Contributed Commentary by Erin Leckrone and Chris McClain, Be The Match BioTherapies

December 8, 2023 | Clinical trial sponsors must strike a balance between budget, speed, and data quality when planning for cell therapy clinical trial costs. These costs can be difficult to predict and budget overruns often happen. However, cell therapy sponsors can better plan for clinical trial costs and lower their risk of exceeding their budgets by taking specific steps early on. 

Create Your Protocol Before You Set Your Budget 

Cell therapy clinical trial sponsors often determine a budget target prior to protocol creation. This can lead to budget challenges from the start because theoretical expectations may not match reality. 

For example, the protocol may change based on input from the investigators who are assessing the design against real-world implementation. Required changes could result in a re-work of endpoints, additional testing or laboratory costs, or an increased patient visit schedule. Collectively these changes can increase vendor costs and the actual reimbursement costs to collect data for each patient. In addition, nonstandard-of-care costs at a site may be higher than assumed so reimbursement costs will rise.  

Designing your protocol and soliciting investigator review before setting your budget will help you more accurately predict your costs. 

In addition, scrutinize your clinical trial design, invest in statistical modeling, and resist the temptation to add elements to your protocol that may not be strictly necessary to achieve your goals. Every patient enrolled and additional data point collected for a clinical trial impacts the budget. Creating a larger sample size or collecting more data or samples than you need for your regulatory submission drives up the cost of your clinical trial. 

Consult With Physicians On Protocol Design 

Physicians experienced in treating the clinical trial’s target patient population are an excellent resource to vet a draft protocol design. Soliciting input from physicians you intend to partner with on your trial—either from a site perspective or at a CRO—is a valuable way to engage key stakeholders early. Additionally, ensuring your trial is well-designed from the beginning ensures the avoidance of costly protocol amendments which can result in increased costs for both vendors and labor. 

A CRO that has physicians on staff who have been investigators on cell therapy clinical trials for blood cancers and blood disorders provide valuable knowledge, too. They can help clinical trial sponsors understand what is standard of care versus investigational per the protocol and advise on how a trial may be received by patients.  

This insight aids a sponsor in designing a protocol that aligns most directly with standard-of-care costs, which means there are likely fewer data points the site will request reimbursement for to collect. 

Choose Centers Based On The Actual Patient Population 

Patient enrollment is one of the main clinical trial cost drivers. Balancing the number of sites to open against the ideal enrollment rate is crucial to maintain budget targets. If clinical trial enrollment is slow, you may need to open additional study sites and incur additional costs. That makes site selection extremely important. 

While a site’s reputation is a factor, you must also consider the actual patient population. Site-reported patient population data may not be fully accurate. However, sponsors of clinical trials treating blood cancers and blood disorders can access a site’s actual patient population data. Most transplant centers in the United States report outcomes data to a central database for patients who receive allogeneic or autologous hematopoietic stem cell transplant. This data includes the indication for which a patient is treated and numerous other factors that could contribute to study design and successful study execution. 

With support from bioinformatics experts, a clinical trial sponsor could use this information to model where the patient population likely lives in the US, which allows the sponsor to identify centers in those areas with the known patient population. 

This kind of visibility into a center’s patient population allows you to select study sites that serve patients who are more likely meet your trial criteria. 

Do Your Homework On Site Costs 

Before you approach a center and begin the contracting process, you need to understand reimbursement costs for a specific center. Clinical trial software can model these numbers based on inputs such as the patient population, therapy type, geographic region and/or state, and procedures associated with the trial. 

This modeling can give you a precise range of costs for that specific center. It is important for you to understand these costs when you enter contract negotiations. If your reimbursement does not match the center’s costs, the budgeting and contracting process can stall and delay site startup by months. 

Recognize How Manufacturing And Logistics Challenges Can Impact Your Clinical Trial 

When designing a cell therapy clinical trial, it is important to not only consider the cost of conducting the clinical trial, but also the logistics complexities. Cell therapy logistics are complicated and getting the product manufactured and to the patient on time is crucial. 

If a cell therapy sponsor consistently fails to deliver on time, clinical study sites will be hesitant to enroll patients, which will extend recruitment timelines and, therefore, stress your budget. To mitigate risk, ask vendors for specific examples of how they have supported cell therapy clinical trials and work with those that have demonstrated experience in cell therapy logistics. 

While cell therapy clinical trials can experience budget challenges and rising costs, careful upfront planning, consultation with physician experts, and modeling both patient populations and site costs can all help you keep your trial on time and on budget. 

Erin Leckrone, MBA, is the vice president of Clinical Trials for NMDP/Be The Match. She manages the activities of CIBMTR CRO Services and its portfolio of internally and externally sponsored clinical studies. Prior to joining NMDP/Be The Match, Erin spent almost 14 years at Boston Scientific leading Clinical Operations and Strategy and served eight years as a medic in the Army National Guard. She holds a Bachelor of Arts in English and Biology from the University of Northwestern in St. Paul, Minn., as well as an MFA from Emerson College and an MBA from the University of Massachusetts Amherst. She can be reached at eleckron@nmdp.org. 

Chris McClain, MBA, is the senior vice president of Sales and Business Development for Be The Match BioTherapies. His team engages with cell therapy companies developing innovative, life-saving therapies that can leverage the organization’s cell sourcing, cord blood unit sourcing, CRO, and bioinformatics capabilities. Previously, Chris was co-Founder of Nora Therapeutics, Inc., a venture-backed biopharmaceutical company developing novel therapies in areas of significant unmet medical need in reproductive health. He can be reached at cmcclai3@nmdp.org.