changing_the_mindset.jpgThere are currently only 314 FDA-approved therapies available among the roughly 7,000 rare diseases that have been identified, according to the Genetic and Rare Diseases Information Center. The global pharmaceutical industry recognizes the unmet need and the opportunity to solve the challenges associated with patients with rare diseases. Current and future pipeline assets offer the pharmaceutical industry an opportunity to bring to market the type of life-changing therapies for patients that add true value where hope is limited.

But the nature of rare disease patients—often highly engaged, knowledgeable and intimately involved in their treatment decision-making process—creates an imperative for pharmaceutical companies to think differently about how they build their organizational capabilities and develop their commercialization strategies. While the actual steps to commercialize these new therapeutics may be the same as more broad-based categories, to succeed in rare diseases, the patient must be the organization’s center of gravity, which means that the organization must take a different approach to the commercialization steps.

Throughout the past several months, I have conducted a series of interviews with commercial leaders to learn how the commercialization process for therapies targeting rare diseases creates significant challenges to the norms. The result was a list of key success factors in orphan drug commercialization, including:

  1. Gaining organizational alignment on how and why the rare disease ecosystem is so different from the typical mass market retail drug markets (where many leaders have “grown up”)
  2. Achieving an actionable understanding of the often arduous, complex and poorly defined rare disease patient journey
  3. Overcoming the lack of robust data
  4. Identifying and acquiring patients in inherently low-prevalence populations
  5. Finding the skillset needed for customer-facing roles, and not only executing but orchestrating activities across a complex set of stakeholders
  6. Creating an empathetic patient support services team and process that enable an individualized approach to support rare disease patients and their caregivers
  7. Establishing optimal pricing and reimbursement strategies, particularly in light of recent pricing pressure in rare disease, specialty therapies and pharmaceuticals in general

During last week’s 2016 World Orphan Drug Congress, my team from ZS and I led a workshop with industry leaders entitled “Achieving Success in Commercializing Rare Disease Drugs.” In this workshop, participants who represented a range of companies and focus areas across the industry provided their views on which of these key success factors they would prioritize and solve for, challenges aside.

If you could prioritize and solve for two key success factors, what would they be?


One interesting result from this poll is that “organizational alignment” tied with “pricing and reimbursement” as the key success factor that the audience would choose to prioritize and solve for. Given the challenges and pressures that the industry faces with regards to pricing strategies and the cost of rare disease therapies, it’s telling that solving for organizational alignment on the implications of working in rare diseases is viewed as being equally important to success. That is, we must educate the broader organization on the ways in which the rare disease ecosystem is different from many traditional pharmaceutical settings. 

As our broader workshop discussion suggested, this includes recognizing the patient and caregiver as a primary driver—if not the primary driver—of decision making; agreeing that successfully commercializing rare disease drugs can only be achieved with the robust and integrated development of the patient journey; and understanding that the success measures typically used by mass-market teams (reach and frequency, etc.) don’t hold water in rare diseases.

We must think differently, change the vernacular, and build solutions that address the patient, families and caregivers at the center.


Topics: rare diseases, Michael Thomas, organizational alignment, WODC, FDA, pricing and reimbursement