February 28, 2024

Trailblazing in Rare Disease Patient Engagement: 5 Lessons From Pharma’s New Playbook

By Eric Peacock, Co-founder and CEO, MyHealthTeam

Twelve years ago, rare diseases were overlooked by pharma, leaving rare disease research underfunded and patients underserved. However, a transformative shift occurred in 2012 when the FDA introduced the Breakthrough Therapy designation for rare disease drug development. This was a pivotal moment, unlocking R&D budgets for rare disease discovery and trials. 

Now, as large pharma companies and small biotechs alike rush to bring rare disease drugs to market, they face a new challenge: How to launch a new rare disease drug when their existing commercialization playbook was built for large chronic conditions and mass media marketing? Fortunately, this newfound need to reach and engage rare disease patients at scale has led to significant investment in creating and supporting rare disease communities like never before.

To date, MyHealthTeam has launched 13 rare disease social networks, partnering with pharma companies and biotech manufacturers to bridge the gap for patients. Patients with rare diseases are typically isolated — there may be no one else facing the same condition within a 100-mile radius and many live far away from the centers of excellence for their disease. Our goal is to make it easy for patients to find the people, trusted medical information, and support they need to best manage their condition. We also want to make it easy for them to engage with pharma partners in research, clinical trials, advertising, and patient support. Here are five key lessons we’ve learned along the way.

1. The Demand for Rare Disease Social Networks Is High Among Patients

Historically, patients with rare diseases found themselves geographically isolated from other patients and disconnected from vital information, specialists who understand their diseases, and any kind of clinical trials available to them. It can feel extremely lonely, frustrating, and scary. Patient social networks like MyHemophiliaTeam, mySMAteam, or MyNarcolepsyTeam can instantly bring together thousands of rare disease patients to share tips and experience. They can participate in a live, virtual Q&A session with one of the nation’s top researchers or clinicians in their disease (often there are fewer than a dozen) or learn about the three or four clinical trials going on near them. They can read medically approved articles and watch videos about their condition that are a safe alternative to “Dr. Google.”  

Technology, expert medical writers, and AI break down the traditional barriers preventing rare disease communities from learning and from engaging with pharma manufacturers. It’s not a “field of dreams” where “if you build it, they will come.” You have to build it, reach out to let them know about it, and then keep delivering value by helping them see around the next corner in their journey with the disease. 

2. Rare Disease Social Networks Are One of the Fastest-Growing Marketing Channels for New Drug Launches

Outside of oncology (which can also benefit from fast-track approvals), the fastest-growing networks on MyHealthTeam are centered around rare diseases. Rare is new for a lot of our pharma partners, and they are partnering with us to launch new social networks well before launching their new rare disease drugs. In fact, pharma partnership revenue from rare disease communities has grown 50 percent year over year at MyHealthTeam. That trend seems to only be increasing as partners engage members in trial recruitment, evidence generation for regulators, and disease state education prior to launch. By the time launch comes around, we already have a built-in community ready to learn about the new therapy. 

Programmatic advertising and mass market advertising typically don’t perform terribly well in rare disease. Advertising directly to a community of patients diagnosed with the disease does work well.

3. Rare Disease Patient Populations Are Educated and Motivated

Common among rare disease communities is a highly educated and motivated patient population. Many patients consider themselves “self-taught Ph.D.s” in their condition, obtaining information from academic journals, peer-to-peer interactions, and podcasts. There is a high ability to engage them and get their input on patient outcomes and burden of disease — or to educate them about new treatments like biologics and gene therapies. Bring the message to them directly and help them understand how to get involved in trials and get insurance companies to pay for cutting-edge new therapies. There are a lot of hurdles in their way at launch, and you can help knock those down with information.

4. Patient Input Can Impact Clinical Trial Design: A MyHemophiliaTeam Case Study

An early inspiration for us came from one of our first rare disease social networks, MyHemophiliaTeam. Research conducted with our members actually influenced the design of our pharma partner’s clinical trial. Up until that point, our client had mainly based their decisions about which outcomes to measure in the trial on KOL input and a panel of five patients with hemophilia. We launched MyHemophiliaTeam and, after a few months, we were able to engage 50 members in a quantitative survey that identified a totally new and important clinical outcome: pain. This hadn’t been considered in the trial design. 

Older hemophilia patients, who had never benefited from prophylactic treatment of their hemophilia at a young age, had suffered debilitating joint damage in their knees and shoulders that even made it hard for them to sleep at night. This collaborative research surfaced an entirely new endpoint to consider in the trial, showcasing the potential for early partnership in the drug development process.

5. Trendsetting Collaborations Get the Best Results

We encourage a collaborative approach with pharma partners for best results. From evidence generation in trials to disease state education and ultimately to B2C market launches, the rare disease social network serves as a platform for engaging and cocreating with the patient community. For brands that are about two to three years shy of a consumer product launch, now is the time to consider partnering with an existing rare disease social network or partnering with us to launch a new social network. And for brands launching into a competitive field, or introducing a new therapy for the first time to a rare disease, differentiate by going direct to patients.  This will help give voice and education to the community while helping you build authentic relationships with the very patients you’ll soon be serving.

As rare diseases step into the spotlight for Rare Disease Day on February 29, MyHealthTeam is not only illuminating the unseen struggles of patients but also reshaping the landscape for pharma partners. Together, we are forging a path where knowledge, support, and groundbreaking advancements intersect to redefine the future of rare disease education and care.