How Advanced Therapies are Changing the Landscape of Rare Disease

Life Sciences, Clinical Trials, Pharmaceutical, Drug Discovery & Development, Cell and Gene Therapy,
  • Thursday, February 28, 2019

After years of innovative research, advanced therapies are dramatically changing the way patients are treated for a wide variety of diseases – particularly for patients with rare diseases and orphan indications. New developments in advanced therapy medicine products (ATMPs) comprising cell and gene therapy products show the potential to modify or even cure severe chronic conditions for which previously no treatment existed. As a consequence, the dynamic market for innovative therapies such as CAR-T, immuno-oncology, and gene and cell-based therapies grows across the globe. Academic institutes and biotech companies are contributing to these developments both in terms of scientific discovery and undertaking clinical trials aiming for regulatory approvals to treat specific patient populations. While we might be in a golden age of medicine due to advancements in ATMPs, innovative clinical research and development remain crucial for getting treatments to patients.

Join experts from Medpace’s medical, regulatory and operational team with hands-on experience in rare disease and ATMP development as they explore:

  • Scientific Advancements: The new and exciting landscape for advanced therapies in rare disease for a variety of indications, with a deeper dive into hematology and ophthalmology
  • Regulatory Landscape: Key characteristics and considerations for obtaining orphan drug development (ODD) designation in the US as well as an overview of the comparative differences among current global orphan product laws and regulations
  • Operational Considerations: Lessons-learned and best practices for operationalizing rare disease clinical trials including identifying, enrolling and retaining patients, as well as special considerations for ATMPs

Even though many ATMPs are in early development, the potential to cure severe chronic conditions suggest that ATMPs have the potential to reach the market earlier than standard therapies. After so many years of innovative research, we are at the point to develop advanced therapies that bring life-changing treatments to patients with rare diseases — join the discussion on Rare Disease Day 2019.


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 with a strong background in thrombosis and hemostasis research, development of anti-thrombotic therapies for a broad range of indications, and development of gene therapy for hemophilia and ophthalmology. Prior to joining Medpace in 2017, Dr. Tangelder held key roles in various biotechnology and pharmaceutical companies where he was responsible for translational medicine, phase 1-4 clinical development programs and regulatory approvals. Most recently, he led the clinical development of gene therapy for hemophilia and contributed to preclinical development and translational research of gene therapy constructs for various rare disease indications. Dr. Tangelder received both his medical degree and PhD at the University of Utrecht in the Netherlands and received his Master’s in Pharmaceutical Medicine at the Karolinska Institutet in Stockholm, Sweden.

Message Presenter

Todd Banks, PharmD, RPh. Director of Regulatory Affairs and Regulatory Intelligence, Medpace

Dr. Todd Banks is a clinical pharmacist with 34+ years of experience in the pharmaceutical industry with a strong background and knowledge in pharmacotherapeutics and FDA regulatory sciences. His healthcare experience is comprehensive and encompasses the complete product life-cycle from product development and formulation, to medical affairs and human safety, pharmacovigilance and regulatory affairs. The breadth of his experience spans cosmetics, medical devices, and non-prescription and prescription medications. He has led numerous scientific advisory meetings on first-in-class product innovations, has provided regulatory support for three successful Rx-to-OTC drug switches, obtained FDA market authorization for the first cleared direct-to-consumer photoepilation medical device and has successfully led numerous pre-IND and IND drug programs in a variety of therapeutic areas, including ultra rare diseases. He is a registered pharmacist, holds a doctorate degree in Clinical Pharmacy with undergraduate degrees in instrumental chemistry, organic chemistry, natural science and pharmacy, all from the University of Cincinnati.

Message Presenter

Madhavi Malladi, PhD, Clinical Trial Manager, Medpace

Madhavi Malladi has 6+ years of clinical research experience which includes working on trials involving advanced therapies for rare disease indications. Prior to joining Medpace, she was an Assistant Research Director at New York Presbyterian/Queens. She has a PhD in Cell and Molecular Biology from The University of Texas at Austin. She completed her Postdoctoral Fellowship in Cancer genetics at Herbert Irving Cancer Center at Colombia University, focusing on molecular classification of diffuse large B-cell lymphomas using transcriptome profiling, next generation sequencing and RNAi technologies. Her therapeutic experience includes ophthalmology, hematology and oncology, cardiovascular, endocrine disorders,obstetrics/gynecology and neonatology.

Message Presenter

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

  • Advanced therapies in rare disease with an emphasis on hematology and ophthalmology
  • Key considerations for obtaining ODD designation in the US
  • Best practices for operationalizing rare disease clinical trials

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. Headquartered in Cincinnati, Ohio, Medpace employs approximately 2,800 people across 36 countries.

You Must Login To Register for this Free Webinar

Already have an account? LOGIN HERE. If you don’t have an account you need to create a free account.

Create Account