The gene therapy marketis predictedto experience rapid growth over the coming years. But with that growth will come many complexities that contract research organizations (CROs) must navigate to keep these unique clinical trials on track.

Despite just 39differentgene therapies recorded on the market in June 2023, according to GlobalData figures, more than 4,450 gene therapy drugs are currently in preclinical and clinical trials.

GlobalDatas State of the Pharmaceutical Industry 2024 report predicts that oncology will account for 44% of the cell and gene therapy (CGT) market by 2029. Trials are also increasing in other therapeutic areas, signalling the increase in research funding and product opportunity for diseasessuchas hemophilia, Alzheimers, primary immunodeficiencies, and even hereditary blindness.

The sponsor portfolio is definitelya lotmore diverse these days, notes Nan Shao, Executive Vice President of Global Operations at Veristat. They have greater confidence in cell and gene therapy. And because of that confidence, a lot more funding gets allocated even in the larger companies now investing in developing CGT.

Despite scientific and technological advances, the cost of gene therapy trials remains a significant hurdle, and reducing expenses is a considerable challengegiventhe inherent complications of such trials. That means CROs must find a middle ground for optimizing results within budget constraints.

With a limited number of patients available for trials of rare disease treatments, ensuring accurate data collection and recordkeeping is vital. All the more reason why executing your clinical trials with the right data management platform iskey for achieving accurate data collection, significant savings on trial costs, and a return on investment for pharma and biotech companies, while also ensuring more patients can access the transformative treatments coming from gene therapy trials.

Many of the genetic disorders thatcan be treatedwith gene therapy areextremely rare with some affecting just one person in a million.Gene therapy trials are limited by the number of patients available with certain conditions.As a result, it is vital tomake full use ofany data gathered.

CGT trials require scrupulous management of patients, sites, and sponsors to deliver successful outcomes.In the modern pharma industry,electronic data capture (EDC) has emerged as the best way to reduce inefficiencies, streamline data capture, and optimize a trials results.

Data is of the most critical value for success, and that dependency on data is only going to increasemore and more, notes Shao. However, in a CGT trial, we identified that it is hard for the typical EDCto be ableto capture all the data and adapt to the changes you need very quickly.

Mismanagement of patient data can trigger trial integrity risk or minimally cause inefficiencies in data collection, and place extra burden on sites and CROs to resolve discrepancies.Withgene therapy trial costs already running high, CROs must do everything they can to eliminate these risks and inefficiencies to ensure trials stay compliant, on schedule, and within budget.

Costing is a critical element for us as a CRO. Because many clients that reach out to us have the budget planned upfront, says Shao. These costs are critical to their decision to outsource to another provider, as they need to know we can keep their studies on time and on budget.

Moreover, because CGT trials often recruit froma very smallpatient pool, patient dropouts can severely disrupt a trial and add further costly delays. For cell and gene therapies, the deployment of an effective ePRO solution is critical for helping to foster patient engagement in trials, solicit patient input conscientiously, and ensure that patients understand everything required of them.

I worked on a Phase I cell therapy trial when I was a statistician myself, and it was very different from a typical Phase I study, explains Shao. There were a lot of unexpected challenges along the way regarding data and patients. You dont know how patientsare going toreact to some of these investigational therapies. So, you need tohave the abilityto quickly adjust your database as the study goes and typical EDC setups arent always able to accommodate this.

For their CGT trials, Shao and her team at Veristat have been using the cloud-based EDC platform from Zelta by Merative. The Zelta platform is uniquely flexible and scalable, allowing users to choose the modules they want for their study, making it compatible with virtually all trial types, regardless of complexity or size.

At its core, a trialsmainpurpose is to produce reliable data to understand the efficacy and safety of new treatments. Accurately recording a patients experience is vital. Yet an overcomplicated, hard-to-use EDC platform can also result in data anomalies due to a lack of user understanding, misinterpretations, or data that are missing entirely. The Zelta platform manages highly complex processes without compromising usability, thanks to features like flexible page layouts and automated queries, enabling CGT trial operators to get the most out of their data.

Shao estimates that Veristat has reduced clinical database costs by approximately 30% incertaintrials, resultinginhighlysignificant savings.Compared to other EDC solutions,Zelta has also helped Veristat reduce the database build duration oftheir complex CGT trialsfrom as long as 10 weeks to eight or less.

Being able to shave a couple of weeks off a trials schedule is huge for us, adds Shao. We save on costs, and it also leads to the quicker launch of a product, which is highly meaningful for those affected by diseases.

Alongside reducing the timeframe for launching life-saving therapiesto the market, Veristat found that the Zelta platform which offers ePRO integrated with EDC reduces the burden of technology integrations, both making managing patient data easier and helping patients engage better with trial processes.

Zelta EDC makes transferring patients from one site to another smoother. In long-term studies, patients regularly move to a different place, requiring their data to be easily transferred. Zelta EDC simplifies this process, thereby enhancing patient engagement.

Additional integrated modules, such as ePRO and eCOA forms, help improve a patientsunderstanding of their own conditions and treatments, while also reducing a CROs workload by automatically transporting inputted data to case report forms (CRFs). Scheduled notifications offered by the ePRO remind patients to input data at therighttimes and ensure that databases remain accurate.

For rare disease studies, you dont have a lot of patients you cant lose them, says Shao. Zeltas translation functionalities areespecially importantfor CGT trials, as patients aredistributed across the globeand speak different languages. This feature has absolutely made comprehension for patients far simpler. The Zelta platform is easy to use and has made the trial experience less stressful for patients, which is also highly important.

Zeltas high performance and the willingness of their team to support customers so that the platform can adapt to any trials needs are furtherkeybenefits from a CRO perspective.

In this industry, you have to be able to scale up and meet the different complexities of trials, notes Shao. Through this partnership with Zelta and with the familiarity of many years of work, thechannels of communication between us are streamlined. We can provide feedback and advance the Zelta product so it can better support our work. The Zelta team are very efficient at addressing questions, and that willingness to collaborate is super important to us as we are always under high pressure to deliver.

Adaptable, scalable, and intuitive for patients, Zelta can help CROs manage data demands for even the most complex cell and gene therapy trials.

To learn more about how Zelta by Merative can support CROs in tackling the biggest challenges facing their clinical trials, download the specially commissioned report below.

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Gene therapy: How the CRO of Veristat cut trial database costs by 30% - Clinical Trials Arena

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