Translating the Patient Experience Into Commercial Success

Key Strategies for Accurate Market Sizing

At the most recent ILAB conference, Chad Holland of Enobia spoke about the challenge of determining accurate market sizing for a small patient population with the rare condition hypophosphatasia. Some of the key commercial challenges the brand was facing included: low disease awareness, misunderstanding of natural history, and very little epidemiology data. In order to determining market sizing, a variety of sources were employed: national and international genetic databases; surveillance projects, especially pediatric; mortality databases (CDC mortality database); disease registries; EMRs; social media mining; market surveys (both U.S. and abroad); market research; competitive information; KOL database audits; patient advocacy groups. In summary, you have to cast a very wide net to get an accurate headcount.

View Chad's Presentation

Effective Collaboration
Between Industry & Advocacy

Patrick Reichenberger of Raptor Pharmaceuticals and Nancy Stack of the Cystinosis Research Foundation (CRF) shared their experiences working together as an example of how both manufacturers and patient advocacy groups stand to benefit by collaborating to bring an orphan drug to market. The key to collaboration is building trust that leads to a true partnership. This requires establishing a personal and ongoing relationship, listening to one another, and finding common goals to work toward. This is exactly the relationship that has developed between Patrick and Nancy, leading the way for Raptor and the CRF to partner on a number of key initiatives, including a patient registry, research funding, and support of family and scientific meetings.

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Strategies to Support Pricing, Reimbursement and Market Access

Douglas Paul of MME gave many concrete examples of how to develop optimal pricing and reimbursement strategies for orphan drugs. Doug talked about how to evaluate the key value drivers in every unique market situation, how to compare against established orphan drug pricing models, and how to avoid pitfalls that can reduce the value of orphan drugs. He also talked about how the price of every orphan drug affects the price of every other orphan drug—and how big pharma is a very real and growing concern for small manufacturers. Finally, Doug talked about the opportunities for pricing outside the U.S., giving examples of both successes and failures.

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Social Media's Unique and Significant Role for Patients and Caregivers

Kristin Keller of Compass Healthcare Marketers talked about the rise of social media networks as a vehicle for patients to exchange health-related information—and ultimately, to influence the way key decisions are made. For the small populations associated with rare diseases, social media is the primary means of connecting with the disease community to share knowledge and experiences. Recent surveys indicate that the two primary reasons why patients/caregivers connect online are: emotional support and practical advice. In order to commercialize an orphan drug, social media is a valuable tool to help determine the brand's key influencers, discover brand perceptions/misperceptions, reveal the patient's unmet needs, uncover the key disease management issues, help evaluate the competitive landscape, and many other key market issues.

View Kristin's Presentation