Procysbi
Launching an orphan drug with a global brand strategy and an individualized patient approach
PROCYSBI® (cysteamine bitartrate) was the first new treatment for nephropathic cystinosis (NC) in 20 years. NC is an ultra-rare genetic lysosomal storage disorder (affecting <2000 people worldwide) where the accumulation of intracellular cystine leads to life-threatening renal and systemic complications.
The Insight
Successful treatment hinges on early initiation and diligent, lifelong adherence to a challenging every-6-hour dosing schedule. (Delays in dosing result in accumulation of cystine, which can become toxic.) Yet data suggested that up to 80% of patients may not comply. PROCYSBI® is a delayed-release formulation of cysteamine that provides continuous control via improved 12-hour dosing.
Unbranded Disease Education Campaigns
Non-branded disease education for consumers (knowcystinosis.com) began one year prior to professional launch advertisements which were designed to educate about the need for continuous cystine clearance as well as the conceptual benefits of a long-acting cystinosis therapy.
KnowCystinosis.com, a robust non-branded disease education site for HCPs, patients and caregivers, delivers an overview of the basic information they need to understand the disease, its causes, effects on the body and potential outcomes.
Children’s Disease Ed Book
The books helped children ages 3-to-5 understand nephropathic cystinosis and feel empowered to fight it. These books also aid caregivers to better explain and understand the disease themselves.
HCP Campaign
Halving the frequency of dosing may seem like a simple idea, but for the hundreds of patients on therapy – and especially their caregivers, it provided a life-changing experience.
So when it came to execution, why obfuscate the obvious? Less frequent dosing meant fewer side effects, more sleep for all concerned, and a general improvement in QoL – for the entire family. Simply put: Less delivered more.
The Result
PROCYSBI achieved 50% of the US market 4 months after launch, and 84% market share one year post-launch. Therapeutic compliance was demonstrated to be 87% for PROCYSBI vs 45% for Cystagon. Procysbi is now available globally and averages a 65% share of market.
In terms of online activity, there were 40K page views in first year of launch with a 15.44% bounce rate. Following consolidation of digital properties, there was a 118% traffic increase, 48% increase in form downloads.
An accompanying SEM campaign resulted in +65% page views, +69% new users; +221% accessing/downloading PEF; and overall bounce rate of only 6%.