Home » Rare diseases » Anticipating a new era of rare disease innovation, driven by patients
Rare diseases Q3 2021

Anticipating a new era of rare disease innovation, driven by patients

iStock / Getty Images Plus

Craig Martin

CEO, Global Genes

The events of the past couple of years have forced us all to think and act differently in many aspects of our lives. For those living with rare diseases this has long been the norm. 


Since the start of the pandemic, we’ve come to appreciate the importance and urgency of innovation in facing critical health challenges. We’ve followed clinical trials and become educated in the underlying science in unprecedented ways. We’ve learned to recognise inequities in healthcare and have sought to become more inclusive.

Adapting during the pandemic 

Rare disease families have understood and struggled with these realities long before COVID-19. The pandemic has amplified challenges, with many patients experiencing constraints in access to critical medical procedures, trials, testing or treatments, as well as severe financial, mental health and wellness challenges. 

Yet rare disease communities, used to responding in real-time to crises, have adapted with resiliency and nimbleness, working together to address issues and help those struggling or left behind. 

The global crisis has also generated new opportunities and ways of connecting with potential positive implications for rare disease communities. 

Examples of pandemic-driven progress include broadened access to telemedicine, virtual and adaptive trials, an increased emphasis on comprehensive testing, accelerated development of treatments and more equitable, global access to critical medicines.

The global crisis has also generated new opportunities and ways of connecting with potential positive implications for rare disease communities. 

A new era for the rare disease community 

“Despite the challenges around us, there’s good reason for optimism,” says Craig Martin, CEO of Global Genes. “We’re entering a new era in which rare disease communities – connected by shared purpose, technology and data – are playing a central role in driving impact across rare diseases, on a more equitable and global scale.” 

Global Genes has adjusted to anticipate changing dynamics and prepare patients, caregivers, and its Foundation Alliance of more than 800 rare disease groups, for what’s ahead. This includes launching a RARE Community Portal, which allows distinct communities to connect, convene and share information virtually around critical topics or initiatives.  

“As leaders, we have a responsibility to be informed, innovative and inclusive in how we work to enable progress across rare diseases,” states Martin. “We need tech to reach communities at scale, identify gaps, and find solutions. But we have to balance that with meaningful, personalised engagement, so we meet people where they are and ensure they’re equipped to take the best next steps forward.”

Next article