Making Cents of Clinical Trials
I graduated with my master’s in medical anthropology from Boston University School of Medicine in 2016. Prior to starting at CISCRP, I worked on research studies focused on patient experiences with health care. Most recently, I worked on several projects related to substance use and people living with HIV. I am incredibly passionate about working with vulnerable populations and increasing access to care for patients through research. As Project Manager in Research Services, I am excited to connect with new patient groups and make research more approachable for vulnerable populations.
Dominantly Inherited Alzheimer’s Disease Brochure
Expanded Registry Flyer (English)
Enfermedad de Alzheimer de herencia dominante
Expanded Registry Flyer (Spanish)
Forgetting the Little Things
Concerned About Memory Loss?
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Krysal Doucet has worked in Research as a CRC for 11 years and as an Associate Site Director for 2 years. She started her path in the health care industry by becoming a C-MA. After completing the Medical Assisting course, she was introduced to a great opportunity working as a Clinical Research Coordinator with Benchmark Research.
Missy Hansen MSN, APRN, CPNP, CPHON, joined ICON in 2019 as a pediatric strategy liaison. She has 25+ years of experience as a pediatric nurse practitioner, including 17+ years in pediatric hematology/oncology at Children’s Minnesota, the largest hematology/oncology service line in the region, providing care for infants through young adults diagnosed with hematologic and/or oncology conditions. Ms. Hansen gained expertise working in inpatient, outpatient, and urgent care type settings. Ms. Hansen has in-depth experience working with the Children’s Oncology Group—the largest consortium with the most open pediatric studies around the world. She also has experience working with other consortia that conduct trials for pediatric to young adult patients.
Lori Ranney DNP, APRN, CPNP, CPHON has worked in pediatric oncology for 20 years and has been a PNP at Children’s Minnesota for over 13 years. She has a special interest in leukemia/lymphoma, patient/family education, AYA population, and oncofertility. She is a member of the Children’s Oncology Group and has actively enrolled patients in clinical trials for over 15 years.
Mark has over 20 years of experience in the pharmaceutical and biotech industries and currently leads ICON’s global pediatric center of excellence. Having earned his medical degree and master’s degree in clinical nutrition from the Chicago Medical School, Dr. Sorrentino completed both a residency in pediatrics and a fellowship in pediatric critical care at Children’s National Medical Center (CNMC) in Washington, DC. Currently, Dr. Sorrentino holds an appointment as an assistant clinical professor at the George Washington University School of Medicine. Prior to joining the industry, Dr. Sorrentino was an attending physician in the CNMC pediatric intensive care unit for 8 years and participated in numerous industry-sponsored critical care trials. He holds active medical licenses in California and Maryland.
Based in Minnesota, Dr. Perkins has 21 years of experience in clinical research across Phases I–III with a focus on hematologic malignancies. Before joining ICON in 2018, she was in clinical practice for 15 years specializing in leukemias, lymphomas, and long-term complications of cancer therapy. Dr. Perkins has supported multiple oncology/hematology trials across indications and phases. She is board certified in pediatrics and pediatric hematology and oncology and holds a master’s degree in clinical research.
Christian oversees the development of new strategic partnerships, products and services to help shorten the diagnostic odyssey, improved access to research and help expand the number of treatments available for all rare disease patients and caregivers.
Dr. Dastgir specializes in pediatric neurology and is the director of the pediatric neuromuscular medicine program at Goryeb Children’s Hospital in Morristown, NJ. Her current practice is primarily composed of patients with rare disorders.
Dr. Dastgir also has experience in being an investigator for clinical trials involving such rare conditions as spinal muscular atrophy, duchenne muscular dystrophy, and infantile neuroaxonal dystrophy.
Generally, chronic health conditions greatly impact the lives of those within a household, not just the patient, but also the caregiver(s) and other members of the family; because of this, Mel has made it her mission to rise above the health obstacles within her own family and help others gain the knowledge and confidence to do the same. Having a chronic condition does not define a person, while it may affect the quality of one’s life, it does not need to become an unsurpassable obstacle preventing one from exploring their dreams and living life to the fullest.
