When people say the world is getting smaller, they cannot exclude a changing pharmaceutical industry in that discussion. Years ago, if a company wanted to be a player in the drug industry, it went blockbuster or it went home. Diabetes, hypertension, depression, and other large therapeutic areas enjoyed an R&D boom during the 1990s and early 2000s. Surprisingly, there were plenty of paying patients for everyone to wet their beaks in the bath. However, in 2011, there are fewer blockbuster drugs on the horizon and even fewer patients and payers willing to pay for them when cheaper alternatives are available. What does a pharmaceutical giant do? It looks to the undiscovered country, which includes rare (formerly known as orphan) diseases. Now that pharma giants are entering the rare disease market, it makes one ask, do they know what they are getting into?

To illustrate this point, let us consider two very different therapeutic areas: diabetes and Pompe disease. In the United States, 23.6 million children and adults—7.8% of the population—have diabetes. The US diabetes marketplace is conservatively valued at more than $9 billion per year and includes medications ranging from generic metformin to branded incretins and insulin. Now, let us look at Pompe, which is a genetic, lysosomal storage disease. Worldwide, there are about 10,000 patients with Pompe disease. The total market for Pompe is estimated at more than $1 billion, assuming 30% to 60% of patients have access to high-value therapeutics and an average cost of therapy that is comparable to other enzyme replacement therapies. Putting this into perspective, if Pompe had the same prevalence as diabetes (in the US only), its market value would be more than $2 trillion. The take-home message: just because sales are high, do not automatically think it is business as usual.

It is easy to see how the business models differ considerably for a disease state with 24 million patients and one that can hold a meeting for all in a Texas high school basketball arena. We should accept that many of our clients may be new to the world of rare diseases and will need our guidance more than ever. This is especially true for those companies who have acquired a compound rather than developed it in-house. Customer Relationship Marketing (CRM) has become the go-to strategy; however it is clear that CRM strategies should never follow a one-size-fits-all approach. So much needs to be different, from what you need to do during the earliest market-shaping activities through the post-approval marketing period. Our own experiences with Pompe disease have taught us that no two patients have identical needs. While many things about successful marketing of rare disease treatments remain fluid, one thing is clear—we cannot allow ourselves to be tempted to squeeze a square peg into a round hole. We need to tailor a proper approach to facilitate success for all involved.

How has your company helped clients adapt to smaller patient populations? Drop me a line at ken.k@core-create.com.