A randomized controlled trial (RCT) is usually held to be the ideal means of generating definitive evidence for the comparison of two or more medical therapies. However, there are several well-known issues with implementation of RCTs in certain settings. The population of patients and sites participating in the trial may not be representative of the full target population for which the therapy is intended; patients may not be in a state of equipoise and may instead have real preferences for treatment; slow enrolling RCTs may provide definitive answers for comparisons between treatments that are no longer meaningful by the end of the trial.

One interesting alternative to traditional RCTs involves the use of objective performance criteria (OPC). Recent developments in collaborative sharing of patient level data by industry competitors have facilitated the application of Bayesian methods in the creation of an adaptive OPC for drug eluting stents. Development of this OPC has been logistically challenging for industry, but offers the prospect of smaller trials and lower cost. Patients can benefit from access to new therapies faster than might happen with RCTs. Regulators on the other hand must ensure that such designs deliver on the promise of providing valid scientific evidence for determining whether there is reasonable assurance that the new therapy being examined is safe and effective.

This session will include an industry perspective on the data access and collaboration necessary in development, and a regulatory perspective on situations where an OPC would be discouraged or encouraged. Participants in the session will be encouraged to discuss the pros and cons of utilizing a one armed trial design for OPCs in particular, or the use of such designs in other settings more generally. Participants would be especially encouraged to debate the use of OPCs in settings beyond just drug eluting stents or bare metal stents.

Roundtable leaders: Ted Lystig (industry; confirmed); Greg Campbell (FDA; confirmed).

Potential discussion questions: What should be the minimal requirements needed in a therapy before use of an OPC would be acceptable? What types of regulatory claims can or cannot be made after successfully beating an OPC? What are examples of therapies without existing OPCs that would be appropriate for development and implementation of an OPC?