Mel loves speaking publicly and jumps at every opportunity to do so, she has spoken at schools, rallies, congregations and even at the California State Capitol; no audience is too big or too small. She is passionate about helping people gain confidence in themselves, to learn self-advocacy, to find their voice and gain the inner strength to effectively be heard, and to live life to its absolute fullest potential.
Jenn McNary is a trusted voice in the rare disease community, as a mother, public speaker and fierce advocate. Her work in the rare disease space as a thought leader earned her the Ryan’s Quest Ryan’s Hero award in 2013, a nomination for the Global Genes Champion of Hope award in 2014, and the prestigious 2017 Meyer- Whalley instrument of change award. Formerly as the director of outreach and advocacy at a Massachusetts based non-profit foundation, she was responsible for the organization of the largest FDA advisory committee hearing in history, with over 1000 duchenne advocates, families, clinicians and researchers in attendance.
Jenn has unique experience in the drug development field, as a parent of children enrolled in the clinical trials, an advocate engaging with the regulators and as a consultant helping to develop programing for patients. Currently, Jenn is consulting in the biotechnology space with an expertise in caregiver/patient engagement, including bringing the patient voice to drug development and solving barriers to access.
Her other activities include serving as the Founder of One Rare, a non-profit formed to meet the needs of young adults with rare and chronic conditions and raising her four children in Massachusetts.
Mr. Schliebner is a clinical development executive with a 25-year background focused on rare diseases, innovation, and patient-focused approaches to drug development. He is passionate about reducing the burden of clinical trial participation and leveraging novel approaches and technologies to bring new therapies to patients faster. Mr. Schliebner also serves as the Co-Chair of Global Genes’ Corporate Alliance, serves on the Board of Directors for Uplifting Athletes, and supports several rare disease panels and committees. He holds a Master’s Degree in Public Health (MPH) from the University of Utah School of Medicine, and completed a Graduate Research Fellowship at The National Institutes of Health.
Cristol Barrett O’Loughlin is a seasoned executive and storyteller. As Founder and CEO of Angel Aid Cares, Cristol is fiercely passionate about providing social, emotional, physical and financial relief to mothers of children with rare disease. A former UCLA instructor, she co-founded the advertising firm, The Craftsman Agency, and is humbled to advise global brands; NBA, Disney, Fox, Cisco Systems and Google. During her tenure at IBM Life Sciences, she helped accelerate advancements in cheminformatics and biotechnology. She presented a TEDx talk “Caring for the Caregivers: 3 Tools for Self-Care”.
Rosamund Round leads Parexel’s Patient Innovation Center, focused on improving research access and experiences for patients and caregivers. This includes leadership of the decentralized clinical trials service, activities to improve diversity in clinical research, and creation of other tools and services that reduce practical, financial and geographical barriers to participation.
She has 17 years of leadership experience in patient engagement and innovation roles, and regularly presents and authors articles on the topic.
Liam Paschall is a transgender man who has found immense courage, power, and pride in accepting the truth of who he is. His pronouns are he and him.
He is an entrepreneur, public speaker, and facilitator, with a lifelong goal of changing hearts and minds – one person at a time. He is a passionate servant to the LGBTQIA+ community with the ability to motivate and inspire others to live out loud.
Having started his female to male transition at the beginning of last year, Liam knows first-hand how critical and life-altering inclusive healthcare and clinical trials can be to members of the LGBTQIA+ community.
Dr. Sebastian Barr is a licensed psychologist currently based in Western Massachusetts. He received his PhD in Counseling Psychology and completed advanced training in health service psychology and psychotherapy research at Cambridge Health Alliance under the auspices of Harvard Medical School. His scholarship centers on the experiences, mental health, and mental healthcare needs of members of the trans community, with a particular recent focus on the impacts of bias and non-affirmation. His research has been recognized nationally and internationally and published and cited across multiple journals and textbooks. Additionally, he specializes in helping institutions and healthcare providers adopt affirming, inclusive, and evidence-based practices in both clinical work and research with members of the trans community. He is a proud transgender man and incorporates personal lived and community experience into his work.
