The Orphan Impact: Small drugs drive big changes

Seems everybody’s talking orphan drugs these days. No wonder, according to EvaluatePharma’s 2013 Orphan Drug Report, orphan drugs are estimated to reach 15.9% of total worldwide prescription sales by 2018. But the orphan drug impact goes beyond sales numbers. I believe that the patient centricity blossoming in the rare disease divisions will eventually spill over into the primary care divisions of pharmaceutical companies.

With the big blockbuster primary care drugs, development success lay primarily with company research departments. The halls of Pharma echo with stories of researcher heroics—how the lone scientist kept a molecule from hitting the drug dustbin only to become the next blockbuster.

But in orphan drugs, the heroes are as likely to be individual patient family members as researchers. In some cases the drug literally starts with patient families. Thanks to advances in digital technology and social media, orphan drug patient families play a major role in every aspect of bringing a drug to market, going far beyond traditional advocacy roles. Now individuals are able to leap tall barriers with a click of a mouse to accomplish superhero feats formerly reserved for massive organizations like the NIH and pharmaceutical companies.

Consider the role of patients, families and organizations in:

Disease discovery: Matt Might, a father whose son had a disease entirely unknown to science, leapt over barriers of scientific self-interest to find other patients and give his son’s illness a name.

A well-known blogger in his field, Matt’s post about his quest helped identify patients like his son across the globe. In the New Yorker article, which describes Matt’s disease naming odyssey, a Duke geneticist, who worked with Matt, sums up the new patient paradigm with this quote:

“It’s kind of a shift in the scientific world that we have to recognize—that, in this day of social media, dedicated, educated, and well-informed families have the ability to make a huge impact…Gone are the days when we could just say, ‘We’re a cloistered community of researchers, and we alone know how to do this.’ ”

Research direction: John Crowley funded individual scientists to fill the treatment void when he learned his daughter had Pompe disease. Ultimately John ended up partnering with one scientist to form a company that eventually was folded into Genzyme. And while his story is certainly one of the most dramatic (to the point of being the subject of a major motion picture starring Harrison Ford), John’s ability to drive the course of scientific discovery is becoming more commonplace in the rare disease space.

Product approval parameters: In June 2014, the Parent Project Muscular Dystrophy (PPMD) patient group, submitted the first ever-patient advocacy-initiated draft guidance for a rare disease to the FDA for Duchenne muscular dystrophy. Patients, through organizations like the PPMD, are now directly driving how Pharma should be conducting their research.
Because of the outsized role patients and their families play in bringing a drug to market, building strong patient relationships is a key marketing investment for orphan drug marketers. For example, Biogen Idec, deployed over 15 community managers to support people with living with hemophilia even before they had an approved product.
There are already signs of an “orphan drug spillover effect” on primary care marketing. Consider Sanofi’s community manager position in their Diabetes franchise. Or more recently, that Sanofi appointed a Chief Patient Officer. And this I believe, is just the start of the orphan drug effect.

Soon the patient centric tactics of Rare Disease marketers will be highlighted in “marketing excellence” meetings all over Pharma. Then questions will come during marketing plan presentations about “why can’t primary care teams start building patient relationships like their rare disease counter parts?” And before you know it, the small seeds of patient centricity will finally blossom throughout Pharma.