Adding rare disease therapies to medicine portfolios: 7 steps to follow

Many established pharmaceutical and biotech companies are moving into (ultra-) rare disease markets. However, as more companies pursue these opportunities – they often discover that commercializing rare disease products introduces a different set of challenges, particularly in the realm of transport requirements, patient services, and commercial distribution.

Traditional supply chains are generally designed for medium- to high- volume products, targeting larger patient populations and standard storage and handling requirements. In contrast, rare disease products often involve low volumes due to low numbers of patients, stringent storage, handling and distribution requirements, and special needs when it comes to patient support and/or home care services.

These characteristics often result in little to no inventory being held in the distribution channels or at the point of care, making reliable fulfillment significantly more difficult without purpose-built, rare disease focused solutions.

For pharmaceutical or biotech companies seeking guidance on how to successfully add rare disease products to its existing portfolio, experts from AIM – an award-winning supply chain consultancy, specialized in pharmaceutical and life science supply chains – have developed a 7-step process tailored to rare disease product requirements:

1: Achieve Strategic Alignment: Align leadership on the role of rare disease therapies and future scalability needs.

2: Conduct Initial Data Gathering: Assess current capabilities and define project governance and charter.

3: Gather Specialized Information: Leverage internal stakeholders and healthcare professionals to understand “last mile” needs.

4: Analyze Commercial Distribution Requirements: Develop a 360-degree distribution profile specific to the rare disease product.

5: Fit-Gap Analysis: Compare existing networks and vendors to new product needs, identifying gaps.

6: Create High-Level Solution Options: Define implementation paths and assess impact across cost, licensing, and operations.

7: Prepare and Present the Recommended Solution: Select a path forward, build a roadmap, and secure approval to proceed.

Typical outcomes

According to analysis by AIM, a company’s existing distribution network can meet the requirements of the new rare disease products in about 50% of cases.

In the remaining cases, new or partially-new solutions are required. Here, companies can run into a number of common pitfalls, such as failing to assess fit early enough (leading to launch delays), underestimating the complexity of seemingly “small” products, and inadequate backing from senior management (meaning there is insufficient buy-in).

Conclusion

Rare disease products often have specific commercial distribution requirements, which usually differ from traditional therapies and thus require a different approach to market, as well as different operational and change management approaches to ensure their commercial success.

Using the 7-step approach from AIM, biopharma companies can address and master how they add rare disease products to their medicine portfolios.