From social distancing and shuttered businesses to personal health concerns, COVID-19 has changed daily life for the foreseeable future. There are nearly 4.75 million diagnoses worldwide, with 1.53 million in the United States alone. On one hand, we understand how this viral pandemic is impacting the medical field: lack of resources, heavily-hit hospitals. But how is it affecting other medical organizations? In an interview on PharmaPhorum, rare disease biotech Cure Rare Disease (CRD) explores the impact of COVID-19 and the future of rare disease research.

Understanding Cure Rare Disease

The Interviewee: Rich Horgan

Rich Horgan is the President and Founder of Cure Rare Disease, a nonprofit that uses CRISPR to create specialized treatment solutions. He grew up in a tight-knit family with a genetic history of Duchenne Muscular Dystrophy (DMD). After his brother Terry was diagnosed with DMD, Rich wanted to make a change: performing research and finding a cure. Now, his organization collaborates with researchers and doctors to create customized drug therapies for rare diseases. Discover the Cure Rare Disease mission here.

Exploring This Rare Disease Biotech

Studying rare diseases and developing treatments is especially important during COVID-19. Many patients with rare diseases or conditions are immunocompromised. This could lead to more severe reactions and worse patient outcomes. But with clinical trials being delayed and financing being more difficult to find how can biotechs survive COVID-19?

According to Horgan, the varied medical industry changes decrease company efficacy. He notes that, to receive FDA approval for drug and gene therapies, the company needs to perform research. However, many clinical trials are canceled or delayed due to safety issues. Rare disease therapy progress is halted. This leaves patients with rare diseases who have not responded to previous treatments without assistance.

Extra funding is being redirected towards creating a vaccine or treatment for patients with COVID-19. So, for patients with one of the 7,000 rare diseases and their families COVID-19 might feel hopeless. Yet Horgan believes that companies will adapt moving forward, creating a better path forward for the rare disease community.

Current Challenges

Many clinical trials rely on in-person patient visits, both for the initial therapeutic application and for continued follow-ups and data collection. However, COVID-19 is extremely contagious. According to the Cleveland Clinic, the virus also survives on surfaces for up to 5 days. So, it is dangerous for patients to visit hospitals or clinics for trials as it may expose them to the virus.

Additionally, COVID-19 spurred a redistribution of people and resources. Many people who previously ran clinical trials are now assisting hospitals overrun with patients.

Adapting to COVID-19 as a Rare Disease Biotech

Adaptability is crucial to surviving COVID-19. So, as Horgan explains, Cure Rare Disease is taking the following measures:

• Enacting new safety guidelines and training procedures.
• Redeveloping core business strategies to take future changes, COVID-19, and medical resources into account.
• Applying for emergency capital (money) to keep the business alive, provide resources to patients, and continue championing any research.
• Moving their medical research out of academic labs.
• Modifying fundraising plans and strategies.
• Raising brand awareness to grow engagement between patients and medical researchers, to help grow patient trust and ensure patient motivations drive research.
• Holding virtual team meetings to keep every team member connected.
• Developing contingency plans in case employees, or study participants, become sick.

Cure Rare Disease began developing their contingency plans as early as January or February to ensure the protection of their studies. Additionally, the organization has strengthened cross-functional collaborations within the organization and with partners.

Future Changes in Research and Business

First, Horgan believes that nonprofit fundraising will change. Nonprofit organizations like Cure Rare Disease rely on the support of others. However, it is difficult to connect with individuals, or stimulate engagement, without in-person events. So, in addition to adaptability, rare disease biotechs will require creativity in connecting with others. For Cure Rare Disease, Horgan is looking to raise funding online. Some of the organization’s creative solutions include:

• Building partnerships with pro gamers,
• Holding your own eSports fundraiser, and
• Partnering with grocery stores to donate $0.10 for every special purchase.

COVID-19 Benefits: Finding the Silver Lining

Ultimately, the way we live life is going to change moving forward. But finding the silver lining in all of this craziness helps us look to the future. Horgan sees COVID-19 as a learning opportunity for medical providers and rare disease researchers.

First, COVID-19 stimulates creativity and innovation. As a rare disease biotech, Cure Rare Disease needs to find a way to move quickly but effectively, develop creative therapeutic solutions, and formulate ideas on how to further research. In the future, this will allow organizations to work well under pressure. It also helps organizations build their accountability, adaptability, and collaboration skills.

Next, COVID-19 highlights the importance of crisis management policies and preparation. If another situation arises, organizations are better prepared to handle the fallout. In particular, Horgan believes contingency plans should be built into business development plans.

Finally, this pandemic strengthens the rare disease community. Although it is a frightening time, this strong and passionate community continues to advocate for change. Additionally, the air of support means nobody feels alone. In the future, Horgan hopes that:

we [can] apply the same focus, collaboration and sense of urgency to developing treatments for less well-known disease.