4) Advocacy Partnerships
Vertex Pharmaceuticals recently announced results of the Phase III Strive study which demonstrated that the experimental drug
VX-770 dramatically improved lung function in cystic fibrosis patients with the G551D genetic mutation, which affects about
4 percent of cystic fibrosis patients in the US. According to Mark Schoenebaum, an analyst at ISI Group, "These are potentially
game-changing data in cystic fibrosis. VX-770 is the first drug to show good data in a Phase III trial that actually modifies
the disease by binding to a defective protein and fixing it." Vertex is currently testing VX-770 in combination with VX-809,
another drug in development for cystic fibrosis patients with a far more common gene mutation. If these drugs are approved,
Sanford Bernstein analyst Geoffrey Porges estimates annual total sales of the two orphan drugs (VX-770 and VX-809) to top
$3 billion worldwide.
The discovery and development of VX-770 and VX-809 would not have been possible without the novel collaboration between Vertex
and the Cystic Fibrosis Foundation, a non-profit, donor-supported patient advocacy organization. The foundation helped identify
the underlying genetic defect; characterize the biochemical process and its link to the disease; leveraged its clinical trial
network to expedite patient enrollment in Vertex trials; and invested more than $75 million with Vertex for the drug's discovery
phase. Presumably, once the products are approved, the foundation, which receives a small royalty on product sales, will complement
Vertex's product promotions with its own communication initiatives.
5) Patient Support Programs
Novartis is launching the first oral multiple sclerosis therapy, Gilenya, worldwide. While Gilenya has the advantage of oral
administration versus injections for competitors, Novartis faces concerns regarding the product's relative efficacy, patient
compliance rates, and higher pricing. According to Bloomberg, Gilenya costs about $4,000 per month, nearly $1,000 more per month than competing drugs such as Teva's Copaxone.
To address these concerns in the US, Novartis has one of the industry's most ambitious patient copay and support programs.
The company is offering to pay out-of-pocket drug costs for non-Medicare patients. These costs include copayments (up to $800)
for those patients with insurance and full coverage of treatment costs for selected patients without insurance who earn less
than 500 percent of federal poverty levels. Novartis will also pay as much as $600 for FDA-required testing and monitoring.
To expedite patient starts, Novartis is offering patients free starter product during the benefit's investigation period and
"nurse navigators" to provide logistical support, educational materials, and a call-in hotline.
These cases represent a small sample of the many ways that companies can win beyond the molecule. Companies should think holistically
and consider a wide range of competitive approaches, including technologies, regulations, manufacturing, distribution, pricing,
reimbursement, public policies, partnerships, and legal actions. As pharmaceutical competition intensifies, savvy professionals
are appreciating the need to differentiate their products through a combination of clinical and non-clinical approaches, with
the latter becoming increasingly important.
Stan Bernard is President of Bernard Associates, a global competition consulting firm. He can be reached at SBernardMD@BernardAssociatesLLC.com