PRINCETON, N.J., September 6, 2012 — Edimer Pharmaceuticals, a biotechnology company focused on developing an innovative therapy for the rare genetic disorder, X-linked Hypohidrotic Ectodermal Dysplasia (XLHED), today announced it has selected patient marketing agency, Compass Healthcare Marketers as their partner to drive patient identification for EDI200, the company’s novel, proprietary, recombinant protein being developed for the treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED). XLHED is a rare orphan disease that causes a range of symptoms including lack of sweat glands, poor temperature control, respiratory problems, and hair and tooth malformations.  EDI200 represents the first potential treatment for this rare genetic disorder and one that could give newly born children with XLHED a chance to live a more normal life.

“We need to increase the awareness of XLHED as a serious medical issue,” said Neil Kirby, PhD, President and CEO at Edimer. “Our goal is to be connected with as many families affected by XLHED as possible and increase awareness of the genetics of this disorder. We chose Compass to help us engage this community because of their unique experience reaching and uniting individuals impacted by rare and orphan diseases.”

Edimer has teamed up with Compass to assist and expand current patient community outreach efforts and to help with recruitment for their upcoming clinical trials.  Initial projects undertaken by Compass will include the redesign and relaunch of XLHEDNetwork.com as well as the creation of the “Have the Conversation” website. Both websites are aimed at patients and caregivers as well as women who may be carriers of, or affected by, the genetic mutation responsible for XLHED.

“Identifying and engaging those mothers and potential mothers who may carry the XLHED gene is not a simple task,” said Peter H. Nalen, president and CEO of Compass Healthcare Marketers. “Yet given the intimate knowledge that Compass has of the rare disease space and our expertise in patient and community outreach, we are confident we can help Edimer succeed in connecting with this community and bringing this very important drug to market.”

About XLHED

XLHED (also known as Christ-Siemens-Touraine Syndrome) is a rare disorder of development resulting from genetic mutations in the ectodysplasin gene (EDA).  Patients affected by XLHED are at risk for life-threatening hyperthermia based on their inability to regulate body temperature, and for clinically-significant pneumonias resulting from their abnormality in respiratory secretions.  Cardinal signs and symptoms in XLHED include diminished/absent sweat, reduced and abnormal airway secretions, few and often misshapen teeth, and absent or early hair loss from face and scalp.

XLHED patients surviving infancy are predisposed to atopy presenting with eczema and asthma, chronic sinusitis, recurrent nose bleeds, and dry eye complications.  Almost uniformly they require dental interventions including early prostheses and later implants.  Their susceptibility to hyperthermia, facial appearance, abnormal dentition and hair loss may impact normal participation in outdoor activities, sports and school attendance.  Both medical and self-esteem issues are life-long in this disorder. As is generally true with X-linked inheritance, males are fully affected while females are variably affected.

About Edimer Pharmaceuticals

Edimer is a privately held biotechnology company based in Cambridge, Massachusetts dedicated to delivering a significant and durable improvement in the health and quality of life for future generations affected by XLHED. Edimer was established in 2009 with investment from Third Rock Ventures and VI Partners.

For further information on Edimer Pharmaceuticals, please visit www.edimerpharma.com. To receive quarterly updates about Edimer Pharmaceuticals’ progress please join the XLHED network at www.xlhednetwork.com.

About Compass Healthcare Marketers — www.compasshc.com

Compass Healthcare Marketers, with offices in Princeton, NJ and San Francisco CA, is a patient marketing agency focused exclusively on pharmaceutical and biopharmaceutical companies that serve the rare disease community. Successfully engaging the patient community positively impacts all stages of commercialization from clinical development to product positioning to post approval patient support. As such Compass services include:

• Interactive Marketing
• Patient Branding
• Patient Programs
• Research, Strategy, & Analytics
• Community Engagement & Social Media

CONTACT:

Edimer Pharmaceuticals
Neil Kirby, Ph.D, President & CEO
617-758-4300

The presentation discussed the elements of value-based patient services programs for patients and healthcare providers, and considered the best practices for designing and delivering such programs. The presenters tried to answer the following questions:

• Are we all talking about the same thing?
• What should these programs deliver?
• What is the difference between some value and more value? How much value do you need to deliver and invest in to achieve real results?
• And when it comes to communicating with the patient—either directly or via the healthcare professional—what do we need to know in order to be effective?

