Too often discussions about the price of orphan drugs focus on the most basic unit of measure: a therapy exchanged for currency. Industry reinforces this notion through their mission statements by repeatedly saying they’re committed to developing a specific therapy for a targeted disease.

So, what would the world look like if rare disease companies stepped back and considered what they’re really selling? Amazing things are happening in the rare disease space: resurrecting molecules to treat previously untreatable diseases, finding solutions for nearly impossible distribution challenges, educating healthcare providers to suspect certain conditions, implementing genetic testing systems, access assistance, and an overriding hope for patients and their families who previously had none.

In reality, each rare disease company is in fact in the business of providing healthcare solutions to patients and families with rare diseases. We can all look around and see what these solutions look like, and they come in all shapes and sizes:

  • Thinking outside the box to see if an abandoned molecule might work
  • Doing the groundwork to convince investors that there may be merit to an idea
  • Working through complex logistical issues
  • Educating healthcare providers on the importance of “looking for zebras” and how to do it
  • Ensuring patients are tested and diagnosed
  • Matching patients with clinical trials
  • Helping disease specific associations grow so that they can help patients
  • Assisting with insurance, co-insurance, drug coverage, travel to appointments

As the world of orphan drug development continues to evolve, it’s time to think about what we’re offering to the patients and families that we aim to serve. If the conversation is reduced to just a therapy for currency exchange, the rare disease patient and family ultimately suffer.

Patti Engel

Patti Engel, President and CEO of Engage Health, has helped define the orphan drug market through her work in market development, research and strategy

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