Pre Conference Workshops
Tuesday, March 10

Workshop A

Natural History Studies: Looking Backwards to Move Forward
08:00 - 11:00

This session will look to highlight how using natural history studies and acquired learnings from drug development progress of the field can inform future decision making.

Harness the power of natural history research and rethink your approach to clinical
trial design to more confidently produce meaningful clinical endpoints.

Explore how collaborative approaches to natural history studies can reduce study
repetition and the burden placed on patients.

Workshop Leader:

16080 - MarciaSellosMoura.OrphanTech

Marcia Sellos-Moura
Senior VP
Orphan Technologies

Workshop B

Identifying Meaningful Clinical Endpoints in Rare Metabolic Disease Trials
11:30 - 14:30

This workshop will look to explore how meaningful clinical endpoints can more confidently be identified in rare metabolic disorder trials. Evaluate current clinical trial design and explore how a more ‘patient centric’ focus can work to better identify and validate meaningful endpoints for the patient. Furthermore, discuss how therapeutic testing protocol can be better designed in order to develop a standardized methodology that more effectively tracks diseases progression and therapeutic outcomes.

Workshop Leader:

16080 - PaoloMartini.ModernaTherapeutics

Paolo Martini
Moderna Therapeutics

Workshop C

Patient Access & Recruitment for Rare & Ultra-Rare Metabolic Diseases
15:00 - 18:00

As the rate of drug development for inherited metabolic diseases continue to increase, access to an already limited population of patients for clinical trials becomes progressively competitive.

This workshop will look to tackle the fundamental challenges that are associated with developing drugs for rare diseases with inherently low patient population numbers. Join this workshop to explore how industry and patient advocacy collaborations can work to maximize patient recruitment and retention. Further your understanding of patient eligibility for clinical trials to more confidently match the right patient to the right trail.

Workshop Leaders:

16080 - LexCowsert.NPKU

Lex Cowsert
Director of Research
National PKU Alliance

16080 - ReenaKartha.UofMinnesota

Reena Kartha
Associate Director
University of Minnesota

17090 - Sue Khan.NTSAD

Sue Khan
Executive Director