From The Editor | September 18, 2021

Can Smarter Study Design Relieve The Recruitment Challenge?

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader
Follow Me On Twitter @EdClinical

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Recruitment has always been a challenge for companies conducting clinical trials. Sponsor companies seeking new ways to engage and recruit patients have seen varied success. But could that challenge be mitigated or eliminated with smarter study design? A recent Town Hall by Halloran Consulting Group in partnership with Exploristics attempted to answer that question. The Town Hall, hosted by Meaghan Powers of Halloran Consulting Group, featured Aiden Fynn of Exploristics, Akil Jackson of BergenBio, and Alan Menius of Spencer Health Solutions. The discussion produced some interesting questions and takeaways regarding clinical trial simulations.

The importance of simulations cannot be ignored. There are problems that exist in clinical trials that are not going to go away. The answer to those problems cannot be spending more money or collecting more data. That, unfortunately, will put more burden on patients at a time when drug companies are trying to make trials more patient centric. The solution could be for companies to have a more informed and holistic approach to study design. Simulations could help provide them with that approach.  

What Are Clinical Trial Simulations?

Clinical trial simulations use mathematical models to describe a system or process and is then used to test and evaluate the process under different sets of conditions. The performance of the process can then be evaluated. A simulation can provide insight on the drivers of success and failure, help quantify risks, and allow for the development of mitigation strategies. 

Several factors have driven the evolution of trial simulation in recent years. The group notes the primary drivers have been interest from regulators, increasing access to data needed to develop realistic simulations, increased computing power, the increased acceptance of cloud computing in pharma, and a new breed of statisticians with multidisciplinary skills who are well-suited to build simulation capabilities.   

Decentralized trials have been a hot topic in the past year with the pandemic forcing many sponsor companies to allow patients to participate in trials remotely. One concern that has been voiced over this approach is the quality of the data being gathered. With data being collected in patient homes rather than clinics, sponsor companies need to know if the data being collected is complete and of good quality. Simulations allow companies to look at these issues, understand what to expect, and know how the data collected would impact the overall study.

Adoption And Challenges

In order to implement a clinical trial simulation, the right expertise is required. The speakers noted the platform is designed for statisticians or someone who is very knowledgeable of statistics. Over time, as an organization develops increased capabilities, it can develop a hybrid model. 

There will certainly be barriers to the adoption of simulations. First, the group notes employees like the status quo. They like doing things a certain way and will be resistant to doing things differently. That is a situation companies will need to overcome. As one panelist noted, the old way does not work anymore. Change is necessary and drug developers will need to invest in the time and effort necessary to put simulations in place. Over time, the investment will lead to cost savings, a decreased burden on patients, and a greater level of confidence going into a study. Hopefully the simulations will also lead to a decreased number of trial failures. 

There is work that will be required to get a simulation tool set up. To be successful, the simulation tool will require early engagement with statisticians to develop the protocol and trial strategy. All available knowledge will be collected at the outset to build a large, virtual, synthetic population that captures risk factors, outcomes, and “what-if” scenarios. 

Regulatory Concerns

With any new technology adoption, there are concerns over whether the FDA and other regulators will be accepting of the changes. The same is true of simulation-based design methods. 

The panel noted the FDA is encouraging companies to use model-based drug development. Simulation is an important element of that. Simulations can be conducted prior to companies even engaging with the FDA. Discussions with the FDA can be viewed as a negotiation, and it is better for those negotiations to be quantified rather than opinionated. Simulations will give companies the data they need for those discussions to be more tangible. 

Of course, the first step to implementing any new tool is getting approval from c-suite executives. The panel had some advice on how to do that as well. 

When presenting the tool to executives, focus on the time savings and the ability to quantify risk. Then turn the discussion to the expected impact of the tool. Demonstrate how a clinical trial simulation will help build a smarter study, generate better data, and minimize the risk of a failed study that will need to be repeated. It can also assist with patient recruitment, as it can better quantify and communicate potential benefits to patients. 

For more information on this or other Town Halls, contact Maria Coakley, events manager, Halloran Consulting Group, at mcoakley@hallorancg.com.