Special Patient Populations
Traditionally, new chemical entities expected to have low toxicity and thus low risk for the participants have been studied in “normal healthy” subjects, usually male, often age 18-45, and (depending on the location of the research unit) primarily Caucasian or Hispanic. While understandable and fairly easy to recruit, the disadvantage is that there are many people in this world who are considerably different from this population, thus the pharmacokinetic or pharmacodynamic data from these studies may not directly apply.
At the other end of the spectrum, new chemical entities expected to have dose related toxicities such as cytotoxic cancer chemotherapy agents were largely studied in patient populations where the ethical considerations of the risk:benefit ratio required some potential for benefit to offset the possible risks. While also understandable, such studies are typically very difficult and time consuming to recruit.
Fortunately there are a variety of alternatives. Most stakeholders recognize the benefit of including a population more diverse than healthy normal young males. Especially with the global trend towards an aging population, the upper age is more typically 50 to 65, and there is more interest in earlier inclusion of women- with appropriate pregnancy precautions to avoid unintended fetal exposure. And as the industry becomes increasingly global, inclusion of Asians and other non-Caucasians is a growing priority.
Earlier inclusion of patients with the target indication is also of increasing interest, at times even in the First Time In Humans study, sometimes as a subset of the total enrollment. Adverse events and basic pharmacokinetic characteristics may both be somewhat different in patients than in normal healthy individuals, and many pharmacodynamic markers cannot be reliably assessed in healthy individuals compared with patients with the target indication. These data may be crucial early in compound development for a well considered decision regarding further compound development.
Dr. Ruckle has experience with study design and conduct involving a variety of special populations: women, Japanese/Asians, healthy elderly, pediatrics, women after menopause, and patients with Diabetes, Hypertension, Obesity, Hyperlipidemia, Osteoporosis, Osteoarthritis, Asthma, Renal Insufficiency, Hepatic Insufficiency, dry eyes, psoriasis, and etc. In collaboration with specialists in the appropriate clinical domain, Pacific Pharma Group is prepared to discuss your target population.
