Defining Standard of Care

EP: 1.Burden of C. Difficile and Novel Therapies

EP: 2.Objectives of Cost Effective Analysis

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EP: 3.Defining Standard of Care

EP: 4.Target Population In Analysis

EP: 5.Cost Inputs and Key Data from Analysis

EP: 6.Study Limitations and Relevance

Dr. Paul Feuerstadt: What’s important to understand with RBL [live JSLM; Rebyota] and any study that considers a live biotherapeutic product is that it’s an add on therapy. So when we talk about the comparator group, the so-called standard of care, that’s a standard antimicrobial course — what the patient would have received previously without the add on therapy. … [The] control group is the standard of care for the antimicrobial and then the comparator group, the study group, is the group that has that antimicrobial plus a treatment like RBL. Within the pivotal phase 3 trial considering RBL, patients with one or more recurrences of C. diff all receive standard of care, had a washout period and then either received the placebo or RBL. Through a Bayesian analysis, the overall efficacy for RBL at eight weeks was 70.6% versus 57.5% in the placebo arm, achieving a statistically significant difference between the two which is why it was granted FDA approval. So with that foundational knowledge in mind, can you explain to us a little bit about what a Markov model is and how it applies to this study?

Dr. Michael Kobernick: [Yes,] Markov model is … a really complex statistical model that has been developed over the years … to apply statistics to clinical science and clinical evaluation. What it does is it takes potential clinical outcomes and it creates groups of them and then it creates a likelihood that one or the other will occur and the cost associated with that. … [In] C. diff, it really talks about the likelihood in cost of developing complications in groups that have, as you mentioned, the standard-of-care antibiotics in groups that have RBL as the add-on therapy. It generates cost associated with them that can then be interpreted to … [mean] we know this treatment is clinically effective but how much does it cost? Does it cost way too much to get a desired result? … There’s a lot to argue back and forth about the desired result and the cost or does it cost an appropriate amount? I think that’s the essence of the Markov model. It’s statistically valid. It’s an acceptable way, a very acceptable way to compare costs between treatment groups and it’s a very good adjunct, the clinical trial that you mentioned.

Transcript is AI-generated and edited for clarity and readability.