Tips and Tools for Biotherapy Trials

October 19, 2021

In a strong final day of the 2021 AABB Virtual Annual Meeting, attendees in the session titled “Tips and Tools to Overcome Challenges and Negotiate Partnerships for Biotherapy Trials” learned useful information on overcoming the challenges in, and negotiating partnerships for, biotherapy trials. 

As more and more biotherapy research is being conducted and more and more biotherapy products are being developed, there is an increasing level of activity in cell collection, typically through apheresis of healthy donors or patients. 

Many blood centers are partnering with study sponsors to meet the demand for source material. However, this is sometimes unfamiliar territory for blood centers that are expanding their services into cell collection for biotherapies. Jennifer Chain, PhD, from the Oklahoma Blood Institute in Oklahoma City, welcomed attendees to the session and outlined the session goals as threefold: identify the challenges encountered when participating in a biotherapy trial, provide advice on conducting negotiations with study sponsors, and offer examples of solutions from experienced colleagues.  

Why Are These Trials So Challenging?
Federico Rodriguez Quezada, SBB, MLSi(ASCP)CM, from UF Health Shands Cancer Hospital in Gainesville, Fla., provided a wealth of practical information. After briefly discussing institutional review and approval of research trials, he highlighted unique aspects of apheresis collections that can prove to be challenging, including potential risks of mobilizing drugs, need for good venous access, loss of platelets during the procedure, donor discomfort and the need for staff to monitor for adverse reactions during lengthy collection procedures.  

Rodriguez Quezada also explored the major initial phases of research studies and detailed several of the associated challenges, including the following:
• Invitation from study sponsor. If lacking in detail, it should prompt many questions by the collection team.
• Feasibility meeting. Address personnel, schedules, workloads, costs, support from staff other than collections.
• Stakeholder meeting. Attendees might include the sponsor’s representative(s), the principal investigator, collection staff and institutional review board members. 
• Approval process. This involves not only the protocol itself, but also the budget and input from the stakeholders.
• Establishing eligibility and exclusion criteria. This would include likelihood of collecting enough cells.
• Development of informed consent. This sometime requires two types of translations—one from English to a different language and one from medical terminology into wording a lay person can understand.

Noting how easy it is to underestimate costs, Rodriguez Quezada listed items not directly related to the collection procedure that may be overlooked (disposable supplies, lab testing, shipping supplies, labor costs, administrative overhead). He also reviewed what he considered to be essential steps in preparing for the trial, emphasizing the importance of a “dry run.” His remaining comments focused on tips for negotiating with the study sponsor, including the following:
• Clarify everything through repeated cycles of questions and answers. 
• Don’t underestimate the hours of labor in preparing for the study, conducting the collections, and any follow-up reporting.
• Don’t accept the study if it isn’t possible to do.
• Define responsibilities.
• Avoid pressure from the sponsor and getting caught up in the excitement about being involved.
• Read the fine print of any agreement; better yet, share the document with your legal team.
• Remember that open and effective communication with the sponsor is of the utmost importance.

Preparing for the Next Study
Ronit Slotky, PhD, MSc, from Hackensack University Medical Center in Hackensack, N.J., continued with the emphasis on communication and collaboration. In fact, getting connected was the first step she listed in her advice on how to get organized for a biotherapy trial. Her list included the following pointers:
• Build relationships with stakeholders (pharmacy, finance, research personnel, others who are part of the facility’s process for trial participation).
• Get involved as early as possible, preferably at the feasibility meeting. Ask lots of questions.
• Create templates, standard operating procedures, forms, checklists, etc—these tools will make many tasks easier.
• Create a process that is as standardized as possible. This is key if your facility participates in several concurrent or overlapping trials.
• Create a budget that covers everything, which means
- Spell out the responsibilities, especially for regulatory compliance.
- Costs are usually more than anticipated.
- Include work performed preparing for the study, not just the procedures.
- Include expenses incurred after the trial itself (close-out tasks, storage, reporting). 
• Assign tasks, even if you need other facility departments or the study sponsor to handle some of the responsibilities.

Slotky also discussed examples of the level of detail required for preparing to conduct a biotherapy trial. What if the sponsor’s containers do not fit your freezer racks? Who is responsible for compliance with each service provided? Is there enough space and equipment for long-term storage? Will the sponsor conduct on-site training? She shared examples of forms, timelines, checklists, and other tools that can help make biotherapy trials easier to manage.

In summary, Slotky reiterated her key points, which were to connect and communicate the broader team of stakeholders, make sure everything is clear and understood, create a process and the tools for a smoother workflow, budget correctly, and collaborate with all the partners needed to make the study successful.