Amplifying the voices in rare diseases

There are approximately 300 million people around the world living with a rare disease. Yet, until relatively recently, there was limited focus on this heterogenous population. By their nature, rare diseases are just that – rare – with a wide array of symptoms and severities affecting patients, which makes it challenging for pharma companies to identify opportunities to develop effective new treatments.

Yet the very fact these diseases are unusual and often fly below the radar has created a tight-knit rare disease community, comprising patients, carers, family members, advocates, and healthcare professionals (HCPs) who are invested in the health and treatment of patients living with one of more than 6,000 identified rare diseases.

Very few rare diseases have a cure, which leaves many patients in need of treatment options. These patients offer invaluable insight and experience when it comes to understanding the realities of living with a rare disease. Acknowledging and amplifying these voices is essential for pharma companies looking to enter the market but communicating with such a passionate and informed group can be challenging.

“Rare diseases are notoriously heterogeneous,” says Dr Emma Lemon, who leads the Medical practice at AXON, a global healthcare communications agency. “You have to have a tailored approach. You can’t go in and approach a medical communications plan for one rare disease in the same way as you would do it for another rare disease.”

• Read the full article in pharmaphorum's Deep Dive digital magazine