Here, we will briefly discuss procedures designed to evaluate the safety and efficacy of your drug product. Your treatment plan should outline the actions and guidelines that investigators and healthcare professionals will follow to administer the treatment to study participants. A few considerations for the treatment plan include the following. (Please refer to Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry (fda.gov) for more detailed information.)
Staggering administration
- Most first-in-human trials of gene therapies include staggered treatment to limit the number of subjects who might be exposed to an unanticipated safety risk
- With staggered treatment, there is a specified follow-up interval between administration of the product to a subject, or small group of subjects, and administration to the next subject or group of subjects
Cohort size
- For trials that enroll sequential cohorts with dose-escalation between cohorts, the choice of cohort size should consider the amount of risk that is acceptable in the study population
- For gene therapies, manufacturing capacity is often limited, which might place a practical limit on cohort size, particularly early in clinical development
Operator training
- For product delivery that involves a complex administration procedure or a device requiring special training, such as subretinal injection, the skill of the individual administering the product can impact the product’s safety and efficacy
- When individual skill in administering a product may affect its safety or effectiveness, the trial should specify minimum requirements for the operator’s training, experience, or level of proficiency