Do you have any idea how many superheroes are orphans?

SupermanSpiderman, and Batman to name just a few. Thrust into an unjust world as orphans, their righteous response is to use their superhero powers to bring justice to the world. Superheroes make the world right for the “little guy.” And that is what families of children with rare and orphan disease do, too.

The parallels we found between the stories of superheroes and many of the families who face rare and orphan diseases are amazing. What else can you call someone who gains major media attention, gets legislation passed, starts a foundation, and even founds a drug company, other than a superhero? These amazing individuals dedicate their lives to curing a disease that is “orphaned” by its small size and limited commercial potential.

These parents and patients transcend the boundaries of their traditional roles to become:

  • Media MavensTara Blocker and her daughter Ashlyn, who suffers from congenital insensitivity to pain, became skillful at using the media to identify fellow sufferers. So frustrated was Tara that, according the New York Times article The Hazards of Growing Up Painlessly, she sent up a media flare that eventually got Ashlyn’s story on Good Morning America, the Today Show, and Inside Edition. From this media exposure, she got connected to important scientists and parents of children suffering from the same condition
  • EntrepreneursJohn Crowley left a secure job at Bristol-Myers Squibb to start not just 1 but 2 different companies dedicated to finding cures for Pompe and Fabry diseases. John had an amazing journey, from a parent frustrated at the lack of a cure for Pompe, to a serial entrepreneur. His efforts resulted in the development of a medication that saved his children’s lives. His story is the inspiration for a major movie, Extraordinary Measuresstarring Brendan Fraser and Harrison Ford
  • Foundation Builders: Christina Saninocencio, a young woman in her 20s founded the Lennox-Gastaut Syndrome Foundation after having spent her childhood explaining her brother’s unique symptoms (drop seizures) to the world around her. We had the chance to meet Christina when we were doing some research on Lennox-Gastaut and were struck by her dedication in the prime of her life to helping families around the country

So how do you work with one of these superhero family members? By involving them from the beginning to design and recruitment for trials, developing patient materials, understanding and connecting with the disease community, lobbying Congress, and testifying to the FDA.

However, it is as important to understand that bio-pharma goals of developing, gaining approval, and launching an orphan drug will undoubtedly diverge in some important ways from the aspirations and hopes of these superheroes. Sometimes, their need to get help for their family members may be at odds with designing a trial in the most effective way. In other circumstances, their overwhelming desire to push for a solution at any cost may not fit within the FDA’s risk–benefit framework.

And here is where it becomes challenging to be a truly patient-focused organization. Can bio-pharma find a way to work with these superheroes, knowing that there are some areas of disagreement? If you work in the orphan drug or rare disease space, the answer to this question has to be yes. Because you know that their superhero activism plays as big a role in moving the research and support ahead as that of your company.