Speaker
Jay JH Park, PhD, MSc
Assistant Professor
Department of Health Research Methods, Evidence and Impact
Faculty of Health Sciences
McMaster University
Keywords
Platform Trial, Study Design, Pragmatic Clinical Trials
Key Points
- Adaptive trial design is an overarching terminology for trials that use accumulating data in a formal way. In this design, we come up with how we are going to look at the data, how we plan to react to the data, with one or more rounds of internal evaluations or interim reviews where we make the adaptations to the trial design, if the data says we should. The most common types of adaptive trial design are sequential design and response adaptive randomization.
- Platform trial design refers to trials that are designed with the flexibility to add new intervention. They use a series of documents called “master protocols” that outline trial plans and standard operating procedures for evaluation of multiple interventions. You can conduct platform trials using adaptive trial designs or fixed sample trial designs.
- To evaluate these trial methods, we did an economic evaluation to determine what are the costs and time requirements conducting a single platform trial versus multiple independent trials? We administered a survey to international experts with publication record on platform trials and master protocols using purposive sampling. The response rate was low (10%). Participants were asked how long it takes and cost for trail set up, conduct, and analysis for a two-arm multi-trial and in addition for a platform trial how long it takes what it costs to add a new intervention.
- The main outputs were the set-up cost and time, comparing the single study set-up and the total set-up cost and time across different trials and scenarios, and the total cost and time (set-up, conduct, and analysis). We compared a single platform trial to a single 2-arm trial and found it takes considerably less time and cost in setting up a single trial for conventional trials. The findings were similar for cost. There was not much difference in cost between a platform trial and multi-arm trial; setting up a single platform does appear to save money. The single platform trial requires less time measured by total persons.
- The key takeaway from the simulation is that the platform trial has larger set-up requirements, but it can save money and time in the long run. The platform trial model is not easy but not impossible; as we saw during the COVID-19 pandemic, it can be an effective way to discover important therapies and research in a fast and effective manner.
Discussion Themes
-Did the total cost include both coordinating center costs and site-level costs? We did not get into the site-level costs.
-These platform trials are almost a public good so that there is a sustainable model for the infrastructure so it continues to grow and evolve and no one bears the full cost. Does academic environment create some disincentives as well? Who becomes the PI is an important question because their institution gets the overhead. I’m not sure what the right answer is. There’s lots of red tape in the academic world. It’s an example of the kind of barriers there are to innovative approaches. How can we chip away at the barriers? At the individual level there is interest and commitment, and we see the value in this but there are barriers that do get in the way. Calling them out is step one.
Tags
#pctGR, @Collaboratory1