For over a decade, Adam has conducted clinical trials across multiple therapeutic areas as a research coordinator, monitor, project manager, and director at various types of organizations—investigational sites, CROs, academia, pharma, and tech. He received a master’s in clinical research from The George Washington University. In June 2020, he joined as Director of Clinical Operations at Curebase, a provider of software and services purpose-built for decentralized clinical trials (DCTs), where he is responsible for oversight of DCT services.
Arsheen Ali is a Project Manager and has served in this role with Curebase for over two years. In this capacity, she manages multiple decentralized clinical trials. Her background includes a variety of healthcare and research experience. She received her bachelor’s in integrative biology from UC Berkeley. Arsheen’s main interests are public health (population and community health focus), increasing healthcare quality and access (especially for minority/marginalized populations), and maximizing the efficiency of research.
Myra Lane is Lead Virtual Research Coordinator at Curebase with over three years of clinical research experience in the decentralized clinical trial space interacting directly with patients. She has worked in various therapeutic areas including neurology, dermatology, and oncology. She is passionate about the impact that the digital therapeutics approach has on addressing unmet patient needs by providing universal access to clinical research and accelerating the development of treatments.
Ken Getz is the founder of CISCRP and chairs the board of directors. He is also a professor and the Director of the Center for the Study of Drug Development, Tufts University School of Medicine where he conducts grant-funded studies on pharmaceutical R&D management and execution; protocol design optimization; contract service provider and investigative site management; e-clinical technology and data usage; and patient engagement. Ken is the founder of CenterWatch, a leading publisher in the clinical trials industry and one of several companies that he has created and sold. A well-known speaker at conferences, symposia, universities and corporations, Ken has published numerous articles and chapters in peer-review journals, books, and in the trade press. He is the author of two nationally recognized books for patients and their advocates including The Gift of Participation, and the recipient of several awards for innovation and scholarship. Ken has held a number of board appointments in the private and public sectors, including serving on Otsuka’s Patient Assistance Foundation, the Institute of Medicine’s Clinical Research Roundtable, the DIA Foundation, the Consortium to Examine Clinical Research Ethics, and the Clinical Trials Transformation Initiative.
At CISCRP, I manage the design, implementation, analysis and reporting of a variety of CISCRP research studies. I have more than 15 years of experience conducting primary and secondary research studies in the healthcare, life sciences and consumer goods industries. Prior to CISCRP, I worked at Colgate-Palmolive as a research manager. I hold an MBA from the Graduate School of Management at Boston University and a Bachelor of Science degree from Bryant University. I am passionate about helping others, and CISCRP offers ample opportunities in that regard. People often comment that I am a nice and genuine person. And I think those characteristics help me connect to the patients I interact with regularly through my work. I especially enjoy being involved in bringing patients and clinical operation teams together through the CISCRP Patient Advisory Boards; speaking with each other about ways to make clinical trials a better experience overall is very rewarding for me.
As a Peace Corps Volunteer, I served as an English Literacy Facilitator in the Kingdom of Tonga. There, I gained invaluable experience working alongside my community and counterparts through projects such as student-centered learning initiatives within the classroom, grant writing for better access to technology, establishing a school library, as well as promoting girl’s empowerment and opportunities for fitness and healthy living. Throughout my service, my favorite experiences were always connecting with individuals in the community – exchanging stories, cultures, traditions, and laughter. Additionally, I am a graduate of Connecticut College where I earned a B.A. in Psychology and an Elementary Education certification. I am excited to have the opportunity to apply my love of education, psychology, and research through my work at CISCRP. As a Project Manager, I enjoy working with a variety of individuals from diverse backgrounds and experiences to help amplify the patient voice within clinical research trials.
I graduated with a B.A. in Sociology from Skidmore College in May 2020. At Skidmore, I worked on multiple research projects including a journal article I co-authored with a professor focusing on Tor and the Dark Web, as well as my Senior Thesis, which examines the relationships between mental health and access to natural environments. Through my research projects, as well as my education in Sociology, I learned the importance of using research to improve access to crucial resources and institutions that help communities grow and thrive. I also had the opportunity to intern for Habitat for Humanity, which strengthened my interest in working for a non-profit organization that aims to overcome barriers in the way of essential resources such as housing or healthcare information. I am excited to use my research experience and my love for working with people to help improve the clinical research process, and highlight the patient experience as central to the development of clinical trials.
