The Great Orphan Drug Debate

Our first series of debates took place in April during the World Orphan Drug Congress in Washington, DC. Over 40 representatives from rare disease and orphan drug companies such as Pfizer, Shire, Raptor, ViroPharma, and Alexion attended the event.

Unique to many debates we've been exposed to during this election year, these debates were full of FACTS, and despite what was said in the "spin rooms" afterwards, everyone emerged a winner!

Debate #1—"The Value and Influence of Patient Communities: Real or Overrated?"

Kristin Keller, Executive Vice President at Compass Healthcare Marketers, represented the position that patients are not central to a successful commercialization strategy. She asserted that the two critical ingredients to the success of a pharmaceutical product—orphan or otherwise—are data and doctors. First, there is data—a product must have proven efficacy, a well-balanced safety profile, and the belief of the key opinion leaders in that particular space. Second, there are the physicians, who are responsible for driving the treatment decisions and managing patients. This is not to say that patients and caregivers don't have any involvement; certainly, they participate and provide support and access services appropriate to them. However, at the end of the day, patients simply do not have a measureable impact on the commercial success of a product—they don't move the needle.

Kristina Broadbelt, Global Director, Public Relations and Advocacy at ViroPharma, posited that in rare diseases the patient community has enormous influence over the commercial path—and ultimate success—of a product. She cited numerous examples where patients can provide a great deal of support and positive momentum, including the accrual of clinical trials, conversion of patients to the commercial drug, even influence over regulatory perceptions. In scenarios where the product exists in a competitive landscape—a reality for an increasing number of orphan products—the relationship with the patient community and their perception of the company and its intent can be quite impactful.

At the end of the debate it was clear that Kristin represented a traditional position that still echoes through the boardrooms of pharmaceutical companies. Her viewpoint was narrow and simply did not take into account the massive influence that patient communities have in the rare disease space, or how the overall power of patients has increased in the last 20 years. Kristina made a compelling argument for the fact that patients are now a major stakeholder and, when treated as such, can have a positive impact on the commercial success of an orphan product.

Debate #2—"Can the Orphan Drug Pricing Model Be Sustained? A New Beginning or the Beginning of the End?"

Doug Paul and Jack Mycka of Medical Marketing Economics debated the sustainability of current price levels in the rare disease and orphan drug market. While each therapy alone may only be an "unnoticeable" fraction of the budget, when taken together their impact is considerable.

Doug posited that the sky as well as drug prices are going to fall. He cited the entrance of Big Pharma into the market and their historical practice of using lower prices to win markets. In addition, he noted that he feels there is a pricing limit that payors and patient groups will accept, and the industry has reached it.

Jack, referencing popular '70s television programming, challenged Doug and his points, suggesting Doug was an ignorant commoner regarding pricing models and the unique characteristics of the orphan drug market. He cited the increased demand for highly specialized therapies. He went further to point out the elasticity in the market with examples of the HAE market with five entrants and each product similarly (high) priced.

In the end there was agreement that market forces will allow for current pricing models to continue, but challenges do lie ahead. A recent example Doug cited was of an anticipated drug that requires only a single dose, and could be priced above $1,000,000!