Kristin Keller on what value-based patient services programs are and what they are not

Kristin opened by talking about the history of patient services programs offered by pharmaceutical companies and discussed how they evolved into robust, multidimensional programs offering a range of services and support. These offerings range from access and reimbursement to much more progressive, patient-centric programs like regional patient speakers, patient mentors for those new to therapy, and so on.

Kristin suggested there is still great inconsistency in how we define these programs and what their core purpose is. Are they about reimbursement and access, or distribution, or educating patients? Or is it all of these? Are there different programs for each of the components or are they integrated into one umbrella program? While manufacturers may see these offerings as unique from an operational perspective, and there may be many stakeholders in different departments within a company designing and implementing them, Kristin held that if it doesn’t feel seamless, if there isn’t one primary touch point, and if it makes no sense to the patient, it won’t matter how well each piece functions individually.  Kristin summed up her presentation stating, “To be successful, these programs must be integrated and user-centric.”

Chad Holland on designing the program – what’s the right amount of investment

Chad set the stage for his presentation by first asking the audience to consider an analogy contrasting a desolate desert to a tropical paradise. Chad used the analogy to help the audience imagine the experience of rare disease patients likening their lives to a difficult journey across the desert that requires going over a mountain to get to the tropical paradise representing treatment.

Chad used this analogy to contrast the rare disease treatment access challenges with the dilemma faced by manufacturers when deciding their level of investment and approach to improving access to their treatments through patient services programs. He took the audience through several principles for estimating investment in these patient services.

Chad concluded by reviewing the big picture in three ideas:

1) Therapies are expensive to develop and may not exist without the possibility of fair profits.

2) Orphan drug legislation has provided societal and legal permission to treat and care for rare disease patients.

3) Manufacturers should start and end every patient services program budget discussion by considering the patient needs.

Julia Olff on best practices for communicating with patients and caregivers

Julia brought the rare disease patient and caregiver perspective to the presentation with quotes expressing their challenges and frustrations trying to get answers about and support for managing their disease. Julia talked about how people living with rare diseases are some of the most highly engaged patients and caregivers. She described the range of skills they must develop, from getting an accurate diagnosis to giving complex medications, and coordinating their care among multiple providers. To find support they can’t get from the medical community, Julia pointed out there is plenty of data to demonstrate how they endlessly scan Internet and social media sources to find answers, get practical support, and follow the trail of treatments in development.

Julia introduced the concept of health literacy, defined as the ability to read, understand, and act on healthcare information. She noted that the majority of Americans experience health literacy challenges, which affect their ability to make sense of medical information and access healthcare. Julia gave some examples of health literacy principles, such as writing actionable content that helps patients know what steps they can take instead of providing lots of medical facts that may not be relevant. She recommended manufacturers follow health literacy guidelines when developing patient programs and communications. Her call to action to the audience was, “Make it easy if you want patients and caregivers to know your programs exist, understand their value, and are able make use of them.”

What did the Super Bowl show us? The work and dedication they put in since training camp in July is impressive. The steady focus on the ultimate goal—the Vince Lombardi trophy—is admirable. The teamwork and precision between Eli Manning and Mario Manningham that enabled them to make what will be remembered as “the play of the game” is remarkable.

But do you know what’s really impressive, admirable, and remarkable? What true dedication and commitment is? Real Teamwork? I just spent a weekend with a group of rare disease patients and caregivers, and if you’re looking for some heroes, folks committed to the “team” and who are focused on a common goal, look no further.

It seems a bit trivial to celebrate football players, with all their support and money, while these patients and caregivers—like many in the rare disease and orphan drug space—have a much harder game to play, one they didn’t spend their lives training for, and much of the time play alone with few coaches, trainers, or support.

Committed to the team? Speaking with and watching the interaction between caregiver and patient was an inspirational demonstration of commitment. Partners, husbands, and wives who a mere year prior were skiing, hiking, or sailing with their mate, are now helping them walk, eat, and change clothes. Did anyone seem sad or resentful? Not at all.

We think the guys on the field are tough? Not compared to these patients. Tough is knowing today might be the best day, unsure of what tomorrow will bring, but remaining hopeful and optimistic. Tough is Mitch traveling from Minneapolis to Ft. Meyers to visit his mom on the way to our meeting. But along the way someone totaled his car in Ft. Meyers so he took an overnight bus to make it to our meeting in time. Why? To learn more and better ways to take care of his big brother with a rare disease.

Grateful? That’s where they have the NFLers beat by a mile.

