Like many drugs today, KORLYM posted a slow start. Sales hit $2 million a year after launch but rose faster in the fourth
quarter of 2013, where sales hit $4.1 million—a 58% rise over the previous quarter, lifting the full year total to $10.4 million.
The company is projecting sales at more than double that for this year, in the $24 million to $28 million range. There is
additional room to build; due to its orphan drug status, KORLYM has marketing exclusivity in the US until February 2019.
One key learning from the launch was the importance of defining the boundaries of the prescriber network. Early on, the assumption
was that, as in most other rare diseases, the sales potential for KORLYM rested on a select few academic teaching hospitals
and the specialist networks around them. Close review of claims data (Cushing's has its own ICD9 diagnosis code) indicated
that this would also be the norm for KORLYM. So the focus was to create a team of "medical science liaisons" able to bridge
the gap between clinical expertise and the real world of patients.
But actual experience proved otherwise, which slowed uptake in the first few months. "We noticed that, while patients did
interact initially with the academic medical centers, there was a tendency for them to disperse back to caregivers at the
community level—endocrinologists not affiliated with any of the large networked hospital centers," says Lo. "This was a group
of prescribers we weren't covering; in fact, at the time we lacked the bandwidth to do so." Lo noted that Corcept was small
enough to respond quickly, making investments in a new 20-member field force geared to this constituency. "The lesson is
to subject the claims data to a 'strength test' by actually tracking the patient journey through the health system," he said.
Another lesson was finding the right message that worked with payers. Starting early, management briefed key PBMs on the disease
and the drug, focusing on its narrow indication, the small target population, and the huge unmet need along with adjacent
co-morbidities, where the amelioration of symptoms in chronic conditions like diabetes translates into big savings on treatment
costs. At the ex-factory wholesale price of 87 cents per milligram, treatment costs per patient for KORLYMN can average a
pricey $200,000 a year. Nevertheless, the impact is relatively diffuse: according to management, seven large health plans
estimated their patient burden under the drug would amount to no more than 20 people.
"There is a clear unmet need for patients in whom standard treatment approaches haven't worked, so payers are recognizing
this by placing few reimbursement restrictions," says Lyons.
To defer cost concerns, Corcept provides the drug to uninsured patients through a patient assistance program, SPARK (Support
Program for Access and Reimbursement for KORLYM), which also funds co-pays of up to $5,000 annually per patient, based on
need. Cases are referred through partnerships with patient advocates like the National Organization for Rare Diseases (NORD).
Messaging on mifepristone
Finally, management took a forthright stance to counter the stigma associated with mifepristone's dual use in terminating
early-stage pregnancies. The FDA proved to be a central player in deflecting the negative connotations. It took these head
on in its approval release for KORLYM by emphasizing the tight controls on distribution jointly agreed with Corcept—the company
actually mimicked the approach taken when mifepristone is prescribed as an abortifacient—along with a black box warning against
use of the product during pregnancy. According to Belanoff, "what we found is the FDA actually proved to be a sympathetic
ally in establishing the legitimacy of this drug." Corcept is pursuing the same high engagement approach in its pending application
for a marketing license for KORLYM at the European Medicines Agency (EMA).
Corcept has also had success with the argument that precedents like thalidomide offer proof that a repurposed medicine with
a different indication can be administered safely while providing an option for patients without hope. "The ravage this disease
imposes on patients is sufficient to make the case that KORLYM should be available, because alternatives are few and far between,"
said Dr. Pejman Cohan, a Los Angeles-based endocrinologist who specializes in Cushing's. He points to the difficulty in diagnosing
Cushing's as a defense for the drug. "Getting to the root of this disease is a complex, sometimes contradictory task at the
individual patient level," said Cohan. "Lab tests are often not definitive, with many false positives, so access to a drug
treatment that is safer than invasive surgery and also reversible is a choice both I and my patients like to have."
Physician talk therapy
To reach this year's goal to double KORLYM sales, management is continuing to refine the demographics of the prescribing community
while improving its understanding of the patient journey. In contrast to other rare diseases, few endocrinologists treat Cushing's
exclusively, especially now that other metabolic disorders like diabetes attract more patients. On top of that, clinical protocols
for Cushing's are entrenched—this is not a community of early adopters. "Our biggest hurdle in marketing is taking the physician
from zero prescriptions to one, because after they see KORLYM works in that first patient the resistance to trying something
new will fade," Lo told Pharm Exec. In other words, education is the focus: it's going to take a little extra time to show practitioners there is a new kid
on the block.
William Looney is Pharm Exec's Editor-in-Chief. He can be reached at firstname.lastname@example.org