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Our Rare Disease Leaders

Our Rare Disease Leaders

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Meet Our Rare Disease Leaders

Meet Our Rare Disease Executive Leadership team and hear their unique perspectives

Michael Goettler

Global President, Rare Disease Global Innovative Pharma Business

“Helping patients with rare diseases is a mission of global proportions. We are prepared for that challenge. Pfizer is in a unique position to provide medicines that span geographies, transcend patient diversity, and offer meaningful solutions to distinct patient populations struggling with rare diseases. The global medical community—and the pharmaceutical sector in particular—has a responsibility to devise better treatments for patients with rare diseases. It’s a privilege for us to assume a leadership role in that shared mission. At the heart of this work is our concern and compassion for patients and families whose lives are affected by rare disease. They are our true inspiration.”

Brenda Cooperstone, MD

Chief Development Officer, Rare Disease

“A significant part of our work is building partnerships with families who are affected by a rare disease and supporting them throughout the clinical trial process. Many rare diseases are genetic, with greater than 50% affecting children. This reality demands a new model in which partnerships with advocacy organizations to embed patient perspectives become a critical means for us to understand the patient journey and better inform and engage patients, their families, and caregivers throughout the trial. This deep collaboration with our patient populations and their advocacy groups enables us to advance closer to the goal: to develop treatments that improve longevity and quality of life for patients with rare diseases.”

Gregory LaRosa, PhD

Chief Scientific Officer, Rare Disease Research Unit

“Pfizer has a deep heritage in the genetic mechanisms and triggers of disease. Since many rare diseases—perhaps 80%—have a genetic component, we are uniquely positioned to pioneer medicines that can make a difference in the lives of people with rare diseases. By focusing scientific expertise in areas of high unmet need, Pfizer Rare Disease has a pipeline that holds the promise to truly advance rare disease research. We are executing a dual approach: near-term disease management to help patients better manage their disease and quickly improve quality of life, and long-term disease correction to ultimately treat the disease by exploring its underlying causes and informing clinical programs.”

Michael Goettler

Global President, Rare Disease Global Innovative Pharma Business

“Helping patients with rare diseases is a mission of global proportions. We are prepared for that challenge. Pfizer is in a unique position to provide medicines that span geographies, transcend patient diversity, and offer meaningful solutions to distinct patient populations struggling with rare diseases. The global medical community—and the pharmaceutical sector in particular—has a responsibility to devise better treatments for patients with rare diseases. It’s a privilege for us to assume a leadership role in that shared mission. At the heart of this work is our concern and compassion for patients and families whose lives are affected by rare disease. They are our true inspiration.”

Brenda Cooperstone, MD

Chief Development Officer, Rare Disease

“A significant part of our work is building partnerships with families who are affected by a rare disease and supporting them throughout the clinical trial process. Many rare diseases are genetic, with greater than 50% affecting children. This reality demands a new model in which partnerships with advocacy organizations to embed patient perspectives become a critical means for us to understand the patient journey and better inform and engage patients, their families, and caregivers throughout the trial. This deep collaboration with our patient populations and their advocacy groups enables us to advance closer to the goal: to develop treatments that improve longevity and quality of life for patients with rare diseases.”

Gregory LaRosa, PhD

Chief Scientific Officer, Rare Disease Research Unit

“Pfizer has a deep heritage in the genetic mechanisms and triggers of disease. Since many rare diseases—perhaps 80%—have a genetic component, we are uniquely positioned to pioneer medicines that can make a difference in the lives of people with rare diseases. By focusing scientific expertise in areas of high unmet need, Pfizer Rare Disease has a pipeline that holds the promise to truly advance rare disease research. We are executing a dual approach: near-term disease management to help patients better manage their disease and quickly improve quality of life, and long-term disease correction to ultimately treat the disease by exploring its underlying causes and informing clinical programs.”

Meet Our Rare Disease Leaders

Learn how Pfizer colleagues bring their passion and commitment to the rare disease community through their everyday work

Brenda Cooperstone, MD

Head of Development, Rare Disease

At Pfizer, we know the importance of the patient in the development of Rare Disease therapies. In a recent issue of PharmaVOICE, Brenda Cooperstone discussed the unique integration of Rare Disease patients in the treatment development and approval process.

“We have found that patient advocacy is a distinguishing characteristic of the rare disease space, and because of the complexity and smaller affected populations, it often falls to patients to be involved in everything from clinical trial recruitment to access and reimbursement.”

Kelly Knee, PhD

Principle Scientist, Pfizer Rare Disease Research Unit

“I’m a principle scientist in the Rare Disease Research Unit and I’m working on sickle cell disease. Sickle cell is a neglected disease. We’ve known about it since the early 1900’s and really, today, what the patients get in terms of treatment and standard of care is the same as what they got back in 1911, so, it’s important that it’s time for them to have some new therapies.”

Click here to watch a video in the Pfizer Annual Review of Kelly and her efforts in sickle cell research and development.

Joseph Nabhan

Principle Scientist, Pfizer Rare Disease Research Unit

“I’m a principle scientist in the Rare Disease Research Unit at Pfizer. I’m working on Friedreich’s ataxia and we’re hoping to develop a therapy for this debilitating disease. Friedreich’s ataxia is a progressive, neurodegenerative disease that affects young patients and can really influence their quality of life and limit their longevity. So, our hope is to bring a therapy that is disease-modifying, meaning, either slow down disease progression or reverse the course of disease. We realize that we can’t do this alone. This requires the concerted effort from academics, the advocacy groups, as well as the drug discovery companies, such as Pfizer. Every single building block of towers of therapy is worth a lot and allows us to really understand how complicated it is to bring a therapy to the table.”

Click here to learn more about Joe’s research efforts in a video featured in the Pfizer Annual Review.

Bhash Parasuraman, PhD

Vice President, Global Health and Value, Rare Disease and Neuroscience/Pain

“My colleagues and I recently attended the EURORDIS Multi-Stakeholder Symposium on Improving Patient Access to Rare Disease Therapies. We came away with an understanding that collaboration with all our stakeholders will be essential to improving access for Rare Disease patients. Not only do we need multi-stakeholder dialogues between regulatory, payers, manufacturers and patient advocates, but we need agreement on value determination and reimbursement strategies across the EU. Partnerships will be key to creating strong pathways for patients to get access to the medications they need, and we are pulling these insights through our partnership with EURORDIS into our strategy moving forward.”