Key Considerations Accelerating Rare Disease Drug Development



Available On Demand This panel discussion will include an informative conversation among Parexel experts, who will share key considerations in accelerating drug development for rare diseases.

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Event Overview:

There are thousands of identified rare diseases today, yet only a small percentage of them have treatments readily available. Drug development is complex and presents specific challenges: many rare disease patients are children, the patient pool is often very limited for any one disease, and patients are often geographically dispersed. Selection of a development partner with strong patient recruitment and global operational capabilities is extremely important for success. What are the challenges and opportunities in accelerating RD studies?

This panel discussion with Parexel experts will address:

  • Building partnerships and relationships between physicians, site staff, patients, and families
  • Considerations for conducting Rare Diseases trials
  • Strategies for accelerating pathways and natural history studies
  • Marketing authorizations with specific indications for rare diseases

Key Learning Objectives:

  • Partners and partnerships: Building relationships and utilizing advocacy groups
  • Quality natural history studies for FDA approval
  • Considerations for conducting pediatric Rare Diseases trials
  • Regulatory strategies for accelerating pathways and natural history studies
  • The usage of real-world evidence (RWE) and synthetic control arms in trials
  • Identifying the right evidence for marketing approval

Who Should Attend:

  • Small to mid-sized biotech and pharma companies
  • Regulatory and clinical functions
  • Manager level and above


Lisa Dilworth
Vice President, Integrated Strategic Solutions

Recently designated the honor of being named to the list of the 100 Most Inspiring People by PharmaVoice, her breadth of experience encompasses more than 20 years of clinical research experience in all phases of drug development. Ms. Dilworth is a passionate collaborator committed to working directly with patients and their families, physicians, sponsors, and other key stakeholders in the rare and orphan disease space. Ms. Dilworth has extensive experience in study design, eligibility criteria, feasibility, endpoint selection, operations/project management, and overall project/program strategy. She combines her prior work as a study coordinator and neurophysiology technician with a patient-focused mindset and strong relationships with advocacy groups to ensure that the appropriate strategies are employed to successfully execute trials.

Lucas Kempf
Vice President, Regulatory Consulting

Lucas has more than 15 years of industry experience. He was the former Director of Rare Disease program Office of New drugs and former team lead for Psychiatric Products for the CDER FDA. He co-authored inter-center and intra-center draft and final Guidances to Industry on drug development for common issues for rare diseases drug development, natural history studies, pre-IND meetings, rare pediatric priority review voucher programs, and eligibility criteria, among other specific rare disease guidances.

Leanne Larson
Senior Vice President, Real-World Evidence and Access

Leanne brings over 25 years’ experience in healthcare, featuring extensive work in pharmaceutical product development and commercialization, and in healthcare technology and operational consulting. Leanne is an industry leader in designing and leading observational / non-interventional studies and in advancing the science of outcomes research, and publishes and speaks widely on a variety of topics in this area.

Rosamund Round
Vice President, Patient Innovation Center

Rosamund has spent more than 15 years devoted to simplifying the patient journey in clinical trials, with a focus on reducing geographical, financial, and practical barriers to study participation. Her first job in an oncology clinic at Massachusetts General Hospital sparked her passion for putting patients at the center of clinical trial planning and implementation. Subsequent patient recruitment roles in both the pharma and CRO industries allowed her to explore better ways to communicate with patients. Her current emphasis is on addressing literacy and health literacy, exploring technological advances, and generating new ideas to make clinical trial participation more accessible and convenient for patients.

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