Reflections on 2020 and the future of rare disease research

January 2021

Our CEO Jeremy Edwards shares his reflections on 2020, as well as his hopes for the year ahead

2020 truly was a unique year for all of us. For a health tech company like ours dedicated to advancing rare disease research, 2020 proved challenging but also highlighted the vulnerabilities and resilience of people living with chronic and rare conditions. Lockdowns and measures like self-isolation and shielding drove a much-needed shift toward virtual communication, making it a temporary ‘new normal’ in how we socialize, buy and communicate with healthcare providers and clinicians. How will this impact the future of rare disease R&D? Our CEO Jeremy shares his reflections.


The year of unprecedented growth 

Despite the challenges our members and the pharma industry as a whole faced around COVID-19, 2020 was a year of growth for us as a company. Growth in terms of members joining our platform, continued pharma interest in orphan drug research and investor funding. This has allowed us to build a stronger executive team to help us keep the momentum going and expand the business. A key milestone for us in 2020 was launching Xperiome to help the pharma industry deliver new therapies faster and smarter, by providing a deeper understanding of how rare conditions affect people’s lives. 


Trends impacting our members and industry as a whole 

While COVID-19 disrupted people’s lives around the world, our members remained resilient – for them, R&D in rare disease would always be a top priority and they are still very much interested in helping pharma find better treatments. For the industry, the pandemic restrictions led to a renewed interest in decentralized, virtual and hybrid approaches to clinical trials, and speaking to our members, they are open to these trial designs too. 

From a tech perspective, last year saw more and more people become accustomed to engaging with HCPs and clinical researchers in multiple ways - from face-to-face interactions to virtual. Coupled with the shift towards decentralized trials, we expect to see even more innovations in telehealth and virtual communication in the coming year. The data people share about their health will increase as these tools advance, and for rare disease, this will open the door to more insight into how rare diseases work and how they affect patients' lives. It will also provide us with opportunities to use machine learning and AI to find even more trends and patterns in rare disease. We’re already seeing innovations in diagnostics for rare disease through the use of machine learning, and there’s huge potential for advancement in other areas too. It’s something we’re very excited to be a part of. 


Future trends we anticipate 

2020 saw a huge multi-generational shift towards digital communication, to the point where it’s become almost normal to set up a Zoom call to catch up with family and friends - and in fact, I’m sure a lot of you had your end-of-year team celebrations virtually as well. As people of all ages become more and more comfortable with virtual communication, we expect to see the pharma industry adopting tech in various aspects of the healthcare ecosystem - from digital communication apps to talk to HCPs with, all the way to wearables used to collect data for clinical research. This change in digital behavior has also revitalized conversations around decentralized trials - and so we foresee more trials designed with these approaches in mind, as well as solutions that help with implementation. 

We also expect to see more research programs that involve patients earlier on in the orphan drug development process, as patient-centricity becomes even more embedded into the industry mindset. Educating patients and giving them a stronger voice in clinical research will be a key area of focus for the industry – our data show there’s still lots to be done in rare disease to educate people about clinical trials, as well as adapting them to the needs of the rare patient. 


Hopes for year ahead 

We’d like to see more patient-centricity during the drug-development process - in rare disease, patients and carers sometimes know more about their disease than anyone else and so their voice is so important. The industry has already made great strides in this area and we’re excited to see even more progress being made here. 

Based on feedback from our members and hearing about what living with rare diseases is like, we would like to see more sponsors take the trial to the patient rather than the other way around. This will help with retention in trials, and speed up treatment development. 

We’re also looking forward to the coming year bringing some normality back to our members. 


By Jeremy Edwards, CEO


You can learn more about Jeremy and the rest of our team here: https://xperiome.raremark.com/about