Implications of Global Drug Development for Health Care and Patients

Drug development issues related to approval of critically important prescription drugs, patient information, and orphan drug research are highlighted for consideration in the health care environment. The need for quicker approval of therapeutic breakthrough agents has grown considerably through the use of treatment INDs and with regulatory and pharmaceutical efforts to combine Phase II and III studies. The risk to benefit ratio applies to patients and to pharmaceutical sponsors who may spend up to $230 million on drug development and, therefore, may afford fewer and fewer failures. Patient education issues must be reviewed by policy makers to examine the existing mechanisms for conveying information to patients. The Food and Drug Administration (FDA) has proposed orphan drug rules that would look beyond the molecular structure of new drugs to determine uniqueness and that describe the handling of potential exclusivity.