 25, 2007, pp. 1084-1087. doi : 10.1038 / nbt1007-1084 PMID 17921984
  5. ↑ FDA regulation of animal research. In: Lancet Volume 372, Number 9644, September 2008, p. 1122, ISSN  1474-547X . doi : 10.1016 / S0140-6736 (08) 61456-7 . PMID 18926259 .