Create (Smart) Molecules
As early as one year after the launch of the anti-epilepsy treatment Neurontin (gabapentin), small investigator-led clinical
studies suggested that GABA agonists—and Neurontin in particular—offer benefits for patients suffering from various types
of neuropathic pain. Monitoring off-label prescriptions of Neurontin revealed extensive prescribing for these conditions,
with physicians writing an estimated 86 percent of all Neurontin scrips off-label.
Pfizer used this information to create and mitigate the regulatory risk for its follow-on product Lyrica. Pfizer chose to
focus on the safer regulatory path—gaining an indication for epilepsy—since it already had a wealth of clinical data from
Neurontin. However, the company also set out to prove Lyrica's effectiveness for painful diabetic neuropathy, for which received
approval in 2004.
This strategy helped not only mitigated the regulatory risk, but also maximized sales. Generic gabapentin manufacturers could
not afford to bankroll trials for neuropathic pain, which isolated Lyrica from third party reimbursement pressure, despite
its higher price. As a consequence, Lyrica sales reached $1.2 billion in 2006, and grew to $1.8 billion in 2007. More recently,
the company has taken this approach a step further by obtaining a label for fibromyalgia, another indication where Neurontin
data was suggestive of efficacy.
Prove Class Effects
Sometimes, even without a reformulation, establishing efficacy in what is perceived to be a class effect can create tremendous
value. Companies can use this strategy even when they haven't had a significant presence in the past in a particular franchise
For example, in 1998 King Pharmaceuticals acquired the rights to the ACE inhibitor Altace (ramipril)—back then considered
a "me-too" drug, with only 4 percent of sales within the class. At the time, many hypothesized that ACE inhibitors had cardio-protective
effects beyond blood pressure control, but no one had done the clinical trials to prove it. King took on the challenge: it
designed and undertook the landmark Heart Outcomes Prevention Evaluation (HOPE) trial. With that data, FDA approved Altace
for prevention of myocardial infarction, stroke, and death due to cardiovascular causes, and the company parlayed this expanded
label into a 15 percent class share by 2004.