Improving Study Activation Time for Gene Therapy Research

Gene therapies and genetically engineered vaccines are growing exponentially and will continue to become more popular. Per a report from the American Society of Cell and Gene Therapy (ASGCT), gene therapy research is an increasingly important part of an institution’s research portfolio, with over 3500 therapies in preclinical or clinical. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027. As human gene therapies enter the pipeline at an exponentially growing pace, research sites need to consider how prepared they are and take into account any additional review requirements of these studies in their activation programs.

This was just one of many questions we explore in our comprehensive survey of research sites and their internal activation practices. This blog dives deeper into the results of our recent site survey, exploring how research sites are focusing on their institutional biosafety committee (IBC) programs as part of overall study activation time.

Understanding When IBCs Are Needed

First, it’s important to understand what an IBC is and when institutions need their approval. When treatments involve recombinant or synthetic nucleic acids, including messenger RNA (mRNA) or viral vector vaccines, additional oversight by an IBC is necessary. Some of these treatments potentially make permanent changes to the humans they are introduced into. Unlike typical drug treatments, which are metabolized by the body, in some cases, gene therapy treatments make permanent changes to a human’s genetic profile. Additional precautions and IBC oversight are necessary when handling and administering these novel research compounds. The US National Institutes of Health Office of Science Policy (NIH OSP) sets the guidelines for research involving recombinant or synthetic nucleic acid molecules.

Activation Insights

Survey results indicated many larger research sites reported they have an IBC in place to review research subject to the NIH OSP Guidelines. Not surprisingly, these organizations were primarily large academic medical centers with sizable research portfolios. However, simply having an IBC is much different than actively managing the IBC review process as part of an overall strategy to streamline trial activation.

Of the respondents who have an IBC at their organization, 54% indicated the review process for human trials exceeds 30 days. Additionally, more than a third of the institutions with an IBC indicated their institutional review board (IRB) and IBC review process for human trials are not integrated nor run in parallel. For these institutions, human gene therapy trials are likely taking significantly longer to activate than their more traditional drug study counterparts.

What Can You Do to Improve Activation for Gene Therapy Trials?

Know The Process

First and foremost, researchers need to map and understand overall study activation processes, including IBC and other oversight committee reviews beyond IRB. Setting performance targets allows committees to review a trial within the institution’s stated activation timeline objective. An IRB tends to get the most attention since all human trials need IRB approval. However, if an organization does not focus on the efficiency and expertise of the additional committees, like the IBC, it does not matter how quickly the IRB moves. The overall activation time is only as fast as the slowest committee, and that’s assuming the process allows for parallel review.

In the survey, many institutions indicated not having a specific measure for IBC performance. In total, 88% of respondents who have an IBC reported they have no turnaround time goal or key performance metrics for their IBC program. The result is not knowing if a gene therapy-related clinical trial can even be activated within the activation timeline typically given to a sponsor during site initiation.

Build Expertise on the IBC

For many institutional IBCs, the majority of research oversight is preclinical. That paradigm is changing as more therapies are moving out of the lab and into human clinical trials. If an IBC does not have the requisite expertise to apply the NIH OSP Guidelines to human clinical trials, it is generally advised to not try to do the review in-house. At best, an inexperienced in-house IBC review may result in a delay while the committee seeks additional expertise or due to unnecessary questions from the committee back to the investigators. At worst, an inexperienced IBC review may not apply the Guidelines appropriately and introduce regulatory risk for the institution. In either case, if the IBC does not have experience with human clinical trials, study activation time will likely be delayed.

Defer IBC Review to a Commercial Provider, While Keeping the Local Biosafety

As human gene transfer trials are increasingly reaching Phases II and III, industry sponsors are routinely selecting a central IBC and IRB provider to review all sites in a multicenter clinical trial. However, according to our survey, most institutions are not deferring their IBC reviews off to a commercial provider in the same way that most institutions defer oversight to the designated single/central IRB. Very similar to how IRB deferrals work, biosafety programs can defer the formal IBC committee review component (e.g., five members convening an IBC meeting, minutes, etc.) to a commercial IBC provider.

Sites should consider using commercial IBC services selected by the sponsor or potentially engage a provider for all human trials at the organization. Not only are commercial IBC services typically faster, but they also allow the biosafety officer to focus their attention on staff training, physical lab tracking, and other local issues, which are keenly important but can be separated from the formal process of conducting IBC review per the NIH OSP Guidelines.

Conclusion

Innovative treatments using gene therapy are growing, and public perception of genetically engineered therapeutics is changing due to the mRNA COVID-19 vaccines. Ensuring an organization has an efficient activation process through an IBC so participants can take advantage of these new, cutting-edge investigational products.

Read more insights on study startup in our survey report

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