Rare is different: how empowered patients and caregivers are changing marketing for rare disorders

Wendy White

Siren Interactive

In a six-part series titled "Rare is different," we’ll show you how the world of rare diseases is unlike other pharma markets, particularly in the knowledge and influence wielded by patients and caregivers.

You'll learn about these differences through the personal stories of rare disease caregivers. We’ll also examine why physicians usually have little knowledge of rare disorders, discuss the role of organizations like NORD and EURORDIS in raising awareness and empowering patients, and explore solutions for ensuring the success of pharma marketers.

We'll give you critical insights into how rare disease patients and caregivers want pharma to become involved in their communities. And we’ll take a look at innovation that’s happening at the margins.

"...rare disease patients and caregivers want pharma to become involved in their communities."

Join us. We look forward to your participation and your comments.

Part 1

Rare disorders are a fast-growing segment of the biopharmaceutical market, due largely to advances in genomics and proteomics and less reliance by pharma on the “blockbuster” product. But marketing in rare disorders has special requirements. Rare is different in many ways, but especially in the power and influence of patients and caregivers, mothers in particular.

There is no “one” rare disease market, each disorder is its own small and extremely powerful community. Successful marketing requires a deep understanding of each community’s unique needs – and the realization that pharma's participation is important and welcome.

As relationship marketers for rare disorder therapies, we at Siren have gained some distinctive insights into needs and challenges in this space. Many of these insights are brought vividly to life in a collection of personal stories we recently published titled Uncommon Challenges, Shared Journeys: Stories of Love, Hope and Community by Rare Disease Caregivers (http://press.sireninteractive.com/).

One of the most important things we’ve learned is this:

“Many patients with rare disorders are not diagnosed by doctors, nurses, or researchers but by a fierce and driven group of extraordinary detectives known as mothers.”

I should know. I’m one of them.

"There is no “one” rare disease market, each disorder is its own small and extremely powerful community."

When my third child, Casey, was born, there was trouble right from the start. I heard the doctors and nurses talking to each other about a club foot and low blood count. Then they whisked her away to a teaching hospital for more tests.

I felt numb and out of control.

After four months in and out of the hospital, I made an off-hand comment about Casey's dimpled knees. My statement made something click for the surgeon. An MRI determined that she didn’t have kneecaps and, though she would probably walk, climbing stairs could be a problem.

This was a painful moment. I must have visited 10 hospitals, and I read everything I could get my hands on about knees and blood and club feet. I spent a huge amount of time following promising threads on the Internet.

Then I found a paper by a researcher who had identified the genetic marker for a rare disorder called nail-patella syndrome. Tracking him down at Johns Hopkins, I talked with him at length.

At my next visit with Casey’s doctor, I laid out all the information I had found on the Internet and told her about my conversation with the researcher at Johns Hopkins. She took my papers and went into the back of her office. She returned five minutes later and said, “I think you are right, I think she has nail-patella syndrome.”

"Many patients with rare disorders are not diagnosed by doctors, nurses, or researchers but by a fierce and driven group of extraordinary detectives known as mothers."

There are 8,000 rare disorders

Figure 1: Casey walking to school on her first day at school.

My experience with Casey is typical of the stories we hear over and over again from rare disease caregivers. By some estimates, rare disorders affect 1 in 10 people, and 50 percent of them are children. There are potentially 250 million people in the world with rare disorders. And there are millions more mothers, fathers, and grandparents looking for answers, searching for any information they can find to make sure they are doing the best they can for their children, whatever the outcome.

My personal journey with my daughter led me in a new direction for my company, Siren Interactive. Our entire focus is on growing the relationships among all of those who are working to support people with rare disorders.

Figure 2: Casey in goal.

Two years ago I joined the board of the National Organization for Rare Disorders (NORD), the U.S. version of EURORDIS (Rare Diseases Europe). We helped the organization launch Rare Disease Day in the U.S., and last year introduced an awareness campaign called Alone We Are Rare, Together We Are Strong.

"Like many caregivers, I have found that having a child with a rare disorder has become the focal point for everything I do."

Like many caregivers, I have found that having a child with a rare disorder has become the focal point for everything I do.

How pharma can be a trusted partner

Knowledgeable, passionate, and empowered by technology, rare disease patients and caregivers can be extremely influential allies for pharma companies working in the area. They will crusade tirelessly to raise disease awareness, mobilize resources to find new treatments and cures, and push hard for access and funding.

They are also undisputed power users of the Internet in seeking disease information, forming relationships, and sharing resources and support (see http://www.pewinternet.org/Media-Mentions/2011/NPR-People-Coping-With-Rare-Disease-Are-Internet-Power-Users.aspx).

For pharma marketers to succeed in this arena, they must:

• Be part of rare disease communities. Get involved. You share common goals. But never compromise the trust that patients, caregivers, and physicians have in you.

• Gain a deep understanding of patient and caregiver populations. Generalizations won’t work because each rare disease is unique. Listening, however, will work, giving you the insights you need for effective two-way communication.

• Share information with these communities They're desperate for knowledge, and information is scarce and not readily available.

• Support physicians in overcoming the challenges they face. Most have very little training or experience in diagnosing and managing rare disorders, so you can be a valuable resource to them.

Part 2 of this series - 'Rare is different–why doctors are often stumped by rare disorders' - can be viewed here.

About the author:

Since founding Siren Interactive in 1999, Wendy White has been recognized as a thought leader at the intersection of niche pharma brands, patient empowerment and online marketing. Her vision for how the Internet can facilitate interactions and provide crucial information that patients, caregivers and their healthcare providers previously struggled to find has propelled Siren to the forefront of relationship marketing for rare disorder therapies. For more information, email Wendy at wwhite@sireninteractive.com or visit www.sireninteractive.com.

What are some of the challenges in marketing to rare disease communities?