Successful Approaches from Rare Disease and Cellular and Gene Therapy Product Approvals

Life Sciences, Clinical Trials, Patient Recruitment & Retention,
  • Tuesday, February 28, 2023

Clinical development is becoming increasingly complex with continuing innovations in cell and gene therapies, especially when factoring in the hurdles of rare disease research. To achieve cell and gene therapy approvals in rare diseases, it is essential to overcome challenges specific to these trials. In this upcoming webinar, learn common medical, regulatory and operational considerations from a team of Medpace experts through lessons learned in advanced therapy and rare disease case studies.

Cell and gene therapy trials in rare diseases raise the challenge of having one chance to get the trial right, making strategic planning, execution and communication essential. Rare diseases represent a favorable target for gene therapy treatment as a high number of rare diseases are known to be caused by a single gene mutation. Gene therapy has the potential to correct the genetic defect underlying the clinical condition with a single or infrequent administration of therapy, achieving a lifelong cure instead of patients having to receive ongoing treatment for the rest of their lives.


Common clinical challenges to consider when planning trials include the complexities related to gene therapy and underlying conditions, as well as needing to monitor patient visits closely and efficiently after administration to ensure study endpoint protection. Operational considerations are often associated with patient recruitment, retention, compliance and challenges behind long-term patient follow-up. A hub and spoke model for treating and referral sites allows efficient recruitment planning and wide patient study access, however, challenges the study execution with long-distance or cross-border patient management.

Additionally, the regulatory landscape continues to evolve to meet challenges with the ongoing development of innovative types of gene therapy, including gene editing, as well as the need for regulatory guidance and flexibility to ensure that a rare disease therapy’s development strategy will meet regulator expectations. Rare disease studies must consider the constraint of very low patient numbers while also needing to achieve appropriate statistical significance to demonstrate efficacy and meet regulatory expectations; having early and regular interactions with regulators to ensure agreement of proposed study designs is essential. Receiving Orphan Drug Designation for a product in development is an important milestone and provides numerous incentives, including regulatory assistance, which helps to facilitate interactions with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and other authorities from around the world.

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Understanding and proactively addressing these common challenges and considerations ensures that a trial is best positioned for success and will support the future goal of marketing approval.

Register now for the webinar on Rare Disease Day 2023 to join the discussion with Medpace medical, regulatory and operational experts on successful approaches for achieving cell and gene therapy approvals in rare diseases.


Terence Eagleton, MB BS, BSc

Terence Eagleton, MB BS, BSc, Senior Medical Director, Medpace

Dr. Terence Eagleton is a UK-based physician who qualified from University College London Medical School, after which he trained as a general surgeon with a focus on trauma and critical patient care. He has subsequently worked as a senior pharmaceutical physician in the biopharma and CRO industries for over 20 years in global clinical research and medical affairs, in a wide variety of therapeutic areas. He has extensive experience and achievement in international orphan drug development across Phase I to IV, with a specific focus in clinical research with a variety of innovative therapeutic modalities, many in ATMPs. Dr. Eagleton is an affiliate member of the Faculty of Pharmaceutical Medicine (FPM) in the UK. He has authored original papers in peer-reviewed journals and established himself as an industry expert in rare diseases.

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Marco Tangelder, MD, PhD

Marco Tangelder, MD, PhD, Senior Medical Director, Medpace

Dr. Marco Tangelder is a Clinical Epidemiologist with 25+ years of academic, pharmaceutical, and biotech industry experience. His clinical and R&D experience includes vascular surgery and medicine, thrombosis & hemostasis, development of antithrombotic therapies for a broad range of indications and development of gene therapy products. Dr. Tangelder is dedicated at Medpace to serve clients with innovative pipelines in advanced therapy medicinal products (ATMP) and contributes to their development to treat disabling diseases in hematology and other therapeutic areas. Dr. Tangelder received his medical degree, PhD, and clinical epidemiology board certification at the University Medical Center Utrecht in the Netherlands, where he also served for 12 years as Associate Professor of Clinical Epidemiology at the department of Vascular Surgery. Dr. Tangelder received his Master’s in Pharmaceutical Medicine at the Karolinska Institute in Stockholm, Sweden. Dr. Tangelder has authored over 50 scientific publications in peer-reviewed medical journals and medical books.

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Laura Omoboni, MS

Laura Omoboni, MS, Executive Director, Clinical Trial Management, Medpace

Laura Omoboni holds a Master of Science in Chemistry and Pharmaceutical Technologies. She has worked within the CRO industry for 22+ years. Laura started her career as a CRA, progressed through the management levels and is currently an Executive Director. She has covered a wide variety of indications, with more recent focus on neuroscience and rare diseases. Laura has strong global experience, including advanced therapies, and has successfully led Phase I to III ATMP, gene therapy programs through to marketing approval. She is a Medpace Subject-Matter Expert for rare diseases and has a deep insight into the unique rare and ultra-rare disease study challenges and hurdles.

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Trevor Walker, DPhil

Trevor Walker, DPhil, Senior Director, Regulatory Affairs, Medpace

Dr. Trevor Walker has over 16 years’ experience in the pharmaceutical industry, gaining extensive regulatory affairs experience with a number of CROs. Within these global roles, he has been involved in a range of investigational medicinal product types at various stages of clinical development and across several therapeutic indications, and with sponsors ranging from emerging biopharma companies to multinational pharmaceutical companies. Trevor has provided regulatory strategy, oversight and delivery of global clinical development programs, focusing on clinical trials with biological products and ATMPs as well as supporting sponsor engagement with regulators. He also has experience in GMP manufacture of autologous and somatic cell therapies for use as investigational products and supported renewal of an MHRA GMP license for a manufacturing facility.

Trevor is a member of TOPRA (The Organisation for Professionals in Regulatory Affairs), and holds a bachelor’s degree (BSc) in pharmacology from Glasgow University and a doctorate (DPhil) in pharmacology from Oxford University.

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Who Should Attend?

VPs, Directors, Managers and Department Heads working within:

  • Clinical Affairs
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Outsourcing
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

What You Will Learn

Attendees will gain insights into:

  • Successful approaches for achieving cell and gene therapy approvals in rare diseases
  • Overcoming common clinical development challenges
  • Lessons learned in planning and conducting advanced therapy clinical trials in rare diseases

Xtalks Partner


Medpace is a scientifically-driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages local regulatory and deep therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective.

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