Providing Patient Access to Promising Investigational Drugs

While enrolling subjects into a well-controlled clinical study, it is certain that some patients, although they have the disease or condition being studied, will have to be excluded from the study in compliance with certain criteria defined in the clinical protocol. The exclusion may be due to any number of reasons, from age to disease state to personal history or habits (such as smoking cigarettes or drinking alcohol). In most of these situations, even though the patient has been excluded from the clinical trial, the standard of care for the given disease-that is, the marketed prescription or over-thecounter drugs or combinations of such drugs or other means of clinical management of the condition, such as diet modification-is still an option for the patient and is not less desirable for the management of the patient’s health. However, what happens when patients are excluded from clinical studies of investigational drugs and no clinical management technique is known to be effective or no other marketed drug is available to treat the disease or condition? Or what if the patient has already failed to respond to available marketed drugs for the disease or condition? If an investigational drug exists that may provide necessary relief to the patient by reducing or curing the symptoms of the disease or that may provide hope of increasing the life of the patient, is it unethical to refuse or withhold treatment to the patient? The answer to these questions and the resulting important issues and considerations are discussed in this chapter.