When rare disease-focused biotech and pharma companies invest in disease state education (DSE), they have the opportunity to meaningfully improve patient experience, while also building a strong foundation for commercial success. That’s a contrast to more common disease states, where new treatments are often later line, and therefore aimed at experienced patients. Moreover, in non-rare disease states, there is no shortage of patient resources, making it harder for organizations to create unbranded information that delivers real value for patients.

Meanwhile, in the rare disease space, patients face a disproportionate burden. Community physicians may have little to no experience with the condition, leaving patients to navigate a life-altering diagnosis on their own. They turn to academic journals and the deep corners of the internet, finding resources that are often either too technical to be practical or so negatively framed that they strip away all hope.

How disease state education sets patients up for success

Practical, patient-centred DSE empowers patients to better navigate their journey and complex decisions with greater confidence by:

• Helping them understand the mechanics of their condition, and the steps and decisions they should anticipate along the way.
• Providing information that is credible, actionable, unvarnished, and honest without being frightening.
• Helping patients explain their disease to friends and family, and connecting patients to additional support and resources.
• Encouraging patients to seek out expert providers and institutions who understand and have experience treating their condition. In the pre-launch context, this can also include helping patients find relevant clinical trials.
• Providing emotional comfort by signalling that other patients have walked similar paths before.

The Case for Early Investment

Biotechs and pharmas who invest in rare DSE get the maximum return from their investment when they create disease state education well ahead of commercial launch. During clinical research, DSE serves as a vital bridge, helping patients navigate existing treatment options, connect with specialized centers, and even discover relevant clinical trials. This, in turn, can help accelerate clinical trial recruitment and put patients in a better position to be aware of and open to newly approved treatments. Done well, DSE can lay the foundation of a community of patients with rare disease, which in turn can help inform more patient-centered drug development.

The Takeaway

Rare disease state patient education provides manufacturers the opportunity to thoughtfully address patients’ critical, unmet needs, signal a genuine, long-term commitment to high-need populations, and foster greater awareness and openness to their emergent treatments.