How does an orphan drug pharma company with offices in the Empire State Building amplify rare disease awareness?
Ask Rocket Pharmaceuticals. The rare disease specialist convinced its famous landmark landlord to turn its outside lights to green, blue, pink and purple for Rare Disease Day.
That was two years ago—and the beginning of its “Light Up for Rare” campaign. This year, the top of the Empire State Building will once again be decked out in multicolor stripes, but so will dozens of other famous landmarks in 35 cities around the world.
Niagara Falls, the Spanish Congress in Madrid and 20 buildings in downtown Chicago are some of the 150 landmarks committed to help Rocket and its collaborator, the National Organization for Rare Disorders (NORD), draw attention to more than 7,000 known rare diseases—and the 30 million people in the U.S. and 300 million worldwide afflicted.
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“Patients will be able to look up and know they’re not alone, at least for that one evening, and give them hope,” Kinnari Patel, president and chief operating officer at Rocket Pharma, said.
She added, "Even though each individual disease might only have a small population, the totality of the impact it has on global healthcare and medical need is significant."
Along with organizing the building light-ups this year, Rocket held virtual educational events every Friday for the month of February. Topics included genetic testing, clinical trials for gene therapies and how to set up an advocacy group.
The Empire State Building is known for changing the colors of its exterior lights to recognize different causes, people and organizations. It's common practice for people who see the lights—visible up to 80 miles away—to search online to find out what the colors that night or week mean.
"We're hoping some people will then ask what is a rare disease and find out more about it. And maybe they'll find something they can do to help the community or that they know someone who has a rare disease," Patel said.
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Rocket Pharma is developing a pipeline of genetic therapies for rare childhood disorders. Its five current programs include candidates to treat Danon disease, Fanconi anemia and leukocyte adhesion deficiency-1.
Rare Disease Day, first celebrated in 2009, is always the last day of February. Every four years it falls on the 29th, which was the initial “rare” date chosen for the event. This year the date is Saturday, Feb. 28.
By 2024, orphan drugs will account for 18% of all prescription sales and will and command $217 billion in sales, according to Evaluate’s most recent orphan drug report. In 2020, more than half—31 of 53, or 58%—of the FDA’s novel drug approvals treat rare diseases.
While it may seem that small drug companies would dominate the space, eight of the top 10 orphan drugs will be in Big Pharma portfolios in 2024, Evaluate says.