When you can see the magic of a drug they used in trial and had success, and hear words like “miracle,” “thank you,” and “you gave me my life back,” that’s grateful. I’m reminded once again why we love what we do; why our industry is such a great one that provides a real and significant impact; why we as partners to the drug manufacturers must continue to innovate and develop tools to support patients so they will continue to partner with us, their physicians, and others with the disease, so together we can assist in ensuring the best possible outcomes. This includes helping patients and caregivers connect with others through mentor programs, providing sources of support and information via interactive tools and destinations, learning their true needs via authentic engagement with them and their communities, assistance programs to help find and obtain the right drug or trial, and supportive tools to ensure the therapies are used appropriately to ensure the best outcome.

While we may be merely people on the sidelines—hypothetical equipment managers or assistant coaches—we are committed to helping patients play their game a little better, a little easier, with as much hope as possible.  It’s a role we love and to which we are equally dedicated as those who performed on Super Sunday.

There are so many stories, so many people who are passionate about impacting the lives of patients with a rare disease. We thought, what better time to share those stories and our support for those patients than on Rare Disease Day? And we invite you to participate with us.

To support Rare Disease Day, we have created a dedicated YouTube channel where you can tell the world why you do what you do. We are reaching out to everyone we know, and we’d like your story to be part of it. Our goal is to have as many videos as possible posted by Rare Disease Day on February 29.

So please grab your iPhone or video camera and join us. Just answer this simple question: Why do you do what you do to help patients with rare diseases?

Thank you for your participation.

-Peter

P.S. Along with your Rare Disease Day video, please sign and submit this release form. (Our lawyers tell us we need them to be able to post the video on YouTube.) Click here to view all the videos.

As a brand manager you have limited resources but want to create a meaningful message. How do you best use those marketing dollars? To do this you should strive to understand the Patient Experience and map marketing initiatives to that experience. In this way you can guarantee that every marketing dollar is spent on the strategies and tactics that will result in the best possible patient outcomes.

There are a few places where you can get sidetracked and the whole project can get derailed. This can happen when you lose focus on whom you should be solving for (hint: it’s the patient). So whom could we wind up solving for if we are not careful? And, how do you know if you are getting sidetracked?

1. Solving for internal stakeholders
   1. Signs of getting sidetracked
      1. “This message and visual does not resonate with me.”
         1. This is said by a 35-year-old male about a brand message targeting elderly women.
      2. “I never click on Paid Search ads, so I do not think they will drive traffic.” (Feel free to change Paid Search to any marketing tool or site functionality.)
         1. Paid Search ads get ~12% of all search clicks; with hundreds of thousands of related searches, this is a ton of traffic.
   2. Signs of solving for the patient experience
      1. “Does this message and visual resonate with the patient?”
      2. “Do we have any evidence that our patients are looking for information and not finding any valuable sources? Can we provide that information and ensure they find it by using Paid Search?”
2. Solving for your competitors
   1. Sign of getting sidetracked
      1. “Competitor X is already doing it, so obviously this is a top priority.”
         1. Is there any evidence this tactic is working for the competitor? Do patients actually use it or find value in it? Most importantly, would your patients use it or find value in it?
   2. Sign of solving for the patient experience
      1. “If our competitor is already providing this service, is there still a patient need for us to provide it? Would our patients appreciate it?”
3. Solving for the agency
   1. Signs of getting sidetracked
      1. “We need to add “Functionality Z” to your site because we have done it on a bunch of other sites and it works great!”
         1. Read: we can make a big profit margin on this because we repurpose work we have already done.
      2. “We know that the industry is heading in this direction, so this is a chance for you to be ahead of the curve.”
         1. Read: “We really want to add this to our capabilities and we need you to do it so we can create a case study.”
   2. Signs of solving for the patient experience
      1. “Will this functionality be used by a large percentage of my patients? It not, will the minority of patients find it extremely useful?”
      2. “Being ahead of the curve is great as long as we have some research to support that this would solve a patient need. Do we have such research?”

These examples show how different the thought process is when you focus on solving for the patient experience. Compass has a few different research strategies that can help provide the knowledge needed to show the value that marketing initiatives could provide to your patients. In that way we do not let our own preferences and beliefs (or yours!) become the catalyst for making decisions. So how do you, as a brand manager, guarantee that the patient remains the focus? Whenever you are faced with a decision, ask the question: “Does this solve for the patient?”

The FDA recently issued draft guidelines that impact how pharma can communicate in social media, but leaves many in the industry underwhelmed.

