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While drug development for rare and orphan disease has taken giant leaps, developers still face many unique challenges and hurdles to bring these products to patients who need them. 

Join us to get insights, expert guidance, and hear practical case studies that address the major challenges of rare disease drug development, from drug candidate early development, through regulatory strategy, trial design, patient engagement and market access. The full day workshop agenda includes:

  • Understanding Current US and EU Regulations - Get insights on endpoint design and drug approval today’s regulatory and HTA environment
  • Patients Insights and Advocacy Groups - Learn how to leverage patient insights for more effective clinical trial design and execution
  • Designing Patient-Based Clinical Trials - Learn how to design patient focused studies that are scientifically robust and address the unique challenges of the disease
  • Successful Rare Disease Trial Execution - Get insights on practical ways to address the unique challenges of running a rare disease trial
  • Patient Engagement and Retention - Hear about ways to ensure patient engagement in clinical trials, expanded access programs and patient registries
  • Strategic Market Access and Reimbursement - Learn strategies for market access and supporting research for rare disease treatments

Refreshments and Networking

Join us for refreshments and registration from 9:30 to 10:00. Lunch will also be provided.

Target Audience

This workshop is intended for pharma and biotech professionals involved in rare disease development, including:

  • Medical Directors
  • Therapy Area Directors
  • Clinical Trial Directors/Managers
  • Regulatory Affairs
  • Market Access
  • Patient Advocacy Groups looking to set up registries and clinical trials

Presenters

William Maier, MD, PhD

Chief Scientific Officer & Head, Rare Disease Research, Commercialisation and Outcomes, ICON

Dr. Maier has over 25 years of experience in drug development and commercialisation at pharmaceutical companies in Europe, Canada, the United States and Asia. In his current role he works with pharmaceutical companies throughout the world to provide regulatory, strategic and scientific guidance on medical treatment development and commercialisation. He is a member of the EMEA’s European Network of Centres for Pharmacoepidemiology
and Pharmacovigilance (www.encepp.eu). In addition, he is a frequent speaker at medical conferences, has had academic appointments in the UK (Dundee) and the USA (North Carolina) and is a member of the Royal Society of Medicine in the UK.

Cynthia Dukes, PA-C, MT

Vice President Drug Development Services, Vaccines, Infectious Disease, Women's Health and Rare Disease, ICON

Cynthia "Cindy" Dukes has more than 40 years of diversified clinical experience including 33 years of clinical research focused in infectious disease, vaccines, oncology, women’s health and rare disease.

Cynthia began her clinical research career as Clinical Team Lead at Baylor College of Medicine’s Influenza and Respiratory Disease Research Center where she gained valuable experience in general medicine, vaccine and infectious disease studies. Following this experience she spent 14 years in the pharmaceutical industry gaining experience in infectious disease, vaccines, immunoglobulins, allergy, ommunology and oncology. During this time, Cynthia participated in more than 40 INDs leading to eight product approvals including five vaccines, two oncology products and one immunoglobulin.

Cindy has spent the last 17 years in the CRO industry At ICON, Cindy founded ICON's Centre of Excellence for Vaccines and Rare Disease. She has also provided strategic and management oversight for numerous studies across a number therapeutic areas, including pivotal Phase III trials that have led to the approval of over 15 drugs/vaccines.

In August of 2016, Cynthia was recognized as among the “Top 100 Leaders in the Industry” by PharmaVoice and in 2014 recognized as one of  the “Top 50 Influential People in Vaccine Development.” In 2015, Cindy was recognized as an ICON Top Leader and as a Distinguished Alumnus by Baylor College of Medicine. Additionally, under Cindy’s leadership ICON was recognized as “Best CRO” at the Vaccine Industry Excellence Award dinner at the World Vaccine Congress in April 2014 and 2017.

Kelly Franchetti, RN CCRN CEN

Vice President, Global Patients Insights and Engagement, ICON

Kelly Franchetti brings extensive experience in partnering with patients, advocacy, and clinical research with a specialty in Rare Disease programs. She is a leading developer of Patient Engagement solutions in the world of both clinical research and in therapeutic practice. Ms. Franchetti has two decades of direct patient experience,gained in both hospital and industry settings. Kelly was an active critical care Registered Nurse for over 18 years and an experienced caregiver for children with rare disease for over 20 years. This experience translates into a keen understanding of how patients think regarding their treatment, giving her a singular perspective regarding the best way to approach patients, their caregivers and Health Care Practitioners. She maintains her nursing status at multiple university health systems to continue to grow her knowledge base regarding patient needs and behaviors. Ms. Franchetti has been involved in the development and moderation of patient partner workshops, advisory boards as well as patient, HCP, and KOL forums. She also has extensive experience collaborating with Patient and Clinical Advocacy groups. Her experience allows her to provide significant insights into how patients regard their treatment options and how to best approach protocol design, patient recruitment and retention strategies as well as factors impacting adherence in the post market. 

Mary Jane Geiger

Vice President, Drug Development, Cardiology, ICON

Dr. Geiger is board certified in internal medicine with over 17 years of experience in cardiometabolic drug development in both large and small pharmaceutical and biotechnology companies. She has expertise in designing and leading global development programs, encompassing all phases of clinical development, and associated regulatory submissions leading to successful drug approvals and new indications. Areas of clinical research have focused on therapies to treat type 2 diabetes, hypercholesterolemia, and hyperkalemia as well as biologics to treat cancer and CV-related rare diseases. She has designed and executed CV outcomes and ABPM trials and is experienced with Scientific Advisory Boards, Steering Committees, Clinical Events Committees and Data Monitoring Boards.  She is knowledgeable regarding adaptive trial design trials, having devised and executed the first ever adaptive, dose-finding, inferentially seamless Phase 2/3 study. She has published more than 60 articles in peer reviewed journals/book chapters.

Matthew McCarty

Global Head, Patient Engagement, Commercialisation & Outcomes, ICON

Matthew McCarty is Global Head of Patient Engagement for ICON’s Commercialisation and Outcomes service area. Matthew is also a leader in Digital Health studies and Virtual Studies and currently serves on the Medical Device Innovation Consortium (MDIC) Science of Patient Input steering committee alongside industry and regulators. Prior to joining ICON in 2017, Matthew spent 10 years at Quintiles (now IQVIA) in a number of global leadership roles where most recently he was Global Head of Direct to Patient Research.

Rory Graham

Senior Director, EU Regulatory Services, ICON

Rory has facilitated numerous aspects of drug and medical device development during his 25 year career in the pharmaceutical, biotechnology and medical device industries. Through global leadership positions in the area of regulatory affairs in companies in Europe and Asia-Pacific Rory has successfully managed global registration projects involving interaction with many regulatory agencies including FDA, EMA, MDA, TGA and European National Agencies. His therapeutic area experience includes CNS, Oncology, Hematology, Immunology, Diabetes, Analgesia and Wound-Care. Rory has achieved orphan designations for pharmaceutical products in the EU, US and Australia. He leads an international team of regulatory professionals that produces client-specific regulatory strategies that address both global and local-market development and product life cycle needs. 

Register Today

Registration is complimentary, but seats are limited. Reserve your spot today.

Register