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Reflecting on 2020 and Finding Hope for the New Year

Leaders in the rare disease community respond to patient concerns and challenges in raising awareness amid a pandemic.

Collage of 17 participants in our year-end survey about HOPE

Editor’s note: We asked patients, leaders of rare disease patient organizations and CSL Behring employees to cast ahead to a more hopeful 2021. We’re sharing their responses in a series of articles. Today, we’re focusing on leaders in the rare disease community.

Leaders in the rare disease community say 2020 taught them valuable lessons that will carry forward in the coming year and help their organizations grow. Others found the pandemic’s silver lining to be the strength and resolve shown by patients and caregivers. Here’s what leaders in the rare disease community say gives them hope for the new year.

Monica Ferrie APARDO director 

Monica Ferrie, a Director of the Asia Pacific Alliance of Rare Disease Organisations (APARDO)

The rare disease community was already incredibly resilient. The COVID life has been an exaggeration of the norm – uncertainty, isolation, inequitable access to services and treatment, difficult access to clinical trials and research studies and more. I am inspired by the collaboration that has occurred in the general community, the acts of kindness and return to appreciation of things that matter.”

APARDO represents a tapestry of organizations across the Asia-Pacific region that serve patients living with rare diseases and rare cancers. The groups work together to facilitate research, share resources and collaborate on various initiatives. Earlier this year, CSL Behring sponsored “Suffering in Silence,” Economic Intelligence Unit study on the scope of rare diseases in the region.

One positive development that may come out of the pandemic, Ferrie says, is that the empathy shown toward COVID-19 patients may extend to those living with rare conditions.

“I am hopeful this will lead to greater tolerance for all, inclusiveness and openness and collaboration and understanding to improve the lives of all people including our vulnerable communities like people living with rare disease.

Lutz Bonacker CSL Behring Senior Vice President/GM for European Commercial Operations

Lutz Bonacker, Senior Vice President/GM for European Commercial Operations

The experiences, challenges and yes, sometimes obstacles COVID put on our path in 2020 have not been easy. In spite of these difficulties, there are three things that I take away from 2020 that give me a lot of hope:

  1. All of my colleagues working at CSL Behring have been able to quickly step up and continue to do what we needed to do to ensure our important products reach patients with rare and serious disorders.
  2. Within the biopharmaceutical industry, we have seen new ways of collaboration, be it on the vaccine side or in the CoVIg-19 Plasma Alliance – all following the theme: in collaboration things move faster.
  3. The rare disease community, including patients, healthcare professionals, governments and payers has found new ways for collaborating, using virtual tools, flexibility in ensuring access and thinking how product utilization via self-administration may remove the need for hospital visits thus reducing infection risk and freeing up valuable nurse and physician time.”

A common theme among leaders in the rare disease community is the hope that newfound collaboration among organizations could be applied to finding new treatments for patients living with rare conditions. In the early days of the pandemic, CSL Behring helped launch the CoVIg-19 Plasma Alliance, an unprecedented collaboration with other plasma industry leaders to create a potential plasma-derived hyperimmune treatment for those suffering serious complications from COVID-19. 

Alain Baumann World Federation of Hemophilia CEO

Alain Baumann, CEO, World Federation of Hemophilia

This year has been a challenging one for the global bleeding disorders community. It has put unprecedented pressure on patients and carers alike. Fortunately, our community is a strong one. We have faced many crises in our history – and we have always adapted to the new reality while remaining united.”

Like Ferrie and Bonacker, Baumann sees the unprecedented collaboration among key organizations in the medical community will pay dividends for rare disease patients as the world returns to some semblance of normal.

“What I do know is that we will certainly leverage and continue using the tools we have developed during the COVID-19 crisis to provide even better care to patients in the future.”

Shane Fitch Lovexair

Shane Fitch, Lovexair Foundation

Fitch, who founded and runs the Lovexair Foundation, which focuses on people living with respiratory disorders, including Alpha 1 antitrypsin deficiency, is among those who hopes digital tools used to address the pandemic are applied to those living with ongoing and chronic conditions.

Understanding of disease management and digital data exchange is so important. Being able to rapidly transfer and share with their healthcare teams, relevant information through self-care monitoring and devices is essential.

Miriam O'Day Alpha-1 Foundation CEO

Miriam O’Day, President & CEO, Alpha-1 Foundation

What gives me hope is the resilience of our patient community and of our research community. Their motivation to help us continue our mission is inspirational. Hope comes from knowing what you want, and patients with a rare disease know what they want... a cure! It is their hope that ignites us to adapt and move forward during these difficult times.”

Dr. Leonard Valentino CEO National Hemophilia Foundation

Leonard A. Valentino, MD, President and CEO of the National Hemophilia Foundation

2020 has indeed been unprecedented except for maybe 1918-1919 when the Spanish influenza pandemic killed upwards of 40 million people worldwide. The difference today compared to 100 years ago is we’re in the era of tremendous technology advances, but what makes me hopeful for the rare disease community is the resilience demonstrated by patients and their families to overcome each challenge they face and come away stronger and more empowered.

Rob Long, Executive Director Uplifting Athletes

Rob Long, Executive Director, Uplifting Athletes

What gives me hope is the resilience of those in the rare disease community. The challenges that we have faced really aren't new, just exacerbated by the pandemic.”

Long, a Syracuse University football star who survived a battle with a rare brain cancer, runs Uplifting Athletes, a non-profit made up of college athletes with member chapters across the U.S. The organization raises awareness about rare disease and each year gives grant funds to emerging rare disease researchers in its Young Investigator Draft, which is presented by CSL Behring. Eventually, knowledge gained through the pandemic could boost rare disease research, Long said.

We will come out of this with lessons learned and new approaches to connect and move research forward.”

Anthony J. Castaldo, US Hereditary Angioedema Association (HAEA)

Anthony J. Castaldo, US Hereditary Angioedema Association (HAEA)

We have observed remarkable resilience within the HAE Association community, despite the extremely unusual challenges posed by the global pandemic. Our HAEA friends inspired us with record setting participation as we transformed our services and events to a virtual format.”

Many said that rare disease patients were uniquely prepared for the adversity of the global pandemic because they had experience fighting everyday battles with rare conditions – most of which lack approved treatments. Castaldo’s group joined other rare disease advocacy organizations in presenting periodic webinars with medical experts to answer specific questions on COVID-19’s impact.

What gives him hope for 2021? Rare disease patients have a foundation of strength to build on as they look to move past the pandemic and toward real solutions for those living with rare and serious conditions across the globe.

The flexibility to embrace change, overcome obstacles, and remain engaged reveals the character of a community committed to a better future.”