How COVID-19 Transformed Our Approach to Rare Disease Clinical Research

In our previous article, we discussed the rare disease pipeline and the complexities in conducting rare disease Inside a CRO. However, the impact of the pandemic did not stop there. COVID-19 sparked a holistic change in the way we approach rare studies.

The Role of Decentralized Strategies During COVID-19

Medicine is evolving from a one-size-fits-all approach to a more targeted one, with timely administration of medical treatments tailored to the individual becoming the increasing norm—particularly for rare indications. The COVID-19 pandemic revealed the need for a new approach to rare disease research. Decentralized clinical trial (DCT) strategies enabled many studies to continue despite COVID-19 restrictions, and they set the focus on facilitating patients’ participation and retention in clinical trials. 

Adaptations During the COVID-19 Pandemic

As regulators, sponsors, CROs, and researchers looked for ways to keep trials going, DCT strategies were implemented with incredible speed. Remote monitoring, which was already broadly implemented in some countries, became more widely adopted. Some previously available electronic tools and systems (e.g., eConsent) were quickly revamped and widely implemented. Other strategies, like home delivery of investigational products, were leveraged to facilitate patient retention and compliance when hospital visits were not possible. Telemedicine and concierge services also played a role in helping to reduce the burden on families and caregivers.

Decentralized strategies can play a role across the patient journey

Electronic consent, remote screening, telemedicine, and traveling nurses are straightforward strategies that can help drive retention. Additionally, researchers can look for sites that are local for eligible patients and initiate meaningful conversations regarding study participation. Visits may then be conducted at home, at the local site, or in another local medical facility. Follow-up visits may be performed by nurses at patients’ homes.  

Ensuring Safety in Rare Disease Clinical Research

Early phase clinical research is often a balancing act between efficacy and toxicity. However, rare diseases present unique challenges as therapies are often life-long and administered to newborns or children. Participants in these studies play an active role which can surface several potential barriers. 

• Data protection laws may post limitations on access to patient data 
• Caregivers face a heavy physical and emotional burdens  
• Access to clinical trial information is restrictive due to complex terminologies and English-only information 

A new approach to rare disease research

DCT strategies can be a viable option for accelerating drug development while maintaining patients’ quality of life. While the increased acceptance and implementation of decentralized strategies is a silver lining of the pandemic, there are still opportunities for further improvements.