The guidance addresses only off-label information. It doesn’t cover on-label promotions, which means the industry can continue to communicate using this channel.

These guidelines provide instructions for pharma on addressing unsolicited off-label questions presented within a public arena, like a forum or chat room.

What industry can do:
If a question about off-label use is stated in a public arena (i.e., within a social media forum), the company may publicly provide a response. This response should:

1. Convey that the question pertains to an unapproved use of the product, noting the approved indication and safety information, and
2. Include the contact information for medical affairs, and
3. Only link to the product’s package insert. Links to promotional websites or third-party websites are prohibited.

Lastly, the firm should record the person requesting information and any follow-up questions from the requestor.

Industry agrees that these guidelines shouldn’t prevent companies from getting involved in social media. As long as they act in the spirit of the law, communicating in social media shouldn’t be inhibited by the lack of more specific additional guidelines.

She was given mere months to live.

Now, I’m sure you’re thinking, “How would I react if I or someone I love were given this sudden news?” and the answer in your mind might be to fold your cards and accept your destiny.

Did this stop my mother from living her life? Not a chance. She had deep roots in faith and religion and she never once complained, blamed God, or even her physician who missed her cancer six months prior. She never stopped fighting cancer. My mother didn’t live for just the six months given to her. She fought ovarian cancer for two entire years, always smiling and spending time with her children.

One could literally see the cancer eating away at her body. She physically was only a shell of the person she was when healthy. But I know that cancer never defeated my mother. It may have taken her body, but I know she was destined for greater things.

She inspires me regularly. There isn’t a day that goes by that I don’t hold a conversation with her, and I know she is listening. She has taught me what it means to be selfless, to cherish the life you’ve been given, and to help others in need.

That’s why I do what I do.

There is a parallel between Ralphie’s situation and the medical community. Some (not all) doctors and regulators believe that sharing medical knowledge and treatment information with patients is not in the best interest of the patient. At best, these physicians consider a patient’s personal medical research to be a waste of time. At worst, they think this information will be harmful to patients or reduce the stature of the medical professional.

But that’s where the parallels end. Unlike a BB gun, information and resources in the hands of patients can only help patients. Patients who research information and therapies may be told by their doctors “you’ll shoot your eye out,” but rest assured, the environment that fosters these interactions is slowly evolving.

The benefits of patient empowerment should challenge those in authority who currently do not value the patient role in medical care. Patients value physicians who work together with them in a partnership. The outcomes from these partnerships will eventually tilt the medical conversation so patients’ needs are better met.

And that leads to better outcomes for everyone involved.

From Compass, we wish you warm holiday gatherings, delicious Chinese turkeys, and for all of your holiday wishes to come true. See you in 2012!

Welcome to Social Media. The party is all your potential customers (or patients, in our case). The people at the party know each other, like or dislike each other, and are, in some way, connected with one another. And, of course, this party never ends.

The problem is nobody knows much about you. And you don’t know them—not really.

So when the big moment comes, what are you going to say? Are you going to walk in the front door and start shouting your brand’s benefits? Who exactly are you going to shout them at? After spending 10 minutes at the party, are you going to head home, feeling like you’ve checked the box?

Many brands do just that. They don’t grasp that to use social media effectively absolutely requires a commitment of time and energy to understand the community you are engaging with. Who are the popular kids (the influencers)? What are they interested in, what do they care about? Is there a good reason for them to care about your brand?

Let’s go back to the party. Grab a brew. Now, the first thing most brands think to do is to ask the Homecoming Queen to the Prom. In other words, we’ve identified a key influencer who is extremely popular and active in the community, and look, she’s standing right over there, and the brand would really love for that influencer to advocate for the brand.

OK, but why would the Homecoming Queen allow you to take her to the Prom? Is that fantasy, or reality? She’s very popular; you’re not. She has her reputation to protect. What’s in it for her?

That’s what you have to understand before you approach any influencer: what are they interested in? How do they feel about your brand? Is there something that you can do for them? What is it? You have to carefully engage with each influencer and try to find common ground. (A social media landscape analysis is a good start.)

Hopefully, you’ll find it—but there’s a lot more you can do. There’s a misperception that the most obvious influencers are the only targets of a social media engagement. Quite the contrary, usually we find that the biggest influencers are only willing to play ball with the brand up to a certain point—they are often too independent, they have their own agenda driving them, and they don’t want to be seen as working for your brand. That could diminish their credibility.

