This month I have the pleasure to highlight Brian Andersen, Group Vice President and General Manager at the Orphan Business Unit at Horizon Pharma.

I met Brian through conversations for upcoming episodes of Behind the Mystery:Rare & Genetic for our 2015 programming.

Horizon Pharma has been an educator on our show, The Balancing Act. They’ve been a part of our health programming for Arthritis Awareness Month, and we were delighted to see them jump in to the rare disease world —which is, of course, my passion!

Brian is dedicated to helping the rare community, and spoke to my partner, Molly Mager and I, about how his company is bringing awareness and support for families affected by Chronic Granulomatous Disease and Severe Malignant Osteopetrosis.

Carri Levy: Can you tell us how working in the world of rare disease   has affected the way you see life?

Brian Andersen: I remember evaluating career options in college and trying to find something where I could do something meaningful and make a difference. When I got into pharmaceuticals, you believed that you were helping patients but you never met them or heard their specific stories to know for sure. Once in biotech, serving the small patient populations, I knew I was in the right place. I remember talking with a patient at a conference while working in acute intermittent porphyria that had numerous unnecessary surgeries because she was misdiagnosed, something that is not uncommon in rare diseases. In my current role we talk with patients who were constantly fighting life-threatening infections before taking our product. With our product and increased disease state education, they have less frequent and less severe infections.

Parents tell us how our product has really made a difference in their child’s life. We also talk with families and hear about those awful stories about the heartaches and the losses they incurred prior to medications being available. It is heart-wrenching…you go home and hug your wife and kids. You really cannot take yourself too seriously when you meet these great people, who are so strong and who persevere through unbelievably tough times and through it all, are so positive.

CL:  Brian, can you explain what you do and how it connects to the rare disease community?

BA: I am the Group Vice President and General Manager of the Orphan Business Unit at Horizon Pharma. What this means is that I am responsible for Horizon’s current product, ACTIMMUNE (interferon gamma-1b) and any future products focused on rare diseases. I came to Horizon through their acquisition of Vidara Therapeutics where we focused on serving two rare disease communities, Chronic Granulomatous Disease (CGD) and severe, malignant osteopetrosis, a combined patient population of ~1600 patients. Prior to Vidara, I spent time working to help patients in two other rare diseases, acute intermittent porphyria affecting about 16,000 people and hereditary antithrombin deficiency affecting about 60,000 patients.

CL: The rare diseases that your company works with are heart wrenching. Are you able to meet face to face the families that are dealing with them?

BA: To best serve the rare disease communities, it is important to get a thorough understanding of the science of the underlying disease, an understanding of where your product can help the rare disease and most importantly, what the patients go through on a daily basis in dealing with the challenges of these rare diseases. I believe it is important for everyone in the Orphan Business Unit to meet patients and caregivers so that they know what impact they can have on the patient and family. It is very inspiring…these patients are so strong and positive. In addition to folks in our business unit meeting patients and caregivers, we have brought in patients and caregivers to talk to the entire company. It is important for everyone to see what we do every day and how we can improve patients’ lives.

CL: Can you give us an example of how rare disease has affected your way of doing business?

BA: I am not sure that working in rare diseases has changed the way I do business. I learned early in my career and have since believed in two principles:

1. Good science is good business.

2. Doing what is right for patients, is doing what is right for business.

When working in rare diseases, I have been able to see the result of these principles interacting with the patients we serve and the physicians with whom we partner.

CL: How have you helped bring awareness to people with CGD and Severe Malignant Osteopetrosis?

BA: Chronic Granulomatous Disease (CGD) is a genetically determined (inherited) disease characterized by an inability of the body’s phagocytic cells (also called phagocytes) to make hydrogen peroxide and other oxidants needed to kill certain microorganisms.  Severe, malignant osteopetrosis is one form of osteopetrosis and is sometimes referred to as marble bone disease or malignant infantile osteopetrosis (MIOP) because it occurs in very young children. Severe, malignant osteopetrosis is a more severe form of malignant osteopetrosis.
*https://www.horizonpharma.com/severe-malignant-osteopetrosis/

As I mentioned before, misdiagnosis is very common with rare diseases. We have partnered with great organizations such as the CGD Association and Immune Deficiency Foundation to raise awareness in CGD and we have partnered with The Osteopetrosis Society, the Ryan Wersten MIOP Foundation and the Mason Shaffer Foundation to raise awareness about severe, malignant osteopetrosis. In addition, we worked with the physician community to educate and support these two ultra-rare diseases.

CL: Who inspires you most?

BA: I am lucky that I have multiple points of inspiration in my life. First and foremost, my wife and four children are the inspiration of everything I do. But in my work, I also have great inspiration. As I mentioned earlier, I cannot believe how strong the patients and caregivers are who have to work through the challenges of rare diseases every day. Also, I have met some many patients and caregivers who have been through so much, much more than people should have to deal with, and yet they are so positive. It really makes you appreciate everything you have in life and also appreciate all the great people we get to work with on a day-to-day basis.

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