Understanding the Power of Patients: Examples of Patient Impact on Clinical Development

Pritha Kuchaculla, Executive Vice President, Client Services, VOZ Advisors

Pritha Kuchaculla has a record of success in aligning business objectives on a broad set of industry clients with patient and professional organizations across a range of therapeutic areas. Her responsibilities include helping companies integrate the patient voice into all activities, from discovery through post-launch access.

Among her achievements in this complex area is the development of the first-ever patient group-initiated guidance to accelerate the development and review of potential therapies for a rare disease — Duchenne Muscular Dystrophy — to The US Food and Drug Administration (FDA). She has also counseled a variety of companies on global advocacy and engagement strategies, best practices in advocacy relations and creating impactful programs. Pritha brings a strong background in industry, non-profit and hospital administration, having worked in healthcare for over 16 years.

She was the Associate Director of Patient Advocacy at Intercept Pharmaceuticals, a company specializing in non-viral liver diseases. There, she helped implement the advocacy strategy for the company’s first product launch, resulting in greater awareness amongst the community for a new treatment option. Pritha has also served the American Liver Foundation as the National Director of Programs, leading education and support services. She has held other positions at the Children’s Health Fund, New York City Health and Hospitals Corporation and Northwell Health.

Pritha holds a Bachelor of Arts in Communications from Rutgers University and a Master of Public Health from the University of Medicine and Dentistry of New Jersey (UMDNJ).

Pat Furlong, Founding President and CEO, Parent Project Muscular Dystrophy

Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Its mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne through research, advocacy, education and
compassion.

When doctors diagnosed her two sons, Christopher and Patrick, with Duchenne in 1984, Pat didn’t accept “there’s no hope and little help” as an answer. Pat immersed herself in Duchenne, working to understand the pathology of the disorder, the extent of research investment and the mechanisms for optimal care. Her sons lost their battle with Duchenne in their teenage years, but she continues to fight — in their honor and for all families affected by Duchenne. In 1994, Pat, together with other parents of young men with Duchenne, founded PPMD to change the course of Duchenne and, ultimately, to find a cure. Today, Pat continues to lead the organization and is considered one of the foremost authorities on Duchenne in the world.

Along with leading PPMD, Pat speaks about Duchenne and related topics at conferences each year worldwide and is an active Board member of the National Health Council. She is also a committee member of CTTI (Clinical Trials Transformation Initiative) and serves on the data safety monitoring board for both the Rare Diseases Clinical Research Network and Cooperative International Neuromuscular Research Group.

Pat graduated from Mt. St. Joseph College in Cincinnati, Ohio with a BS in Nursing. She attended Graduate School at Ohio State University. While attending Ohio State, Pat spent most of her time in the Medical Intensive Care Unit. After marrying Dr. Tom Furlong, Pat ran the renal dialysis unit and patient education center at Akron General Hospital. With four children, Pat continued her career on a part-time basis, teaching patient education classes at Middletown Regional Hospital.

Pat has two daughters, and her perseverance is in honor of her two sons, Christopher and Patrick, who continue to motivate her efforts.

Dr. Alessandra Gaeta, Director of Research, Duchenne UK

Dr. Alessandra Gaeta is the Director of Research at Duchenne UK, the leading Duchenne muscular dystrophy (DMD) charity in the UK. Duchenne UK’s mission is to end the devastating impact of DMD, a progressive and life-limiting muscle-wasting disease. Alessandra joined Duchenne UK in 2021 with nearly 20 years of research and development experience in academia, industry and the public sector, where she held scientific, managerial and leadership roles. At Duchenne UK Alessandra looks after the research and development portfolio of 40+ projects. In just over 10 years, Duchenne UK has committed £20m to research, access and support and the funding has directly contributed to pivotal milestones in DMD research, including making DMD gene therapies a reality.

Following a Pharmacy degree and a PhD in Medicinal Chemistry, she was awarded a 2-year post-doctoral Maplethorpe Fellowship for the promotion of pharmaceutical education and excellence in research at the Department of Pharmacy, King’s College London. Throughout her career, Alessandra has particularly focussed on translational research and the value of cross-sector collaboration.

In previous roles in government-funded organizations, Alessandra led the creation of high-profile, national R&D programmes to accelerate innovative treatments to the clinic. At the Medicines Discovery Catapult, she established a portfolio of patient-focused medicines discovery programmes in areas of unmet medical need, in partnership with charities and industry.

Prior to that, at the NIHR, she was instrumental in the creation of the Translational Research Collaboration pilot programme for early-phase clinical research, which has since been implemented in eight therapeutic areas. Under her leadership, over a period of 4 years, the program attracted over £20 million in industry funding to support a portfolio of early-phase clinical collaborations in UK academia and in the NHS.

Dr. Joe Newman, Clinical Research Fellow, Heart and Lung Research Institute, University of Cambridge

Joe Newman is a Respiratory Physician and a British Heart Foundation Clinical Research Fellow at the Heart and Lung Research Institute, University of Cambridge. He has broad clinical research experience including the delivery of COVID-19 vaccine and therapeutic trials, and a focus on patient and investigator engagement strategies.

His current research is in the design and development of digital and remote endpoints for use in decentralized clinical trials in the rare disease area of pulmonary hypertension and other cardiopulmonary diseases.

Joe works closely with Aparito and Pulmonary Hypertension Association UK through the Patient Group Accelerator programme to co-design, test and deploys novel endpoints with patients.

Sharon Dion, Senior Vice President, Client Services, VOZ Advisors

As part of the VOZ Advisors’ leadership team, Sharon’s responsibilities include client relations, account management, business development, and team development. She works closely with clients to build effective and mutually beneficial relationships and to develop and implement needs-based strategies to support drug development, approval, and marketing activities. Sharon has strong expertise in patient engagement and has worked with several clients to establish and implement successful global patient engagement functions, strategies, and programs. Sharon joined VOZ in 2014 after a decade tenure at a GlaxoSmithKline, where she led the patient marketing and engagement strategy for the portfolio of oncology products. She has been involved in multiple product launches and has experience working in both global and US pharmaceutical marketing roles. Sharon is a skilled market research professional with extensive experience consulting with public and private sector clients to identify key business issues and develop and implement plans to deliver insight-driven solutions.

Sharon holds a Bachelor of Arts in International Relations from Tulane University and a Master of Business Administration from the American Graduate School of International Management. She also completed an executive marketing program at the Wharton School of Business.

Ramona Burress, Head of Patient Engagement & Insights, Takeda

Ramona Burress is Head of Patient Engagement and Insights, Center for Health Equity and Patient Affairs (HEPA) at Takeda Pharmaceuticals. She leads the Center’s work with internal Takeda teams to provide longitudinal patient insights and increased knowledge of patients’ needs, beliefs, and expectations through engagement. Ramona is a licensed clinical pharmacist and accomplished leader with 20+ years of experience in advancing positive patient outcomes and equity within healthcare. Most recently, Ramona served as Director, Diversity & Inclusion in Clinical Trials, at Janssen Pharmaceutical Companies of Johnson & Johnson, where she designed external stakeholder partnerships critical to increasing participation of underrepresented and underserved populations in clinical research. Her results-driven approach was mastered in a former role as a Healthcare Specialty Supervisor with Walgreens Pharmacy in which she employed her extensive medical and business knowledge to serve the needs of patients with complicated health conditions.