Kim Harper, began her clinical research experience as a Clinical Research Coordinator back in 2007. Over the next 7 years she worked as a CRC gaining experience working for an arthritis clinic and a research center. In 2014, Ms. Harper was brought on by Benchmark Research, a leading clinical research firm as a CCRC. She quickly worked her way through the ranks to Assistant Site Director and in 2020 Kim became promoted to Site Director.
During her 14 years in the field, Kim has been a significant contributor to the studies she has worked on which include Phase 1-4 treatment, preventative and diagnostic trials on pediatric, adult, and elderly subjects. Kim has diverse experience in vaccine, drug, and device trials as examples therapeutic areas she has worked on include ebola, anthrax, COVID-19, diabetes, influenza, meningococcal, antipsychotics, depression, alzheimer’s disease, osteoarthritis, and more. During the COVID-19 pandemic, Kim thrived in the high pressure environment and led her North Shore New Orleans research site to successfully conduct 2 coronavirus studies including a high profile vaccine. Kim is known amongst her peers for her proven ability to reach across personal and professional barriers to expedite effective health care.
Stephanie is a patient advocate at The Young Face of Arthritis.
As a rare disease patient living with primary lymphedema (LE) for over three decades, Britta remains determined to challenge her limits rather than to let LE limit her challenges. Despite wearing compression garments 24 hours a day, she has skied Mount Tremblant, completed the 10k Chesapeake Bay Bridge Run, and climbed the Great Wall of China. Britta pays it forward by sharing “advice, comfort, and positive thinking” on her award-winning blog, lymphedemadiary.com. As Senior Director of Communications and Marketing for the EveryLife Foundation for Rare Diseases, Britta initiates strategies that inspire patients and caregivers to engage in advocacy and make their voices heard. Britta lives in Arlington, Virginia with her husband and two very spoiled cats.
Ryan Colburn is a guy with 2(+) typos on his GAA gene on chromosome 17. He has a professional background in engineering and operations management; spending portions of his career working on race cars, airplanes, and rockets. Diagnosed with Pompe disease in 2015, he has spent the time since learning about rare disease topics including research, advocacy, and development of treatments to better understand how to participate in the rare disease ecosystem. He is passionate about patient empowerment and engagement; actively developing relationships with other patients, advocacy groups, researchers, and pharmaceutical companies. He is driven to improve the health of the rare disease ecosystem by shifting the view from patients as “subjects” to one of participants, collaborators and partners who help find the most effective ways to tackle the challenges of rare disease and break down barriers to the acceleration of progress. Ryan has participated in nine clinical studies since he was diagnosed, and has shared his perspectives with industry and advocacy organizations to improve the patient experience within the clinical research space.
Dr. Kim Stephens is the president of Project Alive, a nonprofit organization dedicated to research and advocacy for Hunter syndrome. Project Alive that is a powerful voice for children and adults with Hunter syndrome and brings together families and advocates with researchers, industry, and regulators. Having a son with Hunter syndrome herself, this is also a personal mission for Kim. Her son Cole has been participating in a clinical trial for the past 6+ years. Having gone through the ups and downs of clinical trial life, Kim is a strong advocate for creating better outcome measures, integrating the patient voice in trial design, and considering the entire cycle of the clinical trial – from the patient’s doorstep to the clinic and back home again. She frequently speaks as a rare disease advocate at conferences and events and serves as a mentor and resource for newly-diagnosed families.
Kim is also the founder of Inclusive Thinking – a consulting company dedicated to increasing diversity and inclusion through research, education, and strategic planning. Before founding Inclusive Thinking, she had a twenty-year career with IBM, most recently as the Global Diversity & Inclusion Communication and Education Lead. She has spoken at conferences around the world and conducted workshops on unconscious bias, cultural awareness, women’s leadership, accessibility, and other topics.
Kim earned her doctorate in Business from Georgia State University and focused her dissertation on implicit bias and the role of transformational conversation and social identity on behavioral change.
