Rethinking Rare Disease

How pharma can heighten awareness and enhance engagement with HCPs and patients to address the unique challenges of rare diseases

Rare diseases affect one in 10 Americans, half of whom are children. Yet, of the more than 10,000 rare diseases identified, fewer than 5% have FDA-approved treatments.

MM+M’s new eBook, “Rethinking Rare Disease,” focuses on this special subset of healthcare, exploring how pharma can both improve awareness of rare diseases and engage with healthcare professionals (HCPs) and patients more effectively to address their specific challenges.

Authentic engagement can help pharma establish trust within rare disease communities. As explained in “Why the Story Matters in Rare Disease,” by highlighting narratives depicting the reality of living with a rare condition, brands not only raise awareness of the disease but also lift barriers to care and treatment.

As insurance providers increasingly approve coverage for therapies, drug manufacturers are more willing to invest in ultra-rare conditions, regardless of patient population size. Now, after more than a decade of scientific persistence, the FDA approved Kygevvi, offering a life-changing, substrate-replacement therapy for patients battling ultra-rare TK2d, explained in “The Long Road to ‘Yes.’”

This eBook also explores ways to bridge information gaps by providing the resources patients want and empowering HCPs to educate patients better on treatments and successful patient outcomes.