Sometimes, when rare diseases run in families, people spread the word to relatives. However, like with most rare diseases, there can still be difficulty around the initial diagnoses. And that's where Alnylam Pharmaceuticals saw an opportunity to highlight the power of family.
The company put the spotlight on one extended family in its new disease awareness campaign for hereditary ATTR (hATTR) amyloidosis. Alnylam recently announced FDA acceptance of its new drug application and priority review status for patisiran, an RNA interference therapy to treat hATTR amyloidosis.
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In the new campaign called “Bridge the Gap,” multiple generations of one family talk about their journey since first finding out about hATTR amyloidosis in the 1980s with the initially diagnosed generation. In a series of videos, Angel, Dane, Mark, Daryel, Tonya and others—identified only by their first names—talked about family advocates, symptoms, support and genetic testing. They’ve also created a downloadable booklet featuring the history of the family’s journey with hATTR, dating back to Sarah in the 1960s, who was never diagnosed.
“These are real people and real faces and we need research, we need management, and we need people paying attention to us and knowing this disease applies to real people,” Angel explained in one video.
Alnylam has been working on building its hATTR amyloidosis website with educational material and information for over the past year. The new awareness campaign, which includes a social media push, launches in advance of what the company hopes will be a positive FDA decision, with a verdict due in August. While not an uncommon strategy for drugmakers, seeding the market with information about a disease before drug launch is particularly common in rare diseases.
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“A lot of patients have a pretty intense journey to diagnosis. The time from onset to diagnosis is about four to five years. As you can imagine, patients get bounced around from specialist to specialist trying to figure out what’s going on,” Pritesh Gandhi, Alnylam vice president of medical affairs, said. “It’s an autosomal dominant disease and that means 50% of the family will carry the gene, although not all will have symptoms. If family members speak with each other and share their experience with each other, you can think about having an earlier diagnosis if one member is diagnosed early.”
Wendy Blackburn, executive vice president at Intouch Solutions, which is not involved in the Alnylam campaign, talked to FiercePharma about pre-drug launch disease awareness efforts in general. She said she’s been writing about the rise or proliferation of disease awareness campaigns since 2009, but hasn’t noticed any sudden uptick lately. However, she did say that if there has been an uptick it could be as a result of more new drug approvals, which hit a record high last year.
“The economy is strong, the industry is strong, and marketers are confident in their budgets. In years past, if a marketer had to trim their budget somewhere, they might have trimmed disease awareness efforts because it was difficult to prove ROI. But budgets have become more stabilized, measurements have become more sophisticated. And it’s been proven that—done right, and under the right market circumstances—disease awareness works,” she said via email.