Asuntos regulatorios farmacéuticos: acceso abierto

Rx: Observe, do not treat When is it appropriate not to treat?

Abstract

David V Gauvin and Scott E Boley

The development of a protocol for a nonclinical safety study to assess the safety profile of an investigational treatment, whether a toxicology or safety pharmacology study, should not be undertaken hastily. The evaluation of this new treatment depends on the integrated strategy developed by the scientific team, which may include the pharmaceutical company and the services of a contract research organization. All nonclinical study protocols involving the use of live animals require the inclusion of the Institutional Animal Care and Use Committee (IACUC) and staff veterinarians during the review of the study protocol. The team, as a whole, should endure a frank, honest, and open discussion of study design and animal welfare issues. All available information about the test material should be shared with all parties in a manner and at a time that allows for the constructive development of a protocol and treatment strategy. The intent of nonclinical safety assessments is driven by both administrative guidelines from drug regulatory agencies and statutory (legal) controls from federal laws. When working with test articles, particularly for small molecules, the likelihood of unexpected findings is relatively high. The research team must maintain the highest standards of animal care throughout this process, which is complicated by the fact that when animal health and welfare issues arise, these must be remedied quickly, efficiently, and transparently. We discuss the pitfalls of safety assessment strategies and offer some industry-standard solutions that can help make the path to market a little easier. The article is written with a focus on small molecule development, but many of the points would also apply to biopharmaceuticals.