Marc Yale was diagnosed in 2007 with Cicatricial Pemphigoid, a rare autoimmune blistering skin disease. Like others with a rare disease, he experienced delays in diagnosis and difficulty finding a knowledgeable physician. Eventually, Marc lost the vision in his left eye from the disease. This inspired him to help others with the disease. In 2008, he joined the International Pemphigus and Pemphigoid Foundation (IPPF) as a Peer Health Coach. He worked with people to improve their quality of life and encouraged them to become self-advocates. In 2009, he helped develop the Pemphigus and Pemphigoid Comprehensive Disease Profile giving experts insight into the patient perspective. In 2016, Marc became the Executive Director of the IPPF. He has recently become the Advocacy & Research Coordinator of the IPPF so that he can focus on research and advocate for all of those affected by pemphigus and pemphigoid. He is a member of the American Academy of Dermatology Drug Transparency Task Force, sits on the Executive Board of Directors for The International Alliance for Dermatological Patient Organizations as their Treasurer, serves on the Board of Directors of Haystack Project, and is a committee advisor for Rare Disease Legislative Advocates. Marc recently joined Rare Disease International in the establishment of the WHO Collaborative Global Network for Rare Diseases Panel of Experts to ensure a lasting impact on the lives of people living with a rare disease around the globe.
Marc currently resides in Ventura, California with his wife of 31 years.
Mike Wenger is Global head of Patient Engagement at Informa Pharma Intelligence. As both a patient and software developer, Mike is passionate about patient access to clinical trials. He is spearheading Citeline Connect, the only end-to-end platform for clinical trial education, recruitment and enrollment, enabling sponsors to accelerate study startup and boost engagement. Mike was named one of the 2020 PM360 ELITE 100 in the Patient Advocates category and a 2020 Force for Change Illuminator. He also was a finalist in the Patient Advocate of the Year category in the 2020 Medigy HITMC Awards.
Wes Michael founded Rare Patient Voice in 2013 to give patients and caregivers with rare diseases the opportunity to voice their opinions in research studies. Rare Patient Voice has now conducted thousands of studies and rewarded patients and caregivers with over $8 million for their participation. Many have been recruited in person by Wes and his team at patient events like fund-raising walks and patient conferences, and through a referral program with patient advocacy and support groups. Rare Patient Voice now covers non-rare as well as rare diseases and conditions and has expanded from the United States to Canada, the United Kingdom, France, Germany, Italy, Spain, Australia, and New Zealand.
Before launching Rare Patient Voice, Wes worked for healthcare market research firm Kantar Health. He previously was a brand manager and market research manager at McCormick (the spice company) and General Mills (working on Wheaties, Total and Kix cereals). He has a BA from the University of Pennsylvania (with a year at Edinburgh University) and an MBA from the University of Chicago. Wes was recently inducted into the DTC National Hall of Fame, honoring individuals who have demonstrated extraordinary achievement in the advancement of direct-to-consumer marketing.
Steve Jones, is President of EU-IPFF, the European Idiopathic Pulmonary Fibrosis and Related Disorders Federation and part of the Citeline Connect referral collective. EU-IPFF is dedicated to making clinical trials work better for patients in partnership with pharma and regulators in UK and Europe. Steve also is chair of trustees of the UK patient organization Action for Pulmonary Fibrosis and a council member for the European Lung Foundation. He also sits on the steering committees of four major non-pharma clinical trials funded by the UK’s National Institute of Health Research. Steve himself is a pulmonary fibrosis patient and had a lung transplant five years ago.
Ashley Smock is a woman living with ovarian cancer. She took part in the Rare Patient Voice/Informa survey on patients and clinical trial participation in 2021.
Amy brings a comprehensive understanding of drug discovery and drug development from the research laboratory to patients’ bedside from 20+ years of experience in academic institutions, biotech companies, and CROs. Originally trained as a molecular biologist, she transitioned into drug development and came up through clinical operations before joining the Center for Rare Diseases in 2017. She has contributed to discovery and development of treatments for many rare disease patient communities, from rare cancers to rare neurodegenerative disorders, including development of a gene therapy treatment now commercially available to rare disease patients.
