Rare diseases have historically not attracted significant pharmaceutical investment, but recently big pharma companies are paying more attention to this field. Driven by government incentives and a higher likelihood of success in treating often fatal or severely debilitating diseases, orphan drug sales are expected to see a significant increase in the near future.

However, successfully launching these treatments requires a different approach than the standard framework. Our experience suggests that companies launching rare disease treatments must excel in four strategic pillars:

Engagement with the rare disease community

Investing time with patients, support groups, and therapeutic experts is crucial for generating insights into the patient experience and healthcare ecosystem. These relationships are key to building disease awareness.

Identification of patient populations

Beyond relationships, companies can work with advocacy groups to distribute free diagnostic tests and use algorithms to search claims codes associated with typical disease symptoms to identify undiagnosed patients.

Patient access

Facilitating access through early access programs, minimizing time to treatment initiation, and helping close funding gaps are ways pharmaceutical companies can ensure patient access to these costly treatments.

Patient and caregiver support

Providing nursing services, updates to the treating physician, social network groups, and helplines can enhance the patient experience and prevent treatment discontinuation.

To operationalize these pillars, launch teams must embrace a culture of cross-functional collaboration, with roles spanning multiple functions. Early and careful sizing and deployment of teams is also key. Finally, generating real-world evidence for several years post-launch is essential to support the treatment's value to payers.

While challenging, companies that master these distinctive elements can successfully launch rare disease treatments and help solve challenging medical problems for affected patients.

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