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Pharma: rare diseases
Cunning conditions that rebel against cures, the rare disease landscape is as challenging as it is rewarding to work in. Here's a collection of recent rare disease work to check out, no black box warning required. 

Campaign launch + expanded label + new indication: ORLADEYO struggled with a perception issue. Both patients and HCPs didn't believe that a pill could be as effective as an injection. That led to our new campaign launch-"and/or"-which significantly helped with believability in testing. With over 3,000 patients on the once-daily capsule, the brand has no intention of slowing down. Up next: an expanded label and new indication. Stay tuned. 

Pitch win + new indication + disease education: My partner Jim and I helped pitch and win EVKEEZA, a drug for HoFH (see the concepts below), that treats a rare life-threatening, high-cholesterol condition. Now what we launched with looks nothing like what they said moved them and helped us win the pitch, but, hey, we tried. After launching a refreshed with an HCP website and CVA, we got ready, a few months later to introduce a new indication for younger children and provided disease education that helped HCPs overcome the mental hurdle of realizing 3 year olds can die of high cholesterol and that treat needs to start ASAP.

Pitch win for HCP AOR + future new indication: Instead of focusing on the visual drama of the flare, we won the business by using a strategic approach and focusing on the burden of GPP beneath the skin that dermatologists cannot see. It's still early days, but we're already created some content for their booth that has been a hit with reps and HCPs alike.

Oncology pitch: Although this work—and more importantly the hope of this drug for women living with platinum resistant ovarian cancer—ultimately didn't end up going anywhere because of failed clinical trials, I especially like the tone and emotion of the concept and video we created.

Gene therapy launch: ALS is an especially insidious disease with changes in mood happening years before more classic symptoms like cognitive decline and movement disorders begin. That's why launching to launch AMT-130 was so important and rewarding. The unbranded disease website seeded the ideas that symptoms can begin years before an accurate diagnosis is made and that, while not curable, a disease-modifying treatment is on the horizon.

Gene therapy launch: We helped introduce LUXTURNA, a gene therapy for a rare and debilitating inherited retinal disease, giving hope to people who are affected by this loss of vision.

Drug launch: Zevaskyn is a gene therapy made from patients' own skin that is surgically applied to permanently cover open wounds. It's way to start healing from the stigma of RDEB for children with this rare disease.

Launch print: Some of my favorite concepts for a variety of reasons for a variety of rare diseases.

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