4 central questions for navigating market development for treatments of rare diseases

When GSK and Pfizer pursue orphan drugs it makes headlines:

October 13, 2009: GlaxoSmithKline licenses a drug for the rare Duchenne muscular dystrophy with plans to pursue orphan drug designation.

December 1, 2009: Pfizer licenses a treatment for Gaucher's disease and gains orphan drug designation and fast track status.

But these deals are far from the most significant orphan drug initiatives.  In fact, more than 350 products have been approved under the Orphan Drug Program. Not only are companies meeting important patient medical needs, orphan drugs make good business sense, too — they can provide 7 years of market exclusivity, tax credits, and other advantages.

I try to look beyond the “orphan drug” regulatory designation, which has its own challenges and pitfalls.  I focus on the uniqueness of strategy to develop the brand, develop the market, and develop the company’s support capabilities.

But how do you do it, and does it make sense for your brand?

In our StrategicGPS^® facilitated process, we try to get to the central questions:

1. Who are the “customers”? The treatment of rare diseases is a more complex matrix of health care providers and payers.  And the advocates for the patients are strong, and must be heard.
2. Where are we now?  Consider the current state of the process of care, along with all its components.  A new treatment might fit in or it might require a complete reinvention.
3. Where do we want to be?  This is not a question of sales, share, or “leadership”.  Rather, it’s a vision of what the patients’ and physicians’ world could look like with a new treatment.
4. How do we get there?  The notion that "clinical superiority" alone can get you there has been proven faulty.  A strategy that engages all the relevant issues is essential.  And the earlier the better.