Two Key Questions About Clinical Trials in 2023

One major stumbling block has been the lack of clarity in the regulations regarding principal investigator (PI) oversight of decentralized components. In the clinical sense, a PI is the person responsible for overseeing a clinical trial, research grant, or other sponsored research project.

Current regulations state that the PI is responsible for the conduct of the study at the site. However, as the definition of “site” is changing under DCTs, we are starting to see holes in the guidance. For example, home health agencies are often contracted to the sponsor to help support a site, but current regulations hold that the PI at the site is responsible for the conduct and training of staff which tends to result in a stalemate between PI and sponsor on who goes where on the Delegation of Authority (DOA) and the FDA’s Form 1572.

The 1572 is one of the many important regulatory documents submitted to the FDA in connection with clinical trials. Many common mistakes are made when filling out and maintaining the 1572 form, so the hope is that establishing a shared understanding of responsibilities will be useful to new sites, clinical research coordinators, clinical research associates, and other clinical research professionals.

The biggest challenge has been to avoid adding “another” system into an already complex clinical trial landscape and the burden that this adds to both patients and sites—especially as the goal of decentralization is to open access to patients and remove burden rather than adding in new constraints. DCT adoption has typically been study by study and capability by capability, which has meant a great deal of customization, data integration, and manual effort for each study deployment. Time-constrained site staff are often left with a pile of unfamiliar technology tools to sort through, learn, integrate, and manage.

To solve these issues, the industry will need to increase standardization, re-usability, and templatization as decentralized trials continue to grow in 2023. The next evolution of clinical trials will involve self-service tools that enable sponsors and sites to deploy and operate global DCTs on a common platform using standardized processes. Decentralized platforms will need to play a unifying role across the complex technology landscape across clinical trials so that instead of single-point solution deployments, DCT technologies will enable more elements of the trials, increasing flexibility and optionality for sponsors, sites, and patients.

Most clinical trial participants aren’t motivated by research. They’re patients seeking treatment. Yet, we emphasize research and statistical considerations, relegating participants to research subjects in the hope it provides the best evidence. We must adopt a more nuanced view and more carefully consider the burden involved in a trial. Better trial design tools that leverage lessons learned from historic trial design and real-world data (RWD) can help cut out unnecessary data collection. New technologies such as EHR2EDC can reduce the burden of the data we do need to collect. We must more carefully examine randomization and utilize external control arms in more meaningful ways to reduce our dependence on giving patients control treatments unlikely to work well.

Using RWD and real-world evidence (RWE) in clinical research is becoming more critical for pharma to reevaluate current ways of working. While we see the industry increasingly adopting patient insights from RWE to support clinical trial design and accelerate recruitment and regulatory submission, a lot is still to be done. We see the potential to optimize more than half of capital investment in traditional clinical programs with the use of RWE while also accelerating the program timelines and meeting evidence needs more systematically.

Digital-based clinical trial recruitment and retention methods must become more sophisticated and align with best practices for digital user experiences. Too often, the industry has relied on printed materials, simple banner ads, and social media posts to drive trial awareness. First and foremost, patients are consumers. The person eligible for a clinical trial is the same individual who shops online for groceries or books a trip on their phone. They expect sophisticated yet simple-to-navigate digital experiences and content tailored to their needs.

The industry needs to adopt the same standards that consumer brands have established by creating seamless user experiences from awareness to enrollment. By leveraging qualitative and quantitative data to develop user personas, we can develop communications around trial recruitment tailored to people’s concerns and questions. When we fully understand our patients’ needs and communications profiles, we can reach them at various digital touch points along the journey from diagnosis to enrollment. Lastly, the content must be at-the-ready and easily accessible on their go-to channels. By adopting some of the best practices from consumer brand marketing, the industry can improve the effectiveness and efficiency of trial recruitment.

Many different barriers make it difficult to participate (and remain) in trials. We see financial barriers (especially in this age of hyper-inflation), and personal logistical barriers (transportation and child care as examples) as two of the most common. These are also two of the most obvious, among a myriad of more complex issues, that work to prevent proportionate representation in research across racial and ethnic minorities. Fortunately, the sponsor community is receptive to adopting strategies and solutions that make studies more accessible. The investment case is clear—making trials more accessible will improve recruitment and enrollment rates and will reduce dropout rates, thereby reducing costs and ensuring the quality of the clinical data set.

For example, most patient reimbursement today is based on prepaid cards, offering no patient choice, while so many more efficient solutions are available including direct to debit, Venmo, etc. Prepaid cards also charge inactivity fees, ATM fees, etc., which deprive patients of what they’ve been given. Furthermore, patients are often charged out of pocket for travel costs (a barrier for lower income participants) rather than directly charged to the site or the sponsor. These are just a few examples of how the industry can be more patient centric.