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Alnylam explores the rare disease journey in a new, hard-hitting documentary

The patient voice is a powerful one when it comes to healthcare advocacy, and Alnylam has put together a new documentary that maps the often-difficult journey those suffering with acute hepatic porphyria (AHP) endure.

The documentary is a major departure from the often-upbeat pharma TV ads you’re used to seeing when it comes to a disease awareness campaign. For Alnylam, which only moved from a pure R&D biotech to a commercial biopharma four years ago, this departure was intentional for two reasons, said Genie Dubuk, vice president and rare disease business unit head, in an interview.

The first is to show the reality of being in pain and not having people believe you—and the need for a rare disease marketing message to reach the right patients. There are only around 5,000 AHP patients in the U.S. and Europe, so spending millions of dollars for an all-encompassing upbeat DTC ad would likely just miss the people who need to see it.

A documentary allows for a deeper dive into the pathway of an AHP patient, however gritty that may be. Aiming the launch to coincide with Rare Disease Day on the last day of February, Alnylam teamed up with Emmy-nominated filmmaker Cynthia Lowen to produce “Two of Me: Living with Porphyria,” a film chronicling the lives of seven people around the world living with AHP.

It’s not pulling any punches. “So many times, pharma companies are [in DTCs] intentionally very hopeful, because people like hope,” Dubuk explained. “But our goal here is to highlight the struggle that these patients have, so that not only can patients see themselves in the story, and are validated, but know that their experiences are not just in their heads.”

She says many AHP patients are actively called out by their doctors for exaggerating or even lying about their pain and symptoms, and then shipped from pillar to post with no diagnosis. Alnylam estimates it takes an average of 15 years for AHP patients to be properly diagnosed.

So this documentary is not just for patients, but for doctors, too. “They need to see the pain and the struggle as well and see that these symptoms are real,” Dubuk said. As part of the campaign, Alnylam will be showing the documentary at medical congresses throughout the year to help boost that message.,

Alnylam sales reps and marketers are also sending out links to the documentary for healthcare workers. There is a general social media campaign to help drive rare disease patients to disease education websites alongside this video.

This should help garner more awareness for patients who can ask for a treatment as well as allow prescribers better knowledge of the condition. The main thrust is “to shorten the time to diagnosis,” Dubuk explains.

AHP is an ultra-rare condition that represents a family of rare genetic diseases characterized by potentially life-threatening attacks and, for some people, chronic pain and other symptoms that interfere with their lives.

Alnylam nabbed an FDA approval for its RNAi treatment Givlaari for AHP back in 2019, and the drug made $128 million last year. It costs an eye-watering $575,0000 a year—reduced to around $442,000 after discounts—with about 350 patients having used the drug by the end of last year.

No surprise that Alnylam wants to explain why treatment is so important and wants patients to get access more quickly: Fifteen years to diagnosis is too long for patients—and too long for Alnylam to wait for people to get its drug.

The 30-minute documentary is largely unbranded, but the site hosting it does have Alnylam branding. It’s set up as a global voice: Patients with the condition speak from around the world, including the U.K., Mexico, the U.S. and France.

They take the viewer on the typical journey of a rare disease patient: from symptoms that can dovetail with many other more common conditions, leading to misdiagnosis, wrong treatments and sometimes invasive biopsies or surgeries, all to no avail.

This happens a lot with AHP, because the most common presenting symptom is severe attacks of abdominal pain, something that can happen for many reasons.

All of this creates a perfect storm that delays diagnosis, hence the wait of up to 15 years for an accurate AHP diagnosis.

And that’s the overriding message of “Two of Me: Living with Porphyria,” which predominantly aims to “raise awareness of AHP and the barriers that exist to receiving a timely and accurate diagnosis,” Alnylam said in a statement, by encouraging viewers to “Tell 15” others about the film, echoing the 15 years it takes to get that diagnosis.

Much of the documentary was shot before the pandemic, between 2018 and 2020, to “capture their stories,” Dubuk explained. But when the pandemic hit, “we were struck with a major dilemma: How do we continue to create and share content that best serves the AHP community?”

The best way, she said, was to use the existing footage they had gathered over the two years prior, and that meant Alnylam didn’t have to go out to patients’ homes to film and create the documentary from there.

The next big step, she said, was to find a director, which they did in Lowen, and then figure out how to “weave together” the various story lines, which had been captured without a single connecting theme.

So they tapped Anya, one of the patients featured in the film, and brought her as a narrator to help create a cohesive narrative.