Big Pharma, Big Hope, Big Challenges

The number of rare disease and orphan drugs coming through Big Pharma pipelines, or in development, is expanding so quickly, it’s causing heads to spin. Marketers and physicians suddenly need to get up to speed on diseases and medications for which they may have limited knowledge. At the same time, the heads of patients and advocacy groups as well as payers are spinning, too. For patients with unmet needs, new hope awakens as the breathtaking numbers of M&A deals focused on orphan drugs combined with R&D triumphs yield effective new treatments. Cost, however, raises eyebrows across the healthcare spectrum—particularly those of payers.

In this issue, we present three features and several columns that explore the breadth of this segment with an eye toward market sizing, access, new treatments, marketing, pricing and long-term sustainability, starting with Sizing Up the Orphan Drug Market. Undoubtedly, Big Pharma must be able to recoup R&D investment costs, but backlash from payers and patients is becoming fierce. President Bill Clinton, who keynoted at The Inaugural Klick Ideas Exchange held recently in Philadelphia, counseled the audience, “Drug pricing cannot be done in a vacuum.” Backlash, he noted, could be addressed by making the pricing process more transparent, emphasizing improved patient outcomes and better communicating treatment value.

Great examples of what better creative communication looks like recently emerged from the 2015 Lions Health festival held recently in Cannes at which pharmaceutical companies and their agencies from around the globe walked away with 25 Pharma Lions awards out of 432 entries. AstraZeneca and DigitasLBI New York snagged the top award, the Grand Prix, for its highly creative “Take it From a Fish Campaign.” Go to to see the full story on this year’s winners.

But hold on! Even more great creative is coming your way quickly as PM360 gears up for our 2015 Trailblazer Awards Gala on September 25. Purchase your tickets for the gala at Once again, we will recognize the outstanding achievements and innovation in healthcare marketing in five award categories—at NYC’s singular Gotham Hall.


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