Amy leads the Gene Therapy Think Tank and is a Subject Matter Expert in the Center for Immuno-Oncology, Cellular, and Gene Therapy. She provides full life cycle support for gene therapy programs outside of oncology, from study design through study close out. This includes close collaboration with sponsor teams, study physicians, clinical operations throughout the program, as well as supporting integration of patient partnership into clinical operations. She is passionate about enhancing the efficiency of clinical trials through cooperative productivity and patient-centered design – key elements of “Market Aware” clinical development protocols, and a proven pathway to commercial success.
Laura Iliescu, MSc, has 20 years of experience in development and commercialization of therapies, and patient-centered healthcare services. She has held strategic development roles in rare and complex conditions in leading pharmaceutical, biotechnology, CRO and clinical homecare organizations. Her varied experience comprises strategic development throughout every phase of the product lifecycle from pre-clinical to beyond patent expiry in North America and Europe. She has deep experience in rare metabolic, rare neuromuscular an rare respiratory indications among others.
Her background in human factors engineering and user-centered service design methods informs her passion for patient-centered development and for evolving the clinical development paradigm around patients as key stakeholders.
In her current role within ICON’s Center for Rare Diseases, Laura advises product development companies on patient-centered clinical strategy to optimize both study efficiency and the experience of rare disease patients and caregivers. She leads the Center’s Patient Advocacy Strategy team, who support collaboration between companies and rare disease Patient Advocacy Organizations through study design and conduct.
Laura grew up in Toronto, Canada, after which her career (and love of travel) have taken her to more than 40 countries in North America, Europe and Asia. She currently lives in Toronto, Canada with her partner and has both family and friends who are affected by rare conditions. Laura holds a Masters of Human Factors and Ergonomics from the University of Nottingham.
Kelli Wright is the Senior Manager of Patient Advocacy for Harmony Biosciences working to keep Patients at the Heart of everything we do at Harmony. It’s critical to include the patient voice in drug development efforts and Kelli is honored work with patient communities, people living with rare neurological diseases, caregivers, and patient advocacy organizations to share their lived experiences across functions at Harmony.
With a 15+ year non-profit career, Kelli has a deep understanding of the patient needs and experiences. Most recently Kelli was the National Director of Strategic Initiatives for CureSearch for Children’s Cancer, driving the strategic program of CureSearch by staying in tune with the barriers in drug development. Kelli learned from innovators in the industry, academic and regulatory space, as well as patients and disease-foundations, to address obstacles and develop action plans to close the communication gaps and reduce duplication of efforts.
Kendall is a driven health advocacy professional with strong community outreach, patient association, health care provider, rare disease and chronic illness expertise. She specializes in patient advocacy throughout rare disease clinical drug development. She currently serves as the Patient Advocacy Lead, Early Development , at Spark Therapeutics. In this role she supports Sparks preclinical and early clinical development programs. Previously, Kendall served as a Patient Advocacy Strategy Lead at PRA Health Sciences and as the Director of Strategic Alliances at Global Genes, a leading rare disease patient advocacy organization. She has held key roles in patient advocacy and patient education in both the nonprofit and biopharmaceutical industries. Kendall’s passion is to improve the health outcomes of individuals living with rare diseases while integrating advocacy strategy into clinical drug development. Kendall specializes in cultivating partnerships with patient advocacy organizations, identifying and partnering with Key Opinion and Community Leaders, identifying business needs, developing customized programs to achieve key outcomes and balancing a global focus with national, regional and local patient centric partnerships. Kendall works with leaders in the rare disease nonprofit community & biotechnology space to advance progress in rare disease drug development. Kendall also serves on the board of directors of Team Telomere and as a strategic advisor to the Rare and Undiagnosed Network. She holds a Master’s Degree of Public Health from Michigan State University as well as a Bachelor’s of Science in Psychology from Loyola University, Chicago. Kendall currently lives in Pittsburgh, PA with her husband, son and dachshund.
Sarah Nechama Frisch is a woman living with narcolepsy with cataplexy. She took part in the Rare Patient Voice/Informa survey on patients and clinical trial participation in 2021.
Julie Holtzople is Senior Director, Clinical Transparency and Data Sharing, AstraZeneca.
Christopher Pfitzer is Associate Director, Transparency Operations Lead at UCB Biosciences, Inc.
