Skip to main content

Table 1 Phases/Stages and Methods of Pharmaceutical and Surgical Innovation1, 5, 7

From: Challenges and potential solutions to the evaluation, monitoring, and regulation of surgical innovations

Phase/Stage Pharmaceutical Innovation Surgical Innovation
0 ▪ Preclinical animal studies used to characterize the pharmacology of the agent as well as its safety across a dosage range
▪ Used to support creation of an IND application, which is submitted to the FDA or Health Canada
▪ Initial prehuman (simulator or animal) work and development, which may be reported as preclinical studies
1 ▪ Phase I RCTs conducted within a small group of health volunteers to assess the safety, tolerability, accepted dosage range, and pharmacokinetics of the IND after one or more doses ▪ Technical skills development and/or acquistion and proof of both concept and safety, which may be described in structured case reports
2 ▪ Phase II RCTs that administer the drug over its anticipated target dosage range to patients with the target health condition to assess feasibility and/or the infleunce of the drug on a biomarker or biomarkers  
2a   ▪ Although the technical details of the surgical procedure have not been completey refined, a few surgical practice leaders have adopted the technique and are using it on a small group of patients outside the index hospital or center where it was originally developed
▪ May be described in prospective development studies
2b   ▪ Many of the technical details have been nearly perfected, and the surgeons who adopted the procedure in stage 2a start to broaden patient accrual and procedural indications
▪ May be described in cohort studies, diagnostic performance studies, and RCTs
3 ▪ Phase III RCTs designed to assess the efficacy or effectiveness of the medication in patients with the target health condition on a series of pre-defined efficacy and safety endpoints ▪ The innovative procedure is now becoming part of many surgeon’s practices, and only a select few will not have adopted it
▪ May be evaluated in RCTs or other studies where clinical equipoise exists
4 ▪ Post-marketing studies conducted to monitor for rare adverse effects or to assess the usefulness of the agent among other patient populations or when administered in different dosage forms ▪ Long-term monitoring studies whose aim is to assess for unexpected rare outcomes and restrict or expand indications for the procedure or clarify additional important technical details
  1. Where FDA indicates U.S. Food and Drug Administration; IND Investigational New Drug, and RCT Randomized controlled trial