Ramon Jones

by Ramon Jones

How Social Media Can Help Overcome Challenges with Orphan Disease Research

Orphan diseases account for a large subset of diseases in the United States. An orphan disease is defined as a condition that affects fewer than 200,000 people in a given population. According to the Kakkis EveryLife Foundation, over 7000 orphan diseases in the United States affect 1 in 10 (approximately 25 million) people. Half of the people suffering from these diseases are children. People with these rare diseases depend on researchers to develop new drugs (“orphan drugs”) that may improve quality-of-life. In this blog, we discuss how pharmaceutical companies’ orphan disease research differs from research into more prominent diseases, and how social media is helping shift that paradigm and bring more attention to these rare conditions.

Challenges With Orphan Disease Treatment Development

Considering the thousands of drugs developed for more common diseases, we see that researchers are just scratching the surface in the world of orphan disease. Up until the last 5-10 years, pharmaceutical companies have first defined the need for a drug based on a condition’s prevalence, and then developed a drug treatment program around that need. Historically, given the nature and lack of understanding of some orphan diseases, pharmaceutical companies were reluctant to develop drugs for these diseases. In 1983, the Orphan Drug Act created government incentives for pharmaceutical companies to pursue treatment developments within the scope of orphan disease. Since then, around 600 new orphan disease drugs have been approved by the Food and Drug Administration (FDA) and brought to market. Still, almost 95 percent of orphan diseases have no FDA-approved treatment, and the need for new treatments is often defined on a patient-by-patient basis.

One of the biggest challenges with studying orphan disease is finding the patients to participate in a study. Reaching these patients hasn’t always been straightforward. Patients who have an orphan disease often have feelings of alienation, loneliness, lost hope or defeat from failed diagnoses or treatments, and disempowerment. These feelings can sometimes be attributed to misdiagnosis, being ignored  by healthcare providers, or being geographically isolated from resources and information about potential treatments.

Breakthroughs in the popularity of social media have helped overcome some of these challenges. Previously, orphan drug development depended on advocacy groups and associations to give patients a platform for voicing their concerns. The rise of social media in recent years has broadened and amplified the voice of orphan disease patients.

How Can Social Media Help with Orphan Drug Development?

The intersection between clinical trials and social media use has been a heavily discussed topic due to the meteoric rise in use of social media in clinical research. Social media communities provide a unique opportunity to address some of the concerns with recruitment and access to information. Here, we highlight three key ways that social media can specifically help patients with orphan diseases:

  • Information: Given the communal nature of social media, delivery of information can be almost instantaneous. People who suffer from orphan diseases may feel alone because they know something is wrong but don’t know anything about the symptoms of their disease or where to find out more about it. Social media can serve as a useful tool for sharing new advancements in understanding and continuing education for a disease.
  • Connection: Providing orphan disease patients with a platform where they can interact with others who have had similar experiences can create a sense of community. This can happen on one or more social media platforms (Facebook, Twitter, Instagram, etc.) through support groups, patient advocacy campaigns, or directed advertising. In addition, using social media to connect an orphan disease population with others can allow earlier disease awareness, improve patient experiences when interacting with health care providers, reveal potential recruitment opportunities for clinical studies, and enhance collaboration in the quest for accurate diagnoses.
  • Empowerment: While social media involves some risks, one valuable aspect is that it can empower orphan disease patients. That empowerment may come through testimonials geared toward physicians, fundraising for awareness, a feeling of self-worth by participating in research, or knowing there are others out there looking for information.

These are just a few ways social media can open doors for orphan disease populations. As with all things, though, there are risks involved. To find out more information on special considerations for social media use in clinical trials, view this on-demand webinar where we discuss risks and limitations to social media use and how we can minimize those risks to research participants.

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