In our previous article, we discussed the rare disease pipeline and the complexities in conducting rare disease research. However, the impact of the pandemic did not stop there. COVID-19 sparked a holistic change in the way we approach rare studies.
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.
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.
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.
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.
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.
Next, we will explore what rare disease research looks like in a post-pandemic world through a case study with our VP of Operational Strategy, Esther Mahillo.
Esther Mahillo, PhD, MBA, is Vice President of Operational Strategy and Feasibility at Precision for Medicine in Madrid, Spain. She has more than 25 years of experience in the clinical research arena (22 of them in global oncology trials), and is a master’s program professor at three different universities. In 2015, she was awarded the first prize of the Spanish Society of Medical Oncology contest for her work on the promotion of clinical research.
Precision for Medicine is part of the Precision Medicine Group, an integrated team of experts that extends Precision for Medicine’s therapeutic development capabilities beyond approval and into launch strategies, marketing communication, and payer insights. As one company, the Precision Medicine Group helps pharmaceutical and life-sciences clients conquer product development and commercialization challenges in a rapidly evolving environment.
