Decentralized clinical trials and the future of drug development

The U.S. Food and Drug Administration (FDA) recently released its first-ever guidance on the design and implementation of decentralized clinical trials (DCTs). These trials are noteworthy because they allow some or all clinical trial-related activities to occur at locations other than a traditional hospital or clinic. These in effect may help increase the number of patients participating in clinical trials who would not otherwise be able to take part.

With thoughtful implementation, DCTs can have positive and lasting implications on how medicines are developed to the benefit of patients by:

• Reducing the burden to patients, caregivers and medical providers involved in clinical trials;
• Expanding access to populations that have traditionally been underrepresented in studies; and
• Leading to new ways of studying diseases and fostering innovation.

The ways clinical trials have traditionally been conducted created unintentional barriers to broader patient enrollment. For example, in traditional clinical trials patients may need to travel long distances to a hospital or clinic in order to participate. Understandably, this discourages participation from those who must travel far distances, cannot afford to take time off work or otherwise do not have feasible access to the trial site.

Fortunately, broader acceptance of telemedicine and digital health tools in recent years, due in part to the FDA’s user fee programs and advancements implemented during the COVID-19 pandemic, has spurred adoption of DCTs which can allow for trial-related activities to occur remotely –– including patient enrollment, monitoring and data collection. 

Recently, PhRMA submitted comments to the FDA highlighting the following key considerations that should be made around DCTs:

• DCTs should be held to the same standards as traditional site-based trials. There are instances in the FDA’s draft guidance where DCTs appear to be held to a different standard than traditional clinical trials. Regulatory requirements should be the same for DCTs and traditional site-based clinical trials.
  
  
• New opportunities around data collection and usage of digital health technologies. Digital health technologies allow for remote data collection, potentially offering new ways to study a disease. Such data collection is not inherently less precise or more variable than site-based data collection.
  
  
• Clear direction on the documentation and training needed for different trial personnel involved in a DCT. Since DCT activities can take place at locations other than a traditional hospital or clinic, trial personnel such as local healthcare providers or mobile nurses, who typically do not take part in traditional clinical trials, may be involved in a DCT. In any guidance on DCTs’ design and implementation, there is a need to ensure oversight while providing sufficient flexibility to allow decentralized trials to be effective. 

We look forward to continued engagement with FDA and other stakeholders as the Agency works to expand clinical trial access to more diverse patient populations through the advancement of DCTs.

PhRMA’s full comments to the FDA can be found here.