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.

Follow the Money to Patient Engagement

4 Reasons why Pharma will finally become patient-centric

Pharma Financial Stars Lie with Patient-Centricity

Pharmaceutical marketers have been talking about Empowered Patients ever since I joined Pfizer Pharmaceuticals in the mid-90’s as one of their first consumer marketing hires. But despite all the talk, most pharmaceutical companies are nowhere near being patient-focused.

Pharma marketers know things are changing but are holding onto the HCP-focused status quo for as long as possible. In fact, I was recently asked by a Pharma Exec charged with driving patient engagement, “When do we really have to get serious about patients?” They felt that their primary customer was still the physician.

But ever the optimist, I believe that the next 2-5 years will represent a seismic shift in pharmaceutical marketing. Away from a singular focus on the physician towards a more patient-centric way of being. And that’s because patient-centricity is increasingly critical to a pharmaceutical company’s growth and financial health. As Watergate’s Deep Throat said, “Follow the Money.”

To my mind, there are four market trends that are helping to realign Pharma’s financial stars towards patient-centricity:

1. The Dawn of Health Care Shoppers-Historically, consumers exhibited very little true shopping behavior, even as they became increasingly responsible for their health care costs. This lack of true shopping behavior was largely because consumers had little visibility into costs and quality data and therefore couldn’t make the necessary trade-offs.

But that is changing.

Health care reform, combined with private sector efforts, are increasing transparency around both costs and quality, allowing consumers to start making trade-offs with their health care expenditures, including medications. Patients will move from merely asking a physician for a particular drug they saw advertised on television to making a highly considered decision to pay for drug A or drug B.

With this true decision-making, patients will be able to move markets. As this market moving ability starts to show up in pharma company regression analyses, Pharma companies will be stumbling over each other to be the most patient-centric.

2. The shifting economics of their customers-HCPs, Hospitals and Integrated Delivery Systems won’t be rewarded on the quantity of services they deliver anymore, but rather on the quality of those services and the patient experience. The smart pharmaceutical companies are going to look for ways to help their customers deliver better patient outcomes and experiences. And that is going to require additional investment to prove their patient interventions actually deliver.

3. The exploding orphan drug opportunity-Specialty and orphan drugs now represent the path to financial growth for many Pharma companies. And along with the orphan drug opportunity comes the empowered patient. These patients play a significant role in which drugs get into clinical trials, get approved by the FDA and reimbursed by insurers. If a company is in the Orphan Drug space, then by default they have to be patient centric. I predict that this “patient centricity” will eventually work its way into larger, primary care marketing practices.

4. Patient Reported Outcomes (PROs) in clinical trials will increasingly become commercial differentiators-In many categories, pharmaceutical researchers have already captured patient-reported outcomes, particularly on quality of life. However, these metrics have had little true commercial value since the FDA has been leery of approving claims based on patient reported outcomes. I believe that the FDA’s new focus on patient centricity, as witnessed by their “Patient Focused Drug Development” initiative may signal a growing acceptance of PRO claims. And as PROs become more important to the commercial success of a medication, so will the patients.

It is this alignment of Pharma’s financial stars around patient-centricity, that makes me believe that pharmaceutical companies will finally begin to truly embrace the empowered patient. Just follow the money. It never lies.