So it pays to find other voices in the community who are willing to advocate for your brand—the more, the merrier. These are patients who are already positively predisposed toward the brand, either from what they’ve heard from others, or from what they’ve experienced themselves (by participating in your clinical trial). Note that we’re not looking for patients who are neutral and then trying to “convince them” to advocate for the brand; we are looking for the ones who have already drank the cool-aid. They’re out there. By finding the real brand advocates, listening to them, providing them support and value as part of an ongoing relationship, your brand advocates will reciprocate by communicating the brand’s POV within their community.

These are all extremely valuable connections for the brand to establish. In the rare disease space patients tend to band together and rely heavily on each other for information and advice. Brands that participate and contribute value (beyond just the drug) through social media channels can reach and influence a relatively high percentage of their potential patients.

But it requires an ongoing investment in managing key relationships on an almost daily basis. Think Facebook, Twitter, LinkedIn. Nothing ever stops, and you want to be part of the conversation. Every community has its own personality and influencers, and new issues pop up every day. This is real-time learning that can help you evaluate how your brand overlaps with the needs of the community, and how to talk about (position) your brand to best fit with those unmet needs.

None of this can be done solely by “monitoring” and “observing” the community from afar. Personal relationships are an absolute necessity in order to gain trust, and yes, even friendship. Meeting patients in person (often at advocacy group meetings), listening to what people are dealing with as they attempt to manage their disease (only a small part of which is your brand), and being able to demonstrate that you understand the patient experience are all critically important if you hope to dance with the Homecoming Queen—or anyone else, for that matter.

At 35, while pregnant with my sister, my mom was diagnosed with Type 2 Diabetes. In the 25 years that followed my mom suffered from nearly every complication on the list. It started slowly, but eventually there was a seemingly constant onslaught of medical problems, including high blood pressure, foot infections, hypoglycemia, nerve damage, circulation issues, iron deficiency, eye complications, chronic UTIs, and kidney disease (to name a few). In the last two years of her life she was in and out of the hospital constantly. She’d been diagnosed with end-stage renal failure and continued to struggle with life-threatening wounds due to extreme numbness in her feet. She remained good-natured through it—and even liked to joke about the time she lost a shoe but didn’t realize she was barefoot until she had walked across a parking lot and got into the car with my dad.

My dad was (is) amazing. My mom didn’t want to have to be in the hospital for dialysis treatments so my dad learned how to administer peritoneal dialysis at home (which needed to be administered EVERY night—by my dad, the CARPENTER). He learned how to take care of her wounds—including all of the equipment needed to help stimulate her circulation. He ordered all of her medical supplies and equipment. He dealt with the medical supply companies when things didn’t work properly. He checked her vitals and blood sugar several times per day. He drove her to every appointment. He did all of her shopping. He did all of her cooking. Eventually, he fed and bathed her. He even made a crazy contraption out of wood that enabled him to take her and her wheelchair up and down the stairs—one stair at a time!—so that she could stay at home near the end and sleep in her own bed. (I remember it made a horribly loud noise as is lumbered from one stair to the next and I was always sure he was going to hurt himself.) I never saw anyone more determined.

My mom ultimately succumbed to her disease in 2007 at age 59. It’s hard to say what complication was really the culprit, as her body was so badly ravaged at the end. My dad was destroyed. Not only did he lose his soul mate, but he also felt like he lost his entire purpose in life and his reason for being. He’d spent the last two years caring for my mother 24/7. The concept of “free time” hadn’t existed in his world for over two years. But suddenly there were no medical appointments to fill up his week nor treatments to administer nor vitals to check. Other people were moving on with life but he had to figure out a new life. It was incredibly difficult (and that doesn’t begin to cover it). It took him over two years to find his “new normal”—and of course he still has moments of extreme emotion. In those moments, I just hug him and tell him how much I love him.

In the coming years, I would love to see us (Compass) and our clients do more for the caregivers—not only during the diagnosis and treatment stages, but also after they have lost their loved ones. At the end of life, one hopes that the patient moves on to a better place. But I can tell you from experience that the caregiver moves on to a far worse place. It’s a place filled with despair, guilt, sadness, and an overwhelming feeling of disorientation. I know that we can help them. The opportunity to play a part in that is why I do what I do.

There are two pictures—one of my mom and I at my wedding in 2006 (10 months before she passed) and the other is my dad with my sister at her wedding in 2011. I can see in his eyes that he’s thinking of my mom. I’ve